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ClinicalTrials.gov public records Last synced May 22, 2026, 5:46 AM EDT

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

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Inherited Metabolic Disorders Clear all
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Anti-Thymocyte Globulin, Cyclophosphamide, Fludarabine, Hematopoietic Stem Cell Transplantation, Methylprednisolone, Filgrastim, Cyclosporine, Mycophenolate Mofetil
Biological · Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 59 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 11, 2021 · Synced May 22, 2026, 5:46 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
X-linked Adrenoleukodystrophy
Interventions
exercise training
Behavioral
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
21 Years to 70 Years · Female only
Enrollment
31 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2014
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 28, 2018 · Synced May 22, 2026, 5:46 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Gaucher Disease
Interventions
Standard of Care
Other
Lead sponsor
Takeda
Industry
Eligibility
Up to 5 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Mar 28, 2024 · Synced May 22, 2026, 5:46 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Wilson Disease
Interventions
Bis-Choline Tetrathiomolybdate
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
3
States / cities
Sacramento, California • Ann Arbor, Michigan • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Oct 17, 2024 · Synced May 22, 2026, 5:46 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers
View directory record Official record
Conditions
NAFLD
Interventions
NAFLD-specific weight loss intervention, Wait-list control
Behavioral · Other
Lead sponsor
University of Arizona
Other
Eligibility
18 Years to 64 Years · Male only
Enrollment
15 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2025
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Aug 23, 2025 · Synced May 22, 2026, 5:46 AM EDT
Completed Phase 4 Interventional Results available
View directory record Official record
Conditions
Gaucher Disease
Interventions
Velaglucerase alfa, Vitamin D
Drug · Dietary Supplement
Lead sponsor
Shire
Industry
Eligibility
18 Years to 70 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
8
States / cities
Beverly Hills, California • Los Angeles, California • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Jan 31, 2022 · Synced May 22, 2026, 5:46 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions
Not listed
Lead sponsor
xCures
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Los Altos, California
Source: ClinicalTrials.gov public record
Updated Nov 13, 2024 · Synced May 22, 2026, 5:46 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors, Urea Cycle Disorders
Interventions
Sodium Phenylbutyrate, Arginine
Drug
Lead sponsor
Brendan Lee
Other
Eligibility
5 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 30, 2018 · Synced May 22, 2026, 5:46 AM EDT
Terminated Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Gout
Interventions
ABP-671, Allopurinol
Drug
Lead sponsor
Atom Therapeutics Co., Ltd
Industry
Eligibility
19 Years to 75 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
33
States / cities
Tempe, Arizona • Tucson, Arizona • Anaheim, California + 24 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2025 · Synced May 22, 2026, 5:46 AM EDT
Completed Phase 2 Interventional Results available
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 8, 2013 · Synced May 22, 2026, 5:46 AM EDT
Completed Phase 3 Interventional
View directory record Official record
Conditions
Gout, Arthritis, Gouty, Hyperuricemia, Gout Chronic
Interventions
AR882 50 mg, AR882 75 mg, Placebo
Drug
Lead sponsor
Arthrosi Therapeutics
Industry
Eligibility
18 Years to 85 Years
Enrollment
818 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
52
States / cities
Foley, Alabama • Anchorage, Alaska • Peoria, Arizona + 47 more
Source: ClinicalTrials.gov public record
Updated May 12, 2026 · Synced May 22, 2026, 5:46 AM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases, Urologic Diseases, Genetic Disease, Primary Hyperoxaluria Type 3 (PH3)
Interventions
DCR-PHXC
Drug
Lead sponsor
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Industry
Eligibility
Not listed
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2030
U.S. locations
4
States / cities
San Francisco, California • Boston, Massachusetts • Rochester, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated May 29, 2025 · Synced May 22, 2026, 5:46 AM EDT
Approved For Marketing No phase listed Expanded access
View directory record Official record
Conditions
Fabry Disease
Interventions
Pegunigalsidase Alfa
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
18 Years and older
U.S. locations
11
States / cities
Birmingham, Alabama • Phoenix, Arizona • Orange, California + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2024 · Synced May 22, 2026, 5:46 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Generalised Lipodystrophy, Partial Lipodystrophy
Interventions
Metreleptin
Drug
Lead sponsor
Amryt Pharma
Industry
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2031
U.S. locations
16
States / cities
Birmingham, Alabama • Duarte, California • Atlanta, Georgia + 11 more
Source: ClinicalTrials.gov public record
Updated Jan 19, 2026 · Synced May 22, 2026, 5:46 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Endocrine Disease, Adrenal Insufficiency, Congenital Adrenal Hyperplasia
Interventions
Hydrocortisone Modified Release Capsules
Drug
Lead sponsor
Neurocrine UK Limited
Industry
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 16, 2017 · Synced May 22, 2026, 5:46 AM EDT
Terminated Phase 3 Interventional Results available
View directory record Official record
Conditions
Ataxia Telangiectasia, Genetic Syndrome
Interventions
EryDex System
Combination Product
Lead sponsor
Quince Therapeutics S.p.A.
Industry
Eligibility
6 Years and older
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
4
States / cities
Los Angeles, California • Baltimore, Maryland • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2024 · Synced May 22, 2026, 5:46 AM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
Prader-Willi Syndrome
Interventions
tVNS, intermittent stimulation, tVNS, continuous stimulation
Device
Lead sponsor
Foundation for Prader-Willi Research
Other
Eligibility
10 Years to 40 Years
Enrollment
102 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
18
States / cities
Palo Alto, California • San Diego, California • Aurora, Colorado + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 5, 2026 · Synced May 22, 2026, 5:46 AM EDT
Completed Not applicable Interventional Results available
View directory record Official record
Conditions
Hypercholesterolemia, Hypercholesterolemia, Familial, Hypercholesteremia in Children, Hyperlipidemia in Children
Interventions
Best Practice Alert, Health Maintenance Topic
Behavioral
Lead sponsor
Geisinger Clinic
Other
Eligibility
9 Years to 11 Years
Enrollment
13,340 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Danville, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 6, 2023 · Synced May 22, 2026, 5:46 AM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
DT-216, DT-216 matching Placebo
Drug
Lead sponsor
Design Therapeutics, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Mar 27, 2023 · Synced May 22, 2026, 5:46 AM EDT
Completed Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 22, 2016 · Synced May 22, 2026, 5:46 AM EDT
Recruiting Not applicable Interventional
View directory record Official record
Conditions
Pseudohypoparathyroidism Type 1A, Albright Hereditary Osteodystrophy, Pseudopseudohypoparathyroidism
Interventions
Neurocognitive and psychosocial testing
Behavioral
Lead sponsor
Connecticut Children's Medical Center
Other
Eligibility
2 Months to 89 Years
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2030
U.S. locations
1
States / cities
Hartford, Connecticut
Source: ClinicalTrials.gov public record
Updated May 27, 2025 · Synced May 22, 2026, 5:46 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Wilson Disease
Interventions
UX701, Standard of Care (SOC)
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2034
U.S. locations
11
States / cities
Los Angeles, California • Redwood City, California • Sacramento, California + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced May 22, 2026, 5:46 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
F-18 florbetapir PET
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
23 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 19, 2025 · Synced May 22, 2026, 5:46 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Phenylketonuria
Interventions
Not listed
Lead sponsor
Shoji Yano
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jan 17, 2013 · Synced May 22, 2026, 5:46 AM EDT