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ClinicalTrials.gov public records Last synced Oct 17, 2026, 7:34 PM EDT

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Showing 961–984 of 1,605 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Clear all
Active, not recruiting Phase 3 Interventional

A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

NCT05280548
View directory record Official record
Conditions
Fabry Disease
Interventions
Venglustat (GZ402671), Agalsidase alfa, Agalsidase beta (GZ419828), Migalastat
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
8
States / cities
Birmingham, Alabama • Los Angeles, California • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Oct 17, 2026, 7:34 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders

NCT03812055
View directory record Official record
Conditions
Lysosomal Storage Diseases
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jan 22, 2019 · Synced Oct 17, 2026, 7:34 PM EDT
Active, not recruiting Not applicable Interventional

Randomized Controlled Trial of Alert-Based Computerized Decision Support for Optimizing Low-Density Lipoprotein Management

NCT05088759
View directory record Official record
Conditions
Hyperlipemia, Mixed, ASCVD
Interventions
Alert
Behavioral
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 17, 2026, 7:34 PM EDT
Completed Phase 3 Interventional

Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis

NCT05184088
View directory record Official record
Conditions
Cardiac Amyloidosis, AL Amyloidosis, ATTR Amyloidosis
Interventions
[18F]florbetaben
Drug
Lead sponsor
Lantheus Germany GmbH
Industry
Eligibility
18 Years and older
Enrollment
244 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
3
States / cities
Kansas City, Kansas • Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Oct 17, 2026, 7:34 PM EDT
Terminated Phase 2 Interventional Results available

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia

NCT05116046
View directory record Official record
Conditions
Achondroplasia
Interventions
Recifercept
Biological
Lead sponsor
Pfizer
Industry
Eligibility
15 Months to 12 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
6
States / cities
Irvine, California • Long Beach, California • Torrance, California + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 8, 2024 · Synced Oct 17, 2026, 7:34 PM EDT
Completed Phase 3 Interventional Results available

An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165

NCT01819727
View directory record Official record
Conditions
Phenylketonuria
Interventions
BMN 165
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years to 70 Years
Enrollment
261 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
31
States / cities
La Jolla, California • Oakland, California • Aurora, Colorado + 28 more
Source: ClinicalTrials.gov public record
Updated Feb 26, 2019 · Synced Oct 17, 2026, 7:34 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Role of the Protein Osteoprotegerin in the Bone Health of Women With Congenital Adrenal Hyperplasia

NCT00694525
View directory record Official record
Conditions
Adrenal Hyperplasia, Congenital
Interventions
Not listed
Lead sponsor
Office of Rare Diseases (ORD)
NIH
Eligibility
20 Years to 35 Years · Female only
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 2, 2009 · Synced Oct 17, 2026, 7:34 PM EDT
Completed Phase 3 Interventional Results available

GlycoCholic Acid Treatment for Patients With Inborn Errors in Bile Acid Synthesis

NCT01589523
View directory record Official record
Conditions
Bile Acid Synthesis Defect, Inborn Error of Bile Acid Metabolism, Inborn Error of Bile Acid Conjugation
Interventions
Glycocholic Acid
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
1 Week to 85 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2019
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 8, 2022 · Synced Oct 17, 2026, 7:34 PM EDT
Completed Phase 2 Interventional

Tolerization Reduces Intolerance to Pegloticase and Prolongs the Urate Lowering Effect

NCT02598596
View directory record Official record
Conditions
Gout
Interventions
Pegloticase, Azathioprine
Biological · Drug
Lead sponsor
Ampel BioSolutions, LLC
Industry
Eligibility
18 Years and older
Enrollment
132 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
11
States / cities
Birmingham, Alabama • Huntsville, Alabama • Wheaton, Maryland + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 11, 2021 · Synced Oct 17, 2026, 7:34 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells

NCT00001234
View directory record Official record
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase cDNA
Genetic
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1988 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 17, 2026, 7:34 PM EDT
Completed No phase listed Observational

Genzyme Osteopenia/Osteoporosis Study

NCT02785744
View directory record Official record
Conditions
Gaucher Disease
Interventions
Gaucher disease DNA mutation analysis
Other
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years and older
Enrollment
76 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 23, 2021 · Synced Oct 17, 2026, 7:34 PM EDT
Recruiting Phase 3 Interventional

Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia

NCT06597006
View directory record Official record
Conditions
Familial Hypercholesterolemia - Homozygous
Interventions
Inclisiran, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 11 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
4
States / cities
San Francisco, California • Washington D.C., District of Columbia • St Louis, Missouri + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 24, 2026 · Synced Oct 17, 2026, 7:34 PM EDT
Recruiting No phase listed Observational

Advancing Integrated Therapies for Gaucher Disease

NCT07758816
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
Yale University
Other
Eligibility
3 Months to 75 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Aug 11, 2026 · Synced Oct 17, 2026, 7:34 PM EDT
Enrolling by invitation Phase 3 Interventional

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

NCT04360265
View directory record Official record
Conditions
Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome, Sanfilippo A
Interventions
No Investigational Product, Adjuvant Immunomodulatory (IM) Therapy
Other · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
Not listed
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 17, 2026 · Synced Oct 17, 2026, 7:34 PM 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 17, 2026, 7:34 PM EDT
Terminated Early Phase 1 Interventional

Molecular Imaging of the Underlying Mechanism of Cardiotoxicity in Patients With Light Chain Amyloidosis Using PET/CT

NCT04105634
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
FNOS, Florbetaben
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 12, 2024 · Synced Oct 17, 2026, 7:34 PM EDT
Completed Phase 2 Interventional Results available

Short-term Behavioral Effects of Cholesterol Therapy in Smith-Lemli-Opitz Syndrome

NCT00114634
View directory record Official record
Conditions
Smith-Lemli-Opitz Syndrome
Interventions
Egg yolk preparation with cholesterol, Egg substitute, without cholesterol
Dietary Supplement
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
4 Years to 17 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 29, 2016 · Synced Oct 17, 2026, 7:34 PM EDT
Completed No phase listed Observational

Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

NCT01509768
View directory record Official record
Conditions
Sanfilippo Syndrome Type B
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
1 Year to 10 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
2
States / cities
Oakland, California • Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 17, 2021 · Synced Oct 17, 2026, 7:34 PM EDT
Completed Not applicable Interventional

Ganciclovir: Compassionate Use in Patients With Serious or Life-Threatening Cytomegalovirus Infections

NCT00002024
View directory record Official record
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 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 17, 2026, 7:34 PM EDT
Completed Phase 4 Interventional Results available

Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease

NCT01410890
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD II)
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
4
States / cities
Valhalla, New York • Cincinnati, Ohio • Salt Lake City, Utah + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 28, 2022 · Synced Oct 17, 2026, 7:34 PM EDT
Completed Phase 3 Interventional Results available

Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)

NCT01197378
View directory record Official record
Conditions
Cystinosis
Interventions
Cysteamine Bitartrate Delayed-release Capsules
Drug
Lead sponsor
Amgen
Industry
Eligibility
1 Year and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
5
States / cities
San Francisco, California • Stanford, California • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 17, 2026, 7:34 PM EDT
Completed No phase listed Observational

Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)

NCT02998710
View directory record Official record
Conditions
Homocystinuria Due to CBS Deficiency
Interventions
Not listed
Lead sponsor
Travere Therapeutics, Inc.
Industry
Eligibility
1 Year to 65 Years
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
7
States / cities
Aurora, Colorado • Washington D.C., District of Columbia • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 17, 2026, 7:34 PM EDT
Completed No phase listed Observational

Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies

NCT01451879
View directory record Official record
Conditions
Pompe Disease
Interventions
Rituximab, Miglustat
Drug
Lead sponsor
University of Florida
Other
Eligibility
Up to 65 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Dec 8, 2017 · Synced Oct 17, 2026, 7:34 PM EDT
Completed No phase listed Observational

Primary Hyperoxaluria Mutation Genotyping/Phenotyping

NCT02340689
View directory record Official record
Conditions
Primary Hyperoxaluria
Interventions
Genetic Analysis
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
1 Day and older
Enrollment
1,235 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 19, 2019 · Synced Oct 17, 2026, 7:34 PM EDT