Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 22, 2026, 6:45 AM EDT

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Showing 1393–1416 of 1,711 matching trials from the live ClinicalTrials.gov search.
Conditions
Leigh Syndrome, Leigh Disease, Leigh's Necrotizing Encephalopathy, Subacute Necrotizing Encephalomyopathy, Subacute Necrotizing Encephalomyelopathy
Interventions
Not listed
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
0 Days to 100 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2030
U.S. locations
1
States / cities
Houston, Texas
Active, not recruiting No phase listed Observational

Spinraza in Adult Spinal Muscular Atrophy

NCT03709784
Conditions
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type 3
Interventions
Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care, One time survey
Drug · Other
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years to 70 Years
Enrollment
148 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
10
States / cities
Phoenix, Arizona • Washington D.C., District of Columbia • Baltimore, Maryland + 7 more
Conditions
Friedreich's Ataxia
Interventions
Acetyl-L-Carnitine
Drug
Lead sponsor
University of South Florida
Other
Eligibility
18 Years to 80 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Tampa, Florida
Conditions
Gaucher Disease, Type 1, Type 1 Gaucher Disease, Gaucher Disease
Interventions
Afegostat tartrate
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 74 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
10
States / cities
San Francisco, California • Coral Springs, Florida • Decatur, Georgia + 7 more
Conditions
Pantothenate Kinase-Associated Neurodegeneration
Interventions
Deferiprone oral solution
Drug
Lead sponsor
ApoPharma
Industry
Eligibility
5 Years and older
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Oakland, California
Conditions
Amblyopia, Strabismus
Interventions
Patching therapy, Glasses
Behavioral
Lead sponsor
The Cleveland Clinic
Other
Eligibility
6 Months to 90 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Cleveland, Ohio
Conditions
Angelman Syndrome
Interventions
Alogabat
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
5 Years to 17 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
6
States / cities
Chicago, Illinois • Boston, Massachusetts • New York, New York + 3 more
Conditions
Tourette Syndrome, Obsessive Compulsive Disorder
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
5 Years to 17 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1989
U.S. locations
1
States / cities
New Haven, Connecticut
Conditions
Williams Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Shprintzen Syndrome, Smith-Magenis Syndrome, DiGeorge Syndrome, Chromosome Abnormalities
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
0 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Houston, Texas
Completed Not applicable Interventional Results available

Spinal Stimulation and Mobility Devices

NCT05520359
Conditions
Cerebral Palsy, Stroke, Premature Birth, Hypoxic-Ischemic Encephalopathy, Periventricular Leukomalacia
Interventions
Mobility Device, Spinal Stimulation
Device
Lead sponsor
University of Washington
Other
Eligibility
4 Years to 70 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Seattle, Washington
Conditions
Gaucher Disease, Type 1
Interventions
velaglucerase alfa, imiglucerase
Biological
Lead sponsor
Shire
Industry
Eligibility
2 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Durham, North Carolina
Conditions
Canavan Disease
Interventions
AAV9 BBP-812
Biological
Lead sponsor
Aspa Therapeutics
Industry
Eligibility
Up to 30 Months
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
4
States / cities
Oakland, California • Chicago, Illinois • Boston, Massachusetts + 1 more
Conditions
Friedreich's Ataxia
Interventions
interferon γ-1b
Drug
Lead sponsor
Amgen
Industry
Eligibility
11 Years to 27 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
4
States / cities
Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 1 more
Conditions
Pompe Disease (Late-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenesis 2 Acid Maltase Deficiency
Interventions
Alglucosidase Alfa
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
9
States / cities
Orange, California • Gainesville, Florida • Kansas City, Kansas + 6 more
Conditions
Ryanodine Receptor 1-Related Myopathy, Ryanodine Receptor 1 Related Disorders
Interventions
Not listed
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
7 Years to 100 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Friedreich Ataxia, Rare Diseases
Interventions
Geneticlly confirmed disease causing FXN mutatuion, Healthy Control
Other
Lead sponsor
Friedreich's Ataxia Research Alliance
Other
Eligibility
4 Years to 21 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2028
U.S. locations
3
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania • Memphis, Tennessee
Conditions
Spinal Muscular Atrophy (SMA)
Interventions
onasemnogene abeparvovec
Biological
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
0 Years to 100 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
1
States / cities
Norfolk, Virginia
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
Conditions
Arteriovenous Malformations, Cavernous Angiomas, Brain Aneurysms
Interventions
Doxycycline or Placebo
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
13 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
1
States / cities
San Francisco, California
Conditions
Central Core Disease, Centronuclear Myopathy, Congenital Fiber Type Disproportion, Multiminicore Disease, Myotubular Myopathy, Nemaline Myopathy, Rigid Spine Muscular Dystrophy, Undefined Congenital Myopathy
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
4,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2050
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Brain and Central Nervous System Tumors, Neurofibromatosis Type 1
Interventions
carboplatin, vinblastine sulfate
Drug
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 21 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
19
States / cities
Birmingham, Alabama • Orange, California • Washington D.C., District of Columbia + 16 more
Conditions
Lennox Gastaut Syndrome, Dravet Syndrome, Tuberous Sclerosis Complex
Interventions
Cannabidiol
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
1 Year and older
Enrollment
154 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
26
States / cities
Little Rock, Arkansas • Downey, California • Long Beach, California + 22 more
Conditions
Intracerebral Hemorrhage
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
18 Years and older
Enrollment
2,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2033
U.S. locations
1
States / cities
Atlanta, Georgia
Conditions
Tourette Syndrome, Persistent Tic Disorder
Interventions
CBIT
Other
Lead sponsor
University of Minnesota
Other
Eligibility
10 Years to 17 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
2
States / cities
San Diego, California • Minneapolis, Minnesota