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BEGIN-OB-19: A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN) (BEGIN)
This is a study of highly effective CFTR modulators and their impact in children with CF on endocrine growth factors, the gut microbiome, respiratory microbiome, liver and pancreatic function, lung function, sweat chloride, and inflammatory markers.
Elissa Downs
Up to 18 years old
SITE00000975
Inclusion Criteria:
• For Part A: less than 5 years of age at the first study visit
• For Part B: participated in Part A OR less than 6 years of age at the first study visit, CFTR mutations consistent with FDA labeled indication of highly effective modulator therapy and physician intends to prescribe ivacaftor or elexacaftor/tezacaftor/ ivacaftor
• Documented diagnosis of Cystic Fibrosis (CF)
Exclusion Criteria:
• use of ivacaftor or elexacaftor/tezacaftor/ ivacaftor within the 180 days
• use of an investigational drug within 28 days prior to first study visit
• use of chronic oral corticosteroids within the 28 days prior to first study visit
Rare Diseases
Cystic Fibrosis
COG ACNS1833 - A Phase 3 Randomized Non-Inferiority Study of Carboplatin and Vincristine versus Selumetinib (NSC# 748727, IND# 77782) in Newly Diagnosed or Previously Untreated Low-Grade Glioma (LGG) not associated with BRAFV600E Mutations or Systemic Neurofibromatosis Type 1 (NF1)
The overall goal of this phase 3 non-inferiority study is to assess if selumetinib works as well as the standard treatment using carboplatin and vincristine (called CV) for subjects with low-grade glioma (LGG).
Christopher Moertel, MD
2 years to 21 years old
SITE00000799
Inclusion Criteria:
Patients must be >= 2 years and =< 21 years at the time of enrollment
Patients must have a body surface area (BSA) of >= 0.5 m^2 at enrollment
Patients must have non-neurofibromatosis type 1 (non-NF1) low-grade glioma (LGG) without a BRAFV600E mutation as confirmed by Rapid Central Pathology and Molecular Screening Reviews performed on APEC14B1 (NCT02402244) and that has not been treated with any modality besides surgery. Note: Patients may be newly-diagnosed OR previously diagnosed, and there is no required time frame between biopsy/surgery and treatment initiation.
Patients with residual tumor after resection or progressive tumor after initial diagnosis (with or without surgery) who have not received treatment (chemotherapy and/or radiation) are eligible
Patients must have two-dimensional measurable tumor >= 1 cm^2 to be eligible
Eligible histologies will include all tumors considered low-grade glioma or low-grade astrocytoma (World Health Organization [WHO] grade I and II) by 5th edition WHO classification of central nervous system (CNS) tumors with the exception of subependymal giant cell astrocytoma
Patients with metastatic disease or multiple independent primary LGG are eligible
Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 OR a serum creatinine based on age/gender as follows (performed within 7 days prior to enrollment):
Age: Maximum Serum Creatinine (mg/dL)
2 to < 6 years: 0.8 mg/dL (male); 0.8 mg/dL (female)
6 to < 10 years: 1 mg/dL (male); 1 mg/dL (female)
10 to < 13 years: 1.2 mg/dL (male); 1.2 mg/dL (female)
13 to < 16 years: 1.5 mg/dL (male); 1.4 mg/dL (female)
>= 16 years: 1.7 mg/dL (male); 1.4 mg/dL (female)
Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (performed within 7 days prior to enrollment) (children with a diagnosis of Gilbert's syndrome will be allowed on study regardless of their total and indirect [unconjugated] bilirubin levels as long as their direct [conjugated] bilirubin is < 3.1 mg/dL)
Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L (performed within 7 days prior to enrollment). For the purpose of this study, the ULN for SGPT is 45 U/L
Albumin >= 2 g/dL (performed within 7 days prior to enrollment)
Left ventricular ejection fraction (LVEF) >= 53% (or institutional normal; if the LVEF result is given as a range of values, then the upper value of the range will be used) by echocardiogram (performed within 4 weeks prior to enrollment)
Corrected QT (QTc) interval =< 450 msec by electrocardiography (EKG) (performed within 4 weeks prior to enrollment)
Absolute neutrophil count >= 1,000/uL (unsupported) (performed within 7 days prior to enrollment)
Platelets >= 100,000/uL (unsupported) (performed within 7 days prior to enrollment)
Hemoglobin >= 8 g/dL (may be supported) (performed within 7 days prior to enrollment)
Patients with a known seizure disorder should be stable and should not have experienced a significant increase in seizure frequency within 2 weeks prior to enrollment
Patients 2-17 years of age must have a blood pressure that is =< 95th percentile for age, height, and gender at the time of enrollment (with or without the use of anti-hypertensive medications)
Patients >= 18 years of age must have a blood pressure =< 130/80 mmHg at the time of enrollment (with or without the use of anti-hypertensive medications)
Note for patients of all ages: Adequate blood pressure can be achieved using medication for the treatment of hypertension
All patients must have ophthalmology toxicity assessments performed within 4 weeks prior to enrollment
For all patients, a magnetic resonance imaging (MRI) of the brain (with orbital cuts for optic pathway tumors) and/or spine (depending on the site(s) of primary disease) with and without contrast must be performed within 4 weeks prior to enrollment
Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, or 2. Use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age
Patients must have the ability to swallow whole capsules
All patients have signed an appropriate consent form and Health Insurance Portability and Accountability Act (HIPAA) authorization form (if applicable)
All patients and/or their parents or legal guardians must sign a written informed consent
All patients have been consented and enrolled on APEC14B1 (NCT02402244) followed by enrollment on the ACNS1833 Pre-Enrollment Eligibility Screening (Step 0) on the same day to complete the Rapid Central Review
All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Exclusion Criteria:
Patients must not have received any prior tumor-directed therapy including chemotherapy, radiation therapy, immunotherapy, or bone marrow transplant. Prior surgical intervention is permitted
Patients with a concurrent malignancy or history of treatment (other than surgery) for another tumor within the last year are ineligible
Patients with diffuse intrinsic pontine tumors as seen on MRI (> 2/3 of pons involvement on imaging) are not eligible even if biopsy reveals grade I/II histology
Patients may not be receiving any other investigational agents
Patients with any serious medical or psychiatric illness/condition, including substance use disorders or ophthalmological conditions, likely in the judgment of the investigator to interfere or limit compliance with study requirements/treatment
Patients who, in the opinion of the investigator, are not able to comply with the study procedures are not eligible
Female patients who are pregnant are not eligible since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
Lactating females who plan to breastfeed their infants are not eligible
Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation and for 12 weeks after stopping study therapy are not eligible.
Note: Women of child-bearing potential and males with sexual partners who are pregnant or who could become pregnant (i.e., women of child-bearing potential) should use effective methods of contraception for the duration of the study and for 12 weeks after stopping study therapy to avoid pregnancy and/or potential adverse effects on the developing embryo
Known genetic disorder that increases risk for coronary artery disease. Note: The presence of dyslipidemia in a family with a history of myocardial infarction is not in itself an exclusion unless there is a known genetic disorder documented
Symptomatic heart failure
New York Health Association (NYHA) class II-IV prior or current cardiomyopathy
Severe valvular heart disease
History of atrial fibrillation
Current or past history of central serous retinopathy
Current or past history of retinal vein occlusion or retinal detachment
Patients with uncontrolled glaucoma
If checking pressure is clinically indicated, patients with intraocular pressure (IOP) > 22 mmHg or ULN adjusted by age are not eligible
Supplementation with vitamin E greater than 100% of the daily recommended dose. Any multivitamin containing vitamin E must be stopped prior to study enrollment even if less than 100% of the daily recommended dosing for vitamin E
Surgery within 2 weeks prior to enrollment, with the exception of surgical biopsy, placement of a vascular access device or cerebral spinal fluid (CSF) diverting procedure such as endoscopic third ventriculostomy (ETV) and ventriculoperitoneal (VP) shunt.
Note: Patients must have healed from any prior surgery
Patients who have an uncontrolled infection are not eligibleMT2021-29: Evaluation of intravenous laronidase pharmacokinetics before and after hematopoietic cell transplantation in patients with mucopolysaccharidosis type IH
In this study, the researchers are collecting blood samples to learn more about laronidase treatment in children that receive a hematopoietic cell transplantation. The laronidase dose regimens used after a hematopoietic cell transplantation may differ from those administered before. This study will establish the basis for determining if there is a need to adjust laronidase dosing regimens after receiving a hematopoietic cell transplantation.
Silvia Illamola
Up to 18 years old
STUDY00016560
Inclusion Criteria:
• between 0 to 3 years of age
• meet protocol specific eligibility criteria for allogeneic HCT for MPS IH
• planning to receive laronidase both pre and post-transplant in an inpatient setting as part of standard-of-care treatment. Virtually all patients with MPSIH being considered for transplantation at the University of Minnesota are already receiving enzyme infusions, and it is standard practice to continue to give enzyme infusions to 8 weeks post-transplant. Therefore, participation will not modify the treatment course
Exclusion Criteria:
• patient's parent/ legal guardians are unable to provide informed consent.
Rare Diseases, Cancer
Hematopoietic Cell Transplantation
MT2015-29 : Myeloablative Allogeneic Hematopoietic Cell Transplantation Using a Related or Adult Unrelated Donor for the Treatment of Hematological Disorders
Punita Grover
Up to 60 years old
STUDY00001087
-
Inclusion Criteria:
Age: ≤ 60 years of age
Performance Status: Karnofsky ≥ 70%, Lansky play score ≥ 70
Consent: Voluntary written consent (adult or legally authorized representative; or parental/guardian)
Adequate Organ Function:
Renal: Creatinine <2x upper limit of normal. Patients above this limit must have creatinine clearance ≥ 40 ml/min/1.73m2 as determined by an age-appropriate method, such as cystatin C GFR.
Hepatic: Bilirubin, AST, alkaline phosphatase <4 times the upper limit of institutional normal
Pulmonary: Diffusion capacity of oxygen, corrected for hemoglobin, > 50% of predicted. For pediatric patients not able to undergo PFTs or diffusion testing: O2 sat of >95% on room air
Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 45%. For children not able to cooperate with MUGA or echocardiography, such should be clearly stated in the physician's documentation
HIV Status: HIV infection with undetectable viral load. All HIV+ patients must be evaluated by Infectious Disease (ID) and a HIV management plan establish prior to transplantation
Other Inclusion Criteria:
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.
Donor Availability: Patients considered for transplantation must have a sufficient graft as based on current criteria of the University of Minnesota Blood and Marrow Transplantation Program
Eligible Diseases and Status: Patients are eligible unless their treatment is to be guided by a higher priority protocol.
Acute Leukemias: Must be in remission by morphology (≤5% blasts). 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
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 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
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 achieve 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 one or more tyrosine kinase inhibitors.
Plasma Cell Leukemia after initial therapy, in patients who have achieved at least a partial remission
Myeloproliferative Neoplasms/Myelofibrosis, either primary as a result of polycythemia vera or essential thrombocythemia, with disease risk of intermediate or high-risk according to DIPSS criteria. Blasts must be <10% by bone marrow aspirate morphology.
Myelodysplasia (MDS) IPSS INT-2 or High Risk (i.e. RAEB, RAEBt) or Refractory Anemia with severe pancytopenia, transfusion dependence, or high risk cytogenetics or molecular features. Blasts must be < 10% by a representative 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 debulking chemotherapy before transplant.
Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia are eligible after initial therapy in CR1+ or PR1+.
Diffuse large Cell NHL > CR/> PR: Patients in CR/PR with initial short remission (<6 months) are eligible, or those who have failed/or are not eligible for autologous transplant.
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.
Juvenile myelomonocytic leukemia
Biphenotypic/Undifferentiated/Prolymphocytic Leukemias in first or subsequent CR.
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.
Natural Killer Cell Malignancies
Acquired Bone Marrow Failure Syndromes except for Fanconi Anemia or Dyskeratosis Congenita
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.
Exclusion Criteria:
Chemotherapy refractory large cell and high grade NHL (i.e., progressive disease after > 2 salvage regimens)
CML in blast crisis
Large cell lymphoma, mantle cell lymphoma and Hodgkin disease that is progressing on salvage therapy.
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.
Active central nervous system malignancy
if ≤ 18 years old, prior myeloablative transplant within the last 6 months. If >18 years old prior myeloablative allotransplant or autologous transplant
Active HIV infection or known HIV positive serology
active uncontrolled infection
Pregnant or breastfeeding. 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. Clinics and Surgery Center (CSC)
A multicenter, single arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who have Hb >=10 g/dL in response to anti-C5 antibody and switch to iptacopan
This is a multicenter, single-arm, open label trial, with iptacopan treatment for 24 weeks in adult PNH patients. Eligible participants must have a mean Hb ≥10 g/dL in response to a stable regimen with anti-C5 for at least 6 months and must be transfusion free for the same period.
Joan Beckman
STUDY00018020
Clinics and Surgery Center (CSC)
EPI-MINN: Targeting Cognition and Motivation in Coordinated Specialty Care for Early Psychosis: A National Comparison Study
This is a study enrolling patients receiving care at early psychosis specialty coordinated care programs across the United States. Participants will be randomly assigned to one of two groups. The first group will use two mobile apps, computerized brain training and a motivational smart phone app, for a period of 12 weeks. The other group will participate in their regular clinical care. There will be 4 study timepoints: intake, post-training, 6-month follow up, and 12-month follow up. There is also an optional interview about experiences of loneliness.
Sophia Vinogradov
Not specified
STUDY00018733
Inclusion Criteria:
• aged 15-40 inclusive
• enrolled in an early psychosis coordinated specialty care clinic (PI will determine this)
• in good physical health & stable psychiatric status
• fluent in spoken and written English
• have access to a smart phone (or other mobile device) and computer or tablet
Exclusion Criteria:
• participated in significant cognitive training programs within the last three years
• neurological disorder that may interfere with participation
• substance abuse disorder that would interfere with participation
• risk of suicidal behavior
Brain & Nervous System, Mental Health & Addiction
Psychosis, schizoaffective disorder, schizophrenia
Biologic Abatement and Capturing Kids Outcomes and Flare Frequency in Juvenile Spondyloarthritis (BACK OFF JSpA) (BACK-OFF JSpA)
This study is enrolling participants who have been diagnosed with juvenile spondyloarthritis, are taking a tumor necrosis factor inhibitor (TNFi) and have reached a clinically inactive disease state for a minimum of six months. Researchers want to know if children who have maintained inactive disease for at least 6 months can maintain quiet disease without taking their medication as frequently or stop the TNFi therapy. Quiet disease means that disease related symptoms are not active or being experienced in the patient. Researchers also want to know the safest method to bring patients off medication. If a flare does occur during therapy reduction, researchers want to find out whether they can predict when a flare is most likely to happen, and how quickly an inactive disease state can be recaptured.
Colleen Correll
Not specified
SITE00001260
Inclusion Criteria:
• age 8 to 21 years
• juvenile SpA diagnosis symptom with symptoms starting before their 16th birthday
• currently taking one of the following therapies (Adalimumab, Certolizumab, Etanercept, Golimumab, Infliximab) at standard doses
• have reached a clinically inactive state for at least 6 months
• English speaking or Spanish speaking
• willing to taper off medications
Exclusion Criteria:
• History of inflammatory bowel disease or history of uveitis
• psoriasis that started before TNFi therapy or psoriasis that started after TNFi therapy and has required more than topical therapy for control
Arthritis & Rheumatic Diseases, Children's Health, Rare Diseases
arthritis, Juvenile Spondyloarthritis
An Observational Registry of Abatacept in Patients with Juvenile Idiopathic Arthritis (BMS Protocol IM101240)
The objective of this study is to create an international registry with long-term follow-up to characterize and evaluate the safety of abatacept in juvenile idiopathic arthritis (JIA). The primary objective of the JIA registry is to describe the long-term safety of abatacept treatment for JIA by quantifying the incidence rates of serious infections, autoimmune disorders, and malignancies.
Colleen Correll
Up to 18 years old
1403M48721
Inclusion Criteria:
• less than 18 years of age (unless currently or previously enrolled in an abatacept clinical trial and received abatacept)
• diagnosis of Juvenile Idiopathic Arthritis (JIA)
• currently receiving abatacept per treating physician's decision or received abatacept in a clinical trial
Exclusion Criteria:
• pregnant or breast feeding
• history of cancer unless cancer free for at least 5 years
• any serious acute or chronic medical condition other than JIA
• history of frequently missing clinic appointments
Arthritis & Rheumatic Diseases, Children's Health
Abatacept, Juvenile Idiopathic Arthritis
COG ACNS1831 - A Phase 3 Randomized Study of Selumetinib (IND # 77782) versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)
Christopher Moertel, MD
2 years to 21 years old
SITE00000735
Inclusion Criteria:
Patients must be >= 2 years and =< 21 years at the time of enrollment
Patients must have a body surface area (BSA) of >= 0.5 m^2 at enrollment
Patients must have neurofibromatosis type 1 (NF1) based on clinical criteria and/or germline genetic testing
Patients must be newly diagnosed or have previously diagnosed NF-1 associated LGG that has not been treated with any modality other than surgery
For patients with optic pathway gliomas (OPGs):
Newly-diagnosed patients with OPG are eligible if there are neurologic symptoms (including visual dysfunction, as defined below) or other exam findings associated with the tumor
Previously-diagnosed patients with OPG are eligible if they have new or worsening neurologic symptoms (including visual dysfunction, as defined below) or have tumor growth
For both newly-diagnosed and previously-diagnosed OPG, the patient may be eligible, irrespective of whether there has been tumor growth or other neurological symptoms or worsening, if they meet at least one of the following visual criteria:
Visual worsening, defined as worsening of visual acuity (VA) or visual fields (VF) documented within the past year (by examination or history); OR
Significant visual dysfunction (defined as VA worse than normal for age by 0.6 logMAR [20/80, 6/24, or 2.5/10] or more in one or both eyes)
For patients with LGG in other locations (i.e., not OPGs):
Newly-diagnosed patients with LGG are eligible if there are neurologic symptoms or other exam findings associated with the tumor
NOTE: Newly-diagnosed patients with LGG without associated neurologic symptoms or exam findings are not eligible
Previously-diagnosed patients with LGG are eligible if they have new or worsening neurologic symptoms or have tumor growth
Although not required, if a biopsy/tumor resection is performed, eligible histologies will include all tumors considered LGG or low-grade astrocytoma (World Health Organization [WHO] grade I and II) by 5th edition WHO classification of central nervous system (CNS) tumors with the exception of subependymal giant cell astrocytoma
Patients must have two-dimensional measurable tumor >= 1 cm^2
Patients with metastatic disease or multiple independent primary LGGs are allowed on study
Creatinine clearance or radioisotope glomerular filtration Rate (GFR) >= 70 mL/min/1.73 m^2 OR a serum creatinine based on age/gender (within 7 days prior to enrollment) as follows:
Age; maximum serum creatinine (mg/dL)
2 to < 6 years; 0.8 (male) and 0.8 (female)
6 to < 10 years; 1 (male) and 1 (female)
10 to < 13 years; 1.2 (male) and 1.2 (female)
13 to < 16 years; 1.5 (male) and 1.4 (female)
>= 16 years; 1.7 (male) and 1.4 (female)
Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment) (children with a diagnosis of Gilbert's syndrome will be allowed on study regardless of their total and indirect [unconjugated] bilirubin levels as long as their direct [conjugated] bilirubin is < 3.1 mg/dL)
Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 3 x upper limit of normal (ULN) = 135 U/L (within 7 days prior to enrollment). For the purpose of this study, the ULN for SGPT is 45 U/L
Albumin >= 2 g/dL (within 7 days prior to enrollment)
Left ventricular ejection fraction (LVEF) >= 53% (or institutional normal; if the LVEF result is given as a range of values, then the upper value of the range will be used) by echocardiogram (within 4 weeks prior to enrollment)
Corrected QT (QTc) interval =< 450 msec by electrocardiography (EKG) (within 4 weeks prior to enrollment)
Absolute neutrophil count >= 1,000/uL (unsupported) (within 7 days prior to enrollment)
Platelets >= 100,000/uL (unsupported) (within 7 days prior to enrollment)
Hemoglobin >= 8 g/dL (may be supported) (within 7 days prior to enrollment)
Patients with a known seizure disorder should be stable and should have not experienced a significant increase in seizure frequency within 2 weeks prior to enrollment
Patients 2-17 years of age must have a blood pressure that is =< 95th percentile for age, height, and gender at the time of enrollment. Patients >= 18 years of age must have a blood pressure =< 130/80 mmHg at the time of enrollment (with or without the use of antihypertensive medications).
Note: Adequate blood pressure can be achieved using medication for the treatment of hypertension
All patients must have ophthalmology toxicity assessments performed within 4 weeks prior to enrollment
For all patients, an MRI of the brain (with orbital cuts for optic pathway tumors) and/or spine (depending on the site(s) of primary disease) with and without contrast must be performed within 4 weeks prior to enrollment
For patients who undergo a surgery on the target tumor (not required), a pre- and post-operative* MRI of the brain (with orbital cuts for optic pathway tumors) or spine (depending on the site(s) of primary disease) with and without contrast must also be performed within 4 weeks prior to enrollment
The post-operative MRIs should be performed ideally within 48 hours after surgery if possible
Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, or 2. Use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age
Patients must have the ability to swallow whole capsules
Patients must have receptive and expressive language skills in English or Spanish to complete the quality of life (QOL) and neurocognitive assessments
All patients and/or their parents or legal guardians must sign a written informed consent.
All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met.
Exclusion Criteria:
Patients must not have received any prior tumor-directed therapy including chemotherapy, radiation therapy, immunotherapy, or bone marrow transplant. Prior surgical intervention is permitted
Patients with a concurrent malignancy or history of treatment (other than surgery) for another tumor within the last year are ineligible
Patients may not be receiving any other investigational agents
Patients with any serious medical or psychiatric illness/ condition, including substance use disorders likely in the judgement of the investigator to interfere or limit compliance with study requirements/treatment are not eligible
Patients who, in the opinion of the investigator, are not able to comply with the study procedures are not eligible
Female patients who are pregnant are not eligible since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
Lactating females who plan to breastfeed their infants are not eligible
Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation and for 12 weeks after stopping study therapy are not eligible
Note: Women of child-bearing potential and males with sexual partners who are pregnant or who could become pregnant (i.e., women of child-bearing potential) should use effective methods of contraception for the duration of the study and for 12 weeks after stopping study therapy to avoid pregnancy and/or potential adverse effects on the developing embryo
Cardiac conditions:
Known genetic disorder that increases risk for coronary artery disease. Note: The presence of dyslipidemia in a family with a history of myocardial infarction is not in itself an exclusion unless there is a known genetic disorder documented
Symptomatic heart failure
New York Heart Association (NYHA) class II-IV prior or current cardiomyopathy
Severe valvular heart disease
History of atrial fibrillation
Ophthalmologic conditions:
Current or past history of central serous retinopathy
Current or past history of retinal vein occlusion or retinal detachment
Patients with uncontrolled glaucoma
If checking pressure is clinically indicated, patients with intraocular pressure (IOP) > 22 mmHg or ULN adjusted by age are not eligible
Ophthalmological findings secondary to long-standing optic pathway glioma (such as visual loss, optic nerve pallor, or strabismus) or longstanding orbito-temporal plexiform neurofibroma (PN), such as visual loss, strabismus) will NOT be considered a significant abnormality for the purposes of the study
Treatments and/or medications patient is receiving that would make her/him ineligible, such as:
Supplementation with vitamin E greater than 100% of the daily recommended dose. Any multivitamin containing vitamin E must be stopped prior to study enrollment even if less than 100% of the daily recommended dosing for vitamin E
Surgery within 2 weeks prior to enrollment, with the exception of surgical placement for vascular access or cerebrospinal fluid (CSF) diverting procedures such as endoscopic third ventriculostomy (ETV) and ventriculo-peritoneal (VP) shunt.
Note: Patients must have healed from any prior surgery prior to enrollment
Patients who have an uncontrolled infection are not eligibleMT2005-25 Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia
To determine the incidence of engraftment (defined as achieving donor derived neutrophil count >500/uL by day 42) in young children with leukemia or myelodysplastic syndrome undergoing a partially matched single unit umbilical cord blood transplant (UCBT) after a myeloablative preparative regimen consisting of busulfan, melphalan and fludarabine.
Christen Ebens
Up to 3 years old
0511M77206
Inclusion Criteria:
Matched sibling donor (HLA 8/8), if available, or a unrelated partially HLA matched single unit based on the following priority:
1st priority: 4/6 matched unit, cell dose >5 x 10-7 nucleated cells/kg
2nd priority: 5/6 matched unit, cell dose > 4 x 10-7 nucleated cells/kg
3rd priority: 6/6 matched unit, cell dose > 3 x 10-7 nucleated cells/kg
Patients aged ≤ 3 years at diagnosis (not age of transplant) with hematological malignancy as detailed below:
Acute myeloid leukemia: high risk CR1 as evidenced by:
High risk cytogenetics t(4;11) or other MLL rearrangements; chromosome 5, 7, or 19 abnormalities; complex karyotype (>5 distinct changes); ≥ 2 cycles to obtain complete response (CR); CR2 or higher; Preceding myelodysplastic syndrome (MDS); All patients must be in CR or early relapse (i.e., <15% blasts in BM).
Acute lymphocytic leukemia: high risk CR1 as evidenced by: High-risk cytogenetic: t(4;11) or other MLL rearrangements; hypodiploid; t(9;22); >1 cycle to obtain CR; CR2 or higher; 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%.
Myelodysplasia (MDS) IPSS Int-2 or High risk (i.e. RAEB, RAEBt) or refractory anemia with severe pancytopenia or high risk cytogenetics. Blasts must be < 10% by a representative bone marrow aspirate morphology.
Persistent or rising minimal residual disease (MRD) after standard chemotherapy regimens: Patients with evidence of minimal residual disease at the completion of therapy or evidence of rising MRD while on therapy. MRD will be defined by either flow cytometry (>0.1% residual cells in the blast gate with immune phenotype of original leukemic clone), by molecular techniques (PCR or FISH) or conventional cytogenetics (g-banding).
New Leukemia Subtypes: A major effort in the field of pediatric 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 high risk features are identified after the writing of this protocol, patients can be enrolled with the approval of two members of the study committee.
Recipients must have a Lansky score ≥ 50% and have acceptable organ function defined as:
Renal: glomerial filtration rate > 60ml/min/1.73m^2
Hepatic: bilirubin, AST/ALT, ALP < 5 x upper limit of normal,
Pulmonary function: oxygen saturation >92%
Cardiac: left ventricular ejection fraction > 45%.
Voluntary written informed consent before performance of any study-related procedure not part of normal medical care.
Exclusion Criteria:
Active infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days).
History of HIV infection or known positive serology
Myeloablative transplant within the last 6 months.
Evidence of active extramedullary disease (including central nervous system leukemia).COG AGCT1532 - A Randomized Phase 3 Trial of Accelerated versus Standard BEP Chemotherapy for Patients with Intermediate and Poor-risk Metastatic Germ Cell Tumors (P3BEP)
This trial is an open label, randomized, stratified 2-arm Australian-led multicenter phase 3 clinical trial undertaken in two stages. Participants (age >= 11 years and <= 45 years) with intermediate and poor-risk metastatic germ cell tumors will be randomized into either a “standard BEP” group or “accelerated BEP” group. Participants will be assigned to the two treatment arms in a 1:1 ratio and evaluated weekly, and then for 5 years after completing the study to assess the long-term effects of the chemotherapy. Bleomycin, Etoposide, Cisplatin (BEP) administered 3-weekly x 4 remains standard 1st line chemotherapy for intermediate- and poor-risk metastatic germ cell tumours (GCTs). BEP is accelerated by cycling Cisplatin and etoposide 2-weekly instead of 3-weekly. The aim of this study is to determine if accelerated BEP is superior to standard BEP as first-line chemotherapy for intermediate and poor risk metastatic GCTs.
Emily Greengard
Not specified
SITE00000331
Inclusion Criteria:
• between 11 years and 45 years old
• confirmed germ cell tumour (non-seminoma or seminoma) OR Exceptionally raised tumour markers (AFP equal or greater than 1000ng/mL and/or HCG equal or greater than 5000 IU/L)
• primary arising in testis, ovary, retro-peritoneum, or mediastinum
• metastatic disease or non-testicular primary
• see link to clinicaltrials.gov for completed Inclusion/Exclusion criteria
Exclusion Criteria:
• other primary malignancy (EXCEPT adequately treated non-melanomatous carcinoma of the skin, germ cell tumour, or other malignancy treated at least 5 years previously with no evidence of recurrence)
• significant cardiac disease resulting in inability to tolerate IV fluid hydration for cisplatin
• peripheral neuropathy equal or greater than grade 2 or clinically significant sensorineural hearing loss or tinnitus
Cancer
Germ Cell Tumor
PEPN2113: A Phase 1 and pharmacokinetic study of Uproleselan (GMI-1271, IND #139758, NSC #801708) in combination with fludarabine and cytarabine for patients with acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that expresses E-selectin ligand on the cell membrane and is in second or greater relapse or that is refractory to relapse therapy
A Phase 1 and pharmacokinetic study of Uproleselan (GMI-1271, IND #139758, NSC #801708) in combination with fludarabine and cytarabine for patients with acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that expresses E-selectin ligand on the cell membrane and is in second or greater relapse or that is refractory to relapse therapy
Emily Greengard
Up to 18 years old
SITE00001697
Inclusion Criteria:
• patient must be enrolled on APAL2020SC (NCT04726241)
• patients must be between 1 and 17 years of age at the time of study enrollment
• patients, with or without Down syndrome (DS), and with de novo acute myeloid leukemia, therapy-related acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that expresses E-selectin ligand on the cell membrane
• second or greater relapse or refractory AML OR refractory myelodysplastic syndrome (MDS) OR mixed phenotype acute leukemia (MPAL)
• see link to clinicaltrials.gov for complete criteria
Exclusion Criteria:
• patients who are currently receiving another investigational drug are not eligible
• patients who are currently receiving other anti-cancer agents are not eligible except patients receiving hydroxyurea, which may be continued until 24 hours prior to start of protocol therapy
• study staff will review additional exclusion criteria
Cancer, Cancer
AML, Myelodysplastic Syndrome Post Cytotoxic Therapy, Recurrent Acute Myeloid Leukemia
Determinants of Renal Structural Responses to Enzyme Replacement Therapy (ERT) in Fabry Disease Study (LDN6702)
The effect of enzyme replacement therapy on how well your kidneys are responding to enzyme replacement therapy (ERT) is not clear from blood and urine tests alone, but may be more clear in comparisons of kidney biopsies performed before and some time after ERT has been initiated, and this is what we are focusing our study efforts on. The purpose of this study is to obtain your permission to allow us to study the kidney biopsy tissues (collected for medical reasons) after the regular routine studies have been completed. Through our special research measurements and additional study, we hope to be able to see and measure very specific changes in the kidney tissues from Fabry patients taking ERT. We also hope that through these studies of what happens within the kidney before and after starting ERT, we are able to reveal valuable information about the importance of factors like your age that you started ERT, the amount or dosage of ERT, and any differences seen between males and females.
Michael Mauer
Not specified
1205M14901
Inclusion Criteria:
• diagnosed with Fabry disease and a clinical decision has been made to obtain a kidney biopsy, a GFR, and urinary albumin studies
• have previously completed clinical trials which included measures of renal function and renal biopsies
Exclusion Criteria:
• serum creatinine more than 2.5 mg/dL
• known to have a renal disease other than Fabry
Kidney, Prostate & Urinary, Rare Diseases
Fabry disease, Kidney disease, Renal disease
COG AGCT1531 - A Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients with Germ Cell Tumors
This partially randomized phase III trial studies how well active surveillance, bleomycin, carboplatin, etoposide, or cisplatin work in treating pediatric and adult patients with germ cell tumors. Active surveillance may help doctors to monitor subjects with low risk germ cell tumors after their tumor is removed. Drugs used in chemotherapy, such as bleomycin, carboplatin, etoposide, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
Emily Greengard
Not specified
SITE00000295
Inclusion Criteria:
• newly diagnosed with a Stage I germ cell tumor or metastatic germ cell tumor
• see link to clinicaltrials.gov for detailed inclusion criteria
Exclusion Criteria:
• patients must have had no prior systemic therapy for the current cancer diagnosis
• patients must have had no prior radiation therapy (exception of CNS irradiation of brain metastases for standard risk 1 patients)
• female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs
• lactating females who plan to breastfeed their infants
• there are additional exclusion criteria (study staff will review)
Cancer
Germ Cell Tumor, Malignant Germ Cell Tumor
COG AALL2121: A Phase 2 study of SNDX-5613 in combination with chemotherapy for patients with relapsed or refractory KMT2A-rearranged infant leukemia
This phase II trial tests the safety and best dose of revumenib when given together with chemotherapy, and how well the treatment regimen works for infants and young children with leukemia that has come back (relapsed) or does not respond to treatment (refractory) and is associated with a KMT2A (MLL) gene rearrangement (KMT2A-R). Revumenib is an oral medicine that directly targets the changes that occur in a cell with a KMT2A rearrangement and has been shown to specifically kill these leukemia cells in test tubes and animals. Drugs used in chemotherapy, such as vincristine, prednisone, asparaginase, fludarabine and cytarabine work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. This trial is being done to find out if the combination of revumenib and chemotherapy may help to treat the cancer cells better than either treatment alone.
Peter Gordon
Up to 18 years old
STUDY00021176
Inclusion Criteria:
• Age: Patients must be 1 month to less than 6 years old at the time of study enrollment and must have had initial diagnosis of leukemia less than 2 years old.
• Diagnosis: Patients must have KMT2A-rearranged acute lymphoblastic leukemia (ALL), acute leukemia of ambiguous lineage (ALAL), or mixed phenotype acute leukemia (MPAL), which is determined to be refractory or in first marrow relapse.
• Disease status: First relapse, refractory or failure to achieve remission
Exclusion Criteria:
• Patients with isolated extramedullary leukemia.
• Patients diagnosed with Down syndrome.
Cancer
ALAL, ALL, leukemia, MPAL, relapse
A Phase II, Open Label, Two Arm Study of Therapeutic Iobenguane (131I) as Single Agent or in Combination with Vorinostat for Recurrent or Progressive High-Risk Neuroblastoma Subjects (OPTIMUM Trial) Protocol Number: MIBG 2014-01 (OPTIMUM)
This will be a Phase II, two-arm, nonrandomized, non-comparative, open-label study in participants ≥ 1 year of age with iobenguane avid, recurrent or progressive high-risk neuroblastoma. Participants not eligible for vorinostat treatment may receive 131I-MIBG as monotherapy.
Emily Greengard
1 year and over
STUDY00005792
Inclusion Criteria:
Subjects with a diagnosis of iobenguane avid, high-risk neuroblastoma based on Revised INRC criteria at the time of study enrollment with recurrent or progressive disease at any time prior to enrollment, regardless of overall response to frontline therapy, where frontline therapy includes a minimum of 4 cycles of induction therapy at any time prior to enrollment.
May have had prior 131I-MIBG therapy, provided:
It has been at least 6 months from the date of last 131I-MIBG ;
Response was other than progressive disease on first restaging after 131I-MIBG ;
Prior 131I-MIBG was given as monotherapy and not in combination with systemic anticancer agents;
Cumulative lifetime dose of 131I-MIBG at enrollment does not exceed 18 mCi/kg.
All soft tissue lesions identified on CT/MRI scans must be iobenguane avid lesions on an (123I)-iobenguane scan, or
any progressive non-iobenguane avid lesion is proven by biopsy to be a non-neuroblastoma lesion.
any other non-avid lesion is comprised of a fibrotic or scarred mass as shown by routine imaging and confirmed by the investigator.
Adequate cryopreserved autologous peripheral blood stem cells or bone marrow (at least 2 aliquots of 2.0 × 10exp6 CD34/kg at the time of study enrollment).
If a male, must agree to use an adequate contraception method as deemed appropriate by the Investigator (e.g., vasectomy, condoms) or partner using effective contraception and to not donate sperm during the study and for 90 days after receiving the last dose of study drug.
If a female of childbearing potential, have a negative serum pregnancy test result prior to each dosing and, if sexually active, be practicing an effective method of birth control [e.g., intrauterine device, double-barrier method (i.e., diaphragm, or a cervical cap) with intravaginal spermicidal foam, cream or gel], or male partner sterilization throughout the study.
Age at study entry ≥1 year.
Previous platelet transfusions are permitted, as long as the subject has a platelet count ≥50,000/μL without transfusion support for at least 1 week.
Subjects must have a minimum pulse oximetry measurement of at least 94% at baseline.
An absolute neutrophil count ≥750/μL without growth factor for 5 days.
Liver function parameter results: total bilirubin ≤2 × upper limit of normal for age, and Serum alanine aminotransferase (glutamic-pyruvic transaminase) and serum aspartate aminotransferase (glutamic-oxaloacetic transaminase) ≤ 10 times the upper limit of normal (for all sites, the upper limit of normal for alanine aminotransferase is defined as 45 U/L).
Normal thyroid function as measured by T4 or TSH or have abnormal results that are not considered clinically important by the Investigator or may be receiving levothyroxine.
Cardiac Function: shortening fraction of ≥ 27% by echocardiogram or ejection fraction ≥ 50% documented by echocardiogram or radionuclide angiogram within 1 month prior to Visit 1 (Baseline).
Karnofsky Performance Status (for subjects >16 years of age) or the Lansky Performance Status Performance Status (for subjects 1 to 16 years of age) ≥50%.
Full recovery from the toxic effects of any prior therapy.
Coagulation Function:
International Normalized Ratio (INR) < 1.5
Partial thromboplastin time (PTT) < 1.5 times upper limit of normal.
Exclusion Criteria:
Subjects within 5 half-lives after any antibody-based immunotherapy, or have not recovered from effects of any biologic therapy.
Subjects <12 weeks after myeloablative therapy with autologous stem cell transplant.
Subjects who have had an allogeneic stem cell treatment less than 4 months from Visit 1 are excluded. Those who have received allogeneic stem cell treatment more than 4 months from Visit 1 must have recovered and have no active graft versus host disease (GVHD) to be eligible.
Subjects must not have received radiation for a minimum of 2 weeks prior to study enrollment. Subjects whose only site(s) of disease have been radiated are eligible as long as the subject has MIBG avidity 2 weeks after completion of radiation. A minimum of 12 weeks prior to study enrollment is required following prior large field radiation therapy (ie, craniospinal, whole abdominal, total lung, > 50% marrow space)
History of total body irradiation.
Subjects do not have adequate renal function defined as GFR ≥ 70 mL/min/1.73 m2 either by creatinine clearance or radioisotope direct measurement or by calculation with the Schwartz formula
Subjects who are on hemodialysis.
Pregnancy or breastfeeding.
Significant active infections including active hepatitis B, or hepatitis C infection, or known infection with human immunodeficiency virus (HIV) (testing for HIV is not required prior to study entry).
Clinically important cardiac, pulmonary, and hepatic impairment.
Vorinostat treatment exclusion criteria (subjects, who meet any one of these criteria and otherwise meet eligibility criteria, are still eligible for 131I-MIBG monotherapy)
Since valproic acid has HDAC inhibitory activity, patients must not have received valproic acid within 30 days of study entry.
Since vorinostat may prolong the QT interval, patients must not be receiving other medications known to prolong the QT interval at the time of study entry . Pentamidine must not have been received within 1 week of study enrollment.
Patients with a history of deep venous thrombosis that was not associated with the presence of a central venous catheter.
Patients who are receiving Coumadin.The TrialNet Natural History Study of the Development of Type 1 Diabetes
TrialNet is an international research group dedicated to the study, prevention, and early treatment of Type 1 diabetes. Type 1 diabetes is now understood as a disease that develops over time in stages. Stage 1 starts with the appearance of having two or more autoantibodies. This is followed by Stage 2, which is the development of abnormal blood glucose levels. Stage 3 is the clinical diagnosis of Type 1 diabetes. This study will help us learn more about how Type 1 diabetes occurs through the screening of diabetes-related autoantibodies and monitoring individuals who have tested positive for these autoantibodies.
Antoinette Moran
Not specified
SITE00000016
Inclusion Criteria:
• 2.5 to 45 years old and have an immediate family member with type 1 diabetes (child, parent, or sibling)
• 2.5 to 20 years old who have an extended family member with type 1 diabetes (cousin, niece, nephew, aunt, uncle, grandparent, or half-sibling)
Exclusion Criteria:
• already have diabetes
• history of being treated with insulin or oral diabetes medications
• using systemic immunosuppressive agents (topical and inhaled agents are acceptable)
• have any known serious diseases
Diabetes & Endocrine
T1DM, Type 1 Diabetes
Natural History Study for DNA Repair Disorders
This research is being done to help us better understand the different DNA repair disorders. We will collect data and samples that we will use to develop new therapies and medicine to help treat the disease. We expect that participants will be in this research study for 3 years. Visits will occur every six months and alternate between in-person and remote. Remote visits should be expected to last 1-2 hours, and in-person visits should be expected to last 3-4 hours.
Peter Kang
Not specified
STUDY00015911
Inclusion Criteria:
• at least 6 months old
• diagnosis of Cockayne syndrome (CS), xeroderma pigmentosum (XP), or trichothiodystrophy (TTD), based on genetic testing and/or key clinical characteristics
• have one or more of the neurodevelopmental or neurological complications such as gross motor delay, language delay, altered muscle tone (study staff will review)
• family member of an individual with the above condition
Exclusion Criteria:
• prior history of systemic gene or cell-based therapy
• participation in a clinical trial for treatment
Rare Diseases
Cockayne Syndrome, DNA Repair Disorder, Trichothiodystrophy, Xeroderma Pigmentosum
COG ARST2032: A Prospective Phase 3 Study of Patients with Newly Diagnosed Very Low-risk and Low-risk Fusion Negative Rhabdomyosarcoma
Rhabdomyosarcoma is a type of cancer that occurs in the soft tissues in the body. This phase III trial aims to maintain excellent outcomes in patients with very low risk rhabdomyosarcoma (VLR-RMS) while decreasing the burden of therapy using treatment with 24 weeks of vincristine and dactinomycin (VA) and examines the use of centralized molecular risk stratification in the treatment of rhabdomyosarcoma. Another aim of the study it to find out how well patients with low risk rhabdomyosarcoma (LR-RMS) respond to standard chemotherapy when patients with VLR-RMS and patients who have rhabdomyosarcoma with DNA mutations get separate treatment. Finally, this study examines the effect of therapy intensification in patients who have RMS cancer with DNA mutations to see if their outcomes can be improved.
Emily Greengard
Not specified
SITE00001858
Inclusion Criteria:
• 21 or younger at time of enrollment
• newly diagnosed embryonal rhabdomyosarcoma (ERMS), spindle cell/sclerosing RMS, or FOXO1 fusion negative alveolar rhabdomyosarcoma
• must be enrolled in APEC14B1 (NCT02402244) prior to enrollment and treatment on ARST2032 (this trial)
• contact study team for more detailed criteria
Exclusion Criteria:
• received prior chemotherapy and/or radiation therapy for cancer prior to enrollment
• unable to undergo radiation therapy
• Females who are pregnant
Cancer, Cancer, Children's Health
Rhabdomyosarcoma, Embryonal Rhabdomyosarcoma, Spindle Cell/Sclerosing Rhabdomyosarcoma
Pfizer Registry of Outcomes in Growth hormone RESearch (PROGRES): A multi country, non-interventional prospective cohort study among patients with human growth hormone (hGH) treatments under routine clinical care
The purpose of this study is to learn more about the safety and effectiveness of human growth hormone treatments, in routine practice.
Brad Miller, MD, PhD
Not specified
STUDY00016116
Inclusion Criteria:
• prescribed human growth hormone (hGH) to treat growth hormone deficiency (GHD)
Exclusion Criteria:
• participation in any interventional clinical trials
Rare Diseases
GHD, Growth Hormone Deficiency, hGH, Human Growth Hormone
Cochlear Implantation in Children with Asymmetric Hearing Loss or Single-Sided Deafness Clinical Trial
Kristin Gravel
Up to 18 years old
STUDY00010956
Inclusion Criteria:
• Parents and child fluent in English
• Parents desire functional hearing in both ears for their child
• Severe to profound sensorineural hearing loss in one ear and normal hearing in the other ear
• If older than 5 years, documentation of progressive hearing loss (i.e. passed newborn hearing screening, or significant change in hearing)
Exclusion Criteria:
• Medical condition that contraindicates a cochlear implant, including abnormal hearing nerve
• Already using a cochlear implant
• Inability to complete study procedures
• Unrealistic expectations related to the benefits and limitations of cochlear implantation
• Unwillingness or inability to comply with all investigational requirement
Ear, Nose & Throat
Pediatric audiology, Audiology, Cochlear Implant, Single-Sided Deafness
LTx READY CF 2: A Multi-Site RCT: Lung Transplant Resources for Education And Decision-making for Your CF 2 Study: A Multi-Site Randomized Controlled Clinical Trial
The primary study objective is to determine whether “Take on Transplant” (ToT), a CF-specific Lung Transplant (LTx) educational website, improves patient-reported preparedness for LTx discussions, as measured by the Preparation for Decision Making (PrepDM) Scale at 3 months after randomization, compared to an attention control transplant website (unos.org, UNOS).
Jordan Dunitz
SITE00001827
Rare Diseases
RCT01437: Proactive infliximab optimization using a pharmacokinetic dashboard versus standard of care in patients with inflammatory bowel disease: The OPTIMIZE Trial
The purpose of this study is to find out if using a computer program (called iDose) to guide infliximab dosing is more effective and safer than using standard infliximab dosing over 52 weeks. All patients in this study will be receiving infliximab as part of their medical care, this study is only looking at two different methods of determining the dose and timing of administration.
Eugenia Shmidt
Not specified
STUDY00013632
Inclusion Criteria:
• 16 to 80 years of age
• diagnosis of moderate to severe Crohn's disease (CD) or Ulcerative colitis
• physician intends to prescribe infliximab for treatment
• have not previously taken infliximab
Exclusion Criteria:
• pregnant or breastfeeding
• complications of inflammatory bowel disease (IBD) such as abscess, need for ostomy (study staff review)
• current infection in last 6 months
• other significant medical conditions (heart, lungs, liver, endocrine etc.)
Digestive & Liver Health
Clinics and Surgery Center (CSC), Crohn's disease
MT2012-11C: Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)
This is a treatment guideline for a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) regimen. This regimen, consisting of busulfan, fludarabine, and low dose total body irradiation (TBI), is designed to promote engraftment in patients who failed to achieve an acceptable level of donor-derived engraftment following a previous allogeneic HSCT. While it will primarily be applied for the treatment of non-malignant diseases (NMD), on occasion it may be used to treat patients with malignant disorders as well.
Troy Lund
Up to 55 years old
1207M17641
Inclusion Criteria:
Diagnosis of any disease for which a second or greater hematopoietic stem cell transplant is needed due to insufficient donor chimerism following hematopoietic recovery after previous HSCT. Determination of "insufficiency of donor chimerism" will be made by the treating transplant physician. Occasionally donor derived engraftment may be present, but sustained aplasia or failed recovery of sufficient hematopoiesis requires administration of a second graft. This intervention may be used for both situations.
Donor Availability: Patients considered for transplantation must have a sufficient graft as based on current criteria of the University of Minnesota Blood and Marrow Transplantation Program
Transplantation using sufficiently matched related donors (such as matched siblings) or unrelated donors will be considered. Both granulocyte-colony stimulating factor (GCSF) stimulated peripheral blood grafts and bone marrow grafts will be considered, although bone marrow will be the priority.
Cord blood grafts, both related and unrelated, are also eligible. As this protocol will use a reduced intensity regimen, this protocol will use the current recommendations of the University of Minnesota for choosing cord blood grafts. If a single cord blood unit cell dose is insufficient, double cord transplantation should be considered if sufficiently matched cord blood units are available. The priority of choosing cord blood donors is based on the current institutional recommendations.
Exclusion of Metabolic Disorder or other Inherited Disorder Carrier Status from related donor and unrelated cord blood grafts as appropriate for primary disease.
At the discretion of the treating transplant physician, an allograft from the previous donor may be used, if available.
Age, Performance Status, Consent
Age: 0 to 55 years
Consent: voluntary written consent (adult or parental/guardian)
Exclusion Criteria:
Previous irradiation that precludes the safe administration of an additional dose of 200 cGy of total body irradiation (TBI). Radiation Oncology will evaluate all patients who have had previous radiation therapy or TBI for approval to receive an additional 200 cGy of TBI
Pregnant or breastfeeding
Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted
HIV positive
While it would be advantageous to begin therapy on this second transplant regimen > 6 months following a prior myeloablative regimen or >2 months after a reduced intensity regimen, it is recognized that there are circumstances where this may not be practical.MT2019-09: A randomized trial of low versus moderate exposure busulfan for infants with severe combined immunodeficiency (SCID) receiving TCR alpha beta +/CD19+ depleted transplantation: A Phase II study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC) PIDTC CSIDE Protocol (CSIDE)
To determine the incidence of humoral immune reconstitution by 2 years post-transplant in 2 SCID cohorts (IL2RG/JAK3, RAG1/RAG2) undergoing alternative donor HCT by randomized assignment to a busulfan preparative regimen targeted at cumulative area-under-the-curve (cAUC) exposure of 25-35 mg*h/L vs 55-65 mg*h/ L.
Christen Ebens
0 years to 2 years old
SITE00000541
Inclusion Criteria:
• Infants with SCID, either typical or leaky or Omenn syndrome. Typical SCID is defined as either of the following Absence or very low number of T cells (CD3+ T cells <300/microliter AND no or very low T cell function (<10% of lower limit of normal) as measured by response to phytohemagglutinin OR Presence of maternally derived T cells Leaky SCID is defined as the following • Absence of maternally derived T cells • AND either one or both of the following (i, ii): i) <50% of lower limit of normal T cell function as measured by response to PHA OR <30% of lower limit of normal T cell function as measured by response to CD3 ii) Absent or <10% of lower limit of normal proliferative responses to candida and tetanus toxoid antigens (must document post vaccination or exposure for this criterion to apply) • AND at least two of the following (i through iii): i) CD3 T cells < 1500/microliter ii) >80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR >80% of CD3+ or CD4+ T cells are CD62L negative AND/OR >50% of CD3+ or CD4+ T cells express HLA-DR (at < 4 years of age) AND/OR are oligoclonal T iii) Low TRECs and/or the percentage of CD4+/45RA+/CD31+ or CD4+/45RA+/CD62L+ cells is below the lower level of normal. Omenn syndrome • Generalized skin rash Maternal lymphocytes tested for and not detected. >80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR >80% of CD3+ or CD4+ T cells are CD62L negative AND/OR >50% of CD3+ or CD4+ T cells express HLA-DR (<2 years of age) Absent or low (up to 30% lower limit of normal (LLN)) T cell proliferation to antigens (Candida, tetanus) to which the patient has been exposed IF: Proliferation to antigen was not performed, but at least 4 of the following 8 supportive criteria, at least one of which must be among those marked with an asterisk (*) below are present, the patient is eligible as Omenn Syndrome. Hepatomegaly Splenomegaly Lymphadenopathy Elevated IgE Elevated absolute eosinophil count *Oligoclonal T cells measured by CDR3 length or flow cytometry (upload report) *Proliferation to PHA is reduced to < 50% of lower limit of normal (LLN) or SI < 30 *Low TRECs and/or percentage of CD4+/RA+ CD31+ or CD4+/RA+ CD62L+ cells below the lower level of normal
• Documented mutation in one of the following SCID-related genes a. Cytokine receptor defects (IL2RG, JAK3) b. T cell receptor rearrangement defects (RAG1, RAG2) 3. No available genotypically matched related donor (sibling) 4. Availability of a suitable donor and graft source Haploidentical related mobilized peripheral blood cells 9/10 or 10/10 allele matched (HLA-A, -B, -C, -DRB1, -DQB1) volunteer unrelated donor mobilized peripheral blood cells 5. Age 0 to 2 years at enrollment Note: to ensure appropriate hepatic metabolism, age at time of busulfan start: For IL2RG/JAK3: 8 weeks For RAG1/RAG2: 12 weeks
• Adequate organ function defined as: Cardiac: Left ventricular ejection fraction (LVEF) at rest ≥ 40% or, shortening fraction (SF) ≥ 26% by echocardiogram. Hepatic: Total bilirubin < 3.0 x the upper limit of normal (ULN) for age (patients who have been diagnosed with Gilbert's Disease are allowed to exceed this limit) and AST and ALT < 5.0 x ULN for age. Renal: GFR estimated by the updated Schwartz formula ≥ 90 mL/min/1.73 m2. If the estimated GFR is < 90 mL/min/1.73 m2, then renal function must be measured by 24-hour creatinine clearance or nuclear GFR, and must be > 50 mL/min/1.73 m2. Pulmonary No need for supplemental oxygen and O2 saturation > 92% on room air at sea level (with lower levels allowed at higher elevations per established center standard of care).
Exclusion Criteria:
Presence of any serious life-threatening or opportunistic infection at time of enrollment and prior to the initiation of the preparative regimen. Serious infections as defined below that occur after enrollment must be reported immediately to the Study Coordinating Center, and enrollment will be put on hold until the infection resolves. Ideally enrolled subjects will not have had any infection. If patients have experienced infections, these must have resolved by the following definitions:
a. Bacterial i. Positive culture from a sterile site (e.g. blood, CSF, etc.): Repeat culture(s) from same site must be negative and patient has completed appropriate course of antibacterial therapy (typically at least 10 days).
ii. Tissue-based clinical infection (e.g. cellulitis): Complete resolution of clinical signs (e.g. erythema, tenderness, etc.) and patient has completed appropriate course of antibacterial therapy (typically at least 10 days).
iii. Pneumonia, organism not identified by bronchoalveolar lavage: Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.) and patient has completed appropriate course of antibacterial therapy (typically at least 10 days). If possible, radiographic resolution should also be demonstrated.
b. Fungal i. Positive culture from a sterile site (e.g. blood, CSF, etc.): Repeat culture(s) from same site is negative and patient has completed appropriate course of antifungal therapy (typically at least 14 days). The patient may be continued on antifungal prophylaxis following completion of the treatment course.
c. Pneumocystis i. Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.) and patient has completed appropriate course of therapy (typically at least 21 days). If possible, radiographic resolution should also be demonstrated. The patient may be continued on prophylaxis following completion of the treatment course.
d. Viral i. Viral PCRs from previously documented sites (blood, nasopharynx, CSF) must be re-tested and are negative.
ii. If re-sampling a site is not clinically feasible (i.e. BAL fluid): Complete resolution of clinical signs (e.g. tachypnea, oxygen requirement, etc.). If possible, radiographic resolution should also be demonstrated.
Patients with HIV or HTLV I/II infection will be excluded.Surgical Timing and Rehabilitation for Multiple Ligament Knee Injuries: A Multicenter Integrated Clinical Trial (Protocol # PRO16090503)
This study is being conducted to determine when is the best time to do surgery is and when to start rehabilitation after surgery for the treatment of a multiple ligament knee injury. The study will randomize (i.e. a coin toss) when you have surgery and when you begin rehabilitation (early or delayed surgery and early or delayed rehabilitation). The type of surgery is decided between you and your surgeon. Rehabilitation is tailored to the type of surgery you have. You will fill out some questionnaires and have your knee examined at your normal postsurgery appointments with your surgeon.
Jeffrey Macalena
Not specified
SITE00000183
Inclusion Criteria:
• Ages 16-55
• Has a multiple ligament knee injury (MLKI is defined as a complete grade III injury of two or more ligaments)
Exclusion Criteria:
• Prior knee ligament surgery of the involved knee
• Torn or avulsed patellar or quadriceps tendon
• Periarticular or long bone fracture that is anticipated to preclude weight-bearing after surgery
• Require use of an external fixator for greater than 10 days
• Planned staged surgical treatment
• Unable to weight bear on the contralateral uninjured leg
• Traumatic brain injury (TBI) that limits their ability to participate in their post-operative care
• Surgical procedures that precludes early weight-bearing or range of motion
Bone, Joint & Muscle, Children's Health
Knee injury, Clinics and Surgery Center (CSC)
A Natural History Study of the Gangliosidoses
This is a research study documenting the natural history of disease in patients with GM1 or GM2 gangliosidosis. The information collected will be a way to evaluate disease progression and create a disease stage and severity index. Our goal is to use the data collected to measure the effectiveness of any treatments that are developed in the future.
Jeanine Jarnes
Not specified
1007M85712
Inclusion Criteria:
• documented gangliosidosis disease
• able to complete neuropsychological and neurobehavioral assessments
• Late-onset gangliosidosis subjects must be able to tolerate MRI of the head
Exclusion Criteria:
• none
Rare Diseases
GM1 Gangliosidosis, GM2 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
PEPN2121 : A Phase 1/2 Study of Tiragolumab (NSC# 827799, IND# 161266) and Atezolizumab (NSC# 783608, IND# 161266) in Patients with Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors
This phase I/II trial studies how well tiragolumab and atezolizumab works when given to children and adults with SMARCB1 or SMARCA4 deficient tumors that that has either come back (relapsed) or does not respond to therapy (refractory). SMARCB1 or SMARCA4 deficiency means that tumor cells are missing the SMARCB1 and SMARCA4 genes, which is related to having more aggressive cancers that are harder to treat. Immunotherapy with monoclonal antibodies, such as tiragolumab and atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.
Emily Greengard
Not specified
SITE00001713
Inclusion Criteria:
• patients must be >= 12 months of age at the time of study enrollment. For part A, patients must be <18 years old at enrollment. For part B, there is no upper age limit
• patients must have SMARCB1 (INI1) or SMARCA4 deficient tumors verified through institutional immunohistochemistry (IHC) or molecular confirmation of a pathologic tumor bi-allelic SMARCB1 (INI1) or SMARCA4 loss or mutation from a Clinical Laboratory Improvement Act (CLIA) certified lab
• see link to clinicaltrials.gov for complete eligibility criteria
Exclusion Criteria:
• patients who have undergone allogeneic bone marrow or stem cell transplant are not eligible
• patients with known, untreated CNS metastases will be excluded
Cancer
Malignant Solid Neoplasm
A Phase 1/2a Open-Label Dose-Ranging and Observer-Blind Placebo-Controlled, Safety and Immunogenicity Study of mRNA-1647 Cytomegalovirus Vaccine in Female and Male Participants 9 to 15 Years of Age; mRNA-1647-P104
This study it to test an investigational vaccine called mRNA-1647 that is being developed for preventing cytomegalovirus (CMV) infection in people. CMV is a common virus that can spread easily through an infected person’s saliva or other body fluids such as blood, urine, and breast milk. We want see if the trial vaccine can prevent CMV infection in participants who have not been previously infected, to understand the safety (how many side effects you may have) of the trial vaccine, and to see if the trial vaccine results in participants making antibodies to CMV.
Mark Schleiss
Up to 18 years old
SITE00001871
Inclusion Criteria:
• female or male 9 to 15 years of age
• in good general health
• BMI requirements ( study staff will review)
• female participants of childbearing potential: negative pregnancy test and adequate contraception for at least 28 days prior to receiving vaccine through 3 months following vaccine administration
Exclusion Criteria:
• received, or plans to receive, any nonstudy vaccine less than 28 days prior to or after any study medication
• any diagnosis or condition requiring significant changes in management or medication within the 2 months before starting the study
• contact study staff for review of additional exclusion criteria
Children's Health, Infectious Diseases
CMV, cytomegalovirus, vaccine
MT2020-28: Ruxolitinib, Human Chorionic Gonadotropin (uhCG/EGF), and Dose De-escalated Corticosteroids for Treatment of Minnesota High-Risk Acute GVHD (aGVHD): A Phase I/II Study
The purpose of this study is to learn whether the use of Pregnyl with the drug ruxolitinib is able to reduce the need for high dose steroids to treat severe acute Graft versus Host Disease (GVHD).
Punita Grover
Not specified
STUDY00014365
Inclusion Criteria:
• Hematopoietic Cell Transplant (HCT) recipients over 12 years of age within the first 7 days of initial treatment of high-risk Acute-graft-versus-host Disease (aGVHD)
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
Exclusion Criteria:
• progressive cancer
• uncontrolled bacterial, fungal, parasitic, or viral infection
• current thromboembolic disease requiring full-dose anticoagulation
• active or recent (within prior 3 months) thrombus, irrespective of anticoagulation status
• pregnancy
• women or men of childbearing potential unwilling to take adequate precautions to avoid unintended pregnancy from the start of protocol treatment through 30 days after the last treatment
Cancer
Clinics and Surgery Center (CSC), Acute-graft-versus-host Disease, aGVHD, HCT, Hematopoietic Cell Transplant