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ClinicalTrials.gov public records Last synced Oct 17, 2026, 5:58 PM EDT

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

Showing 937–960 of 1,605 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Clear all
Completed No phase listed Observational

Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment

NCT02311283
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
Not listed
Lead sponsor
University of South Florida
Other
Eligibility
Not listed
Enrollment
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Mar 7, 2018 · Synced Oct 17, 2026, 5:58 PM EDT
Active, not recruiting Phase 3 Interventional

Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants

NCT04988386
View directory record Official record
Conditions
Amyloid Cardiomyopathy, Transthyretin-Related
Interventions
Acoramidis (AG10)
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 90 Years
Enrollment
389 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
26
States / cities
Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 21 more
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced Oct 17, 2026, 5:58 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Copper Histidine Therapy for Menkes Diseases

NCT00001262
View directory record Official record
Conditions
Kinky Hair Syndrome
Interventions
Copper Histidine
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Not listed
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1990 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 30, 2015 · Synced Oct 17, 2026, 5:58 PM EDT
Completed Phase 2 Interventional Results available

Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes

NCT00887562
View directory record Official record
Conditions
MELAS Syndrome
Interventions
Idebenone, Placebo
Drug · Other
Lead sponsor
Michio Hirano
Other
Eligibility
8 Years to 65 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 26, 2016 · Synced Oct 17, 2026, 5:58 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

RDEA3170 PK/PD Study

NCT02608710
View directory record Official record
Conditions
Gout
Interventions
RDEA3170 4.5 mg, RDEA3170 6 mg, RDEA3170 12 mg
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Sep 2, 2016 · Synced Oct 17, 2026, 5:58 PM EDT
Completed Phase 2 Interventional Results available

Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)

NCT01203826
View directory record Official record
Conditions
Hypophosphatasia (HPP)
Interventions
Asfotase Alfa
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
5 Years to 12 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 13, 2019 · Synced Oct 17, 2026, 5:58 PM EDT
Not listed Phase 2 Interventional

Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS)

NCT01773278
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome, Cone-Rod Dystrophy, Hearing Loss
Interventions
Antioxidants, Cholesterol
Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
Up to 65 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2025
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated May 8, 2024 · Synced Oct 17, 2026, 5:58 PM EDT
Not listed Phase 1 Interventional Accepts healthy volunteers

Safety and Efficacy Study of Intravenous Uricase-PEG 20

NCT01021241
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
Uricase-PEG 20
Biological
Lead sponsor
EnzymeRx
Industry
Eligibility
40 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2010
U.S. locations
2
States / cities
Miami, Florida • Duncansville, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 23, 2010 · Synced Oct 17, 2026, 5:58 PM EDT
Completed No phase listed Observational

A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype

NCT01358370
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency, Wolman Disease
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
7
States / cities
Los Angeles, California • Palo Alto, California • Minneapolis, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 27, 2016 · Synced Oct 17, 2026, 5:58 PM EDT
Completed Early Phase 1 Interventional

Ketones & Mitochondrial Heteroplasmy

NCT01252979
View directory record Official record
Conditions
MELAS Syndrome, Mitochondrial Diseases
Interventions
Medium-Chain Triglycerides
Dietary Supplement
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
Not listed
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 6, 2012 · Synced Oct 17, 2026, 5:58 PM EDT
Completed Phase 3 Interventional

Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses

NCT00672022
View directory record Official record
Conditions
GM2 Gangliosidoses, Tay-Sachs, Sandhoff Disease
Interventions
Zavesca (Miglustat)
Drug
Lead sponsor
Children's National Research Institute
Other
Eligibility
6 Months to 5 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated May 6, 2008 · Synced Oct 17, 2026, 5:58 PM EDT
Recruiting Not applicable Interventional

Computerized Decision Support for Identification and Management of Familial Hypercholesterolemia

NCT06743659
View directory record Official record
Conditions
Familial Hypercholesterolaemia
Interventions
Alert-based computerized decision support
Device
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
Not listed
Enrollment
450 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 17, 2026, 5:58 PM EDT
Completed Phase 2 Interventional Results available

A Study to Assess the Effect of Intensive Uric Acid (UA) Lowering Therapy With RDEA3170, Febuxostat, Dapagliflozin on Urinary Excretion of UA

NCT03316131
View directory record Official record
Conditions
Asymptomatic Hyperuricemia
Interventions
Verinurad, Febuxostat, Dapagliflozin, Dapagliflozin matched placebo
Drug · Other
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 99 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
2
States / cities
Glendale, California • Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 28, 2019 · Synced Oct 17, 2026, 5:58 PM EDT
Terminated Phase 4 Interventional Results available

A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

NCT01597596
View directory record Official record
Conditions
Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2, Acid Maltase Deficiency
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Up to 12 Months
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
14
States / cities
Little Rock, Arkansas • Oakland, California • Gainsville, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Jan 18, 2016 · Synced Oct 17, 2026, 5:58 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Burosumab Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With XLH

NCT02915705
View directory record Official record
Conditions
X-Linked Hypophosphatemia
Interventions
burosumab, Oral Phosphate Supplement, active vitamin D
Biological · Drug
Lead sponsor
Kyowa Kirin, Inc.
Industry
Eligibility
1 Year to 12 Years
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
5
States / cities
Los Angeles, California • San Francisco, California • Indianapolis, Indiana + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 29, 2024 · Synced Oct 17, 2026, 5:58 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

NCT04998396
View directory record Official record
Conditions
Canavan Disease
Interventions
AAV9 BBP-812
Biological
Lead sponsor
Aspa Therapeutics
Industry
Eligibility
Up to 30 Months
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
4
States / cities
Oakland, California • Chicago, Illinois • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 17, 2026 · Synced Oct 17, 2026, 5:58 PM EDT
Terminated Phase 1 Interventional Results available

Efficacy, Safety And Tolerability Of PF-06743649 In Gout Subjects.

NCT02187029
View directory record Official record
Conditions
Gout
Interventions
PF-06743649, Placebo
Drug · Other
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 60 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014
U.S. locations
4
States / cities
Miami, Florida • South Miami, Florida • Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated Nov 11, 2016 · Synced Oct 17, 2026, 5:58 PM EDT
No Longer Available No phase listed Expanded access

Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

NCT00962260
View directory record Official record
Conditions
Gaucher Disease
Interventions
Plant cell expressed recombinant glucocerebrosidase (prGCD)
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
U.S. locations
14
States / cities
La Jolla, California • Aurora, Colorado • Coral Springs, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 7, 2018 · Synced Oct 17, 2026, 5:58 PM EDT
Completed Phase 2 Interventional Results available

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

NCT00925054
View directory record Official record
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG 0.001 mg/kg, rAvPAL-PEG 0.003 mg/kg, rAvPAL-PEG 0.01 mg/kg, rAvPAL-PEG 0.03 mg/kg, rAvPAL-PEG 0.1 mg/kg
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 55 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
11
States / cities
Aurora, Colorado • Decatur, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 26, 2019 · Synced Oct 17, 2026, 5:58 PM EDT
Completed Phase 3 Interventional Results available

Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients

NCT01114737
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sapropterin dihydrochloride, Placebo
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
8 Years to 65 Years
Enrollment
206 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
26
States / cities
La Jolla, California • Los Angeles, California • Palo Alto, California + 23 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2016 · Synced Oct 17, 2026, 5:58 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease

NCT04537377
View directory record Official record
Conditions
Wilson's Disease
Interventions
VTX-801
Genetic
Lead sponsor
Vivet Therapeutics SAS
Industry
Eligibility
18 Years to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
6
States / cities
Sacramento, California • New Haven, Connecticut • Orlando, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 30, 2026 · Synced Oct 17, 2026, 5:58 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Validation of Diagnostics to Identify Glucose-6-phosphate Dehydrogenase Activity in the US

NCT04010695
View directory record Official record
Conditions
G6PD Deficiency
Interventions
SD Biosensor G6PD Analyzer, Pointe Scientific Test Kit, HemoCue System
Diagnostic Test
Lead sponsor
PATH
Other
Eligibility
18 Years to 65 Years
Enrollment
250 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated May 10, 2022 · Synced Oct 17, 2026, 5:58 PM EDT
Recruiting No phase listed Observational

A Study of Patients With Fabry Disease (US Specific)

NCT06906367
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl, ERT
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
450 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
8
States / cities
Birmingham, Alabama • Little Rock, Arkansas • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 17, 2026, 5:58 PM EDT
Completed No phase listed Observational

Gait Analysis in MPS IVA

NCT01920828
View directory record Official record
Conditions
MPS IVA, Morquio Syndrome
Interventions
Not listed
Lead sponsor
Oregon Health and Science University
Other
Eligibility
1 Year and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 17, 2019 · Synced Oct 17, 2026, 5:58 PM EDT