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Longitudinal Study of Urea Cycle Disorders

This study is seeking individuals with urea cycle disorders. The study aims to perform a long-term analysis of individuals with a UCD.

All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT00237315
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Inclusion Criteria:

• Diagnosis of NAGS deficiency, defined as the detection of a pathogenic mutation, and/or decreased (less than 20 % of control) NAGS enzyme activity in liver ,and/or hyperammonemia and first degree relative meets at least one of the criteria for NAGS deficiency
• Diagnosis of CPS I deficiency, defined as decreased (less than 20 % of control) CPS I enzyme activity in liver, and/or an identified pathogenic mutation, and/or hyperammonemia and first degree relative meets at least one of the criteria for CPS I deficiency
• Diagnosis of OTC deficiency, defined as the identification of a pathogenic mutation, and/or less than 20% of control of OTC activity in the liver, and/or elevated urinary orotate (greater than 20 uM/mM) in a random urine sample or after allopurinol challenge test, and/or hyperammonemia and first degree relative meets at least one of the criteria for OTC deficiency
• Diagnosis of AS deficiency (Citrullinemia), defined as a greater than or equal to 10-fold elevation of citrulline in plasma, and/or decreased AS enzyme activity in cultured skin fibroblasts or other appropriate tissue, and/or identification of a pathogenic mutation in the AS gene, and/or hyperammonemia and first degree relative meets at least one of the criteria for AS Deficiency
• Diagnosis of AL deficiency (Argininosuccinic Aciduria, ASA), defined as the presence of argininosuccinic acid in the blood or urine, and/or decreased AL enzyme activity in cultured skin fibroblasts or other appropriate tissue, and/or identification of a pathogenic mutation in the AL gene, and/or hyperammonemia and first degree relative meets at least one of the criteria for AL Deficiency
• Diagnosis of ARG deficiency (Hyperargininemia), defined as a greater than or equal to 5-fold elevated arginine levels in the blood, and/or decreased arginase enzyme levels in red blood cells or other appropriate tissue, and/or identification of a pathogenic mutation in the ARG gene, and/or hyperammonemia and first degree relative meets at least one of the criteria for ARG Deficiency
• Diagnosis of HHH Syndrome or ORNT deficiency, defined as a greater than or equal to 5-fold elevated plasma ornithine and homocitrulline levels in the urine, and/or a pathogenic mutation, and/or less than 20% residual labeled ornithine incorporation into protein in cultured fibroblasts, and/or hyperammonemia and first degree relative meets at least one of the criteria for HHH Syndrome or ORNT Deficiency
• Diagnosis of CITR deficiency (Citrullinemia Type II), defined as elevated citrulline levels in the blood and a pathogenic mutation and/or hyperammonemia and first degree relative meets criteria for CITR Deficiency
• Pending diagnosis of a UCD (UCD highly likely), defined as laboratory values highly suggestive of a UCD with symptomatic hyperammonemic episodes but without a verifiable diagnosis
Exclusion Criteria:

• Hyperammonemia caused by an organic academia, lysinuric protein intolerance, mitochondrial disorder, congenital lactic academia, fatty acid oxidation defects, or primary liver disease
• Rare and unrelated comorbidities (e.g., Down's syndrome, intraventricular hemorrhage in the newborn period, and extreme prematurity)
Brain Diseases, Metabolic, Inborn, Amino Acid Metabolism, Inborn Errors, Urea Cycle Disorders
Urea, Inherited metabolic disorders
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Adaptive Phase II Study to Evaluate the Safety & Efficacy of NaBen®

All
12 Years to 17 Years old
Phase 2/Phase 3
This study is NOT accepting healthy volunteers
NCT01908192
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Inclusion Criteria:

• Male or female subjects who are between 12 and 17 years of age inclusive
• Physician confirmed DSM-IV or -V diagnosis of schizophrenia based on MINI International Neuropsychiatric Interview for Schizophrenia and Psychotic Disorders Studies for Children and Adolescents, version 6.0 (MINI-KID, Version 6.0)
• Are clinically stable with residual symptoms, defined as a total score of ≥ 60 of PANSS and a score of ≥ 40 for SANS
• An unchanged antipsychotic medication regimen for at least eight (8) weeks prior to randomization into the study and expected to remain unchanged during the study (longer for depot or long-acting antipsychotics: ten (10) months for Aripiprazole (Maintena®) and Paliperidone (Xeplion®); six (6) months for Olanzapine pamoate monohydrate (Zypadhera®); and at least 6 times duration of the reported half life or minimum four (4) months for other depot or long-acting antipsychotics)
• In good general physical health and all physical exam, neurological exam and laboratory assessments (urine/blood routine, biochemical tests and ECG) are clinically unremarkable per the investigator
• Subject has a negative urine illicit drug screening test
• Subject understands and is willing to sign the Informed Assent Form (IAF) prior to study entry and agrees to be available for all the study visits
• The subject's guardian understands and is willing to sign the Informed Consent Form (ICF) prior to study entry and agrees to be available for all the study visits
• Must not be a danger to self or others and must have family support available to be maintained as outpatients
Exclusion Criteria:

• Meets the DSM-IV or -V criteria at screening for mental retardation, dissociative disorder, bipolar disorder, major depressive disorder, schizoaffective disorder, schizophreniform disorder, autistic disorder, or primary substance induced psychotic disorder. Other comorbid disorders; e.g., attention-deficit hyperactivity disorder (ADHD), are allowed as long as schizophrenia is the primary diagnosis and the comorbid disorder(s) do not require medication.
• Subjects whose illness was resistant to antipsychotics according to prior trials of two different antipsychotics of adequate dose
• History of epilepsy, head trauma, or neurological illness other than Tourette's syndrome
• History of allergic reaction to sodium benzoate
• Serious medical illnesses such as acute or chronic renal disease, liver failure or heart disease that, in the opinion of the investigator, may interfere with the conduct of the study.
• Current substance abuse or positive urine illicit drug screening or history of substance dependence (including alcohol, but excluding nicotine and caffeine) in the past three (3) months.
• Use of depot antipsychotics in the past six (6) months
• Inability to follow protocol
• Body Mass Index (BMI) > 35
• Female subjects who are pregnant (as confirmed by urine pregnancy test performed at screening Visit) or are nursing, or who do not agree to abstinence or birth control during the study
• Cancer within the last three (3) years except for basal cell carcinoma and squamous cell carcinoma
• Previous participation in an intervention trial within 30 days of randomization
• Subjects whose PANSS score has decreased more than 10 percent during the Screening Phase
Drug: NaBen®, Drug: Placebo
Schizophrenia
Sodium Benzoate, Schizophrenia, Adolescent, Antipsychotic, Anti-psychotic, NMDA, NaBen, pediatric
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Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease

Gaucher disease is the most common lysosomal storage disorder due to a mutation in the lysosomal enzyme, glucocerebrosidase. There is increasing evidence that oxidative stress and/or inflammation contribute to the pathophysiology. In order to evaluate oxidative stress and/or inflammation in patients with Gaucher disease, we will analyze a series of blood biomarkers and correlate them with currently used diagnostic biomarkers of this condition. We will determine oxidative stress and/or inflammation related biomarkers in whole blood and/or plasma in adult subjects with Gaucher disease. Fifteen milliliter blood sample will be collected during three independent visits over a period of approximately 3 months. These samples will be processed to separate plasma from red blood cells and frozen until assays are performed. Standardized immunoassay methods and LC/MS based methods will be adopted to assay a series of biomarkers in these samples. These data will be correlated with currently used diagnostic biomarkers.

All
18 Years to 75 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02437396
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Inclusion criteria:
• All participants must be 18 years or older.
• All enrollees must understand and cooperate with requirements of the study in the opinion of the investigators and must be able to provide written informed consent.
• Individuals with Gaucher disease who are medically stable for participation in study in the opinion of the investigator.
• GD subjects must be stable on a specific ERT and/or SRT therapy at a specific dose (for e.g. on a units/kg basis) for at least 2 years or be naïve to these therapies (no therapy for 2 years).
• GD1 patients, who have had a change in therapy i.e. a change in dose or switch from one drug to another, can be enrolled after at least 6 months have elapsed since the change and is considered stable in the opinion of the clinician providing care to the patient.
• All participants must not have taken antioxidants coenzyme Q-10, vitamin C, or vitamin E for 3 weeks prior to the study.
Exclusion Criteria:

• Medically unstable conditions in any group as determined by the investigators
• Concurrent disease; medical condition; or an extenuating circumstance that, in the opinion of the investigator, might compromise subject safety, study compliance, completion of the study, or the integrity of the data collected for the study.
• Females who are pregnant or lactating or of child-bearing age who are not using acceptable forms of contraception
• History of asthma that is presently being treated
• Subjects who cannot or are unwilling to have blood drawn
• Unable to adhere to study protocol for whatever reason
Gaucher Disease Type I, Oxidative Stress, Inflammation
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MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions

Jakub Tolar, MD
All
up to 25 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02582775
1510M79481
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Inclusion Criteria:

• Diagnosis of severe form of EB characterized by collagen, laminin, integrin, keratin or plakin deficiency (by immunofluorescence staining with protein specific antibodies or Western blotting and by mutation analysis).
• Adequate organ function within 4 weeks of study registration defined as:
• Renal: glomerular filtration rate within normal range for age
• Hepatic: Hepatic: bilirubin, AST/ALT, ALP < 5 x upper limit of normal
• Pulmonary: adequate pulmonary function in the opinion of the enrolling investigator
• Cardiac: left ventricular ejection fraction ≥ 45%, normal EKG or approved by Cardiology for transplant
• Sexually active participants must agree to use adequate birth control for the during the study period (from before the start of the preparative chemotherapy through 1 year post-transplant)
• Available donor per section 5: targeted MFI < 1,000 (MFI exceeding 1000 must be approved by the PI and treatment team.)
• Voluntary written consent - adult or parent (with information sheet for minors, if applicable) prior to any research related procedures or treatment
Exclusion Criteria:

• beta 3 laminin JEB mutants
• Active untreated systemic infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days)
• History of HIV infection
• Evidence of squamous cell carcinoma
• Pregnant or breast feeding. Females of child-bearing potential must have a negative pregnancy test prior to study registration as the agents administered in this study are Pregnancy Category C and D.
Drug: Thymoglobulin, Drug: Cyclophosphamide, Drug: Fludarabine, Radiation: Total Body Irradiation, Procedure: Bone marrow infusion, Drug: Tacrolimus, Drug: Mycophenolate Mofetil, Biological: Donor mesenchymal stem cell infusions, Drug: Busulfan
Epidermolysis Bullosa
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Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1): Potential Use of Antioxidant/Anti-inflammatory Medications

The purpose of this study is to measure levels of blood and brain chemicals related to oxidative stress and inflammation in healthy volunteers and individuals with Type 1 Gaucher disease (GD1) to see if these levels are altered by GD1. We will also examine if there is a change in these blood and brain chemicals after participants begin taking oral N-acetylcysteine (NAC), which is available both as a prescription medication and a natural product that has antioxidant and anti-inflammatory effects.

James Cloyd
All
18 Years and over
Phase 2
This study is also accepting healthy volunteers
NCT02583672
1506M74581
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Inclusion Criteria:

• All participants must be 18 years or older.
• All participants must understand and cooperate with requirements of the study in the opinion of the investigators and must be able to provide written informed consent.
• Individuals with GD1 who are medically stable for participation in the study in the opinion of the investigator.
• GD1 patients must be on a stable, specific ERT and/or SRT therapy at a specific dose (e.g. on a units/kg basis) for at least 2 years.
• GD1 patients who have had a change in therapy, i.e. a change in dose or switch from one drug to another, can be enrolled after at least 6 months have elapsed since the change and is considered stable in the opinion of the clinician providing care to the patient.
• Healthy subjects who will be frequency-matched for age.
• All participants must not have taken antioxidants coenzyme Q-10, vitamin C, or vitamin E for 3 weeks prior to the study and during the course of the study.
Exclusion Criteria:

• Medically unstable conditions in any group as determined by the investigators.
• Concurrent disease; medical condition; or an extenuating circumstance that, in the opinion of the investigator, might compromise subject safety, study compliance, completion of the study, or the integrity of the data collected for the study.
• Women who are pregnant or lactating or of child-bearing age who are not using acceptable forms of contraception.
• History of asthma that is presently being treated.
• Patients enrolled in another interventional study.
• Allergy to N-acetylcysteine.
• Patients who cannot or are unwilling to have blood drawn.
• Inability to undergo MRI scanning, including but not limited to: unable to remain still in an MRI scanner for more than 30 minutes, claustrophobia, presence of paramagnetic substances or pacemakers in body, weight over 300 lbs.
• Unable to adhere to study protocol for whatever reason.
Drug: N-acetylcysteine
Gaucher Disease Type 1
Gaucher Disease, GD1, N-acetylcysteine, glutathione, GSH
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Observational Study of Pediatric Rheumatic Diseases: The CARRA Registry

The primary objective for this observational study is to collect general and medical data from children, adolescents, and young adults who had pediatric onset rheumatic disease. This data will be used to evaluate the long-term safety and efficacy of therapeutic agents used to treat these diseases. This information will allow investigators to accurately report and follow changes in current medication use patterns and compare these to proposed standards and current treatment recommendations. The use of a single registry will allow for more analysis of the different therapeutic agents by allowing them to be compared to each other.

Colleen Correll
All
up to 21 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02418442
1506M74443
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Inclusion Criteria:

• Onset of rheumatic disease prior to age 16 years for JIA and onset prior to age 19 years for all other rheumatic diseases (see appendix A).
• Subject (and/or parent/legal guardian when required) is able to provide written informed consent and willing to comply with study procedures.
• Subject and/or parent/legal guardian is willing to be contacted in the future by study staff.
Exclusion Criteria:

• Greater than 21 years of age at the time of enrollment.
Rheumatic Joint Disease
Systemic Arthritis, Oligoarthritis, Polyarthritis (Rheumatoid Factor Negative), Polyarthritis (Rheumatoid Factor Positive), Psoriatic Arthritis, Enthesitis Related Arthritis (ERA), Undifferianted Arthritis, CARRA Consensus Treatment Plans
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CAROTID REVASCULARIZATION AND MEDICAL MANAGEMENT FOR ASYMPTOMATIC CAROTID STENOSIS TRIAL (CREST-2) PROTOCOL (CREST-2)

CREST-2 is a parallel trial to compare carotid endarterectomy + intensive medical management (IMM) vs IMM alone, and carotid artery stenting plus IMM vs IMM alone in patients with asymptomatic carotid stenosis >70%.

Andrew Grande
All
35 Years to 100 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02089217
1508M77101
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General Inclusion Criteria
• Patients ≥35 years old.
• Carotid stenosis defined as:
• Stenosis ≥70% by catheter angiography (NASCET Criteria); OR
• by DUS with ≥70% stenosis defined by a peak systolic velocity of at least 230 cm/s plus at least one of the following:
• an end diastolic velocity ≥100 cm/s, or
• internal carotid/common carotid artery peak systolic velocity ratio ≥4.0, or
• CTA with ≥ 70% stenosis, or
• MRA with ≥ 70% stenosis.
• No medical history of stroke or TIA ipsilateral to the stenosis within 180 days of randomization. Life-long asymptomatic patients will be defined as having no medical history of stroke or transient ischemic attack and negative responses to all of the symptom items on the Questionnaire for Verifying Stroke-free Status (QVSS).18
• Patients must have a modified Rankin Scale score of 0 or 1 at the time of informed consent.
• Women must not be of childbearing potential or, if of childbearing potential, have a negative pregnancy test prior to randomization.
• Patients must agree to comply with all protocol-specified follow-up appointments.
• Patients must sign a consent form that has been approved by the local governing Institutional Review Board (IRB)/Medical Ethics Committee (MEC) of the respective clinical site.
• Randomization to treatment group will apply to only one carotid artery for patients with bilateral carotid stenosis. Management of the non-randomized stenosis may be done in accordance with local PI recommendation. Treatment of the non-study internal carotid artery must take place at least 30 days prior to randomization, or greater than 44 days after randomization and 30 days after the study procedure is completed (whichever is longer).
• Carotid stenosis must be treatable with CEA, CAS, or either procedure. General Exclusion Criteria
• Intolerance or allergic reaction to a study medication without a suitable management alternative.
• GI hemorrhage within 1 month prior to enrollment that would preclude antiplatelet therapy.
• Prior major ipsilateral stroke in the past with substantial residual disability (mRS ≥ 2) that is likely to confound study outcomes.
• Severe dementia.
• History of major symptomatic intracranial hemorrhage within 12 months that was not related to anticoagulation.
• Prior Intracranial hemorrhage that the investigator believes represents a contraindication to the perioperative or periprocedural antithrombotic and antiplatelet treatments necessary to complete endarterectomy or stenting per protocol.
• Current neurologic illness characterized by fleeting or fixed neurologic deficits that cannot be distinguished from TIA or stroke.
• Patient objects to future blood transfusions.
• Platelet count <100,000/microliter or history of heparin-induced thrombocytopenia.
• Anticoagulation with Phenprocoumon (Marcumar®), warfarin, or a direct thrombin inhibitor, or anti-Xa agents.
• Chronic atrial fibrillation.
• Any episode of atrial fibrillation within the past 6 months or history of paroxysmal atrial fibrillation that is deemed to require chronic anticoagulation.
• Other high-risk cardiac sources of emboli, including left ventricular aneurysm, severe cardiomyopathy, aortic or mitral mechanical heart valve, severe calcific aortic stenosis (valve area < 1.0 cm2), endocarditis, moderate to severe mitral stenosis, left atrial thrombus, or any intracardiac mass, or known unrepaired PFO with prior paradoxical embolism.
• Unstable angina defined as rest angina with ECG changes that is not amenable to revascularization (patients should undergo planned coronary revascularization at least 30 days before randomization).
• Left Ventricular Ejection fraction <30% or admission for heart failure in prior 6 months.
• Respiratory insufficiency with life expectancy < 4 years or FEV1 <30% of predicted value.
• Known malignancy other than basal cell non-melanoma skin cancer. There are two exceptions to this rule: patients with prior cancer treatment and no recurrence for >5 years are eligible for enrollment and cancer patients with life expectancy of greater than 5 years are eligible for enrollment.
• Any major surgery, major trauma, revascularization procedure, or acute coronary syndrome within the past 1 month.
• Either the serum creatinine is ≥ 2.5 mg/dl or the estimated GFR is < 30 cc/min.
• Major (non-carotid) surgery/procedures planned within 3 months after enrollment.
• Currently listed or being evaluated for major organ transplantation (i.e. heart, lung, liver, kidney).
• Actively participating in another drug or aortic arch or cerebrovascular device trial for which participation in CREST-2 would be compromised with regard to follow-up assessment of outcomes or continuation in CREST-2.
• Inability to understand and cooperate with study procedures or provide informed consent.
• Non-atherosclerotic carotid stenosis (dissection, fibromuscular dysplasia, or stenosis following radiation therapy).
• Previous ipsilateral CEA or CAS.
• Ipsilateral internal or common carotid artery occlusion.
• Intra-carotid floating thrombus.
• Ipsilateral intracranial aneurysm > 5 mm.
• Extreme morbid obesity that would compromise patient safety during the procedure or would compromise patient safety during the periprocedural period.
• Coronary artery disease with two or more proximal or major diseased coronary arteries with 70% stenosis that have not, or cannot, be revascularized. Specific carotid endarterectomy exclusion criteria Patients who are being considered for revascularization by CEA must not have any of the following criteria:
• Serious adverse reaction to anesthesia not able to be overcome by pre-medication.
• Distal/intracranial stenosis greater than index lesion.
• Any of the following anatomical: radical neck dissection; surgically inaccessible lesions (e.g. above cervical spine level 2 (C2)); adverse neck anatomy that limits surgical exposure (e.g. spinal immobility - inability to flex neck beyond neutral or kyphotic deformity, or short obese neck); presence of tracheostomy stoma; laryngeal nerve palsy contralateral to target vessel; or previous extracranial-intracranial or subclavian bypass procedure ipsilateral to the target vessel. Specific Carotid Artery Stenting Exclusion Criteria Patients who are being considered for revascularization by CAS must not have any of the following criteria:
• Allergy to intravascular contrast dye not amenable to pre-medication.
• Type III, aortic arch anatomy.
• Angulation or tortuosity (≥ 90 degree) of the innominate and common carotid artery that precludes safe, expeditious sheath placement or that will transmit a severe loop to the internal carotid after sheath placement.
• Severe angulation or tortuosity of the internal carotid artery (including calyceal origin from the carotid bifurcation) that precludes safe deployment of embolic protection device or stent. Severe tortuosity is defined as 2 or more ≥ 90 degree angles within 4 cm of the target stenosis.
• Proximal/ostial CCA, innominate stenosis or distal/intracranial stenosis greater than index lesion. Excessive circumferential calcification of the stenotic lesion defined as >3mm thickness of calcification seen in orthogonal views on fluoroscopy.(Note: Anatomic considerations such as tortuosity, arch anatomy, and calcification must be evaluated even more carefully in elderly subjects (≥ 70 years).)
• Target ICA vessel reference diameter <4.0 mm or >9.0 mm. Target ICA measurements may be made from angiography of the contralateral artery. The reference diameter must be appropriate for the devices to be used.
• Inability to deploy or utilize an FDA-approved Embolic Protection Device (EPD).
• Non-contiguous lesions and long lesions (>3 cm).
• Qualitative characteristics of stenosis and stenosis-length of the carotid bifurcation (common carotid) and/or ipsilateral external carotid artery, that preclude safe sheath placement.
• Occlusive or critical ilio-femoral disease including severe tortuosity or stenosis that necessitates additional endovascular procedures to facilitate access to the aortic arch or that prevents safe and expeditious femoral access to the aortic arch. "String sign" of the ipsilateral common or internal carotid artery.
• Angiographic, CT, MR or ultrasound evidence of severe atherosclerosis of the aortic arch or origin of the innominate or common carotid arteries that would preclude safe passage of the sheath and other endovascular devices to the target artery as needed for carotid stenting.
Procedure: Carotid endarterectomy (CEA), Device: Carotid Stenting (CAS), Other: Intensive Medical Management - no CEA, Other: Intensive Medical Management - no CAS
Carotid Stenosis
asymptomatic, carotid, stent, endarterectomy, embolic protection, medical management, hypertension, hyperlipidemia, cognition, risk factor control
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Targeted Therapy Directed by Genetic Testing in Treating Patients With Advanced Refractory Solid Tumors, Lymphomas, or Multiple Myeloma (The MATCH Screening Trial)

Primary Objective To evaluate the proportion of patients with objective response (OR) to targeted study agent(s) in patients with advanced refractory cancers/lymphomas. Secondary Objectives To evaluate the proportion of patients alive and progression free at 6 months of treatment with targeted study agent in patients with advanced refractory cancers/lymphomas. To evaluate time until death or disease progression. To identify potential predictive biomarkers beyond the genomic alteration by which treatment is assigned or resistance mechanisms using additional genomic, RNA and protein-based assessment platforms.

Emil Lou
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT02465060
1603M85141
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Inclusion Criteria:

• ELIGIBILITY CRITERIA FOR SCREENING BIOPSY (STEP 0)
• Patients of childbearing potential must have a negative serum pregnancy test within 2 weeks prior to registration; patients that are pregnant or breast feeding are excluded; a patient of childbearing potential is anyone, regardless of sexual orientation or whether they have undergone tubal ligation, who meets the following criteria:
• Has achieved menarche at some point
• Has not undergone a hysterectomy or bilateral oophorectomy; or
• Has not been naturally postmenopausal for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months)
• Patients must not expect to conceive or father children by using accepted and effective method(s) of contraception or by abstaining from sexual intercourse prior to study entry, for the duration of study participation, and for 4 months after completion of study; should a patient or partner of the patient become pregnant or suspect a pregnancy while participating in this study, the treating physician should be informed immediately
• Patients must have histologically documented solid tumors or histologically confirmed diagnosis of lymphoma or multiple myeloma requiring therapy and meet one of the following criteria:
• Patients must have progressed following at least one line of standard systemic therapy and there must not be other approval/standard therapy available that has been shown to prolong overall survival (i.e. in a randomized trial against another standard treatment or by comparison to historical controls); patients who cannot receive other standard therapy that has been shown to prolong overall survival due to medical issues will be eligible, if other eligibility criteria are met; if the patient is currently receiving therapy, the clinician must have assessed that the current therapy is no longer benefitting the patient prior to enrolling on MATCH, regardless of whether it is considered standard OR
• Patients for whose disease no standard treatment exists that has been shown to prolong overall survival
• NOTE: No other prior malignancy is allowed except for the following:
• Adequately treated basal cell or squamous cell skin cancer
• In situ cervical cancer
• Adequately treated stage I or II cancer from which the patient is currently in complete remission
• Any other cancer from which the patient has been disease-free for 5 years
• Patients must have measurable disease
• Patients must meet the criteria below
• Tumor tissue for the confirmation of "rare variant" by the MATCH assay is to be submitted, preferably from the same time of collection as that used to determine patient candidacy for treatment arm assignment
• Registration to Step 0 must occur after stopping prior systemic anti-cancer therapy. There is no specific duration for which patients must be off treatment prior to registration to Step 0, as long as all eligibility criteria are met
• Patients may have received other non-targeted, immunotherapy or targeted treatment between the prior genetic testing at the outside lab and registration to Step 0. The decision to stop such treatment in favor of participation in MATCH, if no further clinical benefit is expected, is per the treating physician's discretion. Documentation of a lack of response to the prior treatment is not required in these cases
• Patients with an applicable "rare variant" must be able to meet the eligibility criteria for the appropriate subprotocols within 4 weeks following notification of treatment assignment
• Patient meets one of the following criteria:
• Patient is a candidate for Z1M based on local CLIA assessment of MMRd by immunohistochemistry (IHC) or MSI status by polymerase chain reaction (PCR), adequate tumor tissue is available for submission for mandatory central screening IHC and the patient will be able to meet the eligibility criteria for Z1M within 4 weeks following notification of treatment assignment OR
• The sites have received results from one of the designated outside laboratories indicating a "rare variant" that is an actionable Mutation of Interest (aMOI) for specific select subprotocols
• NOTE: There is no particular window of time after receiving the sequencing report notification of potential eligibility from an outside lab in which the patient must be registered to Step 0, but treatment slots will be assigned on a first come, first serve basis to those who do register to Step 0, and are not held for those notified of potential eligibility who do not register to Step 0
• NOTE: Treatment assignment (and the start of the associated deadline for Step 1 registration) may occur shortly after Step 0 registration. Note that certain "rare variant" arms require submission of archival tissue for central IHC testing to determine treatment assignment. For those arms, adequate tissue for the central IHC is required to be available for submission
• NOTE: Other potential aMOIs that would be eligibility criteria for "NON RARE" arms, as determined by the designated laboratories, are not applicable for this process in MATCH
• Patients must have Eastern Cooperative Oncology Group (ECOG) performance status =< 1 and a life expectancy of at least 3 months
• Patients must be able to swallow tablets or capsules; a patient with any gastrointestinal disease that would impair ability to swallow, retain, or absorb drug is not eligible
• Patients who are human immunodeficiency virus (HIV)-positive are eligible if:
• CD4+ cell count greater or equal to 250 cells/mm^3
• If patient is on antiretroviral therapy, there must be minimal interactions or overlapping toxicity of the antiretroviral therapy with the experimental cancer treatment; for experimental cancer therapeutics with CYP3A/4 interactions, protease inhibitor therapy is disallowed; suggested regimens to replace protease inhibitor therapy include dolutegravir given with tenofovir/emtricitabine; raltegravir given with tenofovir and emtricitabine; once daily combinations that use pharmacologic boosters may not be used
• No history of non-malignancy acquired immune deficiency syndrome (AIDS)-defining conditions other than historical low CD4+ cell counts
• Probable long-term survival with HIV if cancer were not present
• Any prior therapy, radiotherapy (except palliative radiation therapy of 30 gray [Gy] or less), or major surgery must have been completed >= 4 weeks prior to start of treatment; all adverse events due to prior therapy have resolved to a grade 1 or better (except alopecia and lymphopenia) by start of treatment; palliative radiation therapy must have been completed at least 2 weeks prior to start of treatment; the radiotherapy must not be to a lesion that is included as measurable disease
• NOTE: Prostate cancer patients may continue their luteinizing hormone-releasing hormone (LHRH) agonist
• NOTE: For patients entering the study via the original screening process, patients may receive non-protocol treatment after biopsy (if clinically indicated) until they receive notification of results; however, lack of response must be documented prior to registration to Step 1; new non-protocol treatment will NOT be permitted as intervening therapy after registration to Step 0; the only intervening treatment permitted is prior therapy that the patient already received prior to Step 0 registration; the decision to stop the intervening non-protocol treatment will be left up to the treating physician if patient has an aMOI; however, patients will need to be off such therapy for at least 4 weeks before receiving any MATCH protocol treatment
• NOTE: For patients entering the study via a designated outside laboratory, no intervening systemic non-protocol treatment is permitted after Step 0 registration; all other eligibility requirements still apply to these patients, including the washouts for prior therapy noted above in this section, the time restrictions outlined, and the eligibility criteria for the intended subprotocol
• Patients with brain metastases or primary brain tumors must have completed treatment, surgery or radiation therapy >= 4 weeks prior to start of treatment
• Patients must have discontinued steroids >= 1 week prior to registration to Step 0 and remain off steroids thereafter, except as permitted; patients with glioblastoma (GBM) must have been on stable dose of steroids, or be off steroids, for one week prior to registration to treatment (Step 1, 3, 5, 7)
• NOTE: The following steroids are permitted (low dose steroid use is defined as prednisone 10 mg daily or less, or bioequivalent dose of other corticosteroid):
• Temporary steroid use: e.g. for computed tomography (CT) imaging in setting of contrast allergy
• Low dose steroid use for appetite
• Chronic inhaled steroid use
• Steroid injections for joint disease
• Stable dose of replacement steroid for adrenal insufficiency or low doses for non-malignant disease
• Topical steroid
• Steroids required to manage toxicity related to study treatment, as described in the subprotocols
• Steroids required as pre- or post-chemotherapy medication for acceptable intervening chemotherapy
• NOTE: Steroids must be completed alongside last dose of chemotherapy
• Leukocytes >= 3,000/mcL (within 2 weeks prior to screening step registration and within 4 weeks prior to treatment step registration)
• Absolute neutrophil count (ANC) >= 1,500/mcL (within 2 weeks prior to screening step registration and within 4 weeks prior to treatment step registration)
• Platelets >= 100,000/mcL (within 2 weeks prior to screening step registration and within 4 weeks prior to treatment step registration)
• NOTE: Patients with documented bone marrow involvement by lymphoma are not required to meet the above hematologic parameters, but must have a platelet count of at least 75,000/mcL and neutrophil count of at least 1,000/mcL
• Total bilirubin =< 1.5 x institutional upper limit of normal (ULN) (unless documented Gilbert's syndrome, for which bilirubin =< 3 x institutional ULN is permitted) (within 2 weeks prior to screening step registration and within 4 weeks prior to treatment step registration)
• Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 2.5 x institutional ULN (up to 5 times ULN in presence of liver metastases) (within 2 weeks prior to screening step registration and within 4 weeks prior to treatment step registration)
• Creatinine clearance >= 45 mL/min/1.73 m^2 for patients with creatinine levels above institutional ULN
• As defined by the Cockcroft-Gault equation (within 2 weeks prior to screening step registration and within 4 weeks prior to treatment step registration)
• Patients must have an electrocardiogram (ECG) within 8 weeks prior to registration to screening step and must meet the following cardiac criteria:
• Resting corrected QT interval (QTc) =< 480 msec
• NOTE: If the first recorded QTc exceeds 480 msec, two additional, consecutive ECGs are required and must result in a mean resting QTc =< 480 msec; it is recommended that there are 10-minute (+/- 5 minutes) breaks between the ECGs
• The following only need to be assessed if the mean QTc > 480 msec
• Check potassium and magnesium serum levels
• Correct any identified hypokalemia and/or hypomagnesemia and may repeat ECG to confirm exclusion of patient due to QTc
• For patients with heart rate (HR) 60-100 beats per minute (bpm), no manual read of QTc is required
• For patients with baseline HR < 60 or > 100 bpm, manual read of QT by trained personnel is required, with Fridericia correction applied to determine QTc
• Patient must not have hypokalemia (value < institutional lower limit of normal)
• No factors that increase the risk of QTc prolongation or risk of arrhythmic events such as heart failure, congenital long QT syndrome, family history of long QT syndrome or unexplained sudden death under 40 years of age or any concomitant medication known to prolong the QT interval
• NOTE: Patient must be taken off prohibited medication prior to registration to the screening step (Step 0, 2, 4, 6) and remain off these medications thereafter, unless permitted on a subprotocol for the management of treatment related toxicity; patient must be off the drug for at least 5 half-lives prior to registration to the treatment step (Step 1, 3, 5, 7); the medication half-life can be found in the package insert for Food and Drug Administration (FDA) approved drugs
• ELIGIBILITY CRITERIA FOR FIRST TREATMENT (STEP 1)
• NOTE: For patients entering step 0 with assay results from outside laboratories, no systemic treatment is allowed after step 0 registration
• As MATCH is designed to add additional subprotocols, implement limited expansions of accrual for certain subprotocols, and/or amend existing arm-specific eligibility criteria, some patients entering under the original screening method may be eligible to have their results rerun in MATCHbox, even if they did not match to a treatment initially or did not receive a treatment assignment due to a lack of available assignment slots; patients whose sequence results will be rerun through MATCHbox must also meet the following criteria:
• Samples must have been collected within 5 months of the activation of the addendum, as there is an additional month needed to get the patients on trial
• Patient has not had treatment within the 5 months that resulted in a PR or better after the performance of the screening assessment
• Patient must meet eligibility criteria, including performance status 1 or better and life expectancy of at least 3 months
• Patients must meet the eligibility requirements with the following exceptions:
• Patients may have received other non-targeted, immunotherapy or targeted treatment, which could be stopped in favor of returning to MATCH, if no response to the interim treatment has occurred and no further benefit is expected from this interim treatment, per the treating physician's discretion; documentation of a lack of response to the interim treatment is not required in these cases; however, the following restrictions apply:
• Enrollment onto another investigational therapeutic study is not permitted
• Patient cannot be responding to interim treatment, since the benefit of the MATCH treatment is unknown and may deprive patient of an effective treatment if it were given when a patient is responding to another treatment
• NOTE: Patients meeting these criteria will NOT be biopsied at this time point; instead, their step 0 results will be re-interrogated to determine if another treatment is available
• ELIGIBILITY CRITERIA FOR SECOND SCREENING (STEP 2)
• Patient's disease
Drug: Adavosertib, Drug: Afatinib, Drug: Afatinib Dimaleate, Drug: Binimetinib, Drug: Capivasertib, Drug: Copanlisib, Drug: Copanlisib Hydrochloride, Drug: Crizotinib, Other: Cytology Specimen Collection Procedure, Drug: Dabrafenib, Drug: Dabrafenib Mesylate, Drug: Dasatinib, Drug: Defactinib, Drug: Defactinib Hydrochloride, Drug: Erdafitinib, Drug: FGFR Inhibitor AZD4547, Drug: Ipatasertib, Other: Laboratory Biomarker Analysis, Drug: Larotrectinib, Drug: Larotrectinib Sulfate, Biological: Nivolumab, Drug: Osimertinib, Drug: Palbociclib, Biological: Pertuzumab, Drug: PI3K-beta Inhibitor GSK2636771, Biological: Relatlimab, Drug: Sapanisertib, Drug: Sunitinib Malate, Drug: Taselisib, Drug: Trametinib, Biological: Trastuzumab, Biological: Trastuzumab Emtansine, Drug: Ulixertinib, Drug: Vismodegib
Advanced Malignant Solid Neoplasm, Bladder Carcinoma, Breast Carcinoma, Cervical Carcinoma, Colon Carcinoma, Colorectal Carcinoma, Endometrial Carcinoma, Esophageal Carcinoma, Gastric Carcinoma, Glioma, Head and Neck Carcinoma, Kidney Carcinoma, Liver and Intrahepatic Bile Duct Carcinoma, Lung Carcinoma, Lymphoma, Malignant Uterine Neoplasm, Melanoma, Ovarian Carcinoma, Pancreatic Carcinoma, Plasma Cell Myeloma, Prostate Carcinoma, Rectal Carcinoma, Recurrent Bladder Carcinoma, Recurrent Breast Carcinoma, Recurrent Cervical Carcinoma, Recurrent Colon Carcinoma, Recurrent Colorectal Carcinoma, Recurrent Esophageal Carcinoma, Recurrent Gastric Carcinoma, Recurrent Glioma, Recurrent Head and Neck Carcinoma, Recurrent Liver Carcinoma, Recurrent Lung Carcinoma, Recurrent Lymphoma, Recurrent Malignant Solid Neoplasm, Recurrent Melanoma, Recurrent Ovarian Carcinoma, Recurrent Pancreatic Carcinoma, Recurrent Plasma Cell Myeloma, Recurrent Prostate Carcinoma, Recurrent Rectal Carcinoma, Recurrent Skin Carcinoma, Recurrent Thyroid Gland Carcinoma, Recurrent Uterine Corpus Cancer, Refractory Lymphoma, Refractory Malignant Solid Neoplasm, Refractory Plasma Cell Myeloma, Skin Carcinoma, Thyroid Gland Carcinoma, Uterine Corpus Cancer
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MT2015-25: Tandem Myeloablative Consolidation Therapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma

Ashish Gupta
All
up to 30 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02605421
1601M82901
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• Less than 30 years of age at diagnosis of neuroblastoma
• End of Induction disease evaluation demonstrating CR, PR, MR or SD
• Hematopoietic Recovery from last induction course of chemotherapy
• No uncontrolled infection
• Minimum frozen PBSCs of 2 x 10^6 CD34 cells/kg for each transplant are mandatory and a PBSC of 2 x 10^6 CD34 cells/kg for back-up are strongly recommended (thus, PBSC of no less than 6 x 10^6 CD34 cells/kg is encouraged). These must all be collected prior to the initiation of consolidation.
• Adequate organ function defined as:
• Hepatic: AST and ALT < 3 x upper limit of institutional normal; ALT ≤ 3 x ULN for age; total bilirubin ≤ 1.5 x ULN for age, if baseline was normal, > 1.0 1.5 x baseline if baseline was abnormal
• Cardiac: shortening fraction ≥ 27% or ejection fraction ≥ 45%, no clinical congestive heart failure
• Pulmonary: no evidence of dyspnea at rest and norequirement for supplemental oxygen
• Renal: Creatinine clearance or GFR > 60 mL/min/1.73m^2. If a creatinine clearance is performed at end induction and the result is < 100 ml/min/1.73m^2, a GFR must then be performed using a nuclear blood sampling method or iothalamate clearance method. Camera method is NOT allowed as measure of GFR prior to or during Consolidation therapy for patients with GFR or creatinine clearance of < 100 ml/min/1.73m^2
• Recovery from acute toxicities of last cycle of induction chemotherapy
• Appropriate written consent - adult or parent/guardian if patient is < 18 years of age and minor information sheet if patient is > 8 years of age
Drug: Thiotepa, Drug: Cyclophosphamide, Drug: Melphalan, Drug: Etoposide, Drug: Carboplatin, Biological: Autologous Stem Cell Infusion, Biological: Granulocyte colony stimulating factor
Neuroblastoma
High-risk neuroblastoma, Neuroblastoma, Myeloablative chemotherapy, Myeloablative consolidation chemotherapy, Autologous peripheral blood stem cell rescue, PBSC, Autologous stem cell rescue
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Basket Study of Entrectinib (RXDX-101) for the Treatment of Patients With Solid Tumors Harboring NTRK 1/2/3 (Trk A/B/C), ROS1, or ALK Gene Rearrangements (Fusions) (STARTRK-2)

To determine the objective response rate (ORR) of entrectinib, as assessed by BICR, in each patient population basket of solid tumors that harbor an NTRK1/2/3, ROS1, or ALK gene rearrangement.

Manish Patel
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT02568267
1512M81181
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Inclusion Criteria:

• Histologically- or cytologically-confirmed diagnosis of locally advanced or metastatic solid tumor that harbors an NTRK1/2/3, ROS1, or ALK gene rearrangement
• For patients enrolled via local molecular testing, an archival or fresh tumor tissue (unless medically contraindicated) is required to be submitted for independent central molecular testing at Ignyta's CLIA laboratory post-enrollment
• Measurable or evaluable disease
• Patients with CNS involvement, including leptomeningeal carcinomatosis, which is either asymptomatic or previously-treated and controlled, are allowed
• Prior anticancer therapy is allowed (excluding approved or investigational Trk, ROS1, or ALK inhibitors in patients who have tumors that harbor those respective gene rearrangements)
• Note: prior treatment with crizotinib is permitted only in ALK- or ROS1-rearranged NSCLC patients presenting with CNS-only progression. Other ALK inhibitors are prohibited.
• At least 2 weeks or 5 half-lives, whichever is shorter, must have elapsed after prior chemotherapy or small molecule targeted therapy
• At least 4 weeks must have elapsed since completion of antibody-directed therapy
• Prior radiotherapy is allowed if more than 14 days have elapsed since the end of treatment
• Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 and minimum life expectancy of 4 weeks
• Adequate organ function as defined per protocol
• Ability to swallow entrectinib intact
• Other protocol specified criteria
Exclusion Criteria:

• Current participation in another therapeutic clinical trial
• Prior treatment with approved or investigational Trk, ROS1, or ALK inhibitors in patients who have tumors that harbor those respective gene rearrangements
• Note: prior treatment with crizotinib is permitted only in ALK- or ROS1-rearranged NSCLC patients presenting with CNS-only progression. Other ALK inhibitors are prohibited.
• History of other previous cancer that would interfere with the determination of safety or efficacy
• Familial or personal history of congenital bone disorders, or bone metabolism alterations
• Incomplete recovery from any surgery
• History of recent (within the past 3 months) symptomatic congestive heart failure or ejection fraction ≤50% observed during screening for the study
• History of non-pharmacologically induced prolonged QTc interval
• History of additional risk factors for torsades de pointes
• Peripheral neuropathy Grade ≥ 2
• Known active infections
• Active gastrointestinal disease or other malabsorption syndromes
• Known interstitial lung disease, interstitial fibrosis, or history of tyrosine kinase inhibitor-induced pneumonitis
• Other protocol specified criteria
Drug: Entrectinib
Breast Cancer, Cholangiocarcinoma, Colorectal Cancer, Head and Neck Neoplasms, Lymphoma, Large-Cell, Anaplastic, Melanoma, Neuroendocrine Tumors, Non-Small Cell Lung Cancer, Ovarian Cancer, Pancreatic Cancer, Papillary Thyroid Cancer, Primary Brain Tumors, Renal Cell Carcinoma, Sarcomas, Salivary Gland Cancers, Adult Solid Tumor
Entrectinib, RXDX-101, TrkA, TrkB, TrkC, NTRK1, NTRK2, NTRK3, ROS1, ALK, Trk Fusions, NTRK Gene Rearrangements, ROS1 Fusions, ROS1 Gene Rearrangements, ALK Fusions, ALK Gene Rearrangements, Basket study, Non-small cell lung cancer, Colorectal cancer, Salivary gland cancers, Primary brain tumors, Melanoma, Sarcomas, Papillary thyroid cancer, Renal cell cancer, Pancreatic cancer, Breast cancer, Cholangiocarcinoma, Head & Neck cancers, Ovarian cancer, Neuroendocrine tumors
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MT2015-32 : Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning (RIC) for the Treatment of Hematological Diseases

This is a phase II trial using a non-myeloablative cyclophosphamide/ fludarabine/total body irradiation (TBI) preparative regimen followed by a related or unrelated donor stem cell infusion. It is a modification of the treatment plan which has been studied extensively over the last 10+ years which has shown consistent engraftment and low transplant related mortality (TRM).

Mark Juckett
All
up to 75 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02661035
1603M85362
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Inclusion Criteria:

• Age, Performance Status, and Graft Criteria
• Age 0 to 70 years of age with Karnofsky score ≥ 70% (≥ 16 years) or Lansky score ≥ 50 (< 16 years)
• Patients ≥ 70 and ≤ 75 years of age may be eligible if they have a HCT-CI Co-Morbidity score ≤ 2
• Must be ≥ 3 months after prior myeloablative transplant, if applicable
• 5/6 or 6/6 related donor match or a 7-8/8 HLA-A,B,C,DRB1 allele matched unrelated donor marrow and/or PBSC donor match per current institutional guidelines Related donors will be evaluated and collected per MT2012-14C; Unrelated donors will be identified and collected per usual procedures
• Eligible Diseases
• Acute Myeloid Leukemia (AML): high risk CR1 (as evidenced by preceding MDS, high risk cytogenetics, ≥ 2 cycles to obtain CR, erythroblastic or megakaryocytic leukemia, FLT-3 ITD +; CR2+. All patients must be in CR as defined by hematological recovery, AND <5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
• Very high risk pediatric patients with AML: Patients <21 years, however, are eligible with (M2 marrow) with < 25% blasts in marrow after having failed one or more cycles of chemotherapy.
• Acute Lymphocytic Leukemia (ALL): factor that define high risk CR1 include but are not limited to cytogenetics demonstrating t(9;22), t (1:19), t(4;11), other MLL rearrangements, hypodiploidy, or IKZF1 abnormalities), DNA index < 0.81, > 1 cycle to obtain CR or presence minimal residual disease (MRD). Patients in CR2+ are eligible. All patients must be in CR as defined by hematological recovery, AND <5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
• Very high risk pediatric patients with ALL: patients <21 years are also considered high risk CR1 if they had M2 or M3 marrow at day 42 from the initiation of induction or M3 marrow at the end of induction. They are eligible once they achieved a complete remission.
• Chronic Myelogenous Leukemia excluding refractory blast crisis: To be eligible in first chronic phase (CP1) patient must have failed or be intolerant to imatinib mesylate.
• Plasma Cell Leukemia after initial therapy, who achieved at least a partial remission
• Myelodysplasia (MDS) requiring transplant as defined as: IPSS INT-2 or High Risk; R-IPSS High or Very High; WHO classification: RAEB-1, RAEB-2; Severe Cytopenias: ANC < 0.8, Anemia or thrombocytopenia requiring transfusion; Poor or very poor risk cytogenetics based on IPSS or R-IPSS definitions; therapy-related MDS. Blasts must be < 5% by bone marrow aspirate morphology.
• Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL), Marginal Zone B-Cell Lymphoma or Follicular Lymphoma are eligible if there was disease progression/relapse within 12 of achieving a partial or complete remission. Patients who had remissions lasting > 12 months, are eligible after at least two prior therapies. Patients with bulky disease (nodal mass greater than 5 cm) should be considered for de-bulking chemotherapy before transplant.
• Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia, NK cell malignancies are eligible after initial therapy in CR1+ or PR1+.
• Large Cell NHL > CR2/> PR2: Patients in CR2/PR2 with initial short remission (<6 months) are eligible.
• Lymphoblastic Lymphoma, Burkitt's Lymphoma, and other high-grade NHL after initial therapy if stage III/IV in CR1/PR1 or after progression if stage I/II < 1 year.
• Multiple Myeloma beyond PR2: Patients with chromosome 13 abnormalities, first response lasting less than 6 months, or β-2 microglobulin > 3 mg/L, may be considered for this protocol after initial therapy.
• Myeloproliferative Syndromes
• Organ Function Criteria Adequate organ function is defined as:
• Liver: AST and ALT < 5 x upper limit of normal and bilirubin < 3 x upper limit of normal
• Renal: Creatinine ≤ 2.0 mg/dl (adults) and estimated glomerular filtration rate (GFR) ≥ 40 mL/min (pediatrics). Adults with a creatinine > 1.2 mg/dl or a history of renal dysfunction must have estimated glomerular filtration rate (GFR) > 40 mL/min.
• Albumin > 2.5 g/dL
• Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 35%.
• Pulmonary: DLCOcorr ≥ 40% predicted, and absence of O2 requirements. For children that are not able to cooperate with PFTs, a pulse oximetry with or without exercise should be attempted. If neither test can be obtained it should be clearly stated in the physician's note.
• If recent mold infection (e.g. aspergillus) must have minimum of 30 days of therapy and responsive disease and be cleared by Infectious Disease
• Females of child bearing potential and sexually active males must agree to use adequate birth control during study treatment
• Voluntary written consent (adult or parent/guardian with presentation of the minor information sheet, if appropriate)
Exclusion Criteria:

• Pregnant or breast feeding. The agents used in this study include Pregnancy Category D: known to cause harm to a fetus. Females of childbearing potential must have a blood test or urine study within 14 days prior to registration to rule out pregnancy.
• Untreated active infection
• Active CNS disease
• Active HIV infection or known HIV positive serology
• Congenital bone marrow failure syndrome
• Previous irradiation that precludes the safe administration of an additional dose of 200 cGy of TBI
• CML in refractory blast crisis
• Intermediate or high grade NHL, mantle cell NHL, and Hodgkin disease that is progressive on salvage therapy. Stable disease is acceptable to move forward provided it is non-bulky.
• Multiple myeloma progressive on salvage chemotherapy
Drug: Allopurinol, Drug: Fludarabine, Drug: Cyclophosphamide, Drug: ATG, Radiation: TBI, Drug: Tacrolimus, Drug: MMF, Biological: Peripheral Blood Stem Cells, Biological: Related or Unrelated Bone Marrow Cells
Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Plasma Cell Leukemia, Myelodysplastic Syndromes, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, B-Cell Lymphoma, Follicular Lymphoma, Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia, Lymphoblastic Lymphoma, Burkitt's Lymphoma, Non-Hodgkin's Lymphoma, Multiple Myeloma, Myeloproliferative Syndromes, Hematological Diseases
AML, ALL, CML, MDS, CLL, SLL, NHL, Clinics and Surgery Center (CSC)
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Choline Supplementation as a Neurodevelopmental Intervention in Fetal Alcohol Spectrum Disorders Study

This is a double-blind randomized placebo-controlled clinical trial examining choline supplementation in 2-5 year old children with Fetal Alcohol Spectrum Disorders. Outcome measures are neurocognitive tests of memory, attention, and behavior.

Jeffrey Wozniak
All
2 Years to 5 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02735473
1506M74642
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Inclusion Criteria:

• Ages 2 years to 5 years of age
• Available parent or legal guardian capable of giving informed consent for participation.
• Documented prenatal alcohol exposure (self-report, social service records, or adoption records).
• Modified Institute of Medicine (IOM) criteria for Fetal Alcohol Syndrome (FAS), Partial Fetal Alcohol Syndrome (PFAS), or Alcohol-Related Neurodevelopmental Disorder (ARND) (Hoyme, May, et al., 2005).
Exclusion Criteria:

• Known history of a neurological condition (ex. epilepsy, traumatic brain injury)
• Known history of a medical condition known to affect brain function.
• Known history of other neurodevelopmental disorder (ex. autism, Down syndrome)
• Known history of very low birthweight (<1500 grams)
Drug: Choline bitartrate, Drug: Placebo
Fetal Alcohol Spectrum Disorders
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A Low Biologically Available Glucose and High Protein Diet for Treatment of Type 2 Diabetes Mellitus

Randomized clinical trial investigating the LoBAG diet versus a control diet for treatment of type 2 diabetes mellitus over a 3 month intervention period.

Anne Bantle
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT02717078
1601M82501
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Inclusion Criteria:
18 years of age or older, Diagnosis of type 2 diabetes mellitus, Hemoglobin A1c of 7.0-9.5%, Taking no medications for diabetes or taking metformin
Exclusion Criteria:
Type 1 diabetes mellitus, Treatment with insulin, BMI < 25 kg/m^2, Change in weight of >5 lbs over the past 3 months, Estimated glomerular filtration rate (GFR) < 60 ml/minute/1.73 m^2, Urine albumin > 300 mg/g creatinine, Anemia, Pregnancy or immediate plans to become pregnant, Current breastfeeding, Use of antibiotics in the past 3 months, Some dietary restrictions
Other: Diet Therapy
Type 2 Diabetes Mellitus, Diabetes & Endocrine, Microbiota, Prevention & Wellness
Carbohydrate, Diet, Nutrition, Protein, Type 2 diabetes mellitus, dstudy, diet, carbohydrate, protein, glucose, type 2 diabetes mellitus, DSTUDY
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MT2015-17 :Transplantation of Umbilical Cord Blood from Unrelated Donors in Patients With Hematological Diseases Using a Non-Myeloablative Preparative Regimen

The primary objective is to estimate the probability of grade II-IV acute GVHD at Day 100 after unrelated donor umbilical cord blood transplantation using a non-myeloablative preparative regimen along with Sirolimus/MMF for GVHD prophylaxis in persons with hematologic malignancies.

Margaret MacMillan, MD
All
up to 75 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02722668
1603M84843
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Inclusion Criteria:

• Age, Performance Status, and Graft Criteria
• <70 years of age with no matched 5/6 or 6/6 sibling donor - patients ≥ 70 and ≤ 75 years of age may be eligible if they have a Co-Morbidity score ≤ 2 (http://www.qxmd.com/calculate-online/hematology/hct-ci)
• Karnofsky score ≥ 70% (≥ 16 years) or Lansky score ≥ 50 (< 16 years)
• UCB graft selected according to current University of Minnesota umbilical cord blood graft selection algorithm
• Eligible Diseases All diseases listed below are advanced hematologic malignancies not curable by conventional chemotherapy. Responses to conventional treatment range from zero to 30% but are typically short lived.
• Acute Leukemias: Must be in remission by morphology (<5% blasts). Note cytogenetic relapse or persistent disease without morphologic relapse is acceptable. Also a small percentage of blasts that is equivocal between marrow regeneration vs. early relapse are acceptable provided there are no associated cytogenetic markers consistent with relapse.
• Acute Myeloid Leukemia (AML) and related precursor neoplasms: 2nd or greater complete remission (CR); first complete remission (CR1) in patients > 60 years old; CR1 in ≤ 60 years old that is NOT considered as favorable-risk. Favorable risk AML is defined as having one of the following:
• t(8,21) without cKIT mutation
• inv(16) or t(16;16) without cKIT mutation
• Normal karyotype with mutated NPM1 and wild type FLT-ITD
• Normal karyotype with double mutated CEBPA
• Acute prolymphocytic leukemia (APL) in first molecular remission at the end of consolidation
• Acute lymphoblastic leukemia (ALL)/lymphoma: second or greater CR; CR1 unable to tolerate consolidation chemotherapy due to chemotherapy-related toxicities; CR1 high-risk ALL. High risk ALL is defined as having one of the following:
• Evidence of high risk cytogenetics, e.g. t(9;22), t(1;19), t(4;11), other MLL rearrangements, IKZF1
• 30 years of age or older at diagnosis
• White blood cell counts of greater than 30,000/mcL (B-ALL) or greater than 100,000/mcL (T-ALL) at diagnosis
• CNS leukemia involvement during the course of disease
• Slow cytologic response (>10% lymphoblasts in bone marrow on Day 14 of induction therapy)
• Evidence of persistent immonophenotypic or molecular minimal residual disease (MRD) at the end of induction and consolidation therapy
• Biphenotypic/Undifferentiated/Prolymphocytic Leukemias in first or subsequent CR
• Chronic myelogenous leukemia in chronic or accelerated phase, or CML blast crisis in morphological remission (<5% blasts): Chronic phase patients must have failed at least two tyrosine kinase inhibitors, been intolerant to all available TKIs, or have T315I mutation.
• Myelodysplastic syndrome: IPSS INT-2 or High Risk; R-IPSS High or Very High; WHO classification: RAEB-1, RAEB-2; Severe Cytopenias: ANC < 0.8, Anemia or thrombocytopenia requiring transfusion; Poor or very poor risk cytogenetics based on IPSS or R-IPSS definitions; therapy-related MDS. Blasts must be < 5% by bone marrow aspirate morphology. If ≥5% blasts, patient requires chemotherapy for cytoreduction to <5% blasts prior to transplantation.
• MRD positive leukemia (AML, ALL or accelerated/blast phase CML). Selected patients in morphologic CR, but with positive immunophenotypic (flow cytometry) or molecular evidence of MRD may be eligible if recent chemotherapy has not resulted in MRD negative status.
• Leukemia or MDS in aplasia. These patients may be taken to transplant if after induction therapy they remain with aplastic bone marrow and no morphological or flow-cytometry evidence of disease ≥ 28 days post-therapy. These high risk patients will be analyzed separately.
• Burkitt's lymphoma in CR2 or subsequent CR
• Relapsed T-Cell Lymphoma that is chemotherapy sensitive in CR/PR that has failed or ineligible for an autologous transplant.
• Natural killer cell malignancies
• Large-cell lymphoma, Hodgkin lymphoma and multiple myeloma with chemotherapy sensitive disease who are ineligible for an autologous transplant.
• Relapsed Chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), marginal zone B-cell lymphoma, follicular lymphoma which have progressed within 12 months of achieving a partial or complete remission. Patients who had remissions lasting > 12 months, are eligible after at least two prior therapies. Patients with bulky disease should be considered for debulking chemotherapy before transplant. Patients with refractory disease are eligible, unless bulky disease and an estimated tumor doubling time of less than one month.
• Lymphoplasmacytic lymphoma, mantle-cell lymphoma, prolymphocytic leukemia are eligible after initial therapy if chemotherapy sensitive.
• Relapsed Multiple Myeloma that is chemotherapy sensitive and has failed or ineligible for an autologous transplant.
• Plasma Cell Leukemia after initial therapy if achieved at least in partial remission; or relapsed and achieved subsequent remission (CR/PR)
• Acquired Bone marrow failure syndromes, except for Fanconi anemia
• Myeloproliferative Neoplasms/Myelofibrosis
• Other Leukemia Subtypes: A major effort in the field of hematology is to identify patients who are of high risk for treatment failure so that patients can be appropriately stratified to either more (or less) intensive therapy. This effort is continually ongoing and retrospective studies identify new disease features or characteristics that are associated with treatment outcomes. Therefore, if new features are identified after the writing of this protocol, patients can be enrolled with the approval of two members of the study committee.
• Additional Criteria for Bulky Disease (lymphomas)
• If stable disease is best response, the largest residual nodal mass must < 5 cm (approximately)
• If response to previous therapy, the largest residual mass must represent a 50% reduction and be < 7.5 cm (approximately)
• Organ Function Criteria Adequate organ function is defined as:
• Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 40%. For children that are not able to cooperate with MUGA and echocardiography, such should be clearly stated in the physician's note.
• Pulmonary: DLCO, FEV1, FVC ≥ 40% predicted, and absence of O2 requirements. For children that are not able to cooperate with PFTs, a pulse oximetry with exercise should be attempted. If neither test can be obtained it should be clearly stated in the physician's note.
• Liver: Transaminases ≤ 5 x upper limit of normal (ULN) and total bilirubin ≤ 2.5 mg/dL except for patients with Gilbert's syndrome or hemolysis
• Renal: Creatinine ≤ 2.0 mg/dl (adults) and creatinine clearance ≥ 40 mL/min (pediatrics). Adults with a creatinine > 1.2 mg/dl or a history of renal dysfunction must have estimated creatinine clearance ≥ 40 ml/min/1.73m^2. Adequate performance status is defined as Karnofsky score ≥ 70% (≥ 16 years of age) or Lansky score ≥ 50 (pediatrics)
• Sexually active females of childbearing potential and males with partners of child-bearing potential must agree to use adequate birth control during study treatment.
• Voluntary written consent (adult or parent/guardian with presentation of the minor information sheet, if appropriate)
Exclusion Criteria:

• Pregnant or breast feeding. The agents used in this study include Pregnancy Category D: known to cause harm to a fetus. Females of childbearing potential must have a negative pregnancy test prior to starting therapy.
• Untreated active infection
• Active HIV infection or known HIV positive serology
• Less than 3 months since prior myeloablative transplant
• Evidence of progressive disease by imaging modalities or biopsy - persistent PET activity, though possibly related to lymphoma, is not an exclusion criterion in the absence of CT changes indicating progression.
• CML in blast crisis
• Large cell lymphoma, mantle cell lymphoma and Hodgkin disease that is progressing on salvage therapy.
• Active central nervous system malignancy
Drug: Fludarabine, Drug: Cyclophosphamide, Drug: MMF, Drug: Sirolimus, Radiation: TBI, Biological: Umbilical cord blood cell infusion, Biological: ATG
Acute Leukemia, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia/Lymphoma, Burkitt's Lymphoma, Natural Killer Cell Malignancies, Chronic Myelogenous Leukemia, Myelodysplastic Syndrome, Large-cell Lymphoma, Hodgkin Lymphoma, Multiple Myeloma, Relapsed Chronic Lymphocytic Leukemia, Relapsed Small Lymphocytic Lymphoma, Marginal Zone B-cell Lymphoma, Follicular Lymphoma, Lymphoplasmacytic Lymphoma, Mantle-cell Lymphoma, Prolymphocytic Leukemia, Bone Marrow Failure Syndromes, Myeloproliferative Neoplasms/Myelofibrosis, Biphenotypic/Undifferentiated/Prolymphocytic Leukemias, MRD Positive Leukemia, Leukemia or MDS in Aplasia, Relapsed T-Cell Lymphoma, Relapsed Multiple Myeloma, Plasma Cell Leukemia
Clinics and Surgery Center (CSC), AML, ALL, CML, CLL, SLL
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MT2014-30R: Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995 (PIDTC 6903)

Christen Ebens
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT02082353
1503M66962
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Inclusion Criteria:

• Participant Inclusion Criteria (Part 1 - Longitudinal Analysis)
• CGD Patients Undergoing Transplant 1995 to Present with Birth Year In or After 1988
• CGD Patients will be Defined by both Defective Neutrophil NADPH Oxidase Function and by Clinical History Consistent with CGD Patients must have both of: A functional assay demonstrating abnormal NADPH oxidase function (see A below); AND Clinical history consistent with CGD (see B below). ************************************************************************* Patients must have both "A" and "B": A. Function: Assays of NADPH Oxidase Function I. Dihydrorhodamine (DHR) Assay:
• Blood sample was obtained at a time when patient was clinically stable and not critically ill, with control samples performed simultaneously indicating a qualified assay; and
• Assay unequivocally demonstrates CGD with an stimulation index (SI) SI < 35 or equivalent. Assay report, including mean fluorescence intensity (MFI) from unstimulated and stimulated samples and gating strategy, must be de-identified and provided. OR II. Nitroblue Tetrazolium Oxidation Test (NBT): o Diagnostic of CGD (reported as reduced granulocyte oxidative response). Report must be de-identified and provided. AND B. Clinical History: One or More of the Following:
• Severe and/or recurrent infection (liver, perirectal or lung abscess; pneumonia; adenitis; or osteomyelitis) due to, for example, Staphylococcus aureus, Burkholderia sp, Serratia marcescens, non-albicans Candida sp, Aspergillus sp or other mold; or Nocardia sp or other deep tissue infection characteristic of CGD
• Sterile granulomatous disease in respiratory, gastrointestinal or urogenital tracts; or Crohn's disease-like colitis
• A family history consistent with either X-linked or autosomal recessive CGD In cases where either functional assay (A) or history (B) is equivocal, one or more of the following may be used to confirm a diagnosis of CGD: C. Absent or significantly reduced in expression or abnormal size of any of the 5 phox components (gp91 phox, p47 phox, p22 phox, p67phox, and p40phox) of NADPH oxidase, by either:
• Western blot
• Northern blot OR D. Mutation in a gene encoding one of the 5 phox components (gp91 phox, p47 phox, p22 phox, p67 phox, and p40 phox) of NADPH oxidase that is predictive of a decreased or absent oxidative burst. (Nonsense, frameshift, or previously described missense mutation associated with CGD). Molecular Diagnosis is Desirable In addition, molecular diagnosis (gene sequencing and expression analysis) of CGD is desirable and should be performed when possible.
• Further Characterization of Oxidase Level, Longitudinal Study, Prospective Cohort Patients who are to undergo transplantation during the study period must be further characterized as oxidase-null or oxidase positive by level of oxidase production by either:
• DHR assay stimulation Index: where SI ≤ 2.5 will be classified as oxidase-null CGD. Those with SI > 2.5 will be classified as oxidase positive CGD. A single validated test that is accepted by the PID-CGD Review Panel is adequate, but testing on two occasions for validation is desirable. OR
• Ferricytochrome C reduction assay of granulocytes with O2 < 2.3 nmoles /106 cells/h classified as oxidase-null CGD. A single validated test that is accepted by the PID-CGD Review Panel is adequate, but testing on two occasions for validation is desirable. OR o Genetic sequencing reporting a mutation that is unequivocally associated to absent oxidase production. (e.g. null mutations) will be classified as oxidase-null CGD (See discussion in Appendix I for how family history, genotype and CGD mutation information will be applied to assigning patients lacking any quantitative oxidase activity measurements to residual oxidase-null or residual oxidase-positive groups).
• Longitudinal Study, Retrospective Cohort Patients who have already been transplanted will be included regardless of whether further characterization by oxidase level (or genotype/mutation data) is possible or not.
• Non-Transplanted CGD Patients with Birth Year In or After 1988 A non-transplant (conventional therapy) group of CGD subjects will be enrolled in the longitudinal study. The non-transplant subjects will be selected from the potentially eligible (retrospective) patient cohort with diagnosis of CGD treated with conventional non-transplant therapy. Participating sites will enter their entire retrospective cohort of CGD patients having birth year in or after 1988 into the registration cohort for this protocol. Baseline for both non-transplant subjects and HCT subjects for the purpose of comparing survival will be the year of birth. However, for non-transplant subjects, many of the detailed analyses such as infection and autoimmune complication rates will be assessed in the year preceding the date of last contact.
• Participant Inclusion Criteria (Part 2 - Cross-Sectional Analysis) To participate in the Cross-Sectional Analysis, patients must have previously been enrolled into the Longitudinal Analysis of Protocol 6903. All transplanted subjects in the Cross-Sectional Analysis are surviving and shall have at least 3 years of follow-up post-transplant to be included. Non-transplanted CGD subjects will become eligible for consideration for the Cross-Sectional Analysis if they were eligible and enrolled in the retrospective cohort of the Longitudinal Analysis, and if/when they are > 3 years post-diagnosis of CGD. Provision of written informed consent will be required for inclusion in the Cross-Sectional Analysis.
Exclusion Criteria:

• Participant Exclusion Criteria (Longitudinal and Cross- Sectional Analyses)
• Presence of other primary immunodeficiency syndromes that do not meet the clinical and laboratory criteria for CGD.
• Rac2 Deficiency
• Myeloperoxidase Deficiency (MPO Deficiency)
• Glutathione deficiency
• Leukocyte adhesion deficiency syndrome
• Non-transplant subjects:
• The above exclusions pertain.
• In addition, non-transplant subjects will be excluded if the only assessment of oxidase function available is the nitroblue tetrazolium (NBT) test (a non-quantitative test).
Granulomatous Disease, Chronic
Granulomatous Disease, Chronic, Hematopoietic Stem Cell Transplantation (HSCT), bone marrow transplant (BMT), non-transplant, factors associated with best outcomes of transplant in CGD, Clinics and Surgery Center (CSC)
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Safety and Acceptability of Patient -administered Sedation During Mechanical Ventilation

The primary objective of the study is to assess the efficacy of patient controlled sedation (Self-management of sedative therapy) using dexmedetomidine to reduce anxiety, delirium incidence and duration of mechanical ventilation compared to usual sedation practices in mechanically ventilated subjects.

Craig Weinert
All
18 Years and over
Phase 2/Phase 3
This study is NOT accepting healthy volunteers
NCT02819141
1605M88241
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Inclusion Criteria:

• Subject is acutely mechanically ventilated during the current hospitalization.
• Subject is currently receiving a continuous intravenous infusion of a sedative/opioid medication(s) or has received at least one intravenous bolus dose of a sedative/opioid medication in the previous 24 hours (fentanyl, hydromorphone, ketamine, morphine, midazolam, diazepam, lorazepam, propofol, haloperidol, dexmedetomidine).
• Subject must pass pre-Patient-Controlled Sedation (PCS) screening test and be assessed Richmond Agitation-Sedation Scale (RASS) -2 to +1
• Subject Age ≥ 18 years
• Subject or their proxy is capable of providing informed consent
Exclusion Criteria:

• Aggressive ventilatory support or prone ventilation.
• Hypotension (systolic blood pressure < 85 mmHg) requiring a vasopressor at a dose greater than norepinephrine or epinephrine 0.15 mcg/kg/min or vasopressin > 2.4 units per hour. Subjects will be excluded if they require more than one continuous infusion of a catecholamine vasopressor medication simultaneously. Subjects will be excluded if the vasopressor dose was higher than norepinephrine or epinephrine 0.15 mcg/kg/min, vasopressin > 2.4 units per hour, phenylephrine >3 mcg/kg/min, dopamine >10 mcg/kg/min or dobutamine at any dose in the prior 6 hours. If dopamine is being used to increase heart rate, rather than as a vasopressor for hypotension, subject will be excluded.
• Second or third degree heart block or bradycardia (heart rate < 50 beats/min).
• Paralysis or other condition preventing the use of push button device
• Positive pregnancy test or lactation
• Acute hepatitis or liver failure (direct bilirubin >5 mg/dL)
• Acute stroke or uncontrolled seizures.
• Acute myocardial infarction within 48 hours prior to enrollment.
• Severe cognition or communication problems (such as coma, deafness without signing literacy, physician-documented dementia)
• Assessed RASS -3, -4, -5 or RASS +2,+3, +4
• Chronic ventilator support in place of residence prior to current hospitalization.
• Imminent extubation from mechanical ventilator support.
Drug: Dexmedetomidine
Critical Illness, Anxiety, Respiratory Failure
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Measurement of Glucose Metabolism in Humans: Effect of Recurrent Hypoglycemia on Hypothalamic GABA (GABA)

The purpose of this study is to determine the effects of altered glucose metabolism on the brain. For example, patients with long duration diabetes mellitus lose their ability to secrete the hormones necessary to protect them against hypoglycemia, which may be due to alterations in glucose availability to the human brain.

All
18 Years to 65 Years old
N/A
This study is also accepting healthy volunteers
NCT02829593
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Inclusion Criteria:

• well controlled type 1 diabetes (hemoglobin A1c <7.5%)
• age 18-65
• healthy controls
Exclusion Criteria:

• history of stroke, seizures, neurosurgical procedures, or arrhythmias
• use of drugs that can alter GABA metabolism (such as benzodiazepines).
• Subjects must also meet requirements for a study in the magnet, which includes weight less than 300 lbs and the absence of metallic substances in their body.
Other: hypoglycemia (low blood sugar) and MRI
Type 1 Diabetes, Healthy
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CAR-T Long Term Follow Up (LTFU) Study (PAVO)

The purpose of this study is to monitor all patients exposed to CD19 directed CAR T-cells (CD19 CART) for 15 years following last CD19 CART (e.g. CTL019) infusion to assess the risk of delayed adverse events (AEs), monitor for replication competent lentivirus (RCL) and assess long-term efficacy, including vector persistence.

Veronika Bachanova, MD
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT02445222
1512M81861
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Inclusion Criteria:

• All patients who have received a CAR-T therapy and completed or discontinued early from a Novartis sponsored treatment protocol that utilized CAR-T cells or from any CAR-T trial sponsored by the University of Pennsylvania with which Novartis has a contractual agreement to co-develop the CAR technology.
• Patients who have provided informed consent for the long term follow up study prior to their study participation .
Exclusion Criteria:

• There are no specific exclusion criteria for this study.
Genetic: Previously treated CAR-T patients
Long Term Safety of Patients Receiving CAR-T in an Eligible Clinical Trial or Managed Access Program
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Improving Outcome for Family Caregivers of Older Adults With Complex Conditions: The Adult Day Plus (ADS Plus) Program (ADS Plus)

To evaluate the efficacy of ADS Plus, a family-based intervention program, in 30 adult day service programs throughout the United States among 240 family caregivers over a 12-month evaluation period.

All
21 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT02927821
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Inclusion Criteria:
Caregivers are eligible to participate at time of intake at an Adult Day Service (ADS) if they:
• are initially enrolling their relative in one of the 30 participating ADS sites
• expect to use ADS for a minimum of 1 week for 6 months
• have primary responsibility for care of the ADS client
• speak English
• provided > 8 hours of assistance to client in past week
• have a telephone and are willing to participate in 4 telephone interviews (baseline, 3 month check-in; 6 and 12 month follow-ups)
• are 21 years of age or older (male or female).
Exclusion Criteria:
caregivers and older adult clients are not eligible if:
• they plan to move from the area within 6 months
• either caregiver or client has been hospitalized >3 times in past year
• either caregiver or client is in active treatment for a terminal illness or are in hospice
• caregiver is involved in other caregiver support services/trials.
Behavioral: ADS Plus
Stress
Adult Day Care Centers, Caregivers
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MT2016-15 :Reduced Intensity (RIC) Conditioning And Transplantation of HLA-Haploidentical Related Hematopoietic Cells (Haplo-HCT) For Patients With Hematologic Malignancies

This is a two stage phase II trial of HLA-haploidentical related hematopoietic cells transplant (Haplo-HCT) using a reduced intensity conditioning (cyclophosphamide, fludarabine, melphalan, total body irradiation) with modifications based on factors including age and comorbidities. Bone marrow is the donor graft source. This study is designed to estimate disease-free survival (DFS) at 1 year posttransplant. Since the goal is to estimate the DFS at a long-term time-point (1 year post-transplant), the design is a generalization of the Simon design. Rather than suspension of the trial for evaluation after stage 1, this design uses an optimal interim analysis for futility without suspension of accrual.

Najla El Jurdi
All
up to 75 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02988466
1610M96901
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Inclusion Criteria:

• Karnofsky performance status of ≥70% or Lansky play score ≥ 70%
• A related haploidentical bone marrow donor with up to 2 or 3 HLA locus-mismatches
• The donor and recipient must be HLA identical for at least one haplotype (using high resolution DNA based typing) at the following genetic loci: HLA-A, HLA-B, HLA-C, and HLA-DRB1.
• Adequate liver and renal function
• Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction ≥ 40%
• Diffusion capacity corrected (DLCOcorr) > 40% predicted, and absence of O2 requirements
• > 6 months after prior autologous transplant (if applicable)
• Agrees to use contraception during study treatment
• Voluntary written consent (adult or parent/guardian with presentation of the minor information sheet, if appropriate)
• Patients who are HIV+ must have undetectable viral load. All HIV+ patients must be evaluated by Infectious Disease (ID) and a HIV management plan establish prior to transplantation
Exclusion Criteria:

• < 70 years with an available 5-6/6 HLA-A, B, DRB1 matched sibling donor
• Pregnancy or breastfeeding
• Current active and uncontrolled serious infection
• Acute leukemia in morphologic relapse/persistent disease defined as > 5% blasts in normocellular bone marrow OR any % blasts if blasts have unique morphologic markers (e.g. Auer rods).
• CML in blast crisis
• Large cell lymphoma, mantle cell lymphoma and Hodgkin disease that is progressive on salvage therapy.
• stable non-bulky disease is acceptable.
• Active central nervous system malignancy Criteria For Donor Selection:
• Donors must be HLA-haploidentical relatives of the patient, defined as having a shared HLA haplotype between donor and patient at HLA-A, -B, -C, and -DRB1.
• Eligible donors (14-70 years old) include biological children, siblings or half siblings, or parents, able and willing to undergo bone marrow harvesting.
• For donors <18 years, the maximum recipient weight (actual body weight) should not exceed 1.25 times the donor weight (actual body weight)1 In addition, bone marrow product volume should be limited to 20 ml/kg donor weight for donors <18 years.
Biological: Haplo HCT <55 years old, Biological: Haplo HCT ≥55 years old, Drug: GVHD Prophylaxis, Biological: Haplo HCT ≥55 and < 65 years old, Biological: Haplo HCT ≥65 and ≤75 years old
Hematologic Malignancies
Acute Leukemias, Acute myeloid leukemia (AML), Acute lymphoblastic leukemia (ALL)/lymphoma, Biphenotypic/Undifferentiated/Prolymphocytic Leukemias, Myelodysplastic syndrome, Chronic myelogenous leukemia, Minimal Residual Disease (MRD) positive leukemia, Leukemia or Myelodysplastic Syndromes (MDS) in aplasia, Myeloproliferative neoplasms/myelofibrosis, Relapsed large-cell lymphoma, mantle-cell lymphoma and Hodgkin lymphoma, Burkitt's lymphoma, Relapsed T-cell lymphoma, Natural Killer cell malignancies, Relapsed chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), marginal zone B-cell lymphoma, follicular lymphoma, Lymphoplasmacytic lymphoma, Relapsed multiple myeloma, Bone marrow failure syndromes, Clinics and Surgery Center (CSC)
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Podocyturia - Predictor of Renal Dysfunction in Fabry Nephropathy

All
up to 90 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02994303
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Inclusion Criteria:

• Patients must have been diagnosed with Fabry disease
• Patients must be between the ages of 1 day-90 years
Exclusion Criteria:

• Fabry disease patients who have had a renal transplant
• Fabry disease patients who are, or have been, subjects in any investigational drug study
Fabry Disease
Fabry disease, Fabry nephropathy, end-stage renal disease, Anderson-Fabry disease, Fabry's disease
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Circuit-Based Deep Brain Stimulation for Parkinson's disease; Udall Clinical Core

The goal of this study is to provide comprehensive longitudinal assessments of a cohort of PD patients before, during, and after DBS surgery, including neurological, neurophysiological, and neuropsychological data.

Scott Cooper
All
22 Years to 85 Years old
N/A
This study is NOT accepting healthy volunteers
NCT03021031
1611M00822
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Inclusion Criteria:

• Age 22-85 years
• Diagnosis of Parkinson's disease
• Candidate for DBS
Exclusion Criteria:

• Other significant neurological disorder
• Diagnosis of dementia
• Prior history of stereotactic neurosurgery for treatment of Parkinson's disease, tremor, or dystonia.
• Pregnant women
Other: Observational
Parkinson Disease
Parkinson's Disease, Clinics and Surgery Center (CSC)
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PARTNER 3 Trial - Aortic Valve-in-Valve (P3-AVIV)

All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT03003299
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Inclusion Criteria:

• Failing surgical or transcatheter bioprosthetic valve in the aortic position demonstrating ≥ moderate stenosis and/or ≥ moderate insufficiency.
• Bioprosthetic valve with a true internal diameter (True ID) of 18.5 mm to 28.5 mm.
• NYHA Functional Class ≥ II.
• Heart Team agrees the patient is low to intermediate risk.
• Heart Team agrees valve implantation will likely benefit the patient.
• The study patient has been informed of the nature of the study, agrees to its provisions and has provided written informed consent.
Exclusion Criteria:

• Surgical or transcatheter valve in the mitral position (mitral rings are not an exclusion)
• Severe regurgitation (> 3+) or stenosis of any other valve
• Failing valve has moderate or severe paravalvular regurgitation
• Failing valve is unstable, rocking, or not structurally intact
• Increased risk of coronary obstruction by prosthetic leaflets of the failing valve.
• Increased risk of embolization of THV
• Known bioprosthetic valve with residual mean gradient > 20 mmHg at the end of the index procedure for implantation of the original valve
• Iliofemoral vessel characteristics that would preclude safe placement of the introducer sheath (Transfemoral)
• Anatomical characteristics that would preclude safe access to the ascending aorta (Transaortic)
• Anatomical characteristics that would preclude safe access to the apex (Transapical)
• Evidence of an acute myocardial infarction ≤ 30 days before enrollment
• Any therapeutic invasive cardiac procedure resulting in a permanent implant that is performed within 30 days prior to the index procedure. Implantation of a permanent pacemaker or implantable cardioverter defibrillator is not considered an exclusion.
• Patients with planned concomitant surgical or transcatheter ablation for Atrial Fibrillation
• Leukopenia, anemia, thrombocytopenia, history of bleeding diathesis or coagulopathy or hypercoagulable states
• Untreated clinically significant coronary artery disease requiring revascularization
• Hemodynamic or respiratory instability requiring inotropic support, mechanical ventilation, or mechanical heart assistance within 30 days of enrollment
• Emergency interventional/surgical procedures within 30 days prior to the procedure
• Any planned surgical, percutaneous coronary, or peripheral procedure to be performed within the 30-day follow-up from the procedure
• Hypertrophic cardiomyopathy with obstruction
• LVEF < 30%
• Cardiac imaging evidence of intracardiac mass, thrombus, or vegetation
• Inability to tolerate or condition precluding treatment with antithrombotic/anticoagulation therapy during or after the valve implant procedure
• Absolute contraindications or allergy to iodinated contrast that cannot be adequately treated with premedication
• Stroke or transient ischemic attack within 90 days of enrollment
• Symptomatic carotid or vertebral artery disease or successful treatment of carotid stenosis within 30 days of enrollment
• Renal insufficiency and/or renal replacement therapy at the time of screening
• Active bacterial endocarditis within 180 days of the procedure
• Patient refuses blood products
• Estimated life expectancy < 24 months
• Positive urine or serum pregnancy test in female subjects of childbearing potential
• Currently participating in an investigational drug or another device study
Device: Edwards SAPIEN 3/SAPIEN 3 Ultra THV
Aortic Stenosis, Aortic Stenosis, Severe
SAPIEN 3, PARTNER 3, cardiovascular disease, heart disease, aortic stenosis, SAVR, TAVR, failing surgical valve, failing bioprosthetic valve, failing valve, SAPIEN 3 Ultra
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Isakos: The impact of tibial tubercle-trochlear groove distance and patellar height on the outcome of isolated medialpatellofemoral ligament reconstruction (TT-TG Outcome)

Our primary objective is to evaluate the influence of increased TT-TG distance and patella height on recurrent dislocation risk and patient-reported outcomes following isolated MPFL reconstruction. This is a multi-center observational trial where subjects already consented to undergo medial patellofemoral ligament reconstruction will agree to be followed for two years to determine their outcome

Elizabeth Arendt
All
13 Years to 90 Years old
N/A
This study is NOT accepting healthy volunteers
NCT03076008
1604M86744
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Inclusion Criteria:

• MPFL Reconstruction
Exclusion Criteria:

• Trochleoplasty Required
• Prior ipsilateral knee surgery
• Iwano grade 2+
• CHondral injury requiring surgical treatment beyond debridement
• Major ligamentous injury to the knee
Other: Observational
Patellar Dislocation
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A Safety Extension Study of Trastuzumab Emtansine in Participants Previously Treated With Trastuzumab Emtansine Alone or in Combination With Other Anti-Cancer Therapy in One of the Parent Studies

Douglas Yee, MD
All
Not specified
Phase 2
This study is NOT accepting healthy volunteers
NCT00781612
1110M05522
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Inclusion Criteria:

• Completed single-agent trastuzumab emtansine or combination trastuzumab emtansine treatment in the parent study or who continue to receive single-agent trastuzumab emtansine or combination trastuzumab emtansine treatment at the time of the parent study closure and received the last study drug dose within the 6 weeks (42 days) prior to the first dose of study therapy on the extension study or Continue to receive treatment in the control arm of study BO21976/TDM4450g (NCT00679341) at the time of the parent study closure if the participant received the last dose of control arm study drug within the 6 weeks (42 days) prior to the first dose of control arm study therapy in the extension study
• Participants in the control arm from Study BO21976/TDM4450g whose disease progression has occurred during the transition interval between the parent study and this extension study may initiate trastuzumab emtansine treatment at the time of enrollment into study TDM4529g (NCT00781612)
• Expectation by the investigator that the participant may continue to benefit from additional single-agent trastuzumab emtansine or combination trastuzumab emtansine treatment or Expectation of the investigator that the participant may continue to benefit from control arm treatment as given in study BO21976/TDM4450g and at the time of disease progression may benefit from single-agent trastuzumab emtansine treatment
• Women of childbearing potential and men with partners of childbearing potential, must be willing to use a highly effective form of non-hormonal contraception or two effective forms of non-hormonal contraception by the participants and/or partner, and to continue the use of contraception for the duration of study treatment and for at least 5 months after the final dose of atezolizumab (if applicable) or 7 months after the final dose of trastuzumab emtansine or pertuzumab, whichever is later. Women must refrain from donating eggs during this same period
• Male participants whose partners are pregnant should use condoms for the duration of the pregnancy. Men must refrain from donating sperm during this same period
Exclusion Criteria:

• AEs leading to single-agent trastuzumab emtansine or combination trastuzumab emtansine treatment discontinuation in the parent study
• Ongoing SAEs from the parent study
• Progressive disease on single-agent trastuzumab emtansine or a trastuzumab emtansine-containing regimen during the parent study or before starting the extension study, with the exception of participants from study TDM4688g (NCT00943670) with early disease progression who went on to receive pertuzumab + trastuzumab emtansine treatment and have not experienced further disease progression on the combination regimen
• Peripheral neuropathy of Grade greater than or equal to (>/=) 3 per the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version
• 0, 4.0 or 5.0, as utilized in the parent study
• History of symptomatic congestive heart failure ([CHF]; New York Heart Association [NYHA] Classes II-IV), ventricular arrhythmia requiring treatment, current unstable angina, or history of myocardial infarction within 6 months prior to study entry
• Severe dyspnea at rest due to complications of advanced malignancy or current requirement for continuous oxygen therapy
• Current severe, uncontrolled systemic disease (for example [e.g.] clinically significant cardiovascular, pulmonary, or metabolic disease)
• Major surgical procedure or significant traumatic injury within 28 days prior to study entry or anticipation of the need for major surgery during the course of study treatment
• Current pregnancy or lactation
• History of receiving any investigational treatment or other systemic therapy directed at controlling cancer (e.g., chemotherapy, trastuzumab, etc.) since the participant's last study drug dose in the parent study
• History of hypersensitivity with previous trastuzumab emtansine or any agent used with trastuzumab emtansine in the parent study, precluding further dosing
• Assessed by the investigator to be unable or unwilling to comply with the requirements of the protocol
Drug: Docetaxel, Drug: Paclitaxel, Drug: Pertuzumab, Drug: Trastuzumab, Drug: Trastuzumab Emtansine, Drug: Atezolizumab
Neoplasm Metastasis
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Graded Motor Imagery for Women at Risk for Developing Type I CRPS following Distal Radius Fractures

Background: Distal radius fractures (DRF) account for nearly one-fifth of all fractures in older adults, and women experience them 5x as often as men. Most DRF occur with low impact injuries to the wrist with an outstretched hand, and are often managed via closed treatment and cast immobilization. Women sustaining a DRF are at risk for upper limb immobility, sensorimotor changes, edema and type I complex regional pain syndrome (CRPS). Since CRPS onset is likely influenced by alterations in the brain’s somatosensory region, a rehabilitation intervention, Graded Motor Imagery (GMI), aims to restore cortical representation, including sensory and motor function, of the affected limb. To date, there are no studies on the use of GMI in reducing risk of or preventing the onset of type I CRPS in women with DRF treated with cast immobilization. Due to a higher likelihood of women with this injury developing type I CRPS, it is important to early intervention is needed. Methods/Design: This article describes a six-week randomized comparative effectiveness trial, where the outcomes of a modified GMI program (mGMI) + standard of care (SOC) group (n=33) are compared to a SOC only control group (n=33). Immediately following cast immobilization, both groups participate in four 1-hour clinic-based sessions, and a home program for 10 minutes three times daily until cast removal. Blinded assessments occur within 1 week of cast immobilization (baseline), at three weeks post cast immbolization, cast removal, and at three months post cast removal. The primary outcomes are patient reported wrist/hand function and symptomology on the Patient Rated Wristand Hand Evaluation, McGill Pain Questionnaire, and Budapest CRPS Criteria. The secondary outcomes are grip strength, active range of motion as per goniometry, circumferential edema measurements, and joint position sense. Discussion: This study will investigate the early effects of mGMI + SOC hand therapy compared to SOC alone. We intend to investigate whether an intervention, specifically mGMI, used to treat preexisiting pain and motor dysfunction might also be used to mitigate these problems prior to their onset. If positive effects are observed, mGMI + SOC may be considered for incorporation into early rehabilitation program.

Corey McGee, PhD, MS, OTR/L, CHT
Female
55 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT02957240
1701M03721
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Inclusion Criteria:

• Women 55 years or older who have received closed treatment of distal radius fractures
Exclusion Criteria:

• Central nervous system disorders (e.g., Brain injury, Spinal Cord Injury, Parkinson's, Multiple Sclerosis)
• Surgical fixation of fracture
• Non english speaking
• Concomitant ipsilateral injuries (i..e., BBFF)
• Other injuries to the affected limb interfering with baseline affected limb function
• Cognitive disorders which would preclude from following the testing commands and home program participation
• Conditions of the contralateral upper limb which would result in painful and markedly limited active hand, wrist and forearm motion as this may impact the brain's ability to perceive safe and proficient movement during mirror therapy.
• Visual impairments resulting in the inability to participate in GMI components
Behavioral: Standard Care, Behavioral: Motor Representation Techniques
Musculoskeletal Pain, Fractures, Closed, Distal Radius Fracture, Complex Regional Pain Syndromes
Forearm [A01.378.800.585], Radius [A02.835.232.087.090.700], Motor Skills [F02.808.260], Task Performance and Analysis [F02.808.600], Casts, Surgical [E07.858.442.660.430.500], Splints [E07.858.690.725.430.750], motor representation techniques, mirror therapy, Women [M01.975], Clinics and Surgery Center (CSC)
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Use of Entresto (sacubitril/valsartan) for the treatment of peripheral arterial disease

The primary objective of this study is to assess the effects of 12 weeks of Entresto on the functional clinical parameters maximal walking duration, pain free walking duration and 6 minute walk test of patients with PAD. In addition, to determine the effects of Entresto on peak VO2, cardiac output and systemic vascular resistance.

Otto Sanchez
All
18 Years to 80 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02636283
1702M08001
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Inclusion Criteria:

• Subject with symptoms of intermittent claudication, such as exercise-induced pain, cramps, fatigue, or other equivalent discomfort, involving large muscle groups of the leg(s) (calf, thigh, buttocks), relieved by rest.
• Ankle-brachial index ≤ 0.90 acquired according to the American Heart Association guidelines.
• Highest ankle pressure reduced by at least 25 mm Hg after exercise compared to resting pressure (or loss of previously present Doppler signal for both the posterior tibial and anterior tibial arteries immediately after exercise if arteries were incompressible).
• Patients on medical treatment for PAD without significant improvement in intermittent claudication within the last 6 months.
Exclusion Criteria:

• Age < 18 and > 80 years.
• Patients with physician diagnosed chronic kidney disease or heart failure stage II or IV or unstable angina.
• Echocardiographic evidence of cardiomyopathies and pulmonary hypertension.
• Patients that have received cancer treatment within the last year (except skin cancer).
• Severe limitations in mobility due to osteomuscular disorders present at time of interview.
• Dementia or other mental disorders that prevent patients from following a research protocol present at time of interview
• Patients engaged in an exercise rehabilitation program within the past 6 months.
• Patients schedule to undergo an arterial revascularization procedure during the study or have undergone one within the past 6 months.
• Inconsistent maximal walking distance on the treadmill test.
Drug: Entresto, Drug: Placebo group
Peripheral Arterial Disease
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MT2016-11 :Autologous Stem Cell Transplant In Patients with Hodgkin Lymphoma (HL) and Non-Hodgkin Lymphomas (NHL)

Veronika Bachanova, MD
All
up to 75 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03125642
1611M99805
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Inclusion Criteria:

• Eligible Diseases
• Non-Hodgkin's Lymphoma (NHL)
• Patients with chemo-sensitive histologically confirmed NHL will be eligible for this treatment protocol contingent on histologic sub-classification.
• Patients in partial or complete remission following cell therapy will also be eligible.
• NHL patients with resistant or refractory lymphoma (no PR following up to three cycles of combination chemotherapy) will not be eligible for transplant in this trial.
• Lymphoblastic Lymphoma:
• All patients will be eligible in second or greater complete remission (CR) or first or subsequent partial remission (PR)
• Patients with any high-risk features will be eligible in first complete remission
• High risk features include: Stage IV, LDH >2 x upper limit of normal, ≥ 2 extranodal sites
• Mature B-cell Lymphoma
• Follicular Lymphoma and other indolent lymphoma in ≥ second CR2/PR2
• Diffuse Large B-Cell Lymphoma: in ≥ CR2 or ≥ PR1; a high intermediate or high IPI (≥ 2 for age-adjusted IPI or ≥3 for IPI) at diagnosis and double-hit or triple-hit lymphoma will be eligible in first CR; transformed lymphoma from FL (or other indolent lymphoma) or chronic lymphocytic leukemia will be eligible if chemosensitive and bone marrow is negative
• Mantle Cell Lymphoma: in first or greater CR or PR
• Burkitt's/Burkitt's like: all patients except localized lymphoma will be eligible any time after initial therapy (after achievement of first complete remission), or in partial remission if they fail to achieve CR; patients with localized (stage I or Ziegler stage A) will be eligible only if they fail to achieve CR1 or after relapse
• Mature T-Cell Lymphoma
• Chemosensitive T-cell lymphomas including Primary T-cell not otherwise specified angioimmunoblastic, and ALK-positive anaplastic large cell, will be eligible after initial therapy, whether or not CR is achieved.
• Mycosis fungoides/Sezary syndrome will be eligible in ≥CR2/PR2
• Hodgkin Lymphoma (HL)
• Patients with histologically proven HL will be eligible for transplantation after failing prior therapy.
• Patients with resistant disease (initial or at relapse): those who fail to achieve an objective partial response to three cycles of combination non-cross resistant chemotherapy will not be eligible for transplant in this trial.
• For stage I/II patients treated with primary chemotherapy-radiation, they must have failed (no CR or progression after CR) at least one salvage combination chemotherapy treatment regimen
• For advanced (stage III/IV) Hodgkin disease, patients must have failed an Adriamycin containing regimen (ABVD) or an alternative non-cross resistant regimen (e.g. MOPP)
• Patients with any high-risk features will also be eligible, including those who:
• fail to achieve complete remission with initial combination chemotherapy
• have bulky disease after initial therapy (chemotherapy or radiation) defined as residual mediastinal mass ≥ 5 cm or other residual mass ≥ 10 cm accompanied by other features of persisting disease (e.g., PET scan positive; high LDH; enlarging on serial x-rays or biopsy positive) will be eligible - if feasible, persistent disease should be proven by biopsy
• Patients should receive chemotherapy to attempt to achieve CR or minimal disease state for all patients pre-transplantation. The use of up to three cycles of non-cross resistant combination chemotherapy is advised.
• Residual areas of limited disease should be considered for radiotherapy after and not prior to transplantation.
• HIV positive patients who are otherwise eligible for this study may be enrolled if they meet the following requirements:
• Are seen in the infectious disease (ID)/HIV clinic prior to enrollment on study for the purpose of determining eligibility and for local coordination of HIV care during the peri-transplant period.
• Are on maximally active anti-HIV regimen to control disease as determined appropriate by the ID/HIV physicians. For the majority of patients, this will be a highly active anti-retroviral therapy (HAART)-type therapy including a protease inhibitor.
• CD4+ ≥ 50/µL
• HIV RNA viral load ≤ 100,000 copies per mL on each of samples 4 weeks apart. The most recent level must be within 30 days of enrollment.
• Performance Status: Karnofsky Performance Status ≥ 80% for patients ≥ 16 years of age or Lansky Play Score ≥ 80 for patients < 16 years of age. Note: if poor performance status is due to lymphoma - KPS ≥ 60% or LPS ≥ 60 is acceptable
• Organ Function
• No evidence of serious organ dysfunction that is not attributable to tumor including:
• Hematologic:
• hemoglobin > 8 gm/dL
• WBC > 2.5 x 109/L with an ANC > 1.5 x 109/L off G-CSF or GM-CSF for 10 days or Neulasta for 21 days
• platelets > 100 x 109/L without transfusion
• bone marrow cellularity of > 20% with <5% involvement with tumor
• Renal: GFR > 50 ml/min/1.73m2 or serum creatinine ≤ 2.5 x ULN for age
• Hepatic: no history of severe prior or ongoing chronic liver disease. Total bilirubin ≤ 2.0 mg/dl, AST and alkaline phosphatase <5x upper limit of normal
• Cardiac: free of symptoms of uncontrolled cardiac disease including unstable angina, decompensated congestive heart failure, or arrhythmia. The ejection fraction by gated cardiac blood flow scan (MUGA) or Echocardiogram must be >40%
• Pulmonary: no significant obstructive airways disease (FEV1 must be ≥ 50%) and must have acceptable diffusion capacity (corrected DLCO > 50% of predicted)
• Central Nervous System: Patients with a history of CNS involvement by lymphoma or with relapsed primary CNS lymphoma will be eligible for Cy/TBI arm. Patients with active CNS disease are eligible if they have completed a standard treatment for CNS lymphoma and have no evidence of progressive CNS disease at the time of enrollment
• Other Inclusion Criteria
• At least 4 weeks from previous chemotherapy; 6 weeks from nitrosoureas
• Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment
• Patients who are carriers of Hepatitis B will be included in this study
• Voluntary written consent
Exclusion Criteria:

• Pregnant or breastfeeding: Females of childbearing potential must have a blood test or urine study within 14 days prior to registration to rule out pregnancy
• Eligible for any higher priority transplant protocols
• Chemotherapy resistant disease
• Unrelated active infection
Drug: Etoposide, Drug: BCNU, Drug: AraC, Drug: Melphalan, Procedure: Peripheral blood stem cell transplantation, Biological: G-CSF, Drug: Cyclophosphamide, Radiation: Total Body Irradiation
Non-Hodgkin Lymphoma, Hodgkin Lymphoma
Lymphoblastic Lymphoma, Mature B-cell Lymphomas, Follicular Lymphoma, Diffuse Large B-Cell Lymphoma, Mantle Cell Lymphoma, Burkitt's/Burkitt's like, Mature T-Cell Lymphoma, Clinics and Surgery Center (CSC)
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RARE-OB-16: Rare CFTR Mutation Cell Collection Protocol (RARE) (RARE)

We are doing the RARE study to learn more about Cystic Fibrosis (CF). CF is caused by mutations in a gene that produces a protein called the cystic fibrosis transmembrane conductance regulator (CFTR). In people with CF, the CFTR does not function correctly. Medications are being developed to help the CFTR function better, but those medications mostly benefit people with common CFTR mutations. There are more than 1,900 mutations of the CF gene. Some of these mutations are rare and found only in a few people. The goal of this research study is to collect specimens (blood, nasal cells, rectal cells) from people with rare CFTR mutations. Another purpose of this study is to create induced pluripotent stem cells or iPS cells. “Pluripotent” stem cells are cells that can be changed into almost any cell type of the body (such as lung or intestine). They can be kept alive and stored indefinitely. There are different kinds of pluripotent stem cells. Inducted pluripotent stem cells can be created from many different kinds of specimens (such as blood, nasal cells, rectal cells). This is different from embryonic stem cells, which can only be derived from embryos. The specimens collected during this study and iPS cells created from them will be stored for use in future research to learn more about CF and study the effect of new medications. This could identify new medications that may help people with rare CFTR mutations.

Joanne Billings
All
12 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT03161808
1702M07621
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Inclusion Criteria:

• Male or female ≥ 12 years of age at time of consent
• Documentation of a CF diagnosis as evidenced by one or more clinical features consistent with CF and one or more of the following criteria (1. Sweat chloride ≥ 60 milliequivalents/Liter (mEq/L) by quantitative pilocarpineiontophoresis test (QPIT) OR upon permission of the RARE Investigator- Sponsors, 2. Two well-characterized mutations in the cystic fibrosis transmembrane conductive regulator (CFTR) gene,
• Abnormal nasal potential difference (NPD) (change in NPD in response to a low chloride solution and isoproterenol of lessthan -6.6 mV)
• Confirmed genotype of the current recruitment focus for certain target rare mutations. The initial recruitment focus will be CF patients who are homozygous for pre-mature stop codons. Operations Memos will detail any future current genotype targets.
• Written informed consent (and assent when applicable) obtained from participant or participant's legal representative and ability to comply with the requirements of the study.
• Willing to travel (if needed) to a regional study site for cell collection.
Exclusion Criteria:

• Presence of a medical condition, abnormality, or laboratory value(s) that in the opinion of the onsite principal investigator and/or collaborating gastroenterologist may compromise the quality of the data or place the subject at significant risk by undergoing the research related biopsy, including: Significantly diseased distal rectal/GI tissue that could place the participant at risk by participating in the study (as judged by the collaborating gastroenterologist, such as significant hemorrhoids, vascular abnormalities, colonic infection, radiation injury or history of radiation therapy to the rectum, prostate and/or pelvic area) Any of the following abnormal lab values at the study visit: i. Platelets < 50 x 103/µL ii. Hemoglobin < 10 gm/dL iii. Hematocrit < 30% iv. WBC > 20 x 103/µL v. Neutropenia (ANC < 1.5 x 103/µL) vi. Lymphopenia (absolute lymphocyte count < 1.5 x 103/µL) vii. PT/INR > 1.5 viii. Other bleeding diathesis
• Positive pregnancy test (for female of childbearing potential) at the study visit.
• Breastfeeding (if patient opts to use sedation).
• Current use of drugs with significant risks of compromising immunity (e.g. oral steroid use >20 mg/day) for >14 days prior to the rectal biopsy.
• History of organ transplant.
• Use of oral anticoagulant medications (e.g., chronic anticoagulant therapy such as warfarin or platelet inactivators such as aspirin) within seven days prior to rectal biopsy.
• Unable or unwilling to withhold use of oral anticoagulant medications (e.g., chronic anticoagulant therapy such as warfarin or platelet inactivators such as aspirin) within 7 days after rectal biopsy.
Cystic Fibrosis
Clinics and Surgery Center (CSC)
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Circuit-Based Deep Brain Stimulation for Parkinsons disease; Udall Project 1 Aim 2 and 3

Study objectives: -To characterize spontaneous and movement-related LFP changes in STN and GP in externalized patients under conditions that modulates the severity of tremor, bradykinesia and rigidity (off meds/off stim; on meds/off stim; off meds/on stim, on meds/on stim). -To characterize and compare the relative effect of different forms of closed loop stimulation (e.g., triggered at specific thresholds of low beta/HFO PAC or beta band activity) to standard isochronal high frequency DBS on motor signs and performance during movement.

Michael Park
All
22 Years to 85 Years old
N/A
This study is NOT accepting healthy volunteers
NCT03079037
1701M04144
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Inclusion Criteria:

• Diagnosis of idiopathic PD
• A history of a good response to levodopa (carbidopa/levodopa) defined as at least a 30% improvement in motor UPDRS score
• DBS surgery or IPG battery replacement at UMN is planned as part of routine clinical care.
Exclusion Criteria:

• Other significant neurological disorder
• History of dementia
• Prior history of stereotactic neurosurgery
• Patients with post-operative complications or adverse effects (e.g. ON stimulation dystonias) that affect patient safety or confound the experiment will be excluded from further study
• Pregnant women
Device: Stimulation
Parkinson Disease
Clinics and Surgery Center (CSC)
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