| Berry-Kravis, Elizabeth |
NCT05358886: A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome |
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| Recruiting | 3 | 150 | US | BPN14770/ zatolmilast, Placebo | Tetra Discovery Partners | Fragile X Syndrome | 06/25 | 09/25 | | |
NCT05367960: An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome |
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| Enrolling by invitation | 3 | 300 | US | Zatolmilast/ BPN14770 | Tetra Discovery Partners | Fragile X Syndrome | 09/27 | 12/27 | | |
| Recruiting | 3 | 250 | Europe, US, RoW | ZYN002 - transdermal gel, cannabidiol formulated as a clear gel that can be applied to the skin (transdermal delivery), Placebo, Placebo Comparator, Matching Placebo | Zynerba Pharmaceuticals, Inc., Zynerba Pharmaceuticals, Inc. | Fragile X Syndrome | 05/25 | 05/25 | | |
NCT05163808: A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome |
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| Recruiting | 2/3 | 150 | US | zatolmilast, BPN14770, Placebo | Tetra Discovery Partners | Fragile X Syndrome | 09/25 | 12/25 | | |
| Completed | 2 | 15 | US | 5-Hydroxytryptophan, 5-HTP, Basic Vitamins, Ergoloid Mesylates, Medisca, Matching placebo for Ergoloid mesylates, Matching placebo for 5-Hydroxytryptophan | Elizabeth Berry-Kravis, FRAXA Research Foundation, Purposeful IKE | Fragile X Syndrome | 01/23 | 01/23 | | |
REVEAL, NCT05606614: Safety and Efficacy of TSHA-102 in Adolescent and Adult Females with Rett Syndrome ( Adult Study) |
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| Recruiting | 1/2 | 18 | Canada, US | TSHA-102 | Taysha Gene Therapies, Inc. | Rett Syndrome | 01/25 | 01/32 | | |
NCT06152237: Safety and Efficacy of TSHA-102 in Pediatric Females with Rett Syndrome (REVEAL Pediatric Study) |
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| Recruiting | 1/2 | 20 | Europe, Canada, US | TSHA-102 | Taysha Gene Therapies, Inc. | Rett Syndrome | 11/28 | 11/31 | | |
NCT05301361: Sensitivity of the NIH Toolbox to Stimulant Treatment in Intellectual Disabilities |
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| Enrolling by invitation | 1 | 68 | US | Methylphenidate Oral Solution | University of California, Davis, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Intellectual Disability, Fragile X Syndrome, Down Syndrome, Attention Deficit Hyperactivity Disorder | 09/25 | 09/26 | | |
| Available | N/A | | US | Arimoclomol | ZevraDenmark | Niemann-Pick Disease, Type C | | | | |
NCT02461420: Mapping the Genotype, Phenotype, and Natural History of Phelan-McDermid Syndrome |
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| Active, not recruiting | N/A | 205 | US | | Boston Children's Hospital, National Institute of Neurological Disorders and Stroke (NINDS), National Institutes of Health (NIH), Office of Rare Diseases (ORD), National Center for Advancing Translational Sciences (NCATS), Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Phelan-McDermid Syndrome Foundation | Phelan-McDermid Syndrome, Autism Spectrum Disorder, Intellectual Disability | 12/25 | 12/26 | | |
| Recruiting | N/A | 300 | Canada, US | | Boston Children's Hospital, University of California, San Diego, Angelman Syndrome Biomarker & Outcome Measure Consortium (A-BOM), Food and Drug Administration (FDA), Angelman Syndrome Foundation Canada, Foundation for Angelman Syndrome Therapeutics Canada, Foundation for Angelman Syndrome Therapeutics, Angelman Syndrome Foundation, Inc. | Angelman Syndrome | 08/25 | 08/25 | | |
| Active, not recruiting | N/A | 35 | US | Cerliponase Alfa, Brineura, Administration Kit | BioMarin Pharmaceutical | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | 08/30 | 08/30 | | |
