- Conditions
- Friedreich Ataxia
- Interventions
- Omaveloxolone
- Drug
- Lead sponsor
- Biogen
- Industry
- Eligibility
- 16 Years and older · Female only
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2035
- U.S. locations
- 1
- States / cities
- Morrisville, North Carolina
Search Results
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Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- Conditions
- Huntington's Disease
- Interventions
- Not listed
- Lead sponsor
- CHDI Foundation, Inc.
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 35,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2062
- U.S. locations
- 62
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Irvine, California + 54 more
- Conditions
- Urea Cycle Disorder
- Interventions
- Acetohydroxamic Acid Oral Tablet, No treatment
- Drug · Other
- Lead sponsor
- Nicholas Ah Mew
- Other
- Eligibility
- 18 Years to 60 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2018
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Congenital Fibrosis of Extraocular Muscles, Duane Retraction Syndrome, Duane Radial Ray Syndrome, Mobius Syndrome, Brown Syndrome, Marcus Gunn Syndrome, Strabismus Congenital, Horizontal Gaze Palsy, Horizontal Gaze Palsy With Progressive Scoliosis, Facial Palsy, Facial Paresis, Hereditary, Congenital, Third Nerve Palsy, Fourth Nerve Palsy, Sixth Nerve Palsy, Synkinesis, Ocular Motility Disorders, Levator-Medial Rectus Synkinesis, Athabaskan Brainstem Dysgenesis, Tongue Paralysis, Ninth Nerve Disorder, Fifth Nerve Palsy, Seventh Nerve Palsy, Eleventh Nerve Disorder, Twelfth Nerve Disorder, Vagus Nerve Paralysis, Moebius Sequence
- Interventions
- Not listed
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 1 Day and older
- Enrollment
- 20,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2004 – 2030
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Huntington Disease, Huntington's Dementia, Huntington Disease, Late Onset, Huntington; Dementia (Etiology)
- Interventions
- Biospecimen Collection
- Other
- Lead sponsor
- Sanguine Biosciences
- Industry
- Eligibility
- 25 Years to 65 Years
- Enrollment
- 5 participants
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Woburn, Massachusetts
- Conditions
- Phenylketonurias
- Interventions
- Not listed
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 14 Years and older
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 14
- States / cities
- Aurora, Colorado • Gainesville, Florida • Tampa, Florida + 11 more
- Conditions
- Argininosuccinic Aciduria, Carbamoyl-Phosphate Synthase I Deficiency, Citrullinemia, Ornithine Carbamoyltransferase Deficiency, Hyperargininemia, N-Acetylglutamate Synthase Deficiency
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 2 Years to 99 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2013
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Child, Only, Brain Diseases, Sickle Cell Disease, Anemia, Sickle Cell
- Interventions
- Carbon Dioxide
- Drug
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- 4 Years to 21 Years
- Enrollment
- 120 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2026
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Neural Tube Defects
- Interventions
- Not listed
- Lead sponsor
- John P. Hussman Institute for Human Genomics
- Other
- Eligibility
- Not listed
- Enrollment
- 6,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- Started 2009
- U.S. locations
- 1
- States / cities
- Miami, Florida
- Conditions
- Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia 3
- Interventions
- Not listed
- Lead sponsor
- The Methodist Hospital Research Institute
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2023
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Fabry Disease, Healthy
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Male only
- Enrollment
- 24 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2000 – 2001
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Tourette Syndrome
- Interventions
- NBI-98854
- Drug
- Lead sponsor
- Neurocrine Biosciences
- Industry
- Eligibility
- 6 Years to 18 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2019
- U.S. locations
- 9
- States / cities
- Anaheim, California • San Diego, California • Gulf Breeze, Florida + 6 more
- 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
- Conditions
- Constitutional Mismatch Repair Deficiency Syndrome, Lynch Syndrome, Malignant Glioma, Recurrent Brain Neoplasm, Recurrent Childhood Ependymoma, Recurrent Diffuse Intrinsic Pontine Glioma, Recurrent Medulloblastoma, Refractory Brain Neoplasm, Refractory Diffuse Intrinsic Pontine Glioma, Refractory Ependymoma, Refractory Medulloblastoma
- Interventions
- Biospecimen Collection, Conventional Magnetic Resonance Imaging, Diffusion Tensor Imaging, Diffusion Weighted Imaging, Dynamic Contrast-enhanced MR Perfusion, Dynamic Susceptibility Contrast-Perfusion-Weighted Imaging, Magnetic Resonance Spectroscopic Imaging, Pembrolizumab
- Procedure · Biological
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 1 Year to 30 Years
- Enrollment
- 71 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2027
- U.S. locations
- 11
- States / cities
- Los Angeles, California • Palo Alto, California • Aurora, Colorado + 8 more
- 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
- Conditions
- Tourette Syndrome
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 14 Years to 65 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2015
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fabry Disease, Alpha Galactosidase A Deficiency
- Interventions
- agalsidase beta
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- Not listed
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2024
- U.S. locations
- 3
- States / cities
- Decatur, Georgia • Fairfax, Virginia • Milwaukee, Wisconsin
- Conditions
- Osteoarthritis
- Interventions
- tanezumab 10 mg, tanezumab 5 mg, oxycodone, placebo
- Biological · Drug · Other
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 614 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2011
- U.S. locations
- 66
- States / cities
- Birmingham, Alabama • Mobile, Alabama • Phoenix, Arizona + 54 more
- Conditions
- Spinal Muscular Atrophy 1
- Interventions
- AVXS-101
- Biological
- Lead sponsor
- Novartis Gene Therapies
- Industry
- Eligibility
- Up to 6 Months
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2017
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Fabry Disease
- Interventions
- AMT-191
- Drug
- Lead sponsor
- UniQure Biopharma B.V.
- Industry
- Eligibility
- 18 Years to 50 Years · Male only
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2031
- U.S. locations
- 8
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 5 more
- Conditions
- Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
- Interventions
- SPK-3006
- Genetic
- Lead sponsor
- Spark Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2032
- U.S. locations
- 10
- States / cities
- Phoenix, Arizona • Orange, California • Atlanta, Georgia + 7 more
- Conditions
- Unresectable or Metastatic Microsatellite Stable (MSS) Solid Tumor, Microsatellite Stable (MSS) Colon Cancer
- Interventions
- Copanlisib, Nivolumab
- Drug
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- 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
- Conditions
- Primary Cervical Dystonia, DYT 1 Dystonia
- Interventions
- Primary Cervical Dystonia (Trihexyphenidyl), Controls Primary Cervical Dystonia (Trihexyphenidyl), DYT 1 Dystonia (Healthy Control), DYT 1 Dystonia
- Drug · Other
- Lead sponsor
- University of Florida
- Other
- Eligibility
- 7 Years to 70 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2014 – 2017
- U.S. locations
- 1
- States / cities
- Gainesville, Florida