- 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
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 1393–1416
of 1,711
matching trials from the live ClinicalTrials.gov search.
- 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
- 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