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Showing 1–24 of 791 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Metabolic Inborn Brain Diseases Clear all
Completed Phase 2 Interventional Results available

Drug-drug Interaction Study

NCT01380743
View directory record Official record
Conditions
Pompe Disease
Interventions
duvoglustat, rhGAA
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
11
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Orange, California + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 25, 2025 · Synced Sep 24, 2026, 9:53 PM EDT
Terminated Not applicable Interventional

Moderate Intensity Exercise and Phenylketonuria

NCT01904708
View directory record Official record
Conditions
Phenylketonuria, PKU
Interventions
Acute Moderate Intensity Exercise
Other
Lead sponsor
Oregon Health and Science University
Other
Eligibility
14 Years to 17 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Oct 15, 2014 · Synced Sep 24, 2026, 9:53 PM EDT
Completed Phase 2 Interventional Results available

Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes

NCT00887562
View directory record Official record
Conditions
MELAS Syndrome
Interventions
Idebenone, Placebo
Drug · Other
Lead sponsor
Michio Hirano
Other
Eligibility
8 Years to 65 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 25, 2016 · Synced Sep 24, 2026, 9:53 PM EDT
Completed Phase 3 Interventional Results available

Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

NCT00925301
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat hydrochloride, Placebo
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
16 Years to 74 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
13
States / cities
Los Angeles, California • San Francisco, California • Decatur, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Oct 29, 2018 · Synced Sep 24, 2026, 9:53 PM EDT
Completed Phase 2 Interventional

An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease

NCT00071877
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 24, 2026, 9:53 PM EDT
Recruiting Phase 3 Interventional

A Long-Term Study of JNT-517 in Participants With Phenylketonuria

NCT06628128
View directory record Official record
Conditions
Phenylketonuria (PKU)
Interventions
JNT-517
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
4 Years and older
Enrollment
240 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
8
States / cities
Gainesville, Florida • Tampa, Florida • Portland, Oregon + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced Sep 24, 2026, 9:53 PM EDT
Completed Phase 2 Interventional Results available

Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders

NCT00551200
View directory record Official record
Conditions
Urea Cycle Disorders
Interventions
HPN-100, BUPHENYL®
Drug
Lead sponsor
Amgen
Industry
Eligibility
18 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
2
States / cities
Minneapolis, Minnesota • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 9, 2024 · Synced Sep 24, 2026, 9:53 PM EDT
Completed No phase listed Observational

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease

NCT00351156
View directory record Official record
Conditions
Gaucher Disease
Interventions
Blood sample
Procedure
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2007
U.S. locations
7
States / cities
San Francisco, California • Coral Springs, Florida • Decatur, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2010 · Synced Sep 24, 2026, 9:53 PM EDT
Active, not recruiting Not applicable Interventional

Home Based Tele-exercise for People With Chronic Neurological Impairments

NCT04564495
View directory record Official record
Conditions
Neurologic Disorder, Stroke, Spinal Cord Injuries, Cerebral Palsy, Hemiplegia, Hemiparesis, Multiple Sclerosis, Parkinson Disease, Trauma, Brain, Transverse Myelitis, Friedreich Ataxia
Interventions
Seated exercise
Behavioral
Lead sponsor
Burke Medical Research Institute
Other
Eligibility
18 Years to 75 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
White Plains, New York
Source: ClinicalTrials.gov public record
Updated Feb 4, 2025 · Synced Sep 24, 2026, 9:53 PM EDT
Completed No phase listed Observational

Metabolic Abnormalities in Children With Epilepsy

NCT00001325
View directory record Official record
Conditions
Generalized Epilepsy, Infantile Spasms, Metabolic Disease, Partial Epilepsy, Seizures
Interventions
18 FDG
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
80 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1992 – 2004
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 24, 2026, 9:53 PM EDT
Completed No phase listed Observational

Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors

NCT00728676
View directory record Official record
Conditions
Phenylketonuria
Interventions
Not listed
Lead sponsor
Shoji Yano
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jan 17, 2013 · Synced Sep 24, 2026, 9:53 PM EDT
Completed Not applicable Interventional

Simplified Diet Approach in Phenylketonuria

NCT02555579
View directory record Official record
Conditions
Phenylketonuria
Interventions
Dietary Protein Counting and Free Fruits and Vegetables
Behavioral
Lead sponsor
University of Nebraska
Other
Eligibility
13 Years to 65 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
4
States / cities
Omaha, Nebraska
Source: ClinicalTrials.gov public record
Updated Sep 6, 2023 · Synced Sep 24, 2026, 9:53 PM EDT
Completed Phase 2 Interventional Results available

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

NCT01043640
View directory record Official record
Conditions
Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Adrenoleukodystrophy (ALD), Krabbe Disease, Metachromatic Leukodystrophy (MLD), Sphingolipidoses, Peroxisomal Disorders
Interventions
Campath-1H, Cyclophosphamide, Busulfan, Allogeneic stem cell transplantation, Cyclosporine A, Mycophenolate Mofetil
Drug · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 21 Years
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2017
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 4, 2018 · Synced Sep 24, 2026, 9:53 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

NCT04884815
View directory record Official record
Conditions
Wilson Disease
Interventions
UX701, Standard of Care (SOC)
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2034
U.S. locations
12
States / cities
Los Angeles, California • Redwood City, California • Sacramento, California + 9 more
Source: ClinicalTrials.gov public record
Updated Jun 14, 2026 · Synced Sep 24, 2026, 9:53 PM EDT
Completed No phase listed Observational

rhGH Therapy on Hepatic Drug Metabolism

NCT00458991
View directory record Official record
Conditions
Growth Hormone Deficiency, Dwarfism
Interventions
Dextromethorphan and Caffeine
Drug
Lead sponsor
University of Louisville
Other
Eligibility
4 Years to 14 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2008
U.S. locations
4
States / cities
San Diego, California • Louisville, Kentucky • Kansas City, Missouri + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 26, 2017 · Synced Sep 24, 2026, 9:53 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

NCT01230801
View directory record Official record
Conditions
Pompe Disease
Interventions
BMN 701
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
13 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
3
States / cities
La Jolla, California • Gainesville, Florida • Kansas City, Kansas
Source: ClinicalTrials.gov public record
Updated Jun 10, 2018 · Synced Sep 24, 2026, 9:53 PM EDT
Completed Phase 3 Interventional Results available

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

NCT01074944
View directory record Official record
Conditions
Gaucher Disease
Interventions
Eliglustat tartrate
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
170 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
10
States / cities
San Diego, California • New Haven, Connecticut • Decatur, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 5, 2017 · Synced Sep 24, 2026, 9:53 PM EDT
Recruiting No phase listed Observational

Cognitive and Neurological Pathologies in Pompe Disease

NCT04639336
View directory record Official record
Conditions
Pompe Disease
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
0 Years to 18 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2029
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 3, 2026 · Synced Sep 24, 2026, 9:53 PM EDT
Not listed Phase 1 Interventional

Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

NCT03893071
View directory record Official record
Conditions
Niemann-Pick Disease, Type C1
Interventions
Hydroxypropyl-β-cyclodextrin
Drug
Lead sponsor
Cyclo Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jun 17, 2021 · Synced Sep 24, 2026, 9:53 PM EDT
Recruiting Not applicable Interventional

Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)

NCT07521930
View directory record Official record
Conditions
Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS), Muscular Disorders, Atrophic, Brain Stem Stroke, Spinal Cord Injuries (SCI), Muscular Dystrophies, Muscular Atrophy, Progressive, Pontine Hemorrhage, Pontine Infarction
Interventions
INTENT Neural Interface System
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 80 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Sep 24, 2026, 9:53 PM EDT
Completed Phase 2 Interventional Results available

A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

NCT00214500
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 55 Years · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
5
States / cities
Los Angeles, California • Decatur, Georgia • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 29, 2018 · Synced Sep 24, 2026, 9:53 PM EDT
Completed Phase 3 Interventional Results available

Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU

NCT00838435
View directory record Official record
Conditions
Phenylketonuria
Interventions
sapropterin dihydrochloride
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
Up to 6 Years
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2018
U.S. locations
12
States / cities
La Jolla, California • Orange, California • Tampa, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Dec 21, 2020 · Synced Sep 24, 2026, 9:53 PM EDT
Completed Phase 2 Interventional Results available

A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia

NCT02255435
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone Capsules, 2.5 mg, Omaveloxolone Capsules, 5 mg, Omaveloxolone Capsules, 10 mg, Placebo, Omaveloxolone Capsules, 20 mg, Omaveloxolone Capsules, 40 mg, Omaveloxolone Capsules, 80 mg, Omaveloxolone Capsules, 160 mg, Omaveloxolone Capsules, 300 mg, Omaveloxolone Capsules, 150 mg
Drug
Lead sponsor
Biogen
Industry
Eligibility
16 Years to 40 Years
Enrollment
172 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2025
U.S. locations
7
States / cities
Los Angeles, California • Gainesville, Florida • Tampa, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 24, 2026, 9:53 PM EDT
Recruiting Phase 1 Interventional

Study of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma or Relapsed/Refractory Osteosarcoma

NCT03721068
View directory record Official record
Conditions
Neuroblastoma, Osteosarcoma
Interventions
iC9.GD2.CAR.IL-15 T-cells, Cyclophosphamide, Fludarabine
Biological · Drug
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
18 Months and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2044
U.S. locations
2
States / cities
Atlanta, Georgia • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 26, 2026 · Synced Sep 24, 2026, 9:53 PM EDT