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ClinicalTrials.gov public records Last synced Oct 10, 2026, 6:05 PM EDT

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Showing 97–120 of 1,599 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Genetic Inborn Errors of Metabolism Clear all
Recruiting Phase 1Phase 2 Interventional

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD)

NCT07282847
View directory record Official record
Conditions
Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
Interventions
AB-1009 (GAA Gene)
Genetic
Lead sponsor
AskBio Inc
Industry
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
9
States / cities
Phoenix, Arizona • Irvine, California • Palo Alto, California + 6 more
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Oct 10, 2026, 6:05 PM EDT
Completed No phase listed Observational

Pompe Telemedicine Developmental Study

NCT02950298
View directory record Official record
Conditions
Pompe Disease, Glycogen Storage Disease II
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
6 Years to 18 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Oct 28, 2021 · Synced Oct 10, 2026, 6:05 PM EDT
Completed Phase 3 Interventional

A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

NCT00074971
View directory record Official record
Conditions
Fabry Disease
Interventions
Fabrazyme (agalsidase beta)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2004
U.S. locations
14
States / cities
Los Angeles, California • San Francisco, California • Coral Springs, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2013 · Synced Oct 10, 2026, 6:05 PM EDT
Recruiting No phase listed Observational

Longitudinal Prospective Natural History Study of Retinopathy in Zellweger Spectrum Disorder

NCT06190626
View directory record Official record
Conditions
Zellweger Spectrum Disorder
Interventions
Not listed
Lead sponsor
McGill University Health Centre/Research Institute of the McGill University Health Centre
Other
Eligibility
Not listed
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 10, 2026, 6:05 PM EDT
Terminated Phase 1Phase 2 Interventional

Liver Cell Transplant for Phenylketonuria

NCT01465100
View directory record Official record
Conditions
Phenylketonuria
Interventions
Preparative Radiation Therapy, Hepatocyte Transplant, Immunosuppression, Liver Evaluation, Neuro-psychological Assessment, Whole body Phe oxidation testing, Liver Biopsy
Radiation · Procedure · Drug + 3 more
Lead sponsor
Ira Fox
Other
Eligibility
14 Years to 55 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 16, 2023 · Synced Oct 10, 2026, 6:05 PM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

Study of Febuxostat Effect on Blood Pressure in Patients With High Normal Blood Pressure

NCT01472692
View directory record Official record
Conditions
Prehypertension, Gout, Pulse Wave Velocity, Hypertension, 24 Hour Blood Pressure
Interventions
Febuxostat
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
18 Years to 50 Years
Enrollment
47 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2014
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Dec 16, 2019 · Synced Oct 10, 2026, 6:05 PM EDT
Active, not recruiting No phase listed Observational

Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

NCT04098211
View directory record Official record
Conditions
Neuronal Ceroid-Lipofuscinoses, Neuronal Ceroid Lipofuscinosis CLN2, Spinocerebellar Ataxia, Autosomal Recessive 7
Interventions
Not listed
Lead sponsor
Children's Hospital of Orange County
Other
Eligibility
4 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 10, 2026, 6:05 PM 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 10, 2026, 6:05 PM EDT
Completed No phase listed Observational

Genetic Analysis of the Chiari I Malformation

NCT00004738
View directory record Official record
Conditions
Syringomyelia, Type I Arnold Chiari Malformation
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
1 Year and older
Enrollment
152 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 10, 2026, 6:05 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 10, 2026, 6:05 PM EDT
Completed Not applicable Interventional Results available

Repeated Eye Injections of Aflibercept for Treatment of Wet Age Related Macular Degeneration

NCT01773954
View directory record Official record
Conditions
Age-Related Macular Degeneration, Macular Degeneration
Interventions
Intravitreal Aflibercept Injection
Drug
Lead sponsor
MidAtlantic Retina
Other
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
3
States / cities
Cherry Hill, New Jersey • Huntingdon Valley, Pennsylvania • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 2, 2018 · Synced Oct 10, 2026, 6:05 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 10, 2026, 6:05 PM EDT
Terminated No phase listed Observational

Oxidative Stress, Inflammation and Acute Decompensation in Urea Cycle Disorders

NCT01541722
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
Not listed
Lead sponsor
Mark Batshaw
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
10
States / cities
Los Angeles, California • Aurora, Colorado • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 2, 2015 · Synced Oct 10, 2026, 6:05 PM EDT
Completed Phase 4 Interventional Results available

A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders

NCT02246218
View directory record Official record
Conditions
Urea Cycle Disorder
Interventions
RAVICTI
Drug
Lead sponsor
Amgen
Industry
Eligibility
1 Day to 2 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
16
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 13 more
Source: ClinicalTrials.gov public record
Updated Jul 1, 2024 · Synced Oct 10, 2026, 6:05 PM EDT
Terminated No phase listed Observational

Study of Inborn Errors of Cholesterol Synthesis and Related Disorders

NCT00046202
View directory record Official record
Conditions
Lysosomal Storage Disease, Cholesterol Metabolism
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Day to 99 Years
Enrollment
344 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Oct 10, 2026, 6:05 PM EDT
Terminated Phase 1Phase 2 Interventional

VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease

NCT02898753
View directory record Official record
Conditions
Pompe Disease
Interventions
VAL-1221, RhGAA
Drug
Lead sponsor
Valerion Therapeutics, LLC
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
2
States / cities
Orange, California • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 2, 2020 · Synced Oct 10, 2026, 6:05 PM EDT
Completed Phase 3 Interventional Results available

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

NCT00891202
View directory record Official record
Conditions
Gaucher Disease, Type 1
Interventions
Eliglustat tartrate, Placebo
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2016
U.S. locations
6
States / cities
San Francisco, California • New Haven, Connecticut • Decatur, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 3, 2017 · Synced Oct 10, 2026, 6:05 PM EDT
Completed Not applicable Interventional

A Multiple Dose Crossover Pharmacokinetics Study to Evaluate the Effects of Food on the Absorption of Oral Ganciclovir

NCT00002251
View directory record Official record
Conditions
Cytomegalovirus Infections, HIV Infections
Interventions
Ganciclovir
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
13 Years to 60 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
2
States / cities
San Francisco, California • Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 10, 2026, 6:05 PM EDT
Completed Phase 1 Interventional

Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP)

NCT00739505
View directory record Official record
Conditions
Hypophosphatasia (HPP)
Interventions
Asfotase Alfa
Biological
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
2
States / cities
St Louis, Missouri • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 29, 2019 · Synced Oct 10, 2026, 6:05 PM EDT
Completed Not applicable Interventional Results available

Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease

NCT01472874
View directory record Official record
Conditions
Wilson Disease
Interventions
Once a day Trientine
Drug
Lead sponsor
Yale University
Other
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Apr 15, 2020 · Synced Oct 10, 2026, 6:05 PM EDT
Completed Phase 4 Interventional Results available

Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease

NCT02574286
View directory record Official record
Conditions
Gaucher Disease
Interventions
Velaglucerase alfa, Vitamin D
Drug · Dietary Supplement
Lead sponsor
Shire
Industry
Eligibility
18 Years to 70 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
8
States / cities
Beverly Hills, California • Los Angeles, California • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 1, 2022 · Synced Oct 10, 2026, 6:05 PM EDT
Completed Phase 2 Interventional Results available

Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder

NCT00345605
View directory record Official record
Conditions
Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors, Urea Cycle Disorders
Interventions
Sodium Phenylbutyrate, Arginine
Drug
Lead sponsor
Brendan Lee
Other
Eligibility
5 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 31, 2018 · Synced Oct 10, 2026, 6:05 PM EDT
Withdrawn Phase 2 Interventional

Apremilast Therapy for Acute Gouty Arthritis

NCT00997581
View directory record Official record
Conditions
Acute Gout
Interventions
apremilast, indomethacin SR
Drug
Lead sponsor
Dartmouth-Hitchcock Medical Center
Other
Eligibility
18 Years and older · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010
U.S. locations
1
States / cities
Lebanon, New Hampshire
Source: ClinicalTrials.gov public record
Updated Apr 2, 2018 · Synced Oct 10, 2026, 6:05 PM EDT
Recruiting Phase 1Phase 2 Interventional

A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia

NCT06525636
View directory record Official record
Conditions
X-linked Hypophosphatemia
Interventions
KK8123
Drug
Lead sponsor
Kyowa Kirin Co., Ltd.
Industry
Eligibility
18 Years to 65 Years
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
5
States / cities
San Francisco, California • New Haven, Connecticut • Indianapolis, Indiana + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 10, 2026, 6:05 PM EDT