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ClinicalTrials.gov public records Last synced May 21, 2026, 10:14 PM EDT

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

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Brain Diseases, Metabolic, Inborn Clear all
Not listed No phase listed Observational
View directory record Official record
Conditions
Gaucher Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 12, 2019 · Synced May 21, 2026, 10:14 PM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Wilson Disease
Interventions
Not listed
Lead sponsor
Prime Medicine, Inc.
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
7
States / cities
Los Angeles, California • Sacramento, California • New Haven, Connecticut + 4 more
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced May 21, 2026, 10:14 PM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Idebenone, Placebo
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
8 Years to 17 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
2
States / cities
Los Angeles, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 10, 2023 · Synced May 21, 2026, 10:14 PM EDT
Terminated Phase 1 Interventional
View directory record Official record
Conditions
Fabry Disease
Interventions
Iohexol
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
5 Years to 25 Years · Male only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
5
States / cities
La Jolla, California • Decatur, Georgia • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 15, 2020 · Synced May 21, 2026, 10:14 PM EDT
Completed Not applicable Interventional Results available
View directory record Official record
Conditions
Phenylketonuria
Interventions
MRI
Other
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years to 55 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 18, 2015 · Synced May 21, 2026, 10:14 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Gaucher Disease
Interventions
eliglustat
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years to 79 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
2
States / cities
Miami, Florida • Knoxville, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 9, 2017 · Synced May 21, 2026, 10:14 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Phenylketonurias
Interventions
Pegvaliase
Drug
Lead sponsor
Boston Children's Hospital
Other
Eligibility
18 Years to 65 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 3, 2021 · Synced May 21, 2026, 10:14 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 7, 2025 · Synced May 21, 2026, 10:14 PM EDT
Withdrawn No phase listed Observational
View directory record Official record
Conditions
Mild Cognitive Impairment (MCI), Alzheimer Disease (AD), Dementia With Lewy Bodies (DLB), Frontotemporal Lobar Degeneration (FTLD), Parkinsons Disease With Dementia (PDD), Transient Epileptic Amnesia (TEA), Temporal Lobe Epilepsy (TLE), Spinocerebellar Ataxias (SCA), HIV Associated Neurocognitive Disorder (HAND), Amyotrophic Lateral Sclerosis (ALS), Primary Lateral Sclerosis (PLS)
Interventions
Neuraceq
Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Timeline
2019 – 2025
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 11, 2019 · Synced May 21, 2026, 10:14 PM EDT
Recruiting No phase listed Observational
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 23, 2026 · Synced May 21, 2026, 10:14 PM EDT
Active, not recruiting No phase listed Observational
View directory record Official record
Conditions
Urea Cycle Disorder, Ornithine Transcarbamylase Deficiency, Citrullinemia 1, ARGI Deficiency, ASL Deficiency, Argininosuccinic Aciduria, ASS Deficiency, Hyperargininemia
Interventions
Not listed
Lead sponsor
Baylor College of Medicine
Other
Eligibility
6 Years to 65 Years
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
5
States / cities
Aurora, Colorado • Washington D.C., District of Columbia • Philadelphia, Pennsylvania + 2 more
Source: ClinicalTrials.gov public record
Updated Apr 26, 2026 · Synced May 21, 2026, 10:14 PM EDT
Completed No phase listed Observational
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 22, 2021 · Synced May 21, 2026, 10:14 PM EDT
Completed Phase 4 Interventional
View directory record Official record
Conditions
Wilson Disease
Interventions
zinc acetate
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1993
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced May 21, 2026, 10:14 PM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Healthy, Mental Disorder, Obsessive Compulsive Disorder, Schizophrenia, Tourette Syndrome
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
Not listed
Enrollment
112 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1998 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 21, 2026, 10:14 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced May 21, 2026, 10:14 PM EDT
Completed Phase 2 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Gaucher Disease Type 1
Interventions
N-acetylcysteine
Drug
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
33 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2025
U.S. locations
2
States / cities
Minneapolis, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced May 21, 2026, 10:14 PM EDT
Completed No phase listed Observational
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 14, 2025 · Synced May 21, 2026, 10:14 PM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Pelizaeus-Merzbacher Disease, PMD
Interventions
HuCNS-SC transplant in the lead-in phase
Biological
Lead sponsor
StemCells, Inc.
Industry
Eligibility
Male only
Enrollment
4 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 May 12, 2016 · Synced May 21, 2026, 10:14 PM EDT
Available No phase listed Expanded access
View directory record Official record
Conditions
Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
Interventions
CliniMACS CD34 Reagent System
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
Up to 65 Years
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 19, 2025 · Synced May 21, 2026, 10:14 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Phenylketonuria
Interventions
rAvPAL-PEG
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
16 Years to 70 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
9
States / cities
Aurora, Colorado • Gainesville, Florida • Louisville, Kentucky + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 25, 2019 · Synced May 21, 2026, 10:14 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Krabbe Disease
Interventions
FBX-101
Biological
Lead sponsor
Forge Biologics, Inc
Industry
Eligibility
1 Day to 12 Months
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jan 29, 2025 · Synced May 21, 2026, 10:14 PM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Phenylketonuria, Galactosemia, Inborn Errors of Metabolism
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2008
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Aug 19, 2014 · Synced May 21, 2026, 10:14 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jun 23, 2024 · Synced May 21, 2026, 10:14 PM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available
View directory record Official record
Conditions
Pompe Disease
Interventions
Thoracic MRI, Spirometry, Inspiratory Load Compensation, Maximal Inspiratory Pressure, Resting Breathing Pattern, Respiratory Muscle Endurance Test
Other
Lead sponsor
University of Florida
Other
Eligibility
20 Years to 65 Years
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2016
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jun 23, 2024 · Synced May 21, 2026, 10:14 PM EDT