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ClinicalTrials.gov public records Last synced Oct 12, 2026, 7:55 PM EDT

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Showing 169–192 of 1,605 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inborn Errors of Metabolism Clear all
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants

NCT04526210
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840, Bupropion Hydrochloride
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
54 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Aug 31, 2023 · Synced Oct 12, 2026, 7:55 PM EDT
Completed Phase 4 Interventional Results available

High Dose or High Dose Frequency Study of Alglucosidase Alfa

NCT00483379
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency
Interventions
alglucosidase alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
6 Months and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
9
States / cities
Birmingham, Alabama • Stanford, California • Washington D.C., District of Columbia + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 7, 2014 · Synced Oct 12, 2026, 7:55 PM EDT
Completed Not applicable Interventional

Open Label Ganciclovir Therapy for Sight- or Life-Threatening Cytomegalovirus Disease in the Immunocompromised Patient

NCT00002025
View directory record Official record
Conditions
Cytomegalovirus Infections, Cytomegalovirus Retinitis, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
3 Months 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 12, 2026, 7:55 PM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

NCT01994889
View directory record Official record
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
Tafamidis, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 90 Years
Enrollment
441 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
50
States / cities
Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 24 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2019 · Synced Oct 12, 2026, 7:55 PM EDT
Terminated Phase 2 Interventional Results available

Vision Response to Dopamine Replacement

NCT01663935
View directory record Official record
Conditions
Albinism, Oculocutaneous Albinism
Interventions
Levodopa/carbidopa
Drug
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
3 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jun 11, 2019 · Synced Oct 12, 2026, 7:55 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

MRI Spectroscopy and Neuropsychological Functioning in Phenylketonuria

NCT03097250
View directory record Official record
Conditions
Phenylketonuria
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
12 Years to 25 Years
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 19, 2018 · Synced Oct 12, 2026, 7:55 PM EDT
Withdrawn No phase listed Observational

A Screening Study Evaluating Disease Status of Gaucher Type I Patients

NCT00795197
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
Not listed
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 8, 2015 · Synced Oct 12, 2026, 7:55 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 12, 2026, 7:55 PM EDT
Completed No phase listed Observational

An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype

NCT01528917
View directory record Official record
Conditions
Cholesterol Ester Storage Disease(CESD), Lysosomal Acid Lipase Deficiency
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
5 Years and older
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
6
States / cities
Stanford, California • Chicago, Illinois • Minneapolis, Minnesota + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 20, 2016 · Synced Oct 12, 2026, 7:55 PM EDT
Terminated Phase 4 Interventional Results available

A Phase 4 Safety and Efficacy Study to Evaluate Lesinurad 200 mg in Participants With Gout and Renal Impairment

NCT03226899
View directory record Official record
Conditions
Gout, Chronic Kidney Disease (CKD)
Interventions
Lesinurad, XOI, Placebo, colchicine, corticosteroids
Drug
Lead sponsor
Ironwood Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 85 Years
Enrollment
242 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
70
States / cities
Birmingham, Alabama • Tucson, Arizona • Covina, California + 59 more
Source: ClinicalTrials.gov public record
Updated Nov 4, 2021 · Synced Oct 12, 2026, 7:55 PM EDT
Completed Phase 3 Interventional Results available

Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

NCT00712348
View directory record Official record
Conditions
Gaucher Disease
Interventions
Taliglucerase alfa
Drug
Lead sponsor
Pfizer
Industry
Eligibility
2 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
3
States / cities
Coral Springs, Florida • Decatur, Georgia • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 4, 2018 · Synced Oct 12, 2026, 7:55 PM EDT
Completed Not applicable Interventional

The Safety and Effectiveness of Ganciclovir Plus Interferon Beta in Preventing the Return of Cytomegalovirus (CMV) of the Eyes in Patients With AIDS

NCT00002299
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Interferon beta-1b, Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years to 60 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
6
States / cities
Los Angeles, California • Orange, California • San Diego, California + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 12, 2026, 7:55 PM EDT
Completed Phase 3 Interventional Results available

Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

NCT00104247
View directory record Official record
Conditions
Phenylketonurias
Interventions
sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
8 Years and older
Enrollment
89 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
13
States / cities
Los Angeles, California • Oakland, California • New Haven, Connecticut + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 23, 2014 · Synced Oct 12, 2026, 7:55 PM EDT
Completed No phase listed Observational

Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation

NCT01316549
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
Interventions
Fludarabine
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 4, 2021 · Synced Oct 12, 2026, 7:55 PM EDT
Completed Phase 2 Interventional

The Safety and Effectiveness of Ganciclovir in the Prevention of Cytomegalovirus (CMV) of the Eyes and Disease of the Stomach and Intestines in Patients With HIV

NCT00001034
View directory record Official record
Conditions
Cytomegalovirus Retinitis, HIV Infections, Gastrointestinal Diseases
Interventions
Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
13 Years and older
Enrollment
850 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Ends 1995
U.S. locations
17
States / cities
San Francisco, California • Denver, Colorado • Wilmington, Delaware + 12 more
Source: ClinicalTrials.gov public record
Updated Nov 4, 2021 · Synced Oct 12, 2026, 7:55 PM EDT
Completed No phase listed Observational

Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)

NCT01136304
View directory record Official record
Conditions
Gaucher Disease
Interventions
Imiglucerase
Drug
Lead sponsor
University Research Foundation for Lysosomal Storage Diseases, Inc.
Other
Eligibility
18 Years and older
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
2
States / cities
Beverly Hills, California • Coral Springs, Florida
Source: ClinicalTrials.gov public record
Updated Jun 1, 2015 · Synced Oct 12, 2026, 7:55 PM EDT
Terminated Phase 3 Interventional

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

NCT00654433
View directory record Official record
Conditions
Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
Interventions
ALD-101
Biological
Lead sponsor
Aldagen
Industry
Eligibility
Up to 16 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
3
States / cities
Los Angeles, California • New York, New York • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 8, 2014 · Synced Oct 12, 2026, 7:55 PM 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 12, 2026, 7:55 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 12, 2026, 7:55 PM EDT
Suspended Phase 1Phase 2 Interventional

RGX-111 Gene Therapy in Patients With MPS I

NCT03580083
View directory record Official record
Conditions
Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
Interventions
RGX-111
Genetic
Lead sponsor
REGENXBIO Inc.
Industry
Eligibility
4 Months and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2029
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Oct 12, 2026, 7:55 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

NCT05152914
View directory record Official record
Conditions
Neuronal Ceroid Lipofuscinosis Type 2
Interventions
Cerliponase Alfa
Drug
Lead sponsor
David L Rogers, MD
Other
Eligibility
24 Months to 72 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced Oct 12, 2026, 7:55 PM EDT
Completed Phase 3 Interventional Results available

Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C

NCT03814187
View directory record Official record
Conditions
ASCVD, Elevated Cholesterol, Heterozygous Familial Hypercholesterolemia, Homozygous Familial Hypercholesterolemia
Interventions
Inclisiran Sodium
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
3,275 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
141
States / cities
Birmingham, Alabama • Foley, Alabama • Huntsville, Alabama + 109 more
Source: ClinicalTrials.gov public record
Updated Mar 6, 2024 · Synced Oct 12, 2026, 7:55 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 12, 2026, 7:55 PM EDT
Completed Phase 4 Interventional Results available

Examining the Effect of Burosumab on Muscle Function

NCT04146935
View directory record Official record
Conditions
X-linked Hypophosphatemia
Interventions
Burosumab Injection [Crysvita]
Drug
Lead sponsor
Yale University
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Aug 24, 2023 · Synced Oct 12, 2026, 7:55 PM EDT