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ClinicalTrials.gov public records Last synced Oct 10, 2026, 5:56 PM EDT

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Showing 145–168 of 2,374 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Congenital Metabolic Disorder Clear all
Active, not recruiting No phase listed Observational

Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

NCT04098211
View directory record Official record
Conditions
Neuronal Ceroid-Lipofuscinoses, Neuronal Ceroid Lipofuscinosis CLN2, Spinocerebellar Ataxia, Autosomal Recessive 7
Interventions
Not listed
Lead sponsor
Children's Hospital of Orange County
Other
Eligibility
4 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 10, 2026, 5:56 PM EDT
Completed Not applicable Interventional Results available

Zinc & Bone Health in Thalassemia: The Think Zinc Study

NCT00459732
View directory record Official record
Conditions
Thalassemia
Interventions
Zinc, Placebo
Dietary Supplement
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
6 Years to 30 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2011
U.S. locations
3
States / cities
Oakland, California • San Francisco, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 4, 2020 · Synced Oct 10, 2026, 5:56 PM EDT
Completed Phase 3 Interventional Results available

Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

NCT00599326
View directory record Official record
Conditions
Porphyria Cutanea Tarda
Interventions
Deferasirox
Drug
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Feb 10, 2014 · Synced Oct 10, 2026, 5:56 PM EDT
Active, not recruiting Phase 1 Interventional

Pembrolizumab in Treating Younger Patients With Recurrent, Progressive, or Refractory High-Grade Gliomas, Diffuse Intrinsic Pontine Gliomas, Hypermutated Brain Tumors, Ependymoma or Medulloblastoma

NCT02359565
View directory record Official record
Conditions
Constitutional Mismatch Repair Deficiency Syndrome, Lynch Syndrome, Malignant Glioma, Recurrent Brain Neoplasm, Recurrent Childhood Ependymoma, Recurrent Diffuse Intrinsic Pontine Glioma, Recurrent Medulloblastoma, Refractory Brain Neoplasm, Refractory Diffuse Intrinsic Pontine Glioma, Refractory Ependymoma, Refractory Medulloblastoma
Interventions
Biospecimen Collection, Conventional Magnetic Resonance Imaging, Diffusion Tensor Imaging, Diffusion Weighted Imaging, Dynamic Contrast-enhanced MR Perfusion, Dynamic Susceptibility Contrast-Perfusion-Weighted Imaging, Magnetic Resonance Spectroscopic Imaging, Pembrolizumab
Procedure · Biological
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Year to 30 Years
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2027
U.S. locations
11
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Oct 10, 2026, 5:56 PM EDT
Completed Not applicable Interventional

Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD

NCT03064048
View directory record Official record
Conditions
Argininosuccinate Lyase Deficiency, Urea Cycle Disorder, Urea Cycle Disorders, Inborn, Argininosuccinic Aciduria
Interventions
Neo-ASA, Placebo
Dietary Supplement
Lead sponsor
Baylor College of Medicine
Other
Eligibility
6 Years to 50 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 27, 2023 · Synced Oct 10, 2026, 5:56 PM EDT
Completed Phase 3 Interventional Results available

Evaluate the Neurological Effects of EryDex on Subjects With A-T

NCT06193200
View directory record Official record
Conditions
Ataxia Telangiectasia
Interventions
Dexamethasone sodium phosphate, Placebo
Drug · Other
Lead sponsor
Quince Therapeutics S.p.A.
Industry
Eligibility
6 Years and older
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
4
States / cities
Los Angeles, California • Baltimore, Maryland • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced Oct 10, 2026, 5:56 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Obesity and Oral Contraceptive Failure

NCT01944306
View directory record Official record
Conditions
Contraception, Fetal Growth Retardation, Infant, Small for Gestational Age, Obesity
Interventions
Not listed
Lead sponsor
Oregon Health and Science University
Other
Eligibility
18 Years to 35 Years · Female only
Enrollment
26 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2015
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Dec 13, 2019 · Synced Oct 10, 2026, 5:56 PM EDT
Completed No phase listed Observational

Genetic Analysis of the Chiari I Malformation

NCT00004738
View directory record Official record
Conditions
Syringomyelia, Type I Arnold Chiari Malformation
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
1 Year and older
Enrollment
152 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 10, 2026, 5:56 PM EDT
Recruiting Not applicable Interventional

Cooking Skills to Improve Long-Term Weight Loss in Young Adults With Intellectual Disabilities

NCT06961591
View directory record Official record
Conditions
Intellectual Disability, Overweight and Obesity
Interventions
Weight Loss Plus Cooking, Traditional Weight Loss
Behavioral
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
18 Years to 35 Years
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Kansas City, Kansas
Source: ClinicalTrials.gov public record
Updated Aug 11, 2026 · Synced Oct 10, 2026, 5:56 PM EDT
Completed No phase listed Observational

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

NCT01451879
View directory record Official record
Conditions
Pompe Disease
Interventions
Rituximab, Miglustat
Drug
Lead sponsor
University of Florida
Other
Eligibility
Up to 65 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Dec 8, 2017 · Synced Oct 10, 2026, 5:56 PM EDT
Completed Not applicable Interventional Results available

Weight Management for Adolescents With IDD

NCT02561754
View directory record Official record
Conditions
Developmental Disabilities, Weight Loss, Body Weight, Down Syndrome, Body Weight Changes, Intellectual Disability
Interventions
Enhanced Stop Light Diet (eSLD), Conventional Diet (CD), Face-to-face (FTF), Technology (TECH), iPad with FaceTime
Dietary Supplement · Behavioral · Other
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
13 Years to 21 Years
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
2
States / cities
Kansas City, Kansas • Lawrence, Kansas
Source: ClinicalTrials.gov public record
Updated Feb 10, 2023 · Synced Oct 10, 2026, 5:56 PM EDT
Completed Not applicable Interventional Results available

Repeated Eye Injections of Aflibercept for Treatment of Wet Age Related Macular Degeneration

NCT01773954
View directory record Official record
Conditions
Age-Related Macular Degeneration, Macular Degeneration
Interventions
Intravitreal Aflibercept Injection
Drug
Lead sponsor
MidAtlantic Retina
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
3
States / cities
Cherry Hill, New Jersey • Huntingdon Valley, Pennsylvania • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 2, 2018 · Synced Oct 10, 2026, 5:56 PM EDT
Completed Phase 1 Interventional

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

NCT03882437
View directory record Official record
Conditions
Danon Disease
Interventions
RP-A501
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 10, 2026, 5:56 PM EDT
Completed Phase 1 Interventional

Combination Iron Chelation Therapy

NCT00004982
View directory record Official record
Conditions
Iron Overload, Thalassemia
Interventions
Combination Iron Chelation Therapy
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
7 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2002
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 14, 2010 · Synced Oct 10, 2026, 5:56 PM EDT
Terminated No phase listed Observational

Oxidative Stress, Inflammation and Acute Decompensation in Urea Cycle Disorders

NCT01541722
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
Not listed
Lead sponsor
Mark Batshaw
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
10
States / cities
Los Angeles, California • Aurora, Colorado • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2015 · Synced Oct 10, 2026, 5:56 PM EDT
Completed Phase 4 Interventional Results available

A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders

NCT02246218
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
RAVICTI
Drug
Lead sponsor
Amgen
Industry
Eligibility
1 Day to 2 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
16
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 13 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2024 · Synced Oct 10, 2026, 5:56 PM EDT
Terminated No phase listed Observational

Study of Inborn Errors of Cholesterol Synthesis and Related Disorders

NCT00046202
View directory record Official record
Conditions
Lysosomal Storage Disease, Cholesterol Metabolism
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Day to 99 Years
Enrollment
344 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Oct 10, 2026, 5:56 PM EDT
Completed Phase 1Phase 2 Interventional

Beclomethasone in Treating Patients With Graft-Versus-Host Disease of the Esophagus, Stomach, Small Intestine, or Colon

NCT00010283
View directory record Official record
Conditions
Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Graft Versus Host Disease, Kidney Cancer, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Neuroblastoma, Ovarian Cancer, Testicular Germ Cell Tumor
Interventions
beclomethasone dipropionate
Drug
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
5 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2002
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Apr 2, 2010 · Synced Oct 10, 2026, 5:56 PM EDT
Terminated Phase 1Phase 2 Interventional

VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

NCT02898753
View directory record Official record
Conditions
Pompe Disease
Interventions
VAL-1221, RhGAA
Drug
Lead sponsor
Valerion Therapeutics, LLC
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
2
States / cities
Orange, California • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 2, 2020 · Synced Oct 10, 2026, 5:56 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Identifying and Caring for Individuals With Inherited Cancer Syndrome

NCT04494945
View directory record Official record
Conditions
BRCA1/2-Associated Hereditary Breast and Ovarian Cancer Syndrome, Breast Ductal Carcinoma In Situ, Hematopoietic and Lymphoid System Neoplasm, Hereditary Neoplastic Syndrome, Lynch Syndrome, Malignant Solid Neoplasm
Interventions
Biospecimen Collection, Genetic Counseling, Genetic Testing, Survey Administration
Procedure · Other
Lead sponsor
OHSU Knight Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
27,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2027
U.S. locations
2
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Oct 10, 2026, 5:56 PM EDT
Completed Phase 2 Interventional Results available

Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study

NCT00873041
View directory record Official record
Conditions
Non-transfusion Dependent Thalassemia
Interventions
deferasirox, placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
10 Years and older
Enrollment
166 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 9, 2013 · Synced Oct 10, 2026, 5:56 PM EDT
Completed Phase 3 Interventional Results available

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

NCT00891202
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
Eliglustat tartrate, Placebo
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
6
States / cities
San Francisco, California • New Haven, Connecticut • Decatur, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 3, 2017 · Synced Oct 10, 2026, 5:56 PM EDT
Completed Not applicable Interventional

A Multiple Dose Crossover Pharmacokinetics Study to Evaluate the Effects of Food on the Absorption of Oral Ganciclovir

NCT00002251
View directory record Official record
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years to 60 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
2
States / cities
San Francisco, California • Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 10, 2026, 5:56 PM EDT
Completed Phase 1 Interventional

Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP)

NCT00739505
View directory record Official record
Conditions
Hypophosphatasia (HPP)
Interventions
Asfotase Alfa
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
2
States / cities
St Louis, Missouri • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 29, 2019 · Synced Oct 10, 2026, 5:56 PM EDT