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280 Study Matches

A Randomized, Open Label, Phase III Trial to Evaluate the Efficacy and Safety of Palbociclib + Anti-HER2 Therapy + Endocrine Therapy vs. Anti-HER2 Therapy + Endocrine Therapy After Induction Treatment for Hormone Receptor Positive (HR+)/HER2-Positive Metastatic Breast Cancer

Phase III
This study is NOT accepting healthy volunteers
NCT02947685
201707881
Estrogen Receptor Positive Breast Cancer, HER-2 Positive Breast Cancer
HER2+, HR+, breast cancer, malignant tumor of the breast, metastatic breast cancer
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Clinical and Basic Investigations into Congenital Disorders of Glycosylation

Define natural history, validate patient reported outcome and share knowledge on congenital disorders of glycosylation. We will recruit and enroll patients with CDG in this study evaluating clinical variation and natural history when a patient is being seen as part of routine clinical care.

Kyriakie Sarafoglou
All
Not specified
NA
This study is NOT accepting healthy volunteers
NCT04199000
STUDY00009013
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Inclusion Criteria:

• Patients diagnosed with congenital disorders of glycosylation based on genetic confirmatory testing
Exclusion Criteria:

• Patients without congenital disorders of glycosylation
Congenital Disorders of Glycosylation
CDG
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APOL1 Long-term Kidney Transplantation Outcomes Network

The National Institutes of Health (NIH)-sponsored collaborative APOL1 Long-term Kidney Transplantation Outcomes Network (APOLLO) is charged with prospectively assessing the effects of renal-risk variants (RRVs) in the apolipoprotein L1 gene (APOL1) on outcomes for kidneys from donors with recent African ancestry and the recipients of their kidneys, after deceased- and living-donor renal transplantation. For the purposes of APOLLO, recent African ancestry is defined as individuals with similar genetic make-up to those currently residing in Africa. APOLLO will also study the impact of APOL1 RRVs on the health of living kidney donors with recent African ancestry.

Samy Riad
NA
This study is NOT accepting healthy volunteers
NCT03615235
STUDY00007354
Kidney Disease, Chronic, Kidney Diseases, Kidney Failure
Apolopoprotein L1 gene (APOL1), Association of Organ Procurement Organizations (AOPO), Kidney Donor, Kidney Transplantation, Kidney Transplantation Outcomes Network, United Network for Organ Sharing (UNOS)
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University of Minnesota Transplant Registry

This registry is open for people of all ages who have either received or donated an organ at the University of Minnesota. This study aims to evaluate organ transplant outcomes among people who have been part of the transplant program.

All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT01062581
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Inclusion Criteria:

• Received a transplanted organ at the University of Minnesota
• Living donor who donates an organ at the University of Minnesota
Exclusion Criteria:

• Did not receive a transplant at the University of Minnesota
• Did not donate an organ at the University of Minnesota
Transplant Recipient, Transplant Donation
Recipient, Donor, Organ Transplant, Registry, University of Minnesota
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A Prospective, Open-Label, Multicenter Pivotal Study to Evaluate the Safety and Efficacy of GelrinC? for the Treatment of Symptomatic Articular Cartilage Defects of the Femoral Condyle: A Comparison to Historical Control Microfracture

Comparison of microfracture (knee surgery) with GelrinC (a glue like solution to augment the tissue regeneration of microfracture) for cartilage defects of the knee with historical control. This is a multi center, open label, controlled, non-randomized, double arm trial with prospective treatment arm (Gelrin C) and a historical control arm (microfracture). We hope to help the sponsor in learning more information regarding treatment of cartilage defects in the knee.

Jeffrey Macalena
Pivotal
This study is NOT accepting healthy volunteers
NCT03262909
STUDY00003117
Articular Cartilage Defects in the Knee Joint
cartilage damage, cartilage defect, chondral lesion, condyle, femur, osteochondral lesion
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A Phase 1/2 Study of the Oral RET Inhibitor LOXO-292 in Pediatric Patients with Advanced RET-Altered Solid or Primary Central Nervous System Tumors; Protocol Number: LOXO-RET-18036 (J2G-OX-JZJJ) (LIBRETTO-121)

This is an open-label, multi-center, Phase 1/2 study of oral LOXO-292 in pediatric patients with an activating RET alteration and an advanced solid or primary CNS tumor.

Emily Greengard
All
6 Months to 21 Years old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT03899792
STUDY00008874
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Inclusion Criteria:

• Advanced or metastatic solid or primary CNS tumor which has failed standard of care therapies
• Evidence of an activating RET gene alteration in the tumor and/or blood
• Measurable or non-measurable disease
• Karnofsky (participants 16 years and older) or Lansky (participants younger than 16) performance score of at least 50
• Participant with primary CNS tumors or cerebral metastases must be neurologically stable for 7 days prior and must not have required increasing doses of steroids within the last 7 days
• Adequate hematologic, hepatic and renal function.
• Ability to receive study drug therapy orally or via gastric access
• Willingness of men and women of reproductive potential to observe conventional and effective birth control
Exclusion Criteria:

• Major surgery within two weeks prior to planned start of LOXO-292
• Clinically significant, uncontrolled cardiac, cardiovascular disease or history of myocardial infarction within 6 months prior to planned start of LOXO-292
• Active uncontrolled systemic bacterial, viral, fungal or parasitic infection
• Clinically significant active malabsorption syndrome
• Pregnancy or lactation
• Uncontrolled symptomatic hyperthyroidism or hypothyroidism (i.e. the participant required a modification to current thyroid medication in the 7 days before start of LOXO-292)
• Uncontrolled symptomatic hypercalcemia or hypocalcemia
• Known hypersensitivity to any of the components of the investigational agent, LOXO-292 or Ora-Sweet® SF and OraPlus®, for participants who will receive LOXO-292 suspension
• Prior treatment with a selective RET inhibitor(s) (including investigational selective RET inhibitor[s])
Drug: LOXO-292
Medullary Thyroid Cancer, Infantile Myofibromatosis, Infantile Fibrosarcoma, Papillary Thyroid Cancer, Soft Tissue Sarcoma
Loxo, LOXO-292, KIF5B-RET, M918T, CCDC6-RET, RET-PTC1, NCOA4-RET, RET-PTC, RET-PTC3, RET-PTC4, PRKAR1A-RET, RET-PTC2, GOLGA5-RET, RET-PTC5, ERC1-RET, KTN1-RET, RET-PTC8, HOOK3-RET, PCM1-RET, TRIM24-RET, RET-PTC6, TRIM27-RET, TRIM33-RET, RET-PTC7, AKAP13-RET, FKBP15-RET, SPECC1L-RET, TBL1XR1-RET, BCR-RET, FGRF1OP-RET, RFG8-RET, RET-PTC9, ACBD5-RET, MYH13-RET, CUX1-RET, KIAA1468-RET, FRMD4A-RET, SQSTM1-RET, AFAP1L2-RET, PPFIBP2-RET, EML4-RET, PARD3-RET, G533C, C609F, C609G, C609R, C609S, C609Y, C611F, C611G, C611S, C611Y, C611W, C618F, C618R, C618S, C620F, C620R, C620S, C630R, C630Y, D631Y, C634F, C634G, C634R, C634S, C634W, C634Y, K666E, E768D, L790F, V804L, V804M, A883F, S891A, R912P, CLIP1-RET, Y806C, RET fusion, RET alteration, RET mutation, RET rearrangement, RET translocation, Neoplasms by Site, Neoplasms, Non-Small Cell Lung Cancer, Lung Neoplasms, Carcinoma, Non-Small-Cell Lung, Cancer of Lung, Cancer of the Lung, Lung Cancer, Neoplasms, Lung, Neoplasms, Pulmonary, Pulmonary Cancer, Pulmonary Neoplasms, Respiratory Tract Neoplasms, Lung Diseases, Respiratory Tract Diseases, Carcinoma, Bronchogenic, Bronchial Neoplasms, Medullary Thyroid Cancer, Papillary Thyroid Cancer, Thyroid Diseases, Thyroid Neoplasms, Cancer of the Thyroid, Cancer of Thyroid, Neoplasms, Thyroid, Thyroid Adenoma, Thyroid Cancer, Thyroid Carcinoma, Endocrine System Diseases, Endocrine Gland Neoplasms, Head and Neck Neoplasms, Thoracic Neoplasms, CNS tumor, Primary CNS tumor, Colonic Neoplasms, Cancer of Colon, Cancer of the Colon, Colon Cancer, Colon Neoplasms, Colonic Cancer, Neoplasms, Colonic, Malignant tumor of Breast, Mammary Cancer, Mammary Carcinoma, Human, Mammary Neoplasm, Human, Neoplasms, Breast, Tumors, Breast, Human Mammary Carcinoma, Malignant Neoplasm of Breast, Breast Carcinoma, Breast Tumors, Cancer of the Breast, Breast Neoplasms, Breast Cancer, RET Inhibitor, MTC, NSCLC, Soft tissue sarcoma, Infantile Myofibromatosis, Infantile Fibrosarcoma
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Influence of Cooling duration on Efficacy in Cardiac Arrest Patients

A multicenter, randomized, adaptive allocation clinical trial to determine if increasing durations of induced hypothermia are associated with an increasing rate of good neurological outcomes and to identify the optimal duration of induced hypothermia for neuroprotection in comatose survivors of cardiac arrest.

Benjamin Miller
Phase II/III
This study is NOT accepting healthy volunteers
NCT04217551
STUDY00011864
Cardiac Arrest, Out-Of-Hospital, Hypothermia, Induced, Hypoxia-Ischemia, Brain
Bayesian Adaptive Clinical Trial, Coma, Hypothermia, therapeutic
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Impact of Very Low Nicotine Content Cigarettes in a Complex Marketplace (CENIC2 Project 1)

This is a Phase III randomized, open label, multi-center study that will examine the impact of very low nicotine content (VLNC) cigarettes in a complex tobacco and nicotine product marketplace simulating a real world environment.

Dorothy Hatsukami
Phase III
This study is also accepting healthy volunteers
NCT03272685
STUDY00000937
Nicotine Dependence, Tobacco Smoking
Alternative nicotine products, Biomarkers of tobacco exposure, Reduced nicotine cigarettes
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The Effect of Tirzepatide Versus Dulaglutide on Major Adverse Cardiovascular Events in Patients With Type 2 Diabetes (SURPASS-CVOT)

Study I8F-MC-GPGN (GPGN), also known as SURPASS-CVOT, is a Phase 3, event-driven, multicenter, international, randomized, double-blind, active comparator, parallel-group study. This study will assess the effect of QW tirzepatide (up to 15 mg) versus dulaglutide (1.5 mg) on CV outcomes when added to the standard of care in patients with T2DM with established cardiovascular disease and elevated risk for MACE.

Les Forgosh
18 Years and over
Phase III
This study is NOT accepting healthy volunteers
NCT04255433
STUDY00009070
Diabetes & Endocrine, Heart & Vascular, Type 2 Diabetes Mellitus
Diabetes Mellitus, Diabetes Mellitus, Type 2, Endocrine System Diseases, Gastric Inhibitory Polypeptide, Glucagon-Like Peptide 1, Glucose Metabolism Disorders, Heart Disease, Hormones, Hormones, Hormone Substitutes, and Hormone Antagonists, Hypoglycemic Agents, Incretins, Metabolic Diseases, Physiological Effects of Drugs, Tirzepatide, Type 2 Diabetes
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COG AREN03B2: Renal Tumors Classification, Biology and Banking Study.

• To classify patients (< 30 years old) with renal tumors by histological categorization, surgico-pathological stage, presence of metastases, age at diagnosis, tumor weight and loss of heterozygosity for chromosomes 1p and 16q, to thereby define eligibility for a series of therapeutic studies. • To maintain a biological samples bank to make specimens available to scientists to evaluate additional potential biological prognostic variables and for the conduct of other research by scientists.

Emily Greengard
All
up to 29 Years old
N/A
This study is NOT accepting healthy volunteers
NCT00898365
0708M15261
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Inclusion Criteria:

• Patients with the first occurrence of any tumor of the kidney identified on CT scan or MRI are eligible for this study; histologic diagnosis is not required prior to enrollment but is required for all patients once on study
• Eligible tumors include (but are not limited to):
• Nephroblastic tumors
• Nephroblastoma (Wilms' tumor) (favorable histology, anaplasia [diffuse, focal])
• Nephrogenic rests and nephroblastomatosis
• Cystic nephroma and cystic partially differentiated nephroblastoma
• Metanephric tumors (metanephric adenoma, metanephric adenofibroma, metanephric stromal tumor)
• Mesoblastic nephroma (cellular, classic, mixed)
• Clear cell sarcoma
• Rhabdoid tumor (any malignant rhabdoid tumor occurring outside the central nervous system [CNS])
• Renal epithelioid tumors of childhood (papillary renal cell carcinoma, medullary renal cell carcinoma, renal tumors associated with Xp11.2 translocations, oncocytic renal neoplasms after neuroblastoma)
• Angiolipoma
• Ossifying renal tumor of infancy
• Patients with the first occurrence of the following tumors are also eligible:
• Extrarenal nephroblastoma or extrarenal neprogenic rests
• Malignant rhabdoid tumor occurring anywhere outside the central nervous system
• Required specimens, reports, forms, and copies of imaging studies must be available or will become available for submission and the institution must intend on submitting them as described in the protocol procedures
• For ALL patients, (with exception of bilateral, bilaterally predisposed, multicentric, or unilateral tumor in solitary kidney planning to enroll without biopsy***), the following submissions are required:
• A complete set of recut hematoxylin and eosin (H & E) slides (including from sampled lymph nodes, if patient had upfront nephrectomy)
• * Tissue must be from diagnosis, prior to any renal tumor directed chemotherapy or radiation (only exception is for presumed favorable histology Wilms tumor [FHWT] patients discovered to have diffuse anaplastic Wilms tumor [DAWT] at delayed nephrectomy and plan to enroll at delayed nephrectomy)
• Representative formalin-fixed paraffin-embedded tissue block or if a block is unavailable, 10 unstained slides from a representative block of tumor, if available.
• Tissue must be from diagnosis, prior to any renal tumor directed chemotherapy or radiation (only exception is for presumed FHWT patients discovered to have DAWT at delayed nephrectomy and plan to enroll at delayed nephrectomy)
• Institutional pathology report, Specimen Transmittal Form, and Pre-Treatment Pathology Checklist
• Copies of images and institutional reports of CT and/or MRI abdomen and pelvis, and Pre Treatment Imaging Checklist
• Copies of images and institutional report of chest CT for all malignant tumors
• Institutional surgical report(s) and Pre-Treatment Surgical Checklist
• CRFs: Staging Checklist and Metastatic Disease Form (if metastatic disease is noted on imaging)
• Patients with bilateral, bilaterally predisposed, multicentric, or unilateral tumor in solitary kidney planning to enroll without biopsy via imaging only - these patients will not have central review or have a risk assignment issued, but may contribute to specimen banking for future research. However, if biopsy is done, tissue must be submitted as for other renal tumors, and initial risk assignment will require pathology and surgical rapid central reviews. The Specimen Transmittal Form and Pre Treatment Pathology Checklist are also needed.
• Please note: if the above required items are not received within 120 days of study enrollment, the patient will be considered off study
• 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
Other: Cytology Specimen Collection Procedure, Other: Laboratory Biomarker Analysis
Adult Cystic Nephroma, Anaplastic Kidney Wilms Tumor, Angiolipoma, Cellular Congenital Mesoblastic Nephroma, Classic Congenital Mesoblastic Nephroma, Clear Cell Sarcoma of the Kidney, Congenital Mesoblastic Nephroma, Cystic Partially Differentiated Kidney Nephroblastoma, Diffuse Hyperplastic Perilobar Nephroblastomatosis, Extrarenal Rhabdoid Tumor, Kidney Medullary Carcinoma, Kidney Neoplasm, Kidney Oncocytoma, Kidney Wilms Tumor, Metanephric Adenofibroma, Metanephric Adenoma, Metanephric Stromal Tumor, Metanephric Tumor, Mixed Congenital Mesoblastic Nephroma, Ossifying Renal Tumor of Infancy, Papillary Renal Cell Carcinoma, Renal Cell Carcinoma, Renal Cell Carcinoma Associated With Xp11.2 Translocations/TFE3 Gene Fusions, Rhabdoid Tumor of the Kidney, Wilms Tumor
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A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Compare the Efficacy and Safety of Sotatercept Versus Placebo When Added to Background Pulmonary Arterial Hypertension (PAH) Therapy for the Treatment of PAH

The objective of this study is to evaluate the efficacy and safety of sotatercept treatment (plus background P AH therapy) versus placebo (plus background PAH therapy) at 24 weeks in adults with PAH.

Thenappan Thenappan
Phase III
This study is NOT accepting healthy volunteers
NCT04576988
STUDY00012824
Pulmonary Arterial Hypertension
Clinics and Surgery Center (CSC), Hypertension, Pulmonary, sotatercept
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Theta Burst Stimulation for Headaches after Traumatic Brain Injury

The primary objective of this study is to investigate the safety and efficacy of theta burst stimulation (TBS) for the management of post-traumatic headaches to improve outcomes and quality of life for individuals who have suffered a traumatic brain injury (TBI). To improve tolerability and logistical burden, we have developed a novel design whereby participants will receive three doses of TBS on alternate days of the week. This design will allow us to assess efficacy while leveraging an accelerated treatment course (nine stimulation sessions per week). We have three specific aims: Specific Aim 1. To determine the efficacy and safety of TBS for the treatment of post-traumatic headache among individuals who have sustained a mild TBI. Hypothesis 1a: TBS will be safe, well-tolerated, and reduce the number of headache days. Hypothesis 1b: TBS will improve function and quality of life outcomes. Specific Aim 2: To determine the efficacy and safety of an accelerated time-course of TBS for the management of post-traumatic headache. Hypothesis 2a: The accelerated-time course will be safe, welltolerated, and improve quality of life outcomes. Hypothesis 2b: The accelerated time-course will produce greater and faster improvement in headache symptoms than that reported in the literature for standard repetitive transcranial magnetic stimulation (rTMS) protocols. Specific Aim 3: To examine the durability of treatment response to accelerated TBS during a one-month observational period. Hypothesis 3: Accelerated TBS will result in enduring treatment response of posttraumatic headache symptoms over the follow-up period.

Cristina Albott
18 Years and over
NA
This study is NOT accepting healthy volunteers
NCT04906603
STUDY00013016
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Inclusion Criteria:
? Veterans receiving services through the MVAHCS; ? History of mild to moderate TBI according to VA/DoD Clinical Practice Guidelines (2009); ? Post-traumatic headaches defined by International Classification for Headache Diagnosis 3rd Edition (ICHD-3) guidelines with the following criteria present: o Headaches developing within seven days following trauma or injury to the head and/or neck o Headaches persisting beyond three months; ? Chronic daily headaches defined by clinical standards with the following criterion present: o 15 or more headache days per month; ? Men and women 18-75 years of age; ? Possess a smartphone and agree to use the EMA application on their personal device; ? Capable and willing to provide voluntary informed consent
Exclusion Criteria:
? History of severe TBI according to VA/DoD Clinical Practice Guidelines (2009); ? Current (within six months of enrollment) psychosis and mania; ? Current (within one month of enrollment) substance dependence: o Does not include dependence on opioids for chronic pain when the medication is taken as prescribed; ? Personal history of epilepsy or seizure disorder: o Does not include seizures therapeutically-induced by electroconvulsive therapy (ECT) or identified as a single seizure event (based on the principal investigator?s judgement); ? Metal particles in the eye or head (exclusive of the mouth) (e.g., shrapnel, fragments from welding or metalwork, etc.); ? Implanted medical device controlled by physiologic signals (e.g., pacemakers, defibrillators, etc.) or implanted medical device above the clavicle (e.g., aneurysm clips, shunts, stimulators, cochlear implants, electrodes, etc.); ? Significant neurological disorder/injury or abnormal structural brain imaging that would impact risk (based on the principal investigator?s judgement and research literature); ? Unstable physical disease (e.g., severe heart disease); ? Current use of medications with significant potential for lowering seizure threshold; ? Current benzodiazepine usage at a dose higher than 3mg of lorazepam or equivalent; ? ECT or cortical energy exposure within one month of enrollment (including participation in any other neuromodulation treatments or studies); ? Current (within one month of enrollment) participation in another interventional study that would impact the results of this research; ? Inadequate communication (e.g., language barrier); ? Women who are pregnant, trying to become pregnant, or breastfeeding; ? Women of childbearing age/potential who are not using a medically-accepted form of contraception when sexually active
Brain & Nervous System, Brain Injuries, Traumatic, Post-Traumatic Headache, Quality of Life, Transcranial Magnetic Stimulation
TBI, TMS, headache
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SHP620-302: A Phase 3, Multicenter, Randomized, Double-blind, Double-dummy, Active-controlled Study to Assess the Efficacy and Safety of Maribavir Compared to Valganciclovir for the Treatment of Cytomegalovirus (CMV) Infection in Hematopoietic Stem Cell Transplant Recipients

Drug study - Maribavir in HSCT patients with CMV infections

Jo-Anne Young, MD
All
16 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT02927067
1703M11501
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Inclusion Criteria:

• Be able to provide written, personally signed, and dated informed consent to participate in the study before completing any study-related procedures. As applicable, a parent/both parents or legally authorized representative (LAR) must provide signature of informed consent and there must be documentation of assent by the participants before completing any study-related procedures. During the COVID-19 public health emergency, informed consent from a potential or current trial participant may, if permitted by local laws and regulations, be obtained via electronic informed consent (eIC) capabilities or an electronic face-to-face consent interview when these individuals are unable to travel to the site (FDA COVID-19 Guidance, 27 January 2021, Q11).
• Be greater than or equal to (>=) 16 years of age at the time of consent.
• Be a recipient of hematopoietic stem cell transplant.
• Have a documented asymptomatic CMV infection, with a screening value of CMV DNA >=1365 International Units per millilitre (IU/mL) to less than or equal to (<=) 273000 IU/mL in whole blood or >=455 IU/mL to <=91000 IU/mL in plasma in 2 consecutive assessments, separated by at least 1 day, as determined by local or central specialty laboratory quantitative polymerase chain reaction (qPCR) or comparable quantitative CMV DNA results. Both samples should be taken within 14 days prior to randomization with second sample obtained within 5 days prior to randomization. Same laboratory and same sample type (whole blood or plasma) should be used for these assessments. Asymptomatic CMV infection is defined as an infection that does not present with tissue invasive CMV disease, as assessed by the investigator. Participants with CMV DNA less than (<) 910 and >=455 IU/mL in plasma or <2730 and >=1365 IU/mL in whole blood will also need to meet at least 1 of the following criteria for high-risk CMV infection to be eligible:
• Human leukocyte antigen (HLA)-related (sibling) donor with at least 1 mismatch at 1 of the following 3 HLA-gene loci: HLA-A, -B or -DR,
• Haploidentical donor
• Unrelated donor with at least 1 mismatch at 1 of the following 4 HLA -gene loci: HLA-A, -B, -C and -DRB1,
• Use of umbilical cord blood as stem cell source,
• Use of ex vivo T-cell-depleted grafts,
• Grade 2 or greater graft-versus-host-disease (GVHD), requiring the use of systemic corticosteroids (defined as the use of >=1 milligram per kilogram per day (mg/kg/day) of prednisone or equivalent dose of another corticosteroid).
• Have the current CMV infection as the first episode of CMV viremia after HSCT, either primary or reactivation, which in the investigator's opinion requires treatment.
• Per investigator's judgment, be eligible for treatment with valganciclovir.
• Have all of the following results as part of screening laboratory assessments (results from either the central laboratory or a local laboratory can be used for qualification):
• Absolute neutrophil count to >=1000 per cubic millimeter (/mm^3) [1.0*10^9/L].
• Platelet count >=25,000/mm^3 [25*10^9/L].
• Hemoglobin >=8 grams per deciliter (g/dL).
• Estimated creatinine clearance >=30 milliliters per minute (mL/min).
• Have a negative serum beta human chorionic gonadotropin (beta-HCG) pregnancy test at screening, if a female of child bearing potential. Urine pregnancy tests may be done per institutional requirements; however they are not sufficient for eligibility determination. Sexually active females of child bearing potential must agree to comply with any applicable contraceptive requirements of the protocol. If male, must agree to use an acceptable method of birth control, as defined in the protocol, during the study treatment administration period and for 90 days afterward the last dose of study treatment.
• Be able to swallow tablets.
• Have life expectancy of >=8 weeks.
• Weigh >=40 kilograms (kg).
• Be willing and have an understanding and ability to fully comply with study procedures and restrictions defined in the protocol.
Exclusion Criteria:

• Have CMV tissue invasive disease as assessed by the investigator at the time of screening and randomization at Visit 2/Day 0.
• Have a CMV infection that is known to be genotypically resistant to ganciclovir, valganciclovir, foscarnet, or cidofovir based on documented evidence.
• Be presenting with recurrent CMV infection (defined as a new detection of CMV infection in a participants who had at least one previously documented episode of CMV infection post-transplant, and who has had at least 2 weeks of undetectable CMV DNA between the episodes during active surveillance, based on same local laboratory and same sample type). The Participants must also have been off any anti-CMV treatment between the current and prior infection. Otherwise, the current infection may be considered continuation of the prior infection.
• Require ganciclovir, valganciclovir, foscarnet, or cidofovir administration for conditions other than CMV when study treatment is initiated (example: herpes simplex virus [HSV] co-infection requiring use of any of these agents after the randomization) or would need a co-administration with maribavir for CMV infection.
• Be receiving leflunomide, letermovir, or artesunate when study treatment is initiated. Note: Participants who may be receiving leflunomide must discontinue the use at least 14 days prior to randomization at Visit 2/Day 0 and the first dose of study treatment. Participants receiving letermovir must discontinue use 3 days prior to first dose of study treatment. Participants receiving artesunate must discontinue the use prior to the first dose of study treatment.
• Be on treatment with anti-CMV agents (ganciclovir, valganciclovir, foscarnet or letermovir) for the current CMV infection for longer than 72 hours.
• Have known hypersensitivity to the active substance or to an excipient of the study treatments.
• Have severe vomiting, diarrhea, or other severe gastrointestinal illness within 24 hours prior to the first dose of study treatment that would preclude administration of oral medication.
• Require mechanical ventilation or vasopressors for hemodynamic support at the time of randomization.
• Be female and pregnant or nursing.
• Have previously completed, discontinued, or have been withdrawn from this study.
• Have received any investigational agent with known anti-CMV activity within 30 days before initiation of study treatment or CMV vaccine at any time.
• Have received any unapproved agent or device within 30 days before initiation of study treatment.
• Have any clinically significant medical or surgical condition that, in the investigator's opinion, could interfere with interpretation of study results, contraindicate the administration of the assigned study treatment, or compromise the safety or well-being of the participant.
• Have previously received maribavir.
• Have serum aspartate aminotransferase (AST) greater than (>) 5 times upper limit of normal (ULN) at screening, or serum alanine aminotransferase (ALT) >5 times ULN at screening, or total bilirubin >= 3.0*ULN at screening (except for documented Gilbert's syndrome), as analyzed by local or central laboratory.
• Have known (previously documented) positive results for human immunodeficiency virus (HIV). Participants must have a confirmed negative HIV test result within 3 months of study entry or, if unavailable, be tested by a local laboratory during the screening period.
• Have active malignancy with the exception of nonmelanoma skin cancer, as determined by the investigator. Participants who experience relapse or progression of their underlying malignancy (for which HSCT was performed), as determined by the investigator, are not to be enrolled.
• Be undergoing treatment for acute or chronic hepatitis C
Drug: Maribavir, Drug: Valganciclovir, Other: Placebo
Cytomegalovirus (CMV)
Clinics and Surgery Center (CSC)
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A Phase 2/3, Two-Part, Open-Label, Dose Escalation, Age De-escalation and Randomized, Observer-Blind, Placebo-Controlled Expansion Study to Evaluate the Safety, Tolerability, Reactogenicity, and Effectiveness of mRNA-1273 SARS-CoV-2 Vaccine in Healthy Children 6 months to < 12 Years of Age (mRNA-1273-P204) - COVID-19

The Sponsor of this study, ModernaTX, is studying the mRNA-1273 vaccine for the prevention of COVID-19 in children. This study is being conducted to learn about the safety, any side effects, and how your child’s body responds to the study vaccine (the “immune response”).

Shane McAllister
Phase II/III
This study is also accepting healthy volunteers
NCT04796896
STUDY00012613
SARS-CoV-2
COVID-19, COVID-19 Vaccine, Coronavirus, Messenger RNA, Moderna, SARS-CoV-2, SARS-CoV-2 Vaccine, Virus Diseases, mRNA-1273, mRNA-1273 vaccine
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An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose PR001A in Infants with Type 2 Gaucher Disease

This is a study to assess the safety and efficacy of PR001A, an Aden-associated (AAV9) viral vector to treat neuronopathic Gaucher disease type 2 (GD2) in infants. PRA001A will be administered via suboccipital injection to the cisterna magna during a single neurosurgical session. GD2 is a fatal disease of early infancy that does not have any therapeutic options beyond palliative care. This study will enroll infants 0-24 months of age.

Chester Whitley, MD, PhD
All
up to 24 Months old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04411654
STUDY00008823
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Inclusion Criteria:

• Bi-allelic GBA1 mutations consistent with a diagnosis of GD2 confirmed by the central laboratory.
• Neurological signs and/or symptoms consistent with diagnosis of GD2
• Parent/legal guardian has the ability to understand the purpose and risks of the study and provide written informed consent and authorization to use protected health information in accordance with national and local privacy regulations.
• Patient has a reliable informant (i.e., parent/legal guardian) willing and able to participate in the study as a source of information on the patient's health status and cognitive and functional abilities (including providing input into the rating scales).
Exclusion Criteria:

• Diagnosis of a significant CNS disease other than GD2 that may be a cause for the patient's GD symptoms or may confound study objectives.
• Achieved independent gait.
• Severe peripheral symptoms of GD which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.
• Concomitant disease, condition, or treatment which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.
• Use of any GD treatment-related substrate reduction therapy.
• Use of strong inhibitors or inducers of cytochrome P450 3A4 (CYP3A4) or P-glycoprotein (P-gp) medications, herbals, or over-the-counter agents.
• Any type of prior gene or cell therapy.
• Immunizations (live vaccines) in the prior 4 weeks.
• Use of blood thinners. Antiplatelet therapies are acceptable if the patient is medically able to temporarily stop them from 7 days prior to dosing and through at least 48 hours after the intracisternal injection and lumbar puncture.
• Use of systemic immunosuppressant or corticosteroid therapy other than protocol-specified (topical preparations for dermatological conditions are allowed).
• Participation in another investigational drug or device study within the past 6 months.
• Brain MRI (magnetic resonance imaging) and MRA (magnetic resonance angiography) showing clinically significant abnormality considered to prevent intracisternal injection.
• Clinically significant laboratory test result abnormalities assessed at screening.
• Contraindications or intolerance to radiographic visualization methods (e.g. MRI, MRA, CT), and intolerance to contrast agents used for MRI or CT scans.
• Contraindications to general anesthesia or sedation. Other protocol-defined inclusion/exclusion criteria may apply.
Biological: PR001, Drug: Methylprednisolone, Drug: Sirolimus, Drug: Prednisone
Gaucher Disease, Type 2
Gaucher Disease, GD, Gaucher, Type 2 Gaucher, Neuronopathic Gaucher, nGD, AAV9, GBA, Gene Therapy, Glucocerebrosidase, GBA1 mutation, Infants
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MT2020-35 - COG AAML1831 - A Phase 3 Randomized Trial for Patients With De Novo AML Comparing Standard Therapy Including Gemtuzumab Ozogamicin (GO) to CPX-351 With GO, and the Addition of the FLT3 Inhibitor Gilteritinib for Patients With FLT3 Mutations

The overall goal of this study is to compare the effects, good and/or bad, of CPX-351 with daunorubicin and cytarabine on people with newly diagnosed AML to find out which is better, and to find out what effects, good and/or bad, the drug gilteritinib has when given with chemotherapy to children and young adults with newly diagnosed AML and the FLT3/ITD mutation or non-ITD FLT3 activating mutations.

Peter Gordon
All
up to 22 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04293562
STUDY00010751
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Inclusion Criteria:

• All patients must be enrolled on APEC14B1 and consented to Eligibility Screening (Part A) prior to enrollment and treatment on AAML1831. Submission of diagnostic specimens must be done according to the Manual of Procedures). Risk stratification will not be possible without the submission of viable samples. Given there are multiple required samples, bone marrow acquisition techniques such as frequent repositioning or performing bilateral bone marrow testing should be considered to avoid insufficient material for required studies. Consider a repeat marrow prior to starting treatment if there is insufficient diagnostic material for the required studies
• Patients must be less than 22 years of age at the time of study enrollment
• Patient must be newly diagnosed with de novo AML according to the 2016 World Health Organization (WHO) classification with or without extramedullary disease
• Patient must have 1 of the following:
• >= 20% bone marrow blasts (obtained within 14 days prior to enrollment)
• In cases where extensive fibrosis may result in a dry tap, blast count can be obtained from touch imprints or estimated from an adequate bone marrow core biopsy
• < 20% bone marrow blasts with one or more of the genetic abnormalities (sample obtained within 14 days prior to enrollment)
• A complete blood count (CBC) documenting the presence of at least 1,000/uL (i.e., a white blood cell [WBC] count >= 10,000/uL with >= 10% blasts or a WBC count of >= 5,000/uL with >= 20% blasts) circulating leukemic cells (blasts) if a bone marrow aspirate or biopsy cannot be performed (performed within 7 days prior to enrollment)
• ARM C: Patient must be >= 2 years of age at the time of Late Callback
• ARM C: Patient must have FLT3/ITD allelic ratio > 0.1 as reported by Molecular Oncology
• ARM C: Patient does not have any congenital long QT syndrome or congenital heart block
• ARM C: Females of reproductive potential must agree to use effective contraception during treatment and for at least 6 months after the last dose of gilteritinib
• ARM C: Lactating women must agree not to breastfeed during treatment with gilteritinib and for 2 months after the last dose of gilteritinib
• ARM C: Males of reproductive potential must agree to use effective contraception during treatment and for at least 4 months after the last dose of gilteritinib
• ARM D: Patient must be >= 2 years of age at the time of Late Callback
• ARM D: Patient must have one of the clinically relevant non-ITD FLT3 activating mutations as reported by Foundation Medicine
• ARM D: Females of reproductive potential must agree to use effective contraception during treatment and for at least 6 months after the last dose of gilteritinib
• ARM D: Lactating women must agree not to breastfeed during treatment with gilteritinib and for 2 months after the last dose of gilteritinib
• ARM D: Males of reproductive potential must agree to use effective contraception during treatment and for at least 4 months after the last dose of gilteritinib
• NEUROPSYCHOLOGICAL TESTING: Patient must be enrolled on Arm A or Arm B. Patients who transfer to Arm C or Arm D are not eligible
• NEUROPSYCHOLOGICAL TESTING: Patient must be 5 years or older at the time of enrollment
• NEUROPSYCHOLOGICAL TESTING: English-, French- or Spanish-speaking
• NEUROPSYCHOLOGICAL TESTING: No known history of neurodevelopmental disorder prior to diagnosis of AML (e.g., Down syndrome, fragile X, William syndrome, mental retardation)
• NEUROPSYCHOLOGICAL TESTING: No significant visual or motor impairment that would prevent computer use or recognition of visual test stimuli
• 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 with myeloid neoplasms with germline predisposition are not eligible
• Fanconi anemia
• Shwachman Diamond syndrome
• Patients with constitutional trisomy 21 or with constitutional mosaicism of trisomy 21
• Any other known bone marrow failure syndrome
• Any concurrent malignancy
• Juvenile myelomonocytic leukemia (JMML)
• Philadelphia chromosome positive AML
• Mixed phenotype acute leukemia
• Acute promyelocytic leukemia
• Acute myeloid leukemia arising from myelodysplasia
• Therapy-related myeloid neoplasms
• Administration of prior anti-cancer therapy except as outlined below:
• Hydroxyurea
• All-trans retinoic acid (ATRA)
• Corticosteroids (any route)
• Intrathecal therapy given at diagnosis
• In particular, strong inducers of CYP3A4 and/or P-glycoprotein (P-gp) should be avoided from the time of enrollment until it is determined whether the patient will receive gilteritinib. Patients receiving gilteritinib will be required to avoid strong CYP3A4 inducers and/or strong P-gp inducers for the duration of the study treatment
• Female patients who are pregnant 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
• Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
• ARM D: Patients with congenital long QT syndrome or congenital heart block are not eligible for this treatment arm
Procedure: Allogeneic Hematopoietic Stem Cell Transplantation, Drug: Asparaginase, Drug: Asparaginase Erwinia chrysanthemi, Behavioral: Cogstate Assessment Battery, Drug: Cytarabine, Drug: Daunorubicin Hydrochloride, Drug: Dexrazoxane Hydrochloride, Drug: Etoposide, Drug: Gemtuzumab Ozogamicin, Drug: Gilteritinib Fumarate, Drug: Liposome-encapsulated Daunorubicin-Cytarabine, Drug: Methotrexate, Drug: Mitoxantrone Hydrochloride, Drug: Therapeutic Hydrocortisone
Acute Myeloid Leukemia
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NCI COVID-19 in Cancer Patients, NCCAPS Study

-Characterize patient factors, such as pre-existing comorbidities, cancer type and treatment, and demographic factors, associated with short- and long-term outcomes of COVID-19, including severity and fatality, in cancer patients undergoing treatment. -Describe cancer treatment modifications made in response to COVID-19, including dose adjustments, changes in symptom management, or temporary or permanent cessation. -Evaluate the association of COVID-19 with cancer outcomes in patient subgroups defined by clinico-pathologic characteristics.

Robert Kratzke
All
Not specified
NA
This study is NOT accepting healthy volunteers
NCT04387656
STUDY00010346
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Inclusion Criteria:

• NCCAPS STUDY ELIGIBILITY CRITERIA:
• Patient must have a prior or current cancer diagnosis (e.g., solid tumor or hematologic malignancy) and cancer treatment that fits into one of the three following categories:
• Metastatic (stage IV) solid tumor, any hematologic malignancy, or any central nervous system (CNS) malignancy, and:
• Patient is receiving eligible active treatment (defined as current treatment or treatment within the 6 weeks prior to their first positive SARS-CoV-2 test collection) or is expected to begin receiving treatment within 2 weeks of study enrollment
• Eligible active treatment types are chemotherapy, immunotherapy, monoclonal antibody therapy (e.g., rituximab, trastuzumab, cetuximab), targeted therapy (e.g., BRAF/MEK inhibitor, EGF-R inhibitor), endocrine therapy, radiation therapy, or targeted radionuclide therapy; OR
• Non-metastatic (Stage I-III) solid tumor and:
• Patient is receiving eligible active treatment (defined as current treatment or treatment within past 6 weeks prior to their first positive SARS-CoV-2 test collection) or is expected to begin receiving treatment within 2 weeks of study enrollment
• Eligible active treatment types for non-metastatic solid tumor patients are intravenous chemotherapy, immunotherapy, targeted therapy, radiation therapy, targeted radionuclide therapy, or monoclonal antibody therapy (except as noted below)
• HER2-targeted therapy (trastuzumab, pertuzumab, neratinib, ado-trastuzumab) that is not accompanied by chemotherapy is NOT considered an eligible active treatment
• Patients on endocrine therapy alone are not eligible; OR
• Prior or current transplant for the treatment of cancer:
• Patient has received an allogenic stem cell/bone marrow transplant or chimeric antigen receptor (CAR)-T cell or other modified cellular therapy at any time; or
• Patient is currently receiving treatment or prophylaxis for graft graft versus (vs.) host disease; or
• Patient has received an autologous stem cell/bone marrow transplant within the past 2 years
• Patient must have documented positive viral test result for SARS-CoV-2
• For patients 18 years of age or older, the specimen collection for the patient's FIRST positive test must have occurred no earlier than 14 days prior to enrollment
• For patients under 18 years of age, the specimen collection for the patient's first positive test must have occurred after January 31, 2020
• The viral test can be either a nucleic acid (PCR) test or an antigen test. Serological or antibody tests are not allowed
• Any specimen source (e.g., nasopharyngeal swab, oropharyngeal swab, etc.) is allowable for the viral SARS-CoV-2 test
• Patients with prior negative viral SARS-CoV-2 test(s) are eligible if they are being tested again
• The SARS-CoV-2 test must be a validated diagnostic assay performed in accordance with the most recent guidance issued by the FDA in the Policy for Coronavirus Disease-2019 Tests During the Public Health Emergency. This policy is available at: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/policy-c oronavirus-disease-2019-tests-during-public-health-emergency-revised
• Human immunodeficiency virus (HIV)-infected patients are eligible
• Patients with CNS metastases are eligible
• Co-enrollment on other clinical trials (for cancer or for COVID-19) is allowed
• PEDIATRIC COVNET COHORT ELIGIBILITY CRITERIA: Patients should only be enrolled in the pediatric COVNET cohort if they are not eligible for the main NCCAPS Study cohort or decline to participate in the main study
• Patient must be < 18 years of age
• Patient must have a positive SARS-CoV-2 viral test after January 31, 2020
• Patient must have a current or prior diagnosis of cancer. Active cancer treatment is not required
• Note: Patients who enroll on Pediatric COVNET cohort will not be followed longitudinally; study data collection involves only a single questionnaire and research blood collection. A separate consent document is provided for the Pediatric COVNET cohort
Procedure: Biospecimen Collection, Other: Data Collection, Other: Quality-of-Life Assessment, Other: Questionnaire Administration
COVID-19 Infection, Hematopoietic and Lymphoid Cell Neoplasm, Malignant Solid Neoplasm, Metastatic Malignant Solid Neoplasm
COVID-19
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Phase 3 Multicenter, Double-Blind, Placebo-Controlled Trial of Viralym-M;(ALVR105) for the Treatment of Patients With Virus-Associated Hemorrhagic Cystitis After Allogeneic Hematopoietic Cell Transplant

The study hypothesis is that the administration of Viralym-M to patients with virus-associated HC will demonstrate superiority for the time to resolution of HC (as measured by resolution of macroscopic hematuria) compared to patients treated with placebo. The primary hypothesis will be tested in patients with BKV viruria to demonstrate superiority over placebo in this population (BK Intent-to-Treat [ITT] Population). A supplementary analysis will be conducted in all patients with any viral-associated HC (BKV, JCV, AdV, EBV, CMV, and/or HHV-6) in order to evaluate efficacy in this broader population (ITT Population). Further detail is provided in the statistical section below and will be described in the Statistical Analysis Plan (SAP).

Jo-Anne Young, MD
All
Not specified
Phase 3
This study is NOT accepting healthy volunteers
NCT04390113
STUDY00011838
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Key Inclusion Criteria Participants must meet all of the following criteria in order to be eligible to participate in the study:
• Male or female of any age.
• Had an allogeneic hematopoietic cell transplant (HCT) performed ≥21 days and ≤1 year prior to randomization.
• Myeloid engraftment confirmed, defined as an absolute neutrophil count ≥500/mm³ for 3 consecutive laboratory values obtained on different days, and platelet count >10,000/mm³ at the time of randomization.
• Diagnosed with HC based on the following criteria (all 3 criteria must be met):
• Clinical signs and symptoms of cystitis, including dysuria, lower abdominal pain, and/or other bladder-associated pain or spasms.
• Grade ≥3 hematuria, defined as macroscopic hematuria with visible clots.
• Viruria of >5 log10 copies/mL of at least 1 target virus (ie, BKV, JCV, AdV, CMV, EBV, and/or HHV-6).
• At least 1 identified, suitably matched Posoleucel (ALVR105) cell line for infusion is available. Key Exclusion Criteria Participants who meet any of the following criteria will be excluded from participation in the study:
• Ongoing therapy with high-dose systemic corticosteroids (ie, prednisone dose >0.5 mg/kg/day or equivalent).
• Therapy with antithymocyte globulin, alemtuzumab (Campath-1H), or other immunosuppressive T cell-targeted monoclonal antibodies <28 days before randomization.
• Evidence of active Grade >2 acute graft versus host disease (GVHD).
• Uncontrolled or progressive bacterial or fungal infections.
• Uncontrolled or progressive viral infections not targeted by Posoleucel (ALVR105).
• Uncontrolled or progressive EBV-associated post-transplant lymphoproliferative disorder.
• Known or presumed pneumonia secondary to any organism that is not considered to be well-controlled by antimicrobial therapy.
• Pregnant or lactating or planning to become pregnant. Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Biological: Posoleucel (ALVR105), Biological: Placebo
BK Virus Infection, Hemorrhagic Cystitis
Allogeneic Hematopoietic Cell Transplant, ALVR105, Posoleucel, Clinics and Surgery Center (CSC)
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Clinical Study of the ARTISAN Aphakia Lens for the Correction of Aphakia in Children

In this study, subjects who had a cataract removed but due to their eye structure, are not able to have a traditional intraocular lens (IOL) implanted, receive a special type of lens called the ARTISAN IOL. The objective of this study is to determine the effectiveness of the ARTISAN IOL and to precisely define the associated risks and, if possible, identify particular groups of patients who may be at high risk of developing complications resulting from the surgical procedure of implanting the lens.

Jill Anderson
All
2 Years to 21 Years old
Phase 3
This study is also accepting healthy volunteers
NCT01547442
1211M24343
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Inclusion Criteria:

• 2 to 21 years of age
• Have a visually significant cataract or need IOL replacement surgery
• Compromised capsular bag prohibiting implantation of standard posterior IOL
• Subject or parent/guardian must be able to comply with visit schedule and study requirements
• Subject's legal representative must be able to sign the Informed Consent
Exclusion Criteria:

• Under 2 years of age
• Unable to meet Postoperative evaluation requirements
• No useful vision or vision potential in fellow eye
• Mentally retarded patients
• History of corneal disease
• Abnormality of the iris or ocular structure
• ACD less than 3.2 mm
• Uncontrolled glaucoma
• IOP > 25 mmHg
• Chronic or recurrent uveitis
• Preexisting macular pathology that may complicate the ability to assess the benefit of this lens
• Retinal detachment or family history
• Retinal disease that may limit visual potential
• Optic nerve disease that may limit visual potential
• Diabetes mellitus
• Pregnant, lactating or plan to become pregnant
Device: Artisan Aphakia Intraocular Lens
Aphakia
aphakia, secondary intraocular lens, congenital cataract, marfan syndrome, pediatric cataract, ectopia lentis, subluxated lens
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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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The REPLACE Registry

This is a prospective, observational, non-interventional patient registry designed to document product safety and clinical outcomes for 10 years in patients treated with Cholbam/Kolbam, including those who have been using Cholbam/Kolbam for at least one month (existing users) and those who start Cholbam/Kolbam treatment at enrollment (new users). Patients who have been using Cholbam/Kolbam for less than one month and those who had previously been treated and who restart treatment will also be included but will not be counted in the existing or new users groups.

Boris Sudel
All
Not specified
Post Market Monitoring
This study is NOT accepting healthy volunteers
NCT03115086
STUDY00003757
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Inclusion Criteria:

• Male and female patients, of any age.
• The patient and/or the patient's parent/legal guardian is willing and able to provide signed informed consent, and the patient, if less than 18 years of age, is willing to provide assent as appropriate and in accordance with local regulatory, IRB, and EC requirements.
• The patient has a diagnosis for which Cholbam is indicated.
• The patient is or will be treated with Cholbam at the time of signing the informed consent form (ICF) (enrollment).
Exclusion Criteria:

• Patients who, by judgement of the Investigator, will not be able to comply with the requirements of the protocol will be excluded
Drug: Cholbam
Bile Acid Synthesis Disorders
Bile Acid Synthesis Disorder, Zellweger Spectrum Disorder, Peroxisomal Disorder, Cholic Acid, Cholbam, The REPLACE Registry
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A PHASE 3/4 RANDOMIZED, DOUBLE-BLIND, DOSE-RANGING STUDY OF THE SAFETY AND EFFICACY OF DEXMEDETOMIDINE (DEX) USED WITH PROPOFOL (PRO) AS NEEDED FOR PROCEDURAL SEDATION OF PEDIATRIC SUBJECTS ???1 MONTH TO <17 YEARS OF AGE UNDERGOING MRI SCANS

This is a randomized, double-blind, dose-ranging study of the efficacy and safety of DEX when used with PRO as needed, for procedural sedation of pediatric subjects ≥1 month to <17 years of age undergoing MRI scans in the US and Japan.

Kumar Belani
All
1 Month to 16 Years old
Phase 4
This study is NOT accepting healthy volunteers
NCT04237792
STUDY00008334
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Key
Inclusion Criteria:

• Male or female subject ≥1 month and <17 years of age.
• American Society of Anesthesiologists (ASA) Physical Status I, II or III.
• Requires non-intubated, spontaneous breathing, moderate to deep sedation (NI MDS) for a magnetic resonance imaging (MRI) study with an intensivist, anesthesiologist or other proceduralist in attendance.
• Duration of the MRI scan is expected to take at least 20 minutes but no more than 3 hours to complete Key
Exclusion Criteria:

• Pregnant female subjects (including those with an indeterminate or positive pregnancy test); breastfeeding female subjects.
• Weight on Day 1 before randomization is less than the 10th percentile of weight for age and sex in the US and Japan or is greater than the 95th percentile of weight for age and sex in the US or greater than the 97th percentile of weight for age and sex in Japan based on sponsor-provided growth charts.
• Planned medical procedure during the MRI scan or post-MRI recovery period.
• Requires endotracheal intubation or laryngeal mask airway (LMA).
• Known allergy to eggs, egg products, soybeans or soybean products.
• SpO2 <93 % on room air -
Drug: dexmedetomidine, Drug: propofol
MRI Sedation
procedural sedation, dexmedetomidine, propofol, MRI
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HYDROXYCHLOROQUINE (HCQ) FOR PREVENTION OF ABNORMAL GLUCOSE TOLERANCE AND DIABETES IN RELATIVES AT-RISK FOR TYPE 1 DIABETES MELLITUS (Protocol TN-22) (TN-22)

The study is a 2-arm, double blinded, multicenter, 2:1 randomized, placebo-controlled clinical trial. All participants will receive close monitoring for progression of T1D. Participants will receive hydroxychloroquine or placebo and close monitoring for progression to Stage 2 (abnormal glucose tolerance) or Stage 3 (clinically overt) T1D. To assess the efficacy, safety and mode of action of hydroxychloroquine to prevent progression from Stage 1 to Stage 2 or Stage 3 of T1D.

Antoinette Moran
All
3 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT03428945
STUDY00004135
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Inclusion Criteria:

• Participant in TrialNet Pathway to Prevention Study (TN01)
• Age 3 years or greater at the time of randomization
• Willing to provide informed consent
• Normal glucose tolerance by OGTT within 7 weeks (no more than 52 days) of baseline
• Two or more diabetes-related autoantibodies present on two separate samples
• Weight of 12 kg or greater at screening
• If a female participant with reproductive potential, willing to avoid pregnancy and undergo pregnancy testing prior to randomization and at each study visit
• Anticipated ability to swallow study medication.
Exclusion Criteria:

• Abnormal Glucose Tolerance or Diabetes
• History of treatment with insulin or other diabetes therapies
• Ongoing use of medications known to influence glucose tolerance
• Ongoing or anticipated future use of medications known to have untoward interactions with hydroxychloroquine
• Known hypersensitivity to 4-aminoquinoline compounds
• G6PD deficiency
• History of retinopathy
• Have an active infection at time of randomization
• Have serologic evidence of current or past HIV, Hepatitis B (positive for Hepatitis B core antibody or surface antigen), or Hepatitis C infection
• Deemed unlikely or unable to comply with the protocol or have any complicating medical issues, including prolonged QT interval, a disease previously or likely in the future to require immunosuppression, or abnormal clinical laboratory results that interfere with study conduct or cause increased risk.
• Deemed unlikely or unable to comply with the protocol or have any complicating medical issues, including prolonged QT interval, a disease previously or likely in the future to require immunosuppression, or abnormal clinical laboratory results that interfere with study conduct or cause increased risk.
• Be pregnant or breastfeeding.
Drug: Hydroxychloroquine, Drug: Placebo
Type1 Diabetes Mellitus
TrialNet
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MT2020-06: A PHASE 1/2 STUDY TO EVALUATE THE SAFETY, TOLERABILITY, AND EFFICACY OF JSP191 FOR HEMATOPOIETIC CELL TRANSPLANTATION CONDITIONING TO ACHIEVE ENGRAFTMENT AND IMMUNE RECONSTITUTION IN SUBJECTS WITH SCID

Phase 1: To evaluate the safety and tolerability of JSP191 and to determine Phase 2 doses of JSP191 as a conditioning agent prior to allogeneic hematopoietic cell transplantation (HCT) in two populations of subjects with severe combined immunodeficiency (SCID): • SCID subjects with history of prior allogeneic HCT but with poor graft function • SCID subjects who are HCT-naïve Phase 2: • To evaluate the efficacy of JSP191 conditioning to enable engraftment of allogeneic CD34+ hematopoietic cells, as determined by CD15+ donor myeloid chimerism • To evaluate the efficacy of JSP191 conditioning to enable immune reconstitution determined by the production of naïve T cells

Christen Ebens
All
3 Months and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT02963064
STUDY00010559
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Key
Inclusion Criteria:
All patient groups must have:
• Typical SCID as defined by Primary Immune Deficiency Treatment Consortia including but not limited to the following subtypes:
• T-, B+, NK-: IL-2Rcγ deficient, JAK3-deficient
• T-, B-, NK+: RAG1/2 deficient, Artemis-deficient
• T-, B+, NK+: IL7Rα deficient, CD3 subunit deficient, CD45 deficient OR Variant SCID with absent or low T cell function, Omenn syndrome, Leaky SCID, Reticular dysgenesis, Adenosine deaminase deficiency, and Purine nucleoside phosphorylase deficiency may be included after consultation with the medical monitor.
• Patients with human leukocyte antigen (HLA) matched related or unrelated donors
• Adequate end organ function as defined in study protocol Key
Exclusion Criteria:

• Patients with any acute or uncontrolled infections
• Patients receiving any other investigational agents, or concurrent biological, chemotherapy, or radiation therapy
• Patients with active malignancies
• Active GVHD within 6 months prior to enrollment, or on immunosuppressive therapy for GVHD
Biological: Humanized anti-CD117 Monoclonal Antibody (JSP191)
SCID
Immunodeficiency, Pediatric, SCID, Bone Marrow Transplantation, GVHD, Stem Cells, Chimerism, Transplant, BMT
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VX18-445-110: A Phase 3, Open-label Study Evaluating the Long term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

This is an open-label study for patients rolling over from VX18-445-104. This study will evaluate the long-term safety and efficacy of VX-445 combination therapy in subjects with CF.

Joanne Billings
All
12 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT04058366
STUDY00006925
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Key
Inclusion Criteria:

• Completed study drug treatment in parent study (VX18-445-104); or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study Key
Exclusion Criteria:

• History of study drug intolerance in parent study Other protocol defined Inclusion/Exclusion criteria may apply
Drug: ELX/TEZ/IVA, Drug: IVA
Cystic Fibrosis
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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)

This study is a randomized phase 3 study comparing selumetinib to Carboplatin and Vincristine (CV) in previously untreated NF1-associated LGG. This study will compare both the event-free survival (EFS) and visual functional outcomes between the 2 randomized arms.

Christopher Moertel, MD
All
2 Years to 21 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03871257
STUDY00008583
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Inclusion Criteria:

• 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
• 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 7 days prior to enrollment
• Corrected QT (QTc) interval =< 450 msec by electrocardiography (EKG) within 7 days 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 eligible
Drug: Carboplatin, Other: Quality-of-Life Assessment, Other: Questionnaire Administration, Drug: Selumetinib Sulfate, Drug: Vincristine Sulfate
Low Grade Glioma, Neurofibromatosis Type 1, Visual Pathway Glioma
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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
All
2 Years to 21 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04166409
STUDY00009277
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Inclusion Criteria:

• 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 7 days prior to enrollment)
• Corrected QT (QTc) interval =< 450 msec by electrocardiography (EKG) (performed within 7 days 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)
• All patients must have ophthalmology toxicity assessments performed within 4 weeks prior to 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 anti-hypertensive medications)
• Note for patients of all ages: Adequate blood pressure can be achieved using medication for the treatment of hypertension
• 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 eligible
Drug: Carboplatin, Other: Quality-of-Life Assessment, Other: Questionnaire Administration, Drug: Selumetinib Sulfate, Drug: Vincristine Sulfate
Low Grade Astrocytoma, Low Grade Glioma, Metastatic Low Grade Astrocytoma, Metastatic Low Grade Glioma
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Smart Use of Medication for the Treatment of Adolescent Severe Obesity (SMART)

This is a single site, 2-staged sequential multiple assignment randomized trial (SMART) that will systematically examine: 1) the optimal timing (12- versus 24 weeks) for identifying non-responders to lifestyle modification therapy (LSMT) before starting adjunct pharmacotherapy with phentermine and 2) for non-responders to LSMT+phentermine, the relative effect of adding topiramate to LMST+phentermine versus switching to LSMT+topiramate monotherapy. All participants will receive a total of 48 weeks of intervention.

Claudia Fox
All
12 Years to 17 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04007393
STUDY00006824
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Inclusion Criteria:

• Provision of signed and dated informed assent form;
• Provision of signed and dated informed parental consent form from at least 1 legal parent/guardian;
• Stated willingness to comply with all study procedures and availability for the duration of the study;
• BMI >/= 1.2 times the 95th percentile or BMI >/= 35 Kg/m2, whichever is lower;
• Tanner stage >/= 2;
• Male or female, aged 12-17 at time of consenting;
• For females of reproductive potential: when sexually active, agreement to use highly effective contraception (oral contraceptive pill, intra-uterine device (IUD), or implant) during study participation;
• For males of reproductive potential: use of condoms or other methods to ensure effective contraception with partner.
Exclusion Criteria:

• Contraindications to phentermine or topiramate use according to package inserts, including: history of glaucoma; current or recent (< 14 days) use of monoamine oxidase inhibitor; known hypersensitivity to sympathomimetic amines; current pregnancy, plans to become pregnant, or if sexually active refusal to use 2 forms of birth control; history of cardiac disease including coronary artery disease; clinically significant cardiac arrhythmias; heart failure or uncontrolled hypertension;
• Diabetes (type 1 or 2);
• Presence of cardiac pacemaker;
• Current or recent (<6 months prior to enrollment) use of weight loss medication(s);
• Current use of weight-altering medication(s) (e.g., atypical antipsychotic, metformin) unless dose has been stable for past 6 months;
• Current use of other sympathomimetic amine such as attention-deficit hyperactivity disorder (ADHD) stimulants;
• Seizure disorder (other than infantile febrile seizure);
• Previous bariatric surgery;
• Recent initiation of change in dose (< 3 months prior to enrollment) of anti-hypertensive or lipid medication(s);
• Tobacco use
• History of or current diagnosis of schizophrenia, psychosis, mania, chemical dependency;
• Unstable depression or anxiety that has required hospitalization in the past year;
• Any history of suicide attempt;
• Suicidal ideation or self-harm within 12 months prior to enrollment;
• Bicarbonate < 18 mmol/L;
• Creatinine > 1.2 mg/dL;
• History of cholelithiasis;
• History of nephrolithiasis;
• Untreated thyroid disorder;
• Hyperthyroidism;
• Breastfeeding
Behavioral: Lifestyle Modification Therapy (LSMT), Drug: Phentermine Pill, Drug: Topiramate Pill
Adolescent Obesity
Clinics and Surgery Center (CSC)
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Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia (HERO)

All
2 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT04732429
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Inclusion Criteria:

• Confirmed diagnosis of symptomatic PA or MMA (Mutase)
• Ages ≥ 2 years old.
• History of Inadequate metabolic control while receiving standard of care (SoC).
• Plasma MCA concentration > 3x upper limit of normal of the reference range at screening.
• Stable supplementation dose of carnitine for at least 1 week prior to the entry in the study.
Exclusion Criteria:

• Moderate-to-severely impaired cardiac function with LVEF < 45% by ECHO.
• Clinically significant arrhythmia by Holter monitor.
• QTcF > 450 msec
• Moderate to severe chronic kidney disease with estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73m2.
• Exposure to any investigational therapy, apart for a COVID-19 vaccine, within the past 6 months prior to study entry.
• Exposure to gene therapy for PA or MMA at any time prior to study entry.
• History of organ transplantation (Part A and B only)
• History of severe allergic or anaphylactic reactions to any of the components of HST5040.
Drug: HST5040, Drug: Placebo
Methylmalonic Acidemia, Propionic Acidemia
Methylmalonic Acidemia, Propionic Acidemia, Organic Acidemia, Inborn errors of metabolism, PCCA, PCCB, Propionyl-coenzyme A carboxylase, MMUT, Methylmalonyl-CoA mutase, Metabolic disease, Genetic disease, HemoShear
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A Randomized, Double-Blind, Multicenter, Placebo-Controlled Phase 3 Study with Open-Label Period to Evaluate the Efficacy and Safety of Inebilizumab in Adults with Myasthenia Gravis

This is a randomized, double-blind, placebo-controlled, parallel-group study with an optional open-label extension testing the safety and efficacy of inebilizumab in participants with Myasthenia Gravis.

Georgios Manousakis
Phase III
This study is NOT accepting healthy volunteers
NCT04524273
STUDY00010270
Myasthenia Gravis, Rare Diseases
Clinics and Surgery Center (CSC), Myasthenia Gravis
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