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ClinicalTrials.gov public records Last synced Oct 17, 2026, 5:18 AM EDT

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Showing 865–888 of 1,605 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Clear all
Withdrawn Phase 1 Interventional

Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases

NCT01003912
View directory record Official record
Conditions
Lysosomal Storage Diseases, Inborn Errors of Metabolism
Interventions
ALD-601
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
18 Years and older · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2017
U.S. locations
2
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 29, 2017 · Synced Oct 17, 2026, 5:18 AM EDT
Recruiting Not applicable Interventional

Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria

NCT06560736
View directory record Official record
Conditions
Phenylketonurias
Interventions
Show Me FIRST coping skills intervention
Behavioral
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
10 Years to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Dec 23, 2025 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 1Phase 2 Interventional Accepts healthy volunteers

First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria

NCT05781399
View directory record Official record
Conditions
Phenylketonuria
Interventions
JNT-517 Suspension, Placebo Suspension, JNT-517 Tablet, Placebo Tablet
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
163 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
11
States / cities
Gainesville, Florida • Tampa, Florida • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients

NCT01678898
View directory record Official record
Conditions
Fabry Disease
Interventions
PRX-102
Drug
Lead sponsor
Protalix
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
9
States / cities
Sacramento, California • Atlanta, Georgia • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Oct 17, 2026, 5:18 AM EDT
Completed No phase listed Observational

Longitudinal Studies of Brain Structure and Function in MPS Disorders

NCT01870375
View directory record Official record
Conditions
Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type IV, Mucopolysaccharidosis Type VII
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
6 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2019
U.S. locations
3
States / cities
Oakland, California • Minneapolis, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 6, 2019 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 2Phase 3 Interventional

A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset Pompe Disease

NCT00059280
View directory record Official record
Conditions
Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 26 Weeks
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
4
States / cities
Gainesville, Florida • Durham, North Carolina • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2014 · Synced Oct 17, 2026, 5:18 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?

NCT03150069
View directory record Official record
Conditions
Morquio Disease, MPS - Mucopolysaccharidosis
Interventions
Interview
Other
Lead sponsor
Emory University
Other
Eligibility
15 Years to 100 Years · Female only
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Decatur, Georgia
Source: ClinicalTrials.gov public record
Updated Jan 20, 2021 · Synced Oct 17, 2026, 5:18 AM EDT
Terminated Phase 2 Interventional

Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia

NCT04732429
View directory record Official record
Conditions
Methylmalonic Acidemia, Propionic Acidemia
Interventions
HST5040, Placebo
Drug
Lead sponsor
HemoShear Therapeutics
Industry
Eligibility
2 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
14
States / cities
San Diego, California • New Haven, Connecticut • Washington D.C., District of Columbia + 11 more
Source: ClinicalTrials.gov public record
Updated Jan 5, 2024 · Synced Oct 17, 2026, 5:18 AM EDT
Recruiting Phase 4 Interventional

SAVE-Care (Sodium Glucose Cotransporter-2 Inhibitors [SGLT2i] As Novel Gout Care) Trial

NCT06674109
View directory record Official record
Conditions
Gout and Hyperuricemia
Interventions
Empagliflozin 10 mg, Placebo
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 80 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Oct 17, 2026, 5:18 AM EDT
Terminated Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Manipulating the Gut Microbiome Study

NCT03181828
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
Acetohydroxamic Acid Oral Tablet, No treatment
Drug · Other
Lead sponsor
Nicholas Ah Mew
Other
Eligibility
18 Years to 60 Years
Enrollment
4 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Dec 15, 2021 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 3 Interventional

Tigulixostat, Phase 3 Study, Allopurinol Controlled in Gout Patients

NCT05586971
View directory record Official record
Conditions
Gout, Hyperuricemia, Gout Flare, Tophi
Interventions
Tigulixostat, Allopurinol, Placebo
Drug
Lead sponsor
LG Chem
Industry
Eligibility
18 Years to 85 Years
Enrollment
2,202 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
124
States / cities
Birmingham, Alabama • Daphne, Alabama • Mobile, Alabama + 91 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2025 · Synced Oct 17, 2026, 5:18 AM EDT
Recruiting No phase listed Observational

Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C

NCT05588167
View directory record Official record
Conditions
Niemann-Pick Disease, Type C
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
3 Months and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 2 Interventional Results available

Ponatinib for Patients Whose Advanced Solid Tumor Cancer Has Activating Mutations Involving the Following Genes: FGFR1, FGFR2, FGFR3, FGFR4, RET, KIT.

NCT02272998
View directory record Official record
Conditions
Malignant Neoplasm
Interventions
ponatinib hydrochloride, laboratory biomarker analysis
Drug · Other
Lead sponsor
Sameek Roychowdhury
Other
Eligibility
18 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2022
U.S. locations
2
States / cities
Ann Arbor, Michigan • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 24, 2025 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 2 Interventional Results available

A Study to Assess Efficay and Safety of LC350189 Different Doses in Gout Patients With Hyperuricemia

NCT03934099
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
LC350189 formulated capsule, Placebo
Drug
Lead sponsor
LG Chem
Industry
Eligibility
18 Years to 75 Years
Enrollment
156 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
34
States / cities
Birmingham, Alabama • Chandler, Arizona • Mesa, Arizona + 28 more
Source: ClinicalTrials.gov public record
Updated Dec 3, 2025 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 2 Interventional

Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies

NCT00763932
View directory record Official record
Conditions
Pompe Disease Infantile-Onset, Glycogen Storage Disease Type II
Interventions
Myozyme
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
5
States / cities
Oakland, California • Orange, California • Omaha, Nebraska + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2014 · Synced Oct 17, 2026, 5:18 AM EDT
Completed Phase 3 Interventional Results available

Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis

NCT01733316
View directory record Official record
Conditions
Cystinosis
Interventions
RP103, Cystagon®
Drug
Lead sponsor
Amgen
Industry
Eligibility
12 Years and older
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 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 17, 2026, 5:18 AM EDT
Terminated No phase listed Observational

Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Participants With Glycogen Storage Disease Type III

NCT05196165
View directory record Official record
Conditions
Glycogen Storage Disease Type III
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
1 Year and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
3
States / cities
Philadelphia, Pennsylvania • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 28, 2023 · Synced Oct 17, 2026, 5:18 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Biomarker Analysis for GBA Associated Parkinson's Disease

NCT03811496
View directory record Official record
Conditions
Parkinson Disease, Gaucher Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
50 Years and older
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jan 23, 2019 · Synced Oct 17, 2026, 5:18 AM EDT
Terminated Phase 1 Interventional

Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)

NCT00410566
View directory record Official record
Conditions
Acid Sphingomyelinase Deficiency, Niemann-Pick Disease
Interventions
rhASM
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 65 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 19, 2015 · Synced Oct 17, 2026, 5:18 AM EDT
Completed No phase listed Observational

Study of Glycogen Storage Disease Expression in Carriers

NCT02057731
View directory record Official record
Conditions
Glycogen Storage Disease
Interventions
Glycogen Storage Disease markers
Genetic
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 100 Years
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Dec 2, 2016 · Synced Oct 17, 2026, 5:18 AM EDT
Enrolling by invitation Phase 2 Interventional

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

NCT07393373
View directory record Official record
Conditions
Hypochondroplasia
Interventions
Infigratinib
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
3 Years to 18 Years
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2036
U.S. locations
8
States / cities
Oakland, California • Aurora, Colorado • Washington D.C., District of Columbia + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Oct 17, 2026, 5:18 AM EDT
Recruiting Phase 2Phase 3 Interventional

A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)

NCT07159841
View directory record Official record
Conditions
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
Interventions
Atumelnant, Placebo
Drug
Lead sponsor
Crinetics Pharmaceuticals Inc.
Industry
Eligibility
1 Year to 17 Years
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
10
States / cities
San Francisco, California • Chicago, Illinois • Boston, Massachusetts + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 17, 2026, 5:18 AM EDT
Recruiting Phase 3 Interventional

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

NCT06563895
View directory record Official record
Conditions
Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases, Polyneuropathies
Interventions
Acoramidis, Placebo oral tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 75 Years
Enrollment
587 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
40
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 31 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Oct 17, 2026, 5:18 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Genetic and Family Studies of Inherited Muscle Diseases

NCT00001331
View directory record Official record
Conditions
Dermatomyositis, Glycogen Storage Disease Type II, Glycogen Storage Disease Type VII, Myositis, Polymyositis
Interventions
Not listed
Lead sponsor
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
NIH
Eligibility
Not listed
Healthy volunteers
Accepts healthy volunteers
Timeline
1993 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 5, 2008 · Synced Oct 17, 2026, 5:18 AM EDT