39 completed. 39 Phase 3, 9 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 39 | Completed 39 |
| Phase 2 | 9 | Completed 9 |
Follow Chromosome Disorders: new trials by email Literature Review of Chromosome Disorders trials Research Report on Chromosome Disorders
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Open-Label Extension Study of DCCR in Patients With Prader-Willi Syndrome DCCR - Prader-Willi Syndrome | Phase 3 | Completed | Soleno Therapeutics, Inc. | 77 | 2023-01-31 | 2026-10-13 |
| A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome BPN14770/ zatolmilast, Placebo - Fragile X Syndrome | Phase 3 | Completed | Tetra Discovery Partners | 171 | 2022-11-01 | 2026-07-10 |
| Blood Flow and Blood Pressure Investigation in Down Syndrome Exercise - Down Syndrome | Phase 3 | Completed | University of Illinois at Chicago | 48 | 2022-09-01 | 2024-04-18 |
| Effect of Intranasal Oxytocin on Dysphagia in Children and Adolescents With Prader-Willi Syndrome Oxytocin nasal spray, Placebo - Prader-Willi Syndrome | Phase 2 / Phase 3 | Completed | University Hospital, Toulouse | 25 | 2022-04-05 | 2023-06-01 |
| A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome zatolmilast, Placebo - Fragile X Syndrome | Phase 2 / Phase 3 | Completed | Tetra Discovery Partners | 163 | 2022-03-29 | 2026-08-31 |
| Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome ZYN002, Placebo - Fragile X Syndrome, Fragile X Syndrome (FXS) | Phase 3 | Completed | Harmony Biosciences Management, Inc. | 257 | 2021-09-13 | 2026-09-01 |
| Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS somatropin - GH naïve pediatric cohort, somatropin - GH treated cohort, somatropin - adult cohort - Prader-Willi Syndrome | Phase 3 | Completed | Pfizer | 33 | 2021-02-09 | 2026-01-14 |
| Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome OT, Placebo comparator - Prader-Willi Syndrome | Phase 3 | Completed | University Hospital, Toulouse | 52 | 2020-03-10 | 2023-06-01 |
| Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome 3.2 mg intranasal carbetocin, 9.6 mg intranasal carbetocin, placebo - Prader-Willi Syndrome | Phase 3 | Completed | Levo Therapeutics, Inc. | 130 | 2018-11-20 | 2022-07-26 |
| Electromyographic Study for the Help and Guidance of BoNTA Administration in the Treatment of Chronic Pelvic Floor Pain Clostridium botulinum type A (BoNTA) - Electromyography, EMG: Myopathy | Phase 3 | Completed | Jose Alberola-Rubio | 25 | 2018-10-29 | 2020-08-11 |
| Open-Label Extension Study of DCCR in PWS Followed by Double-Blind, Placebo-Controlled, Randomized Withdrawal Period DCCR, Placebo for DCCR - Prader-Willi Syndrome | Phase 3 | Completed | Soleno Therapeutics, Inc. | 115 | 2018-10-01 | 2024-04-19 |
| Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX) ZYN002 - Cannabidiol Transdermal Gel, Placebo Transdermal Gel - Fragile X Syndrome | Phase 2 / Phase 3 | Completed | Zynerba Pharmaceuticals, Inc. | 212 | 2018-06-12 | 2022-07-06 |
| A Study of Diazoxide Choline in Patients With Prader-Willi Syndrome DCCR, Placebo for DCCR - Prader-Willi Syndrome | Phase 3 | Completed | Soleno Therapeutics, Inc. | 127 | 2018-05-09 | 2023-09-21 |
| A Trial of Metformin in Individuals With Fragile X Syndrome Placebo Medication, Metformin - Fragile X Syndrome, Fragile X Mental Retardation Syndrome | Phase 2 / Phase 3 | Completed | University of California, Davis | 120 | 2018-04-30 | 2025-01-17 |
| Clinical Trial for Asses the Safety and Efficacy of Vitamin C and Vitamin E in Combination Versus Placebo for Treating Cognitive and Behavior Disorder in Children With Fragile X Syndrome Vitamin C 10mg/Kg Vitamin E 10 mg/Kg, Placebo - X Fragile Syndrome | Phase 3 | Completed | Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud | 34 | 2016-07-01 | 2022-07-22 |
| Evaluating the Effects of Tasimelteon vs Placebo on Sleep Disturbances in SMS tasimelteon, placebo - Smith-Magenis Syndrome, Circadian | Phase 2 / Phase 3 | Completed | Vanda Pharmaceuticals | 49 | 2015-09-01 | 2022-11-18 |
| Effects of Intranasal Administration of Oxytocin in Adults With Prader-Willi Syndrome Oxytocin (OXT) continuous, Placebo, Placebo continuous - Prader-Willi Syndrome | Phase 2 / Phase 3 | Completed | University Hospital, Toulouse | 50 | 2014-06-01 | 2025-12-05 |
| Study to Assess the Efficacy and Safety of Eutropin in Prader-Willi Syndrome Eutropin, Genotropin - Prader-Willi Syndrome | Phase 3 | Completed | LG Life Sciences | 34 | 2014-06-01 | 2019-06-27 |
| Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities Circadin 2/5/10 mg, Placebo - Sleep Disorders | Phase 3 | Completed | Neurim Pharmaceuticals Ltd. | 125 | 2013-10-01 | 2024-04-23 |
| A Clinical Study to Assess the Efficacy and Safety of DA-3002 DA-3002, Genotropin® - Turner's Syndrome | Phase 3 | Completed | Dong-A ST Co., Ltd. | 58 | 2013-02-01 | 2018-07-26 |
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Prader-Willi Syndrome (10) · Turner Syndrome (10) · Fragile X Syndrome (8) · Down Syndrome (5) · Dwarfism (3) · Genetic Diseases, Inborn (3) · Angelman Syndrome (2) · Autism Spectrum Disorder (2) · Beckwith-Wiedemann Syndrome (2) · Hypogonadism (2) · Smith-Magenis Syndrome (2) · Alzheimer Disease (1) · Behavior (1) · Cognitive Dysfunction (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (39) · Congenital Abnormalities (38) · Genetic Diseases, Inborn (36) · Nervous System Diseases (26) · Neurologic Manifestations (25) · Intellectual Disability (22) · Neurobehavioral Manifestations (22) · Abnormalities, Multiple (20) · Sex Chromosome Disorders (18) · Imprinting Disorders (14) · Female Urogenital Diseases (13) · Female Urogenital Diseases and Pregnancy Complications (13) · Male Urogenital Diseases (13) · Urogenital Diseases (13)
Human Growth Hormone (7) · Growth Hormone (3) · Leucovorin (3) · Oxytocin (3) · BPN14770 (2) · Doxorubicin (2) · Estradiol (2) · Vincristine (2) · Vitamin E (2) · arbaclofen placarbil (2) · Androgens (1) · Ascorbic Acid (1) · Botulinum Toxins, Type A (1) · Calcium Dobesilate (1) · Castor Oil (1)
Novo Nordisk A/S (3) · Soleno Therapeutics, Inc. (3) · University Hospital, Toulouse (3) · Children's Oncology Group (2) · Eli Lilly and Company (2) · Institut Jerome Lejeune (2) · Seaside Therapeutics, Inc. (2) · Tetra Discovery Partners (2) · Children's Mercy Hospital Kansas City (1) · Dong-A ST Co., Ltd. (1) · Ferring Pharmaceuticals (1) · Fundación Pública Andaluza para la Investigación de Málaga en Biomedicina y Salud (1) · Harmony Biosciences Management, Inc. (1) · Ipsen (1) · Jose Alberola-Rubio (1)
United States (21) · France (8) · United Kingdom (7) · Australia (5) · Canada (5) · New Zealand (4) · Spain (4) · Belgium (2) · Czechia (2) · Finland (2) · Germany (2) · Ireland (2) · Israel (2) · Italy (2) · Netherlands (2)
Paul Wang, M.D. (2) · Andrea Isidori, MD, PhD (1) · Andrea Lenzi, MD (1) · Anne L Angiolillo (1) · Anne Slavotinek, MD (1) · Byung Kyu Suh, M.D., Ph.D. (1) · Carlos A. Bacino, MD (1) · Cary Fu, MD (1) · Chang Jong Kim, M.D., Ph.D. (1) · Cheol Woo Ko, M.D., Ph.D. (1) · Choong Ho Shin, M.D., Ph.D. (1)
PMID 37919617, PMID 38671361, PMID 15784709, PMID 16757526, PMID 20084109, PMID 20733274, PMID 21619701, PMID 26994593, PMID 29096777, PMID 31281181, PMID 31511031, PMID 31532522, PMID 31982581, PMID 33232306, PMID 33395098, PMID 33411585, PMID 34863019, PMID 34893062, PMID 35072864, PMID 36434514, PMID 36633570, PMID 36639249, PMID 37890117, PMID 39121442, PMID 39625363, PMID 40315406, PMID 40436776, PMID 40588365, PMID 42486751
U10CA098543 (2), U10CA180886 (2), 2004 (1), 2025 (1), R01AG016381 (1)