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ClinicalTrials.gov public records Last synced Oct 13, 2026, 2:08 AM EDT

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

Showing 241–264 of 1,605 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Clear all
Terminated No phase listed Observational

Kidney Information Network for Disease Research and Education

NCT03321604
View directory record Official record
Conditions
ESRD, Fabry Disease, Rare Diseases, Kidney Diseases, End Stage Renal Disease, Genetic Diseases, X-Linked
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 65 Years
Enrollment
338 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 24, 2021 · Synced Oct 13, 2026, 2:08 AM EDT
Recruiting Phase 1Phase 2 Interventional

An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705

NCT05295433
View directory record Official record
Conditions
Methylmalonic Acidemia
Interventions
mRNA-3705
Drug
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
1 Year and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2034
U.S. locations
3
States / cities
Los Angeles, California • Palo Alto, California • San Diego, California
Source: ClinicalTrials.gov public record
Updated Dec 23, 2025 · Synced Oct 13, 2026, 2:08 AM EDT
Withdrawn No phase listed Observational

International Registry for Primary Hyperoxaluria

NCT00875823
View directory record Official record
Conditions
Primary Hyperoxaluria, Nephrocalcinosis, Kidney Stones
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2009
U.S. locations
4
States / cities
Los Angeles, California • Sacramento, California • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 7, 2015 · Synced Oct 13, 2026, 2:08 AM EDT
Withdrawn Not applicable Interventional

Cross-over Pilot Study of Blood Cholesterol Response to Dietary Saturated Fat in Patients With Autosomal Dominant Hypercholesterolemia

NCT03756779
View directory record Official record
Conditions
Familial Hypercholesterolemia
Interventions
Dietary intervention
Other
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 80 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Nov 28, 2018 · Synced Oct 13, 2026, 2:08 AM EDT
Terminated Phase 2 Interventional Results available

Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia

NCT01597440
View directory record Official record
Conditions
Propionic Acidemia, Methylmalonic Acidemia
Interventions
N-carbamylglutamate, Standard of Care
Drug · Other
Lead sponsor
Mendel Tuchman
Other
Eligibility
1 Hour to 4 Weeks
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
7
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 26, 2017 · Synced Oct 13, 2026, 2:08 AM EDT
Recruiting No phase listed Observational

Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease

NCT02437396
View directory record Official record
Conditions
Gaucher Disease Type I, Oxidative Stress, Inflammation
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 75 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 13, 2026, 2:08 AM 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 13, 2026, 2:08 AM EDT
Recruiting Phase 1Phase 2 Interventional

DFT383 in Pediatric Participants With Nephropathic Cystinosis

NCT06910813
View directory record Official record
Conditions
Nephropathic Cystinosis
Interventions
DFT383
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 5 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2044
U.S. locations
6
States / cities
Phoenix, Arizona • San Diego, California • Stanford, California + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 13, 2026, 2:08 AM EDT
Recruiting Phase 2 Interventional

Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

NCT07169279
View directory record Official record
Conditions
Achondroplasia
Interventions
Infigratinib is provided as a single dose of minitablets for oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration, Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
Drug
Lead sponsor
QED Therapeutics, a BridgeBio company
Industry
Eligibility
0 Years to 32 Months
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
3
States / cities
Oakland, California • Baltimore, Maryland • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Oct 13, 2026, 2:08 AM EDT
Completed Phase 2Phase 3 Interventional Results available

A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome

NCT03098797
View directory record Official record
Conditions
Barth Syndrome
Interventions
Elamipretide, Placebo
Drug
Lead sponsor
Stealth BioTherapeutics Inc.
Industry
Eligibility
12 Years and older · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 16, 2024 · Synced Oct 13, 2026, 2:08 AM EDT
Terminated Not applicable Interventional Results available

SLOS: The Effect of Simvastatin in Patients Receiving Cholesterol Supplementation

NCT01434745
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Simvastatin, Lactose
Drug · Dietary Supplement
Lead sponsor
Oregon Health and Science University
Other
Eligibility
1 Year to 89 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 1, 2019 · Synced Oct 13, 2026, 2:08 AM EDT
Terminated Phase 2 Interventional

Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders

NCT01195753
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HHLivC
Biological
Lead sponsor
Cytonet GmbH & Co. KG
Industry
Eligibility
1 Day to 5 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
4
States / cities
Palo Alto, California • San Diego, California • New Haven, Connecticut + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 8, 2016 · Synced Oct 13, 2026, 2:08 AM EDT
Not listed Phase 2Phase 3 Interventional

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

NCT03612869
View directory record Official record
Conditions
Mucopolysaccharidosis Type IIIA
Interventions
LYS-SAF302
Drug
Lead sponsor
LYSOGENE
Industry
Eligibility
6 Months and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
4
States / cities
Orange, California • Minneapolis, Minnesota • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2021 · Synced Oct 13, 2026, 2:08 AM EDT
Completed No phase listed Observational

Visual Function and Ocular Pigmentation in Albinism

NCT00001153
View directory record Official record
Conditions
Albinism, Albinism, Ocular, Albinism, Oculocutaneous
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1976 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 13, 2026, 2:08 AM EDT
Terminated No phase listed Observational

Cardiovasculorenal Phenotyping in Fabry Disease Through Noninvasive Testing

NCT05699265
View directory record Official record
Conditions
Fabry Disease
Interventions
Measures of arterial stiffness and endothelial function, Ambulatory blood pressure monitoring, Cardiopulmonary exercise testing (CPET), Serum and urine biomarkers
Diagnostic Test
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
8 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 19, 2025 · Synced Oct 13, 2026, 2:08 AM EDT
Completed Phase 2 Interventional Results available

Dosing Study of Replagal in Patients With Fabry Disease

NCT00068107
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
Baylor Research Institute
Other
Eligibility
Male only
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 31, 2015 · Synced Oct 13, 2026, 2:08 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI

NCT05845749
View directory record Official record
Conditions
MPS IVA, MPS VI
Interventions
Vosoritide Injection [Voxzogo]
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
5 Years to 10 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Mar 10, 2026 · Synced Oct 13, 2026, 2:08 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

NCT03921541
View directory record Official record
Conditions
Arginase I Deficiency, Hyperargininemia
Interventions
Pegzilarginase, Placebo
Drug
Lead sponsor
Immedica Pharma AB
Industry
Eligibility
2 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
17
States / cities
Rogers, Arkansas • Orange, California • Stanford, California + 14 more
Source: ClinicalTrials.gov public record
Updated Aug 21, 2026 · Synced Oct 13, 2026, 2:08 AM EDT
Completed No phase listed Observational

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

NCT01387854
View directory record Official record
Conditions
MPS VI
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Apr 2, 2014 · Synced Oct 13, 2026, 2:08 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)

NCT07748403
View directory record Official record
Conditions
Wilson Disease, Wilson's Disease, Wilsons Disease
Interventions
PM577a
Drug
Lead sponsor
Prime Medicine, Inc.
Industry
Eligibility
12 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
3
States / cities
New Haven, Connecticut • Chicago, Illinois • San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 13, 2026, 2:08 AM EDT
Not yet recruiting Phase 1 Interventional

High-Dose Vitamin C in G6PDA and Pyruvate Kinase Deficiency: A Safety Study

NCT07612345
View directory record Official record
Conditions
Pyruvate Kinase Deficiency, Glucose 6 Phosphate Dehydrogenase Deficiency
Interventions
Vitamin C
Drug
Lead sponsor
University of Utah
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Oct 13, 2026, 2:08 AM EDT
Completed No phase listed Observational

Gene Therapy for Tay-Sachs Disease

NCT01869270
View directory record Official record
Conditions
Tay Sachs Disease, Sandhoff Disease, Late Onset Tay Sachs Disease
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
Not listed
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
2
States / cities
Tampa, Florida • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 4, 2014 · Synced Oct 13, 2026, 2:08 AM EDT
Recruiting Phase 4 Interventional

Effect of Theophylline in Pseudohypoparathyroidism

NCT03718403
View directory record Official record
Conditions
PHP Ia, PHP IB, Php1C
Interventions
Theophylline
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
5 Years to 21 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2032
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 22, 2022 · Synced Oct 13, 2026, 2:08 AM EDT
Completed Early Phase 1 Interventional Results available

Calcitriol Monotherapy for X-Linked Hypophosphatemia

NCT03748966
View directory record Official record
Conditions
X-linked Hypophosphatemia, Hypophosphatemic Rickets, Hypophosphatemic Rickets, X-Linked Dominant
Interventions
Calcitriol
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
3 Years to 70 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 7, 2026 · Synced Oct 13, 2026, 2:08 AM EDT