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ClinicalTrials.gov public records Last synced Oct 15, 2026, 10:59 AM EDT

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Showing 433–456 of 1,605 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Clear all
Completed No phase listed Observational

The Effects of Respiratory Muscle Strength Training (RMST) on Inspiratory and Expiratory Muscle Strength in Adults and Children With Pompe Disease

NCT01701154
View directory record Official record
Conditions
Pompe Disease
Interventions
respiratory muscle strength training
Other
Lead sponsor
Duke University
Other
Eligibility
3 Years to 99 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 30, 2023 · Synced Oct 15, 2026, 10:59 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Allergy and Immunology Natural History Study

NCT05584488
View directory record Official record
Conditions
Immune Deficiency, Immune Dysregulation Disorder, Allergic Inflammation
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Up to 99 Years
Enrollment
10,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Oct 15, 2026, 10:59 AM EDT
Completed Phase 3 Interventional Results available

Open-Label Lesinurad Monotherapy Extension Study in Gout

NCT01650246
View directory record Official record
Conditions
Gout
Interventions
lesinurad
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 85 Years
Enrollment
143 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
57
States / cities
Birmingham, Alabama • Glendale, Arizona • Peoria, Arizona + 51 more
Source: ClinicalTrials.gov public record
Updated May 26, 2016 · Synced Oct 15, 2026, 10:59 AM EDT
Completed No phase listed Observational

Clinical Assessment Study in Crigler-Najjar Syndrome

NCT03078881
View directory record Official record
Conditions
Crigler-Najjar Syndrome
Interventions
Not listed
Lead sponsor
Audentes Therapeutics
Industry
Eligibility
1 Year and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
2
States / cities
The Bronx, New York • Strasburg, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 6, 2019 · Synced Oct 15, 2026, 10:59 AM EDT
Completed No phase listed Observational Results available

A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

NCT04281537
View directory record Official record
Conditions
Fabry Disease
Interventions
Enzyme Replacement Therapy (ERT)
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
82 participants
Timeline
2020 – 2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 5, 2026 · Synced Oct 15, 2026, 10:59 AM EDT
Completed Phase 3 Interventional Results available

Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis

NCT01744782
View directory record Official record
Conditions
Cystinosis
Interventions
RP103
Drug
Lead sponsor
Amgen
Industry
Eligibility
Up to 6 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 15, 2026, 10:59 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants

NCT04526210
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840, Bupropion Hydrochloride
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
54 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Aug 31, 2023 · Synced Oct 15, 2026, 10:59 AM EDT
Recruiting No phase listed Observational

Natural History Study of Patients With Excess Androgen

NCT00250159
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
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
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2006
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 15, 2026, 10:59 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Bioequivalence Study of Colchicine Tablets

NCT01021020
View directory record Official record
Conditions
Healthy
Interventions
Colchicine, Colchicine/Probenecid
Drug
Lead sponsor
Mutual Pharmaceutical Company, Inc.
Industry
Eligibility
18 Years to 45 Years
Enrollment
28 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007
U.S. locations
1
States / cities
Fargo, North Dakota
Source: ClinicalTrials.gov public record
Updated Dec 9, 2009 · Synced Oct 15, 2026, 10:59 AM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

Impact of B. Adolescentis iVS-1 Probiotic on Gastrointestinal Outcomes

NCT07408518
View directory record Official record
Conditions
Digestive Disease, Gut Health
Interventions
Probiotic, Placebo
Dietary Supplement · Other
Lead sponsor
Synbiotic Health
Industry
Eligibility
18 Years to 75 Years
Enrollment
92 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 1, 2026 · Synced Oct 15, 2026, 10:59 AM EDT
Completed No phase listed Observational

A Longitudinal Study of Hermansky-Pudlak Syndrome Pulmonary Fibrosis

NCT02368340
View directory record Official record
Conditions
Hermansky Pudlak Syndrome
Interventions
Pulmonary function test, Chest CT, Sample collection
Other
Lead sponsor
Vanderbilt University
Other
Eligibility
12 Years to 90 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
5
States / cities
Maywood, Illinois • Boston, Massachusetts • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2020 · Synced Oct 15, 2026, 10:59 AM 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 15, 2026, 10:59 AM EDT
Completed Not applicable Interventional

A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

NCT04106544
View directory record Official record
Conditions
Sphingomyelin Lipidosis
Interventions
Investigational Procedures
Procedure
Lead sponsor
Sanofi
Industry
Eligibility
Not listed
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
3
States / cities
Atlanta, Georgia • The Bronx, New York • Valhalla, New York
Source: ClinicalTrials.gov public record
Updated Aug 16, 2023 · Synced Oct 15, 2026, 10:59 AM EDT
Completed Phase 1Phase 2 Interventional

Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)

NCT00004454
View directory record Official record
Conditions
Mucopolysaccharidosis II
Interventions
lymphocyte gene therapy
Genetic
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1996
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 15, 2026, 10:59 AM EDT
Terminated No phase listed Observational

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

NCT04628871
View directory record Official record
Conditions
Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
Interventions
SB-318, SB-913, SB-FIX
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 15, 2026, 10:59 AM EDT
Completed Phase 2 Interventional Results available

Study of REGN1500 in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

NCT02265952
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
REGN1500 250 mg SC/15 mg/kg IV/450 mg SC
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
3
States / cities
Los Angeles, California • Philadelphia, Pennsylvania • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Dec 9, 2019 · Synced Oct 15, 2026, 10:59 AM EDT
Completed No phase listed Observational

Screening Protocol for a Gene Therapy Trial in Subjects With Homozygous Familial Hypercholesterolemia

NCT03018678
View directory record Official record
Conditions
Hypercholesterolemia, Familial
Interventions
Not listed
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 6, 2020 · Synced Oct 15, 2026, 10:59 AM EDT
Recruiting No phase listed Observational

Natural History of MADD

NCT07734090
View directory record Official record
Conditions
Multiple Acyl-CoA Dehydrogenase Deficiency
Interventions
Not listed
Lead sponsor
Icahn School of Medicine at Mount Sinai
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
Aurora, Colorado • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Oct 15, 2026, 10:59 AM EDT
Completed No phase listed Observational

Studies of States With Resistance to Vitamin D and Parathyroid Hormone

NCT00001242
View directory record Official record
Conditions
Hypocalcemia, Pseudohypoparathyroidism, Osteomalacia, Rickets, Pseudopseudohpoparathyroidism
Interventions
Not listed
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
2 Months to 100 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 20, 2023 · Synced Oct 15, 2026, 10:59 AM EDT
Not listed No phase listed Observational

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

NCT03227042
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIB
Interventions
Not listed
Lead sponsor
Allievex Corporation
Industry
Eligibility
Up to 18 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Oct 26, 2022 · Synced Oct 15, 2026, 10:59 AM EDT
Completed Phase 1 Interventional

Safety and Pharmacodynamics of SEL-212 (Pegsiticase + SEL-110) in Subjects With Elevated Blood Uric Acid Levels

NCT02648269
View directory record Official record
Conditions
Gout
Interventions
SEL-110, SEL-212, SEL-037
Drug · Biological
Lead sponsor
Selecta Biosciences, Inc.
Industry
Eligibility
21 Years to 70 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
6
States / cities
Little Rock, Arkansas • Miami, Florida • Orlando, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 30, 2017 · Synced Oct 15, 2026, 10:59 AM EDT
Terminated Phase 4 Interventional Results available

The Role of F-18 Florbetapir in the Early Detection of Cardiac Amyloidosis

NCT03040427
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
F-18 florbetapir
Drug
Lead sponsor
The Cleveland Clinic
Other
Eligibility
18 Years to 95 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 27, 2021 · Synced Oct 15, 2026, 10:59 AM EDT
Completed Phase 3 Interventional

Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis

NCT05184088
View directory record Official record
Conditions
Cardiac Amyloidosis, AL Amyloidosis, ATTR Amyloidosis
Interventions
[18F]florbetaben
Drug
Lead sponsor
Lantheus Germany GmbH
Industry
Eligibility
18 Years and older
Enrollment
244 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
3
States / cities
Kansas City, Kansas • Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Oct 15, 2026, 10:59 AM EDT
Active, not recruiting Not applicable Interventional Results available

Gout in the ED and Improving Research Participation

NCT04075903
View directory record Official record
Conditions
Gout
Interventions
Storytelling
Behavioral
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
18 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
4
States / cities
Birmingham, Alabama • Iowa City, Iowa • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced Oct 15, 2026, 10:59 AM EDT