- Conditions
- Facial Angiofibroma, Tuberous Sclerosis
- Interventions
- rapamycin, placebo
- Drug
- Lead sponsor
- Dermatology Specialties Limited Partnership
- Industry
- Eligibility
- 6 Years to 65 Years
- Enrollment
- 107 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 6
- States / cities
- Phoenix, Arizona • La Jolla, California • St. Petersburg, Florida + 3 more
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 889–912
of 1,710
matching trials from the live ClinicalTrials.gov search.
- Conditions
- X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
- Interventions
- Allo-HSCT
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- Up to 17 Years · Male only
- Enrollment
- 59 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 6
- States / cities
- Los Angeles, California • Palo Alto, California • Boston, Massachusetts + 3 more
- Conditions
- Krabbe Disease
- Interventions
- FBX-101
- Biological
- Lead sponsor
- Forge Biologics, Inc
- Industry
- Eligibility
- Up to 18 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2026
- U.S. locations
- 3
- States / cities
- Orange, California • Ann Arbor, Michigan • Durham, North Carolina
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Interventions
- eteplirsen
- Drug
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Eligibility
- 7 Years to 16 Years · Male only
- Enrollment
- 109 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2019
- U.S. locations
- 37
- States / cities
- Phoenix, Arizona • Los Angeles, California • Sacramento, California + 32 more
- Conditions
- Syringomyelia, Type I Arnold Chiari Malformation
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 1 Year and older
- Enrollment
- 152 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2001
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Cancer
- Interventions
- cyproheptadine hydrochloride, placebo
- Drug · Other
- Lead sponsor
- University of South Florida
- Other
- Eligibility
- 2 Years to 21 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2014
- U.S. locations
- 14
- States / cities
- Long Beach, California • Hartford, Connecticut • Wilmington, Delaware + 10 more
- Conditions
- Friedreich Ataxia
- Interventions
- Vatiquinone
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Up to 6 Years
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2024
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Tetraplegia, Spinal Cord Injury, Brachial Plexus Injury, Muscular Dystrophy, ALS, Brainstem Stroke
- Interventions
- Implantation of ECoG sensors on the brain surface
- Device
- Lead sponsor
- University of Pittsburgh
- Other
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2015
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Behcet's Disease, Churg-Strauss Syndrome, Vasculitis, Central Nervous System, Giant Cell Arteritis, Wegener Granulomatosis, Henoch-Schoenlein Purpura, Microscopic Polyangiitis, Polyarteritis Nodosa, Takayasu's Arteritis
- Interventions
- Not listed
- Lead sponsor
- University of South Florida
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 386 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 1
- States / cities
- Tampa, Florida
- Conditions
- Epilepsy, Localization-related Epilepsy, Infantile Spasms, Lennox-Gastaut Syndrome, Polymicrogyria, Periventricular Heterotopias
- Interventions
- Not listed
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 4 Weeks to 60 Years
- Enrollment
- 4,150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2018
- U.S. locations
- 23
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • San Francisco, California + 19 more
- Conditions
- GM1 Gangliosidosis, GM2 Gangliosidosis, Gaucher Disease, Type 2, Tay-Sachs Disease, AB Variant Gangliosidosis GM2, Sandhoff Disease
- Interventions
- Not listed
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.
- Industry
- Eligibility
- 0 Months and older
- Enrollment
- 226 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 3
- States / cities
- Chicago, Illinois • Rochester, Minnesota • Fairfax, Virginia
- Conditions
- Phenylketonuria
- Interventions
- PTC923, Placebo
- Drug
- Lead sponsor
- PTC Therapeutics
- Industry
- Eligibility
- Not listed
- Enrollment
- 157 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 12
- States / cities
- Stanford, California • Aurora, Colorado • Gainesville, Florida + 9 more
- Conditions
- Hypertension
- Interventions
- 6R-BH4 (sapropterin dihydrochloride)
- Drug
- Lead sponsor
- BioMarin Pharmaceutical
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 84 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 24
- States / cities
- Hoover, Alabama • Huntsville, Alabama • Mobile, Alabama + 21 more
- Conditions
- Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
- Interventions
- Not listed
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- Not listed
- Enrollment
- 12,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2030
- U.S. locations
- 23
- States / cities
- Los Angeles, California • Orange, California • Palo Alto, California + 18 more
- Conditions
- Phenylketonurias
- Interventions
- Palynziq
- Drug
- Lead sponsor
- Emory University
- Other
- Eligibility
- 16 Years and older
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2028
- U.S. locations
- 2
- States / cities
- Atlanta, Georgia
- Conditions
- Epilepsy, Epilepsy, Temporal Lobe, Seizures
- Interventions
- anteromesial temporal resection, antiepileptic drugs
- Procedure · Drug
- Lead sponsor
- University of California, Los Angeles
- Other
- Eligibility
- 12 Years and older
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2007
- U.S. locations
- 12
- States / cities
- Phoenix, Arizona • Los Angeles, California • Atlanta, Georgia + 9 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
- Alzheimer's Disease in Down Syndrome
- Interventions
- Not listed
- Lead sponsor
- LuMind IDSC Foundation
- Other
- Eligibility
- 35 Years and older
- Enrollment
- 46 participants
- Timeline
- 2021 – 2024
- U.S. locations
- 5
- States / cities
- San Diego, California • Park Ridge, Illinois • Baltimore, Maryland + 2 more
- Conditions
- Vasculitis, Behcet's Disease, CNS Vasculitis, Cryoglobulinemic Vasculitis, Eosinophilic Granulomatosis With Polyangiitis (EGPA), Churg-Strauss Syndrome (CSS), Granulomatosis With Polyangiitis (GPA), Wegener's Granulomatosis, IgA Vasculitis, Henoch-Schoenlein Purpura (HSP), Microscopic Polyangiitis (MPA), Polyarteritis Nodosa (PAN), Takayasu Arteritis (TAK), Urticarial Vasculitis, Systemic Vasculitis
- Interventions
- Online questionnaires
- Other
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 18 Years to 50 Years · Female only
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2027
- U.S. locations
- 1
- States / cities
- Tampa, Florida
- Conditions
- Advanced Malignant Solid Neoplasm, Recurrent Childhood Ependymoma, Recurrent Childhood Malignant Germ Cell Tumor, Recurrent Childhood Medulloblastoma, Recurrent Childhood Non-Hodgkin Lymphoma, Recurrent Childhood Osteosarcoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Hepatoblastoma, Recurrent Langerhans Cell Histiocytosis, Recurrent Malignant Glioma, Recurrent Malignant Solid Neoplasm, Recurrent Neuroblastoma, Recurrent Primary Central Nervous System Neoplasm, Recurrent Rhabdoid Tumor, Refractory Childhood Malignant Germ Cell Tumor, Refractory Childhood Osteosarcoma, Refractory Childhood Rhabdomyosarcoma, Refractory Childhood Soft Tissue Sarcoma, Refractory Ependymoma, Refractory Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Refractory Hepatoblastoma, Refractory Langerhans Cell Histiocytosis, Refractory Malignant Glioma, Refractory Malignant Solid Neoplasm, Refractory Medulloblastoma, Refractory Neuroblastoma, Refractory Non-Hodgkin Lymphoma, Refractory Primary Central Nervous System Neoplasm, Refractory Rhabdoid Tumor, Wilms Tumor
- Interventions
- Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan, Computed Tomography, Erdafitinib, Laboratory Biomarker Analysis, Magnetic Resonance Imaging, Pharmacological Study, Positron Emission Tomography, Radionuclide Imaging, X-Ray Imaging
- Procedure · Drug · Other
- Lead sponsor
- National Cancer Institute (NCI)
- NIH
- Eligibility
- 12 Months to 21 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2026
- U.S. locations
- 114
- States / cities
- Birmingham, Alabama • Anchorage, Alaska • Mesa, Arizona + 91 more
- Conditions
- Hypopituitarism
- Interventions
- Arimidex (Anastrozole), Placebo, Growth Hormone
- Drug
- Lead sponsor
- Nemours Children's Clinic
- Other
- Eligibility
- 11 Years to 18 Years · Male only
- Enrollment
- 53 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2010
- U.S. locations
- 1
- States / cities
- Jacksonville, Florida
- Conditions
- Huntington Disease
- Interventions
- Pridopidine, Placebo
- Drug
- Lead sponsor
- Prilenia
- Industry
- Eligibility
- 25 Years and older
- Enrollment
- 499 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2024
- U.S. locations
- 26
- States / cities
- Davis, California • San Diego, California • Englewood, Colorado + 22 more
- Conditions
- Spina Bifida, Myelomeningocele
- Interventions
- Phase One: Exploration, Phase Two: Creation, Phase Three: Reflection, Aim 3
- Behavioral
- Lead sponsor
- Indiana University
- Other
- Eligibility
- 8 Years and older
- Enrollment
- 67 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2026
- U.S. locations
- 1
- States / cities
- Indianapolis, Indiana
- Conditions
- Classic Galactosemia
- Interventions
- AT-007
- Drug
- Lead sponsor
- Applied Therapeutics, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Berlin, New Jersey