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ClinicalTrials.gov public records Last synced Oct 18, 2026, 2:52 PM EDT

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

Showing 1105–1128 of 1,605 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Clear all
Completed No phase listed Observational

Study of Smith-Lemli-Opitz Syndrome

NCT00001721
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Cholesterol Suspension, CRH, Cholesterol
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
Not listed
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1998
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 7, 2026 · Synced Oct 18, 2026, 2:52 PM EDT
Recruiting Phase 3 Interventional

A Gaucher Disease Gene Therapy Trial With FLT201

NCT07223944
View directory record Official record
Conditions
Gaucher Disease Type 1
Interventions
FLT201
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
15
States / cities
Los Angeles, California • San Francisco, California • Aurora, Colorado + 11 more
Source: ClinicalTrials.gov public record
Updated Oct 14, 2026 · Synced Oct 18, 2026, 2:52 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth

NCT06193304
View directory record Official record
Conditions
Gaucher's Disease
Interventions
Eliglustat
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 55 Years
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014
U.S. locations
1
States / cities
Evansville, Indiana
Source: ClinicalTrials.gov public record
Updated Jan 5, 2024 · Synced Oct 18, 2026, 2:52 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)

NCT05487599
View directory record Official record
Conditions
Gaucher Disease, Gaucher Disease, Type 1
Interventions
LY3884961
Genetic
Lead sponsor
Prevail Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
2
States / cities
Durham, North Carolina • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 18, 2026, 2:52 PM EDT
Enrolling by invitation Phase 3 Interventional

Long-term Efficacy and Safety of OLE LIB003 in HoFH, HeFH, and High-risk CVD Patients Requiring Further LDL-C Reduction

NCT04798430
View directory record Official record
Conditions
Cardiovascular Disease With Mention of Arteriosclerosis, Elevated Cholesterol, Familial Hypercholesterolemia
Interventions
lerodalcibep
Drug
Lead sponsor
LIB Therapeutics LLC
Industry
Eligibility
10 Years and older
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
4
States / cities
Evanston, Illinois • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 22, 2024 · Synced Oct 18, 2026, 2:52 PM 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 18, 2026, 2:52 PM EDT
Completed Not applicable Interventional Results available

Virtual Gout Clinic

NCT02790463
View directory record Official record
Conditions
Gout
Interventions
Pharmacist-Led Intervention
Behavioral
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
18 Years and older
Enrollment
1,463 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
2
States / cities
Birmingham, Alabama • Pasadena, California
Source: ClinicalTrials.gov public record
Updated Nov 12, 2019 · Synced Oct 18, 2026, 2:52 PM EDT
Completed No phase listed Observational

Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease

NCT01707433
View directory record Official record
Conditions
Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI
Interventions
Enzyme testing
Other
Lead sponsor
Children's Hospitals and Clinics of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
2
States / cities
Minneapolis, Minnesota • Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 4, 2015 · Synced Oct 18, 2026, 2:52 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Diabetes/ Endocrine Surveillance in SDS

NCT04275479
View directory record Official record
Conditions
Shwachman-Diamond Syndrome
Interventions
Oral Glucose Tolerance Test, Modified Oral Glucose Tolerance Test, Modified Mixed Meal Tolerance Test, Continuous Glucose Monitor, Food Diary, Medical History Questionnaires
Diagnostic Test · Other · Device
Lead sponsor
Washington University School of Medicine
Other
Eligibility
3 Years and older
Enrollment
11 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2022
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Aug 19, 2024 · Synced Oct 18, 2026, 2:52 PM 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 18, 2026, 2:52 PM EDT
Terminated Phase 1 Interventional

Gene Transfer Clinical Study in Crigler-Najjar Syndrome

NCT03223194
View directory record Official record
Conditions
Crigler-Najjar Syndrome
Interventions
AT342
Genetic
Lead sponsor
Audentes Therapeutics
Industry
Eligibility
1 Year and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
2
States / cities
The Bronx, New York • Strasburg, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 18, 2022 · Synced Oct 18, 2026, 2:52 PM EDT
Recruiting Phase 3 Interventional

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

NCT06128629
View directory record Official record
Conditions
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Interventions
NTLA-2001, Placebo
Biological · Drug
Lead sponsor
Intellia Therapeutics
Industry
Eligibility
18 Years to 90 Years
Enrollment
1,200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
38
States / cities
Tucson, Arizona • Los Angeles, California • Palo Alto, California + 29 more
Source: ClinicalTrials.gov public record
Updated Mar 27, 2026 · Synced Oct 18, 2026, 2:52 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

NCT05206773
View directory record Official record
Conditions
Fabry Disease
Interventions
Venglustat (GZ402671), Placebo
Drug
Lead sponsor
Sanofi
Industry
Eligibility
16 Years and older
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
11
States / cities
Birmingham, Alabama • Los Angeles, California • Orange, California + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 18, 2026, 2:52 PM EDT
Active, not recruiting Phase 3 Interventional

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

NCT03911505
View directory record Official record
Conditions
Pompe Disease (Late-onset)
Interventions
Cipaglucosidase Alfa, Miglustat
Biological · Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
0 Years to 17 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
9
States / cities
Gainesville, Florida • Jacksonville, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Oct 18, 2026, 2:52 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study

NCT05130437
View directory record Official record
Conditions
Propionic Acidemia
Interventions
mRNA-3927
Biological
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
1 Year and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2031
U.S. locations
7
States / cities
Los Angeles, California • Palo Alto, California • Ann Arbor, Michigan + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2026 · Synced Oct 18, 2026, 2:52 PM 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 18, 2026, 2:52 PM EDT
Completed Phase 1 Interventional

Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease

NCT00004294
View directory record Official record
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase gene into autologous peripheral blood stem cells
Genetic
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 65 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 2:52 PM EDT
Recruiting No phase listed Observational

Ovarian Tissue Cryopreservation in Pre-Pubertal (OTC-Pre Pubertal)

NCT05299710
View directory record Official record
Conditions
Cancers
Interventions
Laparoscopic surgery
Procedure
Lead sponsor
Erin Rowell
Other
Eligibility
Up to 11 Years · Female only
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2035
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 18, 2026, 2:52 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

The NIH UNI Study: Urea Cycle Disorders, Nutrition and Immunity

NCT01421888
View directory record Official record
Conditions
Argininosuccinic Aciduria, Carbamoyl-Phosphate Synthase I Deficiency, Citrullinemia, Ornithine Carbamoyltransferase Deficiency, Hyperargininemia, N-Acetylglutamate Synthase Deficiency
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Years to 99 Years
Enrollment
4 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 9, 2019 · Synced Oct 18, 2026, 2:52 PM EDT
Completed No phase listed Observational

Intravenous ACP-501 for Familial LCAT Deficiency (rhLCAT)

NCT04737720
View directory record Official record
Conditions
Familial LCAT Deficiency
Interventions
Recombinant LCAT
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 18, 2026, 2:52 PM EDT
Completed Phase 1 Interventional

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ABP-671

NCT04060173
View directory record Official record
Conditions
Hyperuricemia, Gout
Interventions
ABP-671, Placebo
Drug · Other
Lead sponsor
Atom Therapeutics Co., Ltd
Industry
Eligibility
18 Years to 65 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Tempe, Arizona
Source: ClinicalTrials.gov public record
Updated Feb 12, 2020 · Synced Oct 18, 2026, 2:52 PM EDT
Completed Phase 2 Interventional

A Randomized Comparison of Intravitreal ISIS 2922 Plus Ganciclovir Versus Ganciclovir as Treatment for Patients With Cytomegalovirus Retinitis ( CMVR )

NCT00002156
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Fomivirsen sodium, Ganciclovir
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
194 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
18
States / cities
Los Angeles, California • Pasadena, California • San Diego, California + 14 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 18, 2026, 2:52 PM EDT
Completed No phase listed Observational

Hunter Outcome Survey (HOS)

NCT03292887
View directory record Official record
Conditions
Hunter Syndrome
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Not listed
Enrollment
1,443 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2023
U.S. locations
1
States / cities
Lexington, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 28, 2024 · Synced Oct 18, 2026, 2:52 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

NCT04093349
View directory record Official record
Conditions
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
Interventions
SPK-3006
Genetic
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2032
U.S. locations
10
States / cities
Phoenix, Arizona • Orange, California • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 27, 2024 · Synced Oct 18, 2026, 2:52 PM EDT