103 completed, 24 recruiting, 23 unknown, 19 terminated. 48 Not applicable, 41 Phase 2, 35 Phase 3, 20 Phase 1, 7 Phase 4.
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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 |
|---|---|---|---|---|---|---|
| Regulating Together for Prader-Willi Syndrome: A Group Behavioral Therapy for Emotion Dysregulation Regulating Together group therapy - Prader-Willi Syndrome | Not applicable | Recruiting | Children's Mercy Hospital Kansas City | 10 | 2027-01-01 | 2026-09-02 |
| CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS) obudanersen - Angelman Syndrome | Phase 3 | Recruiting | Ionis Pharmaceuticals, Inc. | 30 | 2026-09-30 | 2026-10-20 |
| A Natural History Study of Angelman Syndrome Angelman Syndrome, Neurodevelopmental Conditions | Recruiting | Massachusetts General Hospital | 40 | 2026-09-09 | 2026-10-13 | |
| BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome. rugonersen, Sham procedure - Angelman Syndrome | Phase 3 | Recruiting | OHB Pediatrics Ltd. | 165 | 2026-06-23 | 2026-11-06 |
| A Study of CSTI-500 in Patients With Prader-Willi Syndrome CSTI-500 - Prader-Willi Syndrome | Phase 2 | Recruiting | ConSynance Therapeutics | 12 | 2026-05-18 | 2026-10-21 |
| A Study of Multilocus Methylation Abnormalities in Subjects With Imprinting Disorders Methylation study - Imprinting Disorder, MLID | Not applicable | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 96 | 2026-04-01 | 2026-03-27 |
| A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome. BMB-101, Placebo - Prader-Willi Syndrome | Phase 2 | Not yet recruiting | Bright Minds Biosciences Pty Ltd | 16 | 2026-01-01 | 2025-12-05 |
| Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome VYKAT XR - Prader-Willi Syndrome | Enrolling by invitation | Soleno Therapeutics, Inc. | 200 | 2025-11-26 | 2026-10-14 | |
| A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome MVX-220 - Angelman Syndrome | Phase 1 / Phase 2 | Recruiting | MavriX Bio, LLC | 12 | 2025-10-29 | 2026-07-24 |
| A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) No intervention, GTX-102 - Angelman Syndrome | Phase 2 | Terminated | Ultragenyx Pharmaceutical Inc | 55 | 2025-10-13 | 2026-11-03 |
| A Study of Pitolisant in Participants With Prader-Willi Syndrome Pitolisant - Prader-Willi Syndrome | Phase 3 | Enrolling by invitation | Harmony Biosciences Management, Inc. | 150 | 2025-08-20 | 2026-03-17 |
| Brain Olfactory Pathways in Prader-Willi Syndrome olfactory stimulation, functional MRI - Prader-Willi Syndrome | Not applicable | Not yet recruiting | University Hospital, Toulouse | 30 | 2025-06-23 | 2025-06-05 |
| REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome obudanersen, Placebo - Angelman Syndrome | Phase 3 | Active not recruiting | Ionis Pharmaceuticals, Inc. | 156 | 2025-06-10 | 2026-09-29 |
| Tirzepatide in PWS, HO and GNSO Tirzepatide - Prader-Willi Syndrome, Hypothalamic Obesity | Phase 4 | Recruiting | Grace Kim | 36 | 2025-05-01 | 2025-09-16 |
| Autistic Symptomatology and Sensory Profile in Children With Prader-Willi Syndrome psychological and sensorial tests - Prader-Willi Syndrome | Recruiting | University Hospital, Toulouse | 75 | 2025-04-07 | 2025-05-08 | |
| A Study of Setmelanotide in Patients With Prader-Willi Syndrome Setmelanotide - Prader-Willi Syndrome, Obesity | Phase 2 | Active not recruiting | Rhythm Pharmaceuticals, Inc. | 18 | 2025-03-04 | 2026-02-12 |
| Functional Connectome in Prader-Willi Syndrome: Neuroimaging and AI to Assess Therapeutic Impact GH treatment (Somatropin), Eating - Prader Willi Syndrome, Hyperphagia | Completed | Corporacion Parc Tauli | 101 | 2025-03-01 | 2025-04-01 | |
| Silver Russell Syndrome, Parental Fertility and Assisted Reproductive Technology Syndrome, Russell Silver | Not yet recruiting | Assistance Publique - Hôpitaux de Paris | 125 | 2025-03-01 | 2025-03-17 | |
| Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome Eye tracking, Data collection from patients' medical files, questionnaires - Angelman Syndrome | Recruiting | Assistance Publique - Hôpitaux de Paris | 60 | 2025-02-25 | 2026-03-16 | |
| Physical Activity and Community EmPOWERment Project PACE Program - Intellectual Disability, Neurodevelopmental Disorders | Not applicable | Recruiting | University of North Carolina, Chapel Hill | 376 | 2025-01-10 | 2026-02-23 |
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Prader-Willi Syndrome (140) · Angelman Syndrome (51) · Obesity (28) · Hyperphagia (16) · Beckwith-Wiedemann Syndrome (10) · Fragile X Syndrome (7) · Genetic Diseases, Inborn (6) · Rett Syndrome (6) · Autism Spectrum Disorder (5) · Down Syndrome (5) · Intellectual Disability (5) · Rare Diseases (5) · Silver-Russell Syndrome (5) · Smith-Magenis Syndrome (5) · Tuberous Sclerosis (5)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (194) · Abnormalities, Multiple (192) · Chromosome Disorders (192) · Congenital Abnormalities (192) · Genetic Diseases, Inborn (188) · Nervous System Diseases (183) · Neurologic Manifestations (145) · Neurobehavioral Manifestations (144) · Nutritional and Metabolic Diseases (144) · Intellectual Disability (140) · Nutrition Disorders (139) · Overnutrition (139) · Overweight (139) · Obesity (112)
Oxytocin (11) · Human Growth Hormone (6) · Growth Hormone (5) · Blood Specimen Collection (3) · Levodopa (3) · carbidopa, levodopa drug combination (3) · gaboxadol (3) · pitolisant (3) · Absorptiometry, Photon (2) · CKD732 (2) · Cannabidiol (2) · Exenatide (2) · Hematologic Tests (2) · Melatonin (2) · Minocycline (2)
University Hospital, Toulouse (17) · Assistance Publique - Hôpitaux de Paris (9) · Samsung Medical Center (6) · Soleno Therapeutics, Inc. (6) · Istituto Auxologico Italiano (4) · Radius Pharmaceuticals, Inc. (4) · Ultragenyx Pharmaceutical Inc (4) · University of Florida (4) · Aardvark Therapeutics, Inc. (3) · Boston Children's Hospital (3) · Children's Mercy Hospital Kansas City (3) · Corporacion Parc Tauli (3) · Duke University (3) · Harmony Biosciences Management, Inc. (3) · Hoffmann-La Roche (3)
United States (95) · France (36) · Canada (19) · Spain (18) · Italy (17) · United Kingdom (17) · Australia (16) · Germany (9) · Israel (8) · Netherlands (8) · Belgium (7) · South Korea (6) · Denmark (4) · Poland (4) · Sweden (4)
Wen-Hann Tan, BMBS (5) · Carlos A. Bacino, MD (3) · Deepan Singh, MD (3) · Eric Hollander, MD (3) · Steven A. Skinner, MD (3) · Ahmed Elkashef, MD (2) · Andrea M Haqq, MD (2) · Carles Lerin, PhD (2) · David Meeker (2) · Dong-Kyu Jin, M.D (2) · Jennifer L Miller, MD (2)
PMID 38671361, PMID 33232306, PMID 37919617, PMID 10417280, PMID 12520259, PMID 12820840, PMID 16509530, PMID 16814942, PMID 20375405, PMID 20965292, PMID 21310373, PMID 21519036, PMID 21702900, PMID 21827860, PMID 22002941, PMID 22005709, PMID 22281937, PMID 22464072, PMID 22617594, PMID 22742782, PMID 23215555, PMID 23436915, PMID 23613152, PMID 23632904, PMID 23712453, PMID 24135152, PMID 24360789, PMID 24388429, PMID 24406482, PMID 24529259, PMID 24785862, PMID 24916642, PMID 24955611, PMID 25004354, PMID 25071871, PMID 25111587, PMID 25283752, PMID 25479548, PMID 25491305, PMID 26259133, PMID 26994593, PMID 27071367, PMID 27146407, PMID 28100688, PMID 28556449, PMID 28823614, PMID 29096777, PMID 29718281, PMID 30126448, PMID 30346530
2016 (1), 2017 (1), 2019 (1), 2025 (1), K23RR021979 (1), R01AG076678 (1), U10CA098543 (1), U10CA180886 (1), U54HD061222 (1), U54RR019478 (1)