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ClinicalTrials.gov public records Last synced Oct 23, 2026, 1:42 PM EDT

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Showing 457–480 of 1,510 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Disorders Clear all
Completed No phase listed Observational

Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

NCT03609840
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immune Deficiency, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
Interventions
Thiotepa
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Sep 21, 2023 · Synced Oct 23, 2026, 1:42 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 23, 2026, 1:42 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Diagnosis and Treatment of Patients With Inborn Errors of Metabolism

NCT00369421
View directory record Official record
Conditions
Arterial Calcification Due to Deficiency of CD73
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 115 Years
Enrollment
4,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1978
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 23, 2026, 1:42 PM EDT
Terminated Phase 1 Interventional Results available

A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

NCT04669535
View directory record Official record
Conditions
Tay-Sachs Disease, Sandhoff Disease
Interventions
AXO-AAV-GM2 Starting Dose, AXO-AAV-GM2 Low Dose, AXO-AAV-GM2 Middle Dose, AXO-AAV-GM2 High Dose
Biological
Lead sponsor
Terence Flotte
Other
Eligibility
6 Months to 12 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
2
States / cities
Boston, Massachusetts • Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 2, 2025 · Synced Oct 23, 2026, 1:42 PM EDT
Completed Phase 2Phase 3 Interventional Accepts healthy volunteers Results available

Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

NCT02004691
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
placebo (saline), Olipudase alfa
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2023
U.S. locations
3
States / cities
San Francisco, California • Decatur, Georgia • The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Nov 7, 2024 · Synced Oct 23, 2026, 1:42 PM EDT
Recruiting No phase listed Observational

Prospective Clinical Assessment Study in Children With Hypochondroplasia

NCT06410976
View directory record Official record
Conditions
Hypochondroplasia
Interventions
Not listed
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
30 Months to 16 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
8
States / cities
Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Oct 23, 2026, 1:42 PM EDT
Completed Phase 3 Interventional Results available

Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)

NCT00947297
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
21
States / cities
Long Beach, California • Los Angeles, California • Stanford, California + 18 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2024 · Synced Oct 23, 2026, 1:42 PM EDT
Completed No phase listed Observational

DECT Study in Allopurinol-Treated Gout Patients

NCT02393560
View directory record Official record
Conditions
Gout
Interventions
DECT scan
Procedure
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 85 Years
Enrollment
223 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
9
States / cities
Irvine, California • Englewood, Colorado • Wheat Ridge, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2017 · Synced Oct 23, 2026, 1:42 PM EDT
Recruiting Phase 2Phase 3 Interventional

Study of Cannabidiol in Sanfilippo Syndrome

NCT06333041
View directory record Official record
Conditions
Sanfilippo Syndrome, Mucopolysaccharidosis III
Interventions
Epidiolex, Placebo
Drug
Lead sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Other
Eligibility
4 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 23, 2026, 1:42 PM EDT
Completed Phase 1 Interventional

Oral Nifedipine to Treat Iron Overload

NCT00712738
View directory record Official record
Conditions
Thalassemia, Iron Overload, Hemochromatosis
Interventions
Nifedipine, Phlebotomy, Urinalysis, Echocardiogram, Electrocardiogram
Drug · Procedure
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 23, 2026, 1:42 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I

NCT02702115
View directory record Official record
Conditions
MPS I
Interventions
SB-318
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
5 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jan 26, 2023 · Synced Oct 23, 2026, 1:42 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders

NCT03812055
View directory record Official record
Conditions
Lysosomal Storage Diseases
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jan 22, 2019 · Synced Oct 23, 2026, 1:42 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

NCT04020055
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl 150 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
3
States / cities
Atlanta, Georgia • Cleveland, Ohio • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 23, 2026, 1:42 PM EDT
Completed Phase 2 Interventional Results available

Study of the Safety and Effectiveness of Rilonacept for the Prevention of Gout Flares

NCT00610363
View directory record Official record
Conditions
Gout
Interventions
Rilonacept, Placebo (for Rilonacept)
Biological · Other
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
83 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
34
States / cities
San Diego, California • Santa Maria, California • Whittier, California + 30 more
Source: ClinicalTrials.gov public record
Updated May 2, 2017 · Synced Oct 23, 2026, 1:42 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Fabry Disease Gene Therapy Study

NCT04040049
View directory record Official record
Conditions
Fabry Disease, Lysosomal Storage Diseases
Interventions
FLT190
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
4
States / cities
Los Angeles, California • New York, New York • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 23, 2026, 1:42 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Radicle GI Health RDH™: A Study Assessing the Impact of Health and Wellness Products on Gastrointestinal (GI) Health and Related Health Outcomes

NCT07230821
View directory record Official record
Conditions
Gas and Bloating, Diarrhea, Constipation
Interventions
GI Health Active Product 1, GI Health Product Placebo Control
Dietary Supplement
Lead sponsor
Radicle Science
Industry
Eligibility
21 Years to 105 Years
Enrollment
500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
Del Mar, California
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Oct 23, 2026, 1:42 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Genetic Studies of Lysosomal Storage Disorders

NCT00001215
View directory record Official record
Conditions
Lysosomal Storage Disorders, Gaucher Disease, Parkinson Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Week to 110 Years
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1995
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 23, 2026, 1:42 PM EDT
Recruiting No phase listed Observational

NYSCF Scientific Discovery Biobank

NCT06203106
View directory record Official record
Conditions
ALS, Amyotrophic Lateral Sclerosis, Alzheimer Disease, Alzheimer Disease, Early Onset, Alzheimer Disease, Late Onset, Batten Disease, Corticobasal Degeneration, Dementia, Frontotemporal Dementia, Huntington Disease, Lewy Body Disease, Multiple Sclerosis, Multiple System Atrophy, Parkinson Disease, Parkinson's Disease and Parkinsonism, Progressive Supranuclear Palsy, INAD, Diabetes, Diabetes Mellitus, Diabetes Mellitus, Type 2, Diabetes Mellitus, Type 1, Macular Degeneration, Ovarian Cancer, Cervical Cancer, Uterine Cancer, Vaginal Cancer, Vulvar Cancer, PTSD, Post Traumatic Stress Disorder
Interventions
Biological Sample Collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
30 Days and older
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2045
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 3, 2025 · Synced Oct 23, 2026, 1:42 PM EDT
Completed Phase 3 Interventional Results available

Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)

NCT01197378
View directory record Official record
Conditions
Cystinosis
Interventions
Cysteamine Bitartrate Delayed-release Capsules
Drug
Lead sponsor
Amgen
Industry
Eligibility
1 Year and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
5
States / cities
San Francisco, California • Stanford, California • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 23, 2026, 1:42 PM EDT
Completed No phase listed Observational

Identification of Tongue Involvement in Late-Onset Pompe Disease

NCT02765828
View directory record Official record
Conditions
Myopathy, Neuropathy, Glycogen Storage Disease Type II (Late-onset Pompe Disease)
Interventions
Observational study
Other
Lead sponsor
Duke University
Other
Eligibility
12 Years and older
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 6, 2021 · Synced Oct 23, 2026, 1:42 PM EDT
Completed No phase listed Observational

Clinical Studies of Progeria

NCT00094393
View directory record Official record
Conditions
Progeria
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
6 Months to 70 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 15, 2019 · Synced Oct 23, 2026, 1:42 PM EDT
Recruiting Phase 3 Interventional

A Gaucher Disease Gene Therapy Trial With FLT201

NCT07223944
View directory record Official record
Conditions
Gaucher Disease Type 1
Interventions
FLT201
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
15
States / cities
Los Angeles, California • San Francisco, California • Aurora, Colorado + 11 more
Source: ClinicalTrials.gov public record
Updated Oct 14, 2026 · Synced Oct 23, 2026, 1:42 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Oxidative Stress Markers In Inherited Homocystinuria And The Impact Of Taurine

NCT01192828
View directory record Official record
Conditions
Homocystinuria
Interventions
taurine
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
8 Years to 49 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
4
States / cities
Aurora, Colorado • Durham, North Carolina • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 9, 2018 · Synced Oct 23, 2026, 1:42 PM EDT
Completed Phase 2Phase 3 Interventional Results available

The Effect of Diflunisal on Familial Amyloidosis

NCT00294671
View directory record Official record
Conditions
Familial Amyloid Polyneuropathy, Familial Amyloidosis
Interventions
diflunisal, placebo
Drug · Other
Lead sponsor
Boston University
Other
Eligibility
18 Years to 75 Years
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
3
States / cities
Boston, Massachusetts • Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 17, 2017 · Synced Oct 23, 2026, 1:42 PM EDT