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ClinicalTrials.gov public records Last synced Oct 21, 2026, 8:24 AM EDT

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Showing 193–216 of 1,510 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Disorders Clear all
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 21, 2026, 8:24 AM EDT
Completed Phase 3 Interventional Results available

The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome

NCT02658175
View directory record Official record
Conditions
Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency, Hyperlipoproteinemia Type 1
Interventions
Volanesorsen
Drug
Lead sponsor
Akcea Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
8
States / cities
Huntington Beach, California • San Francisco, California • Boca Raton, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 26, 2021 · Synced Oct 21, 2026, 8:24 AM EDT
Completed Phase 2 Interventional Results available

CLINICAL PROTOCOL to Investigate the Long-term Safety and Efficacy of Metreleptin in Various Forms of Partial Lipodystrophy

NCT02654977
View directory record Official record
Conditions
Familial Partial Lipodystrophy, Nonalcoholic Steatohepatitis, NAFLD
Interventions
Metreleptin
Drug
Lead sponsor
University of Michigan
Other
Eligibility
5 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 24, 2020 · Synced Oct 21, 2026, 8:24 AM EDT
Completed Phase 2 Interventional

Phase II Pilot Randomized Study of Sodium Dichloroacetate in Patients With Congenital Lactic Acidemia

NCT00004493
View directory record Official record
Conditions
Lactic Acidosis
Interventions
sodium dichloroacetate
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
3 Months and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2003
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 21, 2026, 8:24 AM EDT
Terminated Phase 1Phase 2 Interventional

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis

NCT04273269
View directory record Official record
Conditions
GM1 Gangliosidosis
Interventions
LYS-GM101
Genetic
Lead sponsor
LYSOGENE
Industry
Eligibility
Up to 3 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Jun 9, 2023 · Synced Oct 21, 2026, 8:24 AM EDT
Completed No phase listed Observational Results available

A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)

NCT01419028
View directory record Official record
Conditions
Hypophosphatasia (HPP)
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Not listed
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
5
States / cities
Los Angeles, California • Indianapolis, Indiana • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2019 · Synced Oct 21, 2026, 8:24 AM EDT
Completed No phase listed Observational

"The MaP Study": Mapping the Patient Journey in MMA and PA

NCT03484767
View directory record Official record
Conditions
Methylmalonic Acidemia, Propionic Acidemia
Interventions
Not listed
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
Not listed
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
11
States / cities
Stanford, California • Atlanta, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 2, 2021 · Synced Oct 21, 2026, 8:24 AM EDT
Withdrawn Phase 4 Interventional

A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)

NCT05494593
View directory record Official record
Conditions
Mucopolysaccharidosis (MPS), Hunter Syndrome
Interventions
ELAPRASE, Rituximab, Methotrexate, Intravenous Immunoglobulin (IVIG)
Drug
Lead sponsor
Takeda
Industry
Eligibility
Up to 6 Years · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
8
States / cities
Phoenix, Arizona • Oakland, California • San Diego, California + 5 more
Source: ClinicalTrials.gov public record
Updated Dec 9, 2025 · Synced Oct 21, 2026, 8:24 AM EDT
Completed Phase 1 Interventional Results available

Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

NCT01173016
View directory record Official record
Conditions
Hurler Syndrome
Interventions
Laronidase
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 14 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 20, 2020 · Synced Oct 21, 2026, 8:24 AM EDT
Completed Not applicable Interventional

Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD

NCT03064048
View directory record Official record
Conditions
Argininosuccinate Lyase Deficiency, Urea Cycle Disorder, Urea Cycle Disorders, Inborn, Argininosuccinic Aciduria
Interventions
Neo-ASA, Placebo
Dietary Supplement
Lead sponsor
Baylor College of Medicine
Other
Eligibility
6 Years to 50 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 27, 2023 · Synced Oct 21, 2026, 8:24 AM EDT
Recruiting No phase listed Observational

Natural History Study of Patients With Hypophosphatasia (HPP)

NCT02237625
View directory record Official record
Conditions
Hypophosphatasia
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2028
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Oct 21, 2026, 8:24 AM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)

NCT01212744
View directory record Official record
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 70 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
9
States / cities
Aurora, Colorado • Gainesville, Florida • Louisville, Kentucky + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 26, 2019 · Synced Oct 21, 2026, 8:24 AM EDT
Completed Phase 4 Interventional

Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq

NCT06780332
View directory record Official record
Conditions
Phenylketonuria, PKU
Interventions
RDD to Palynziq
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Chevy Chase, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 21, 2026, 8:24 AM EDT
Completed Phase 3 Interventional Results available

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

NCT00158600
View directory record Official record
Conditions
Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease, Glycogenosis 2
Interventions
alglucosidase alfa, Placebo
Biological · Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
8 Years and older
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
5
States / cities
Beverly Hills, California • Washington D.C., District of Columbia • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 28, 2015 · Synced Oct 21, 2026, 8:24 AM EDT
Recruiting No phase listed Observational

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

NCT06305234
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
Pegvaliase
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2033
U.S. locations
11
States / cities
Aurora, Colorado • Atlanta, Georgia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Oct 21, 2026, 8:24 AM EDT
Recruiting Phase 3 Interventional

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

NCT06302348
View directory record Official record
Conditions
Phenylketonuria
Interventions
Sepiapterin
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Up to 9 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Aug 28, 2026 · Synced Oct 21, 2026, 8:24 AM EDT
Completed Phase 3 Interventional

Evaluation of the Efficacy, Durability, Safety, and Tolerability of RP-G28 in Patients With Lactose Intolerance

NCT03597516
View directory record Official record
Conditions
Lactose Intolerance
Interventions
RP-G28, Placebos
Drug
Lead sponsor
Ritter Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 75 Years
Enrollment
557 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
35
States / cities
Birmingham, Alabama • Chula Vista, California • Huntington Park, California + 31 more
Source: ClinicalTrials.gov public record
Updated Jul 8, 2019 · Synced Oct 21, 2026, 8:24 AM EDT
Completed Phase 2 Interventional

Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome

NCT01721733
View directory record Official record
Conditions
Leigh Syndrome
Interventions
Placebo, EPI-743 15 mg/kg, EPI-743 5 mg/kg
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
6 Years to 17 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
4
States / cities
Palo Alto, California • Akron, Ohio • Houston, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2020 · Synced Oct 21, 2026, 8:24 AM EDT
Completed Phase 2 Interventional Results available

Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)

NCT01205152
View directory record Official record
Conditions
Hypophosphatasia
Interventions
asfotase alfa
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
24 Weeks to 42 Months
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
5
States / cities
Wilmington, Delaware • Springfield, Missouri • Omaha, Nebraska + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2019 · Synced Oct 21, 2026, 8:24 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Single and Multiple Dose Study in Japanese Subjects

NCT01872832
View directory record Official record
Conditions
Gout
Interventions
RDEA3170 and Placebo
Drug
Lead sponsor
Ardea Biosciences, Inc.
Industry
Eligibility
20 Years to 55 Years · Male only
Enrollment
46 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013
U.S. locations
1
States / cities
Glendale, California
Source: ClinicalTrials.gov public record
Updated Jan 9, 2014 · Synced Oct 21, 2026, 8:24 AM EDT
Terminated Phase 1 Interventional

Brain Stem Gliomas Treated With Adoptive Cellular Therapy During Focal Radiotherapy Recovery Alone or With Dose-intensified Temozolomide (Phase I)

NCT03396575
View directory record Official record
Conditions
Diffuse Intrinsic Pontine Glioma (DIPG), Brain Stem Glioma
Interventions
TTRNA-DC vaccines with GM-CSF, TTRNA-xALT, Cyclophosphamide + Fludarabine Lymphodepletive Conditioning, Dose-Intensified TMZ, Td vaccine, Autologous Hematopoietic Stem Cells (HSC)
Biological · Drug
Lead sponsor
University of Florida
Other
Eligibility
3 Years to 30 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 29, 2025 · Synced Oct 21, 2026, 8:24 AM EDT
Completed Phase 3 Interventional Results available

Randomized Study to Evaluate the Effect of Obicetrapib on Top of Maximum Tolerated Lipid-Modifying Therapies

NCT05142722
View directory record Official record
Conditions
Dyslipidemias, High Cholesterol, Hypercholesterolemia, Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Interventions
Placebo, Obicetrapib
Drug
Lead sponsor
NewAmsterdam Pharma
Industry
Eligibility
18 Years and older
Enrollment
2,530 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
69
States / cities
Anniston, Alabama • Birmingham, Alabama • Saraland, Alabama + 60 more
Source: ClinicalTrials.gov public record
Updated Sep 19, 2025 · Synced Oct 21, 2026, 8:24 AM EDT
Completed Phase 3 Interventional Results available

PREventative Study Against URate-lowering Drug-induced Gout Exacerbations 1

NCT00829829
View directory record Official record
Conditions
Intercritical Gout
Interventions
Placebo, Rilonacept 80 mg, Rilonacept 160 mg
Other · Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years to 80 Years
Enrollment
241 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
67
States / cities
Birmingham, Alabama • Gilbert, Arizona • Peoria, Arizona + 61 more
Source: ClinicalTrials.gov public record
Updated Apr 28, 2017 · Synced Oct 21, 2026, 8:24 AM EDT