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ClinicalTrials.gov public records Last synced Oct 28, 2026, 4:03 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 649–672 of 1,511 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Disorders Clear all
Active, not recruiting Phase 2 Interventional

Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old

NCT04551170
View directory record Official record
Conditions
Pseudohypoparathyroidism, Albright Hereditary Osteodystrophy, Pseudohypoparathyroidism Type 1a
Interventions
Theophylline, Placebo
Drug
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
2 Years to 12 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 29, 2026 · Synced Oct 28, 2026, 4:03 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders

NCT02935283
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
MRI, Behavioral
Procedure · Behavioral
Lead sponsor
Children's National Research Institute
Other
Eligibility
7 Years to 50 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2025
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 25, 2024 · Synced Oct 28, 2026, 4:03 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 28, 2026, 4:03 PM EDT
Suspended Phase 1Phase 2 Interventional

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease

NCT04771416
View directory record Official record
Conditions
Leukodystrophy, Globoid Cell
Interventions
PBKR03
Biological
Lead sponsor
Gemma Biotherapeutics
Industry
Eligibility
1 Month to 9 Months
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
4
States / cities
Chicago, Illinois • New York, New York • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Oct 28, 2026, 4:03 PM EDT
Enrolling by invitation Phase 2 Interventional Accepts healthy volunteers

Theophylline for Treatment of Pseudohypoparathyroidism

NCT04240821
View directory record Official record
Conditions
Pseudohypoparathyroidism, Pseudohypoparathyroidism Type 1a, Albright Hereditary Osteodystrophy
Interventions
Theophylline ER
Drug
Lead sponsor
Jaclyn Tamaroff
Other
Eligibility
2 Years to 99 Years
Enrollment
34 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 28, 2026, 4:03 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

NCT03454893
View directory record Official record
Conditions
Fabry Disease
Interventions
AVR-RD-01
Drug
Lead sponsor
AVROBIO
Industry
Eligibility
16 Years to 50 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
2
States / cities
Hackensack, New Jersey • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 5, 2024 · Synced Oct 28, 2026, 4:03 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Safety and Effect of L. Reuteri on Biomarkers of Inflammation in Healthy Infants With Colic

NCT01849991
View directory record Official record
Conditions
Gastrointestinal, Colic
Interventions
Lactobacillus reuteri, Placebo
Biological
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
3 Weeks to 3 Months
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 30, 2018 · Synced Oct 28, 2026, 4:03 PM EDT
Recruiting No phase listed Observational

Natural History Study of Patients With Hypophosphatasia (HPP)

NCT02237625
View directory record Official record
Conditions
Hypophosphatasia
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2028
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Oct 28, 2026, 4:03 PM EDT
Completed No phase listed Observational

MRI QSM Imaging for Iron Overload

NCT04631718
View directory record Official record
Conditions
Hemochromatosis, Iron Overload
Interventions
Magnetic Resonance Imaging (MRI)-based Quantitative Susceptibility Mapping (QSM)
Radiation
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
5 Years and older
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
2
States / cities
Stanford, California • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated May 9, 2024 · Synced Oct 28, 2026, 4:03 PM EDT
Completed No phase listed Observational

Study of Megakaryocytes From Patients With Abnormal Platelet Vesicles

NCT00086476
View directory record Official record
Conditions
Blood Coagulation Disorders
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2011
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 28, 2026, 4:03 PM EDT
Completed Phase 1 Interventional

Bone Marrow Transplant With Abatacept for Non-Malignant Diseases

NCT01917708
View directory record Official record
Conditions
Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Severe Congenital Neutropenia, Leukocyte Adhesion Deficiency, Shwachman-Diamond Syndrome, Diamond-Blackfan Anemia, Dyskeratosis-congenita, Chediak-Higashi Syndrome, Severe Aplastic Anemia, Thalassemia Major, Hemophagocytic Lymphohistiocytosis, Sickle Cell Disease
Interventions
Abatacept
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 21 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Dec 26, 2019 · Synced Oct 28, 2026, 4:03 PM EDT
Completed Phase 4 Interventional

A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients

NCT00144768
View directory record Official record
Conditions
Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie's Syndrome
Interventions
laronidase
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
3
States / cities
Los Angles, California • Hartford, Connecticut • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated May 5, 2014 · Synced Oct 28, 2026, 4:03 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 28, 2026, 4:03 PM EDT
Recruiting No phase listed Observational

An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

NCT05040178
View directory record Official record
Conditions
Hyperammonemia, Methylmalonic Acidemia, Propionic Acidemia
Interventions
Carglumic Acid
Drug
Lead sponsor
RECORDATI GROUP
Industry
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2032
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Tampa, Florida • Chicago, Illinois + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 15, 2025 · Synced Oct 28, 2026, 4:03 PM EDT
Terminated Phase 4 Interventional Results available

Synergistic Enteral Regimen for Treatment of the Gangliosidoses

NCT02030015
View directory record Official record
Conditions
GM1 Gangliosidoses, GM2 Gangliosidoses, Tay-Sachs Disease, Sandhoff Disease
Interventions
miglustat, Ketogenic Diet
Drug · Other
Lead sponsor
University of Minnesota
Other
Eligibility
Up to 204 Months
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 14, 2021 · Synced Oct 28, 2026, 4:03 PM EDT
Completed Phase 3 Interventional Results available

Three Drug Combination Therapy Versus Conventional Treatment of Children With Congenital Adrenal Hyperplasia

NCT00001521
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia (CAH)
Interventions
Fludrocortisone, Hydrocortisone, Letrozole, Flutamide, Testolactone
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 18 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2024
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 12, 2025 · Synced Oct 28, 2026, 4:03 PM EDT
Recruiting Phase 1 Interventional

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

NCT04532047
View directory record Official record
Conditions
MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
Interventions
Aldurazyme (laronidase)
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Oct 28, 2026, 4:03 PM EDT
Recruiting No phase listed Observational

National Ophthalmic Genotyping and Phenotyping Network (eyeGENE (Registered Trademark)), Stage 3 - Expansion of DNA and Data Repositories for Rare Inherited Ophthalmic Diseases

NCT06491615
View directory record Official record
Conditions
Inherited Ophthalmic Diseases, Hypopigmentation Disorder, Corneal Dystrophy, Blue-cone Monochromacy, Best Disease, Aniridia, Albinism
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
1 Day to 120 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2054
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Oct 28, 2026, 4:03 PM EDT
Recruiting No phase listed Observational

Clinical and Basic Investigations Into Known and Suspected Congenital Disorders of Glycosylation

NCT02089789
View directory record Official record
Conditions
Congenital Disorders of Glycosylation
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 80 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 26, 2026 · Synced Oct 28, 2026, 4:03 PM EDT
Completed Phase 3 Interventional Results available

β-RELIEVED - REsponse in Acute fLare and In prEVEntion of episoDes of Re-flare in Gout - Extension 3 (E3)

NCT01470989
View directory record Official record
Conditions
Acute Gouty Arthritis Flares
Interventions
ACZ885, Triamcinolone acetonide 40 mg
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
136 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
33
States / cities
Anniston, Alabama • Mobile, Alabama • Norwalk, California + 28 more
Source: ClinicalTrials.gov public record
Updated Jul 9, 2021 · Synced Oct 28, 2026, 4:03 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

RDEA3170 Bioavailability Study

NCT02336594
View directory record Official record
Conditions
Gout
Interventions
RDEA3170 10 mg, RDEA3170 2.5 mg
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2015
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Oct 13, 2017 · Synced Oct 28, 2026, 4:03 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders

NCT01624142
View directory record Official record
Conditions
Severe Familial Hypercholesterolemia
Interventions
Evolocumab
Biological
Lead sponsor
Amgen
Industry
Eligibility
12 Years to 80 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
6
States / cities
Los Angeles, California • New York, New York • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated May 28, 2024 · Synced Oct 28, 2026, 4:03 PM EDT
Completed Phase 1 Interventional

Study to Evaluate Safety, Tolerability, PK and PD of DCR-PHXC in PH Type 3 Patients

NCT04555486
View directory record Official record
Conditions
Primary Hyperoxaluria Type 3
Interventions
DCR-PHXC, Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Industry
Eligibility
6 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
3
States / cities
Boston, Massachusetts • Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 28, 2026, 4:03 PM EDT
Completed Not applicable Interventional Results available

Cardiac Amyloidosis Discovery Trial

NCT06469372
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
Cardiac amyloidosis deep learning model
Device
Lead sponsor
Pierre Elias
Other
Eligibility
50 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 4, 2025 · Synced Oct 28, 2026, 4:03 PM EDT