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ClinicalTrials.gov public records Last synced Oct 19, 2026, 4:22 PM EDT

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Showing 1393–1416 of 1,605 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Clear all
Not listed Phase 2 Interventional

Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria

NCT00916747
View directory record Official record
Conditions
Progeria
Interventions
Lonafarnib, Zoledronic Acid, and Pravastatin
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2023
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 27, 2023 · Synced Oct 19, 2026, 4:22 PM EDT
Completed No phase listed Observational

A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease

NCT00515398
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II
Interventions
Observation
Other
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
4
States / cities
Gainesville, Florida • St Louis, Missouri • Lake Success, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 5, 2008 · Synced Oct 19, 2026, 4:22 PM EDT
Completed Not applicable Interventional

A Study of the Safety and Tolerance of Long-Term Therapy With Intravenous Cytovene (Ganciclovir Sodium) for Cytomegalovirus Retinitis in Persons With AIDS

NCT00002034
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Zidovudine, Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
8
States / cities
Birmingham, Alabama • Oakland, California • San Francisco, California + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 19, 2026, 4:22 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)

NCT05394064
View directory record Official record
Conditions
AMN, AMN Gene Mutation, X-ALD
Interventions
SBT101 Treatment
Genetic
Lead sponsor
SwanBio Therapeutics, Inc.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Oct 19, 2026, 4:22 PM EDT
Completed No phase listed Observational

PheCheck Feasibility Study

NCT05998109
View directory record Official record
Conditions
Phenylketonurias
Interventions
PheCheck™, HPLC Amino Acid Analyzer, Dried Blood Spot Cards
Device · Diagnostic Test
Lead sponsor
Lumos Diagnostics
Industry
Eligibility
10 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Oct 19, 2026, 4:22 PM EDT
Terminated Phase 2 Interventional Results available

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

NCT02350816
View directory record Official record
Conditions
Sanfilippo Syndrome, Mucopolysaccharidosis (MPS)
Interventions
HGT-1410
Drug
Lead sponsor
Shire
Industry
Eligibility
12 Months to 48 Months
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
2
States / cities
Minneapolis, Minnesota • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 11, 2021 · Synced Oct 19, 2026, 4:22 PM EDT
Recruiting No phase listed Observational

National Ophthalmic Genotyping and Phenotyping Network (eyeGENE (Registered Trademark)), Stage 3 - Expansion of DNA and Data Repositories for Rare Inherited Ophthalmic Diseases

NCT06491615
View directory record Official record
Conditions
Inherited Ophthalmic Diseases, Hypopigmentation Disorder, Corneal Dystrophy, Blue-cone Monochromacy, Best Disease, Aniridia, Albinism
Interventions
Not listed
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
1 Day to 120 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2054
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Oct 19, 2026, 4:22 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Relative Bioavailability Study to Assess an LC350189 Tablet Compared to an LC350189 Capsule in Healthy Adults

NCT04886050
View directory record Official record
Conditions
Gout, Hyperuricemia
Interventions
LC350189 Tablet, LC350189 Capsule
Drug
Lead sponsor
LG Chem
Industry
Eligibility
18 Years to 50 Years
Enrollment
23 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021
U.S. locations
1
States / cities
Las Vegas, Nevada
Source: ClinicalTrials.gov public record
Updated Jan 6, 2022 · Synced Oct 19, 2026, 4:22 PM 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 19, 2026, 4:22 PM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor

NCT01556906
View directory record Official record
Conditions
Homozygous Familial Hypercholesterolemia
Interventions
Lomitapide
Drug
Lead sponsor
Aegerion Pharmaceuticals, Inc.
Industry
Eligibility
13 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2004
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 10, 2013 · Synced Oct 19, 2026, 4:22 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07082543
View directory record Official record
Conditions
Gangliosidoses, GM2, Gangliosidosis, GM1
Interventions
AZ-3102, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 19, 2026, 4:22 PM EDT
Completed Not applicable Interventional

An Open-Label Safety Study of Oral Ganciclovir Maintenance Treatment of CMV Retinitis in People With Limited Venous Access

NCT00002135
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 19, 2026, 4:22 PM EDT
Completed Phase 3 Interventional Results available

Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks

NCT03180840
View directory record Official record
Conditions
Fabry Disease
Interventions
Pegunigalsidase alfa
Biological
Lead sponsor
Protalix
Industry
Eligibility
18 Years to 60 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
7
States / cities
Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 13, 2023 · Synced Oct 19, 2026, 4:22 PM EDT
Completed Phase 3 Interventional Results available

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency

NCT01757184
View directory record Official record
Conditions
Lysosomal Acid Lipase Deficiency
Interventions
Sebelipase Alfa, Placebo
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
4 Years and older
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
11
States / cities
Tucson, Arizona • Palo Alto, California • San Francisco, California + 8 more
Source: ClinicalTrials.gov public record
Updated Dec 29, 2020 · Synced Oct 19, 2026, 4:22 PM EDT
Recruiting No phase listed Observational

Evaluation of Outcome Metrics in Alexander Disease

NCT02714764
View directory record Official record
Conditions
Alexander Disease
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Not listed
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 15, 2026 · Synced Oct 19, 2026, 4:22 PM EDT
Completed Phase 3 Interventional Results available

PLUTO: Pediatric Lipid-redUction Trial of rOsuvastatin

NCT00355615
View directory record Official record
Conditions
Familial Hypercholesterolemia
Interventions
Rosuvastatin, Placebo
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
10 Years to 17 Years
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
4
States / cities
Los Angeles, California • Hyde Park, New York • Cincinnati, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2011 · Synced Oct 19, 2026, 4:22 PM EDT
Completed Not applicable Interventional

Regeneron 1331 Kinetics Sub-Study HoFH

NCT04722068
View directory record Official record
Conditions
Hypercholesterolemia, Familial
Interventions
Kinetics test
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 17, 2021 · Synced Oct 19, 2026, 4:22 PM EDT
Completed Phase 1 Interventional

Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

NCT03882437
View directory record Official record
Conditions
Danon Disease
Interventions
RP-A501
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 19, 2026, 4:22 PM EDT
Recruiting Not applicable Interventional

CAPACITY (Cardiac Amyloidosis and Physical ACtivITY) Study

NCT06096675
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
• Cardiac Rehabilitation
Behavioral
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Oct 19, 2026, 4:22 PM EDT
Completed Phase 2 Interventional Results available

A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

NCT00813865
View directory record Official record
Conditions
Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1
Interventions
afegostat tartrate
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 15, 2018 · Synced Oct 19, 2026, 4:22 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Radicle GI Health RDH™: A Study Assessing the Impact of Health and Wellness Products on Gastrointestinal (GI) Health and Related Health Outcomes

NCT07230821
View directory record Official record
Conditions
Gas and Bloating, Diarrhea, Constipation
Interventions
GI Health Active Product 1, GI Health Product Placebo Control
Dietary Supplement
Lead sponsor
Radicle Science
Industry
Eligibility
21 Years to 105 Years
Enrollment
500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
1
States / cities
Del Mar, California
Source: ClinicalTrials.gov public record
Updated May 28, 2026 · Synced Oct 19, 2026, 4:22 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Nitisinone for Type 1B Oculocutaneous Albinism

NCT01838655
View directory record Official record
Conditions
Albinism, Vision Loss
Interventions
Nitisinone
Drug
Lead sponsor
National Eye Institute (NEI)
NIH
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 26, 2019 · Synced Oct 19, 2026, 4:22 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy

NCT01855360
View directory record Official record
Conditions
Amyloidosis; Heart (Manifestation), Senile Cardiac Amyloidosis
Interventions
Tauroursodeoxycholic Acid and Doxycycline
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 4, 2020 · Synced Oct 19, 2026, 4:22 PM EDT
Not listed Phase 2Phase 3 Interventional

N-Carbamylglutamate (Carbaglu) In The Treatment Of Hyperammonemia

NCT00843921
View directory record Official record
Conditions
Inborn Errors of Metabolism
Interventions
N-carbamylglutamate
Drug
Lead sponsor
Mendel Tuchman
Other
Eligibility
1 Day to 70 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2020
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 17, 2020 · Synced Oct 19, 2026, 4:22 PM EDT