- Conditions
- Fragile X Syndrome
- Interventions
- AFQ056, Placebo, Language Intervention
- Drug · Other
- Lead sponsor
- Elizabeth Berry-Kravis
- Other
- Eligibility
- 32 Months to 6 Years
- Enrollment
- 110 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2022
- U.S. locations
- 14
- States / cities
- Davis, California • Denver, Colorado • New Haven, Connecticut + 11 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 73–96
of 246
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Danon Disease
- Interventions
- Not listed
- Lead sponsor
- University of California, San Diego
- Other
- Eligibility
- 0 Years to 100 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2024
- U.S. locations
- 1
- States / cities
- San Diego, California
- Conditions
- Mucopolysaccharidosis II
- Interventions
- tividenofusp alfa
- Drug
- Lead sponsor
- Denali Therapeutics Inc.
- Industry
- Eligibility
- Up to 18 Years
- Enrollment
- 99 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2027
- U.S. locations
- 8
- States / cities
- Oakland, California • Chicago, Illinois • Hackensack, New Jersey + 5 more
- Conditions
- Fragile X Syndrome
- Interventions
- AZD7325 (High-Dose), AZD7325 (Low-Dose), Placebo oral capsule
- Drug
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- 18 Years to 50 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Upper Extremity Impairments, Cerebral Palsy (CP), ALS - Amyotrophic Lateral Sclerosis, Rett Syndrome
- Interventions
- One-week trial of Obi Medical device, which provides robotic assistance with feeding to persons with upper extremity impairments who are unable to self-feed in the traditional manner using utensils
- Device
- Lead sponsor
- Desin LLC
- Industry
- Eligibility
- 5 Years and older
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025
- U.S. locations
- 1
- States / cities
- Jacksonville, Florida
- Conditions
- Fragile X Syndrome
- Interventions
- Zatolmilast/ BPN14770
- Drug
- Lead sponsor
- Tetra Discovery Partners
- Industry
- Eligibility
- 9 Years to 45 Years · Male only
- Enrollment
- 314 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 17
- States / cities
- Irvine, California • Orange, California • Sacramento, California + 14 more
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
- Interventions
- Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 55 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2027
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- X-linked Adrenoleukodystrophy
- Interventions
- Lorenzo's Oil
- Drug
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Months to 18 Years · Male only
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, 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 1, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus-Merzbacher-Like Disease, 1, 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
- Up to 18 Years
- Enrollment
- 236 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2024
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Fragile X Syndrome
- Interventions
- SPG601, Placebo
- Drug
- Lead sponsor
- Spinogenix
- Industry
- Eligibility
- 18 Years to 45 Years · Male only
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Autism, Autism Spectrum Disorder, Autistic Disorder, Autism, Akinetic, Autism; Atypical, Autism; Psychopathy, Autism Fragile Syndrome X, Autism With High Cognitive Abilities, Autism-Related Speech Delay, Autism, Susceptibility to, 6, Autism Spectrum Disorder High-Functioning, Autistic Thinking, Autism, Susceptibility to, X-Linked 6, Autistic Behavior
- Interventions
- No Intervention
- Other
- Lead sponsor
- ProgenaBiome
- Other
- Eligibility
- 6 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2030
- U.S. locations
- 1
- States / cities
- Ventura, California
- Conditions
- Fragile X Syndrome (FXS), Idiopathic Intellectual Developmental Disorder (IDD)
- Interventions
- [18F]flumazenil
- Drug
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 18 Years to 30 Years · Male only
- Enrollment
- 17 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2018
- U.S. locations
- 1
- States / cities
- Stanford, California
- Conditions
- Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
- Interventions
- Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D)
- Biological · Drug
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Up to 55 Years
- Enrollment
- 149 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2029
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Rett Syndrome
- Interventions
- Electroencephalogram (EEG) Recording
- Procedure
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 2 Years and older
- Enrollment
- 60 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders, Cholestasis
- Interventions
- Cholic Acids
- Drug
- Lead sponsor
- Mirum Pharmaceuticals, Inc.
- Industry
- Eligibility
- Not listed
- Enrollment
- 85 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1992 – 2009
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Rett Syndrome
- Interventions
- triheptanoin
- Drug
- Lead sponsor
- Center for Rare Neurological Diseases, Norcross, GA
- Other
- Eligibility
- 2 Years and older · Female only
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2022
- U.S. locations
- 1
- States / cities
- Norcross, Georgia
- Conditions
- Intellectual Disability, Epilepsy, Hypogonadisms, Microcephaly, Nervous System Malformations, Obesity
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 1 Week to 100 Years
- Enrollment
- 150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2053
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fragile X Syndrome
- Interventions
- Arbaclofen
- Drug
- Lead sponsor
- Seaside Therapeutics, Inc.
- Industry
- Eligibility
- 6 Years to 40 Years
- Enrollment
- 45 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 9
- States / cities
- Los Angeles, California • Sacramento, California • Chicago, Illinois + 6 more
- Conditions
- Fragile X Syndrome
- Interventions
- arbaclofen, placebo
- Drug
- Lead sponsor
- Seaside Therapeutics, Inc.
- Industry
- Eligibility
- 12 Years to 50 Years
- Enrollment
- 125 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2011
- U.S. locations
- 24
- States / cities
- Phoenix, Arizona • Long Beach, California • Sacramento, California + 20 more
- Conditions
- Fragile X Syndrome
- Interventions
- AFQ056
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 12 Years to 18 Years
- Enrollment
- 119 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 7
- States / cities
- Sacramento, California • Decatur, Georgia • Chicago, Illinois + 4 more
- Conditions
- Fragile X Syndrome
- Interventions
- Sertraline, Placebo
- Drug
- Lead sponsor
- Randi J. Hagerman, MD
- Other
- Eligibility
- 24 Months to 68 Months
- Enrollment
- 57 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2015
- U.S. locations
- 1
- States / cities
- Sacramento, California
- Conditions
- Fragile X Syndrome
- Interventions
- STX209, Placebo
- Drug
- Lead sponsor
- Seaside Therapeutics, Inc.
- Industry
- Eligibility
- 6 Years to 40 Years
- Enrollment
- 63 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2010
- U.S. locations
- 12
- States / cities
- Phoenix, Arizona • Los Angeles, California • Sacramento, California + 9 more
- Conditions
- Mucopolysaccharidosis II
- Interventions
- JR-141, Idursulfase, JR-141 or Idursulfase
- Drug
- Lead sponsor
- JCR Pharmaceuticals Co., Ltd.
- Industry
- Eligibility
- Not listed
- Enrollment
- 86 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 7
- States / cities
- Phoenix, Arizona • Oakland, California • Chicago, Illinois + 4 more
- Conditions
- Autism Spectrum Disorders, Psychosis, Fragile X Syndrome
- Interventions
- Not listed
- Lead sponsor
- University of North Carolina, Chapel Hill
- Other
- Eligibility
- 3 Years to 45 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 1
- States / cities
- Chapel Hill, North Carolina