77 completed, 16 unknown, 15 terminated, 14 recruiting. 39 Not applicable, 31 Phase 2, 25 Phase 3, 12 Phase 1, 7 Phase 4.
Follow Prader-Willi Syndrome: new trials by email Literature Review of Prader-Willi Syndrome trials Research Report on Prader-Willi Syndrome
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 |
| 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 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 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 |
| 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 | |
| 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 |
| ARD-101 for Treatment of PWS: The Hunger Elimination or Reduction Objective Trial Placebo, ARD-101 - Hyperphagia, Prader-Willi Syndrome | Phase 3 | Terminated | Aardvark Therapeutics, Inc. | 68 | 2024-12-20 | 2026-06-30 |
| The Hunger Elimination or Reduction Objective (HERO ) Open -Label Extension (OLE) Trial ARD-101 - Hyperphagia, Prader-Willi Syndrome | Phase 3 | Terminated | Aardvark Therapeutics, Inc. | 19 | 2024-07-23 | 2026-06-30 |
| Institutional Registry of Rare Diseases Rare Diseases, Amyloidosis | Recruiting | Hospital Italiano de Buenos Aires | 380 | 2024-07-01 | 2026-01-14 | |
| A Study of Pitolisant in Patients With Prader-Willi Syndrome Pitolisant tablet, Placebo tablet - Prader-Willi Syndrome | Phase 3 | Recruiting | Harmony Biosciences Management, Inc. | 134 | 2024-05-28 | 2026-08-25 |
| Oromyofunctional Therapy: a Rehabilitation Program for OSA in Children With Down Syndrome and Prader-Willi Syndrome Orofacial myofunctional therapy - Obstructive Sleep Apnea (OSA), Orofacial Myofunctional Disorders | Not applicable | Recruiting | University Ghent | 60 | 2024-04-15 | 2025-08-14 |
| A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment Part A: RM-718 or placebo (matched to specific RM-718 dose cohort), Part B: RM-718 or placebo (matched to specific RM-718 dose cohort), Part C: RM-718 - Hypothalamic Obesity, Prader-Willi Syndrome | Phase 1 / Phase 2 | Active not recruiting | Rhythm Pharmaceuticals, Inc. | 111 | 2024-03-05 | 2026-10-09 |
| Adolescent Projections During Transition in Prader-Willi Syndrome Phone interview - Prader-Willi Syndrome | Not applicable | Unknown | University Hospital, Toulouse | 20 | 2024-03-01 | 2024-02-28 |
| Respiratory Restriction in Prader-Willi Syndrome Spirometry and optoelectronic plethysmography - Prader-Willi Syndrome | Completed | Istituto Auxologico Italiano | 30 | 2024-01-16 | 2025-02-28 | |
| A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People With PWS tVNS, intermittent stimulation, tVNS, continuous stimulation - Prader-Willi Syndrome | Phase 3 | Active not recruiting | Foundation for Prader-Willi Research | 102 | 2024-01-08 | 2026-04-06 |
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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 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 25111587, PMID 25479548, PMID 26259133, PMID 26994593, PMID 27071367, PMID 27146407, PMID 28100688, PMID 28556449, PMID 28823614, PMID 29718281, PMID 30346530, PMID 30407275, PMID 30833118, PMID 31315600, PMID 31511031, PMID 31684997, PMID 33066107, PMID 33950234
2016 (1), 2017 (1), 2025 (1), K23RR021979 (1), R01AG076678 (1), U54HD061222 (1)