17 completed, 5 terminated, 4 withdrawn, 2 active not recruiting. 31 Phase 2, 7 Phase 1, 4 Phase 3.
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| 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 |
| An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001) NNZ-2591 - Prader-Willi Syndrome | Phase 2 | Withdrawn | Neuren Pharmaceuticals Limited | 2023-09-01 | 2024-12-09 | |
| Mitochondrial Complex I Dysfunction in PWS Coenzyme Q10, Placebo - Prader-Willi Syndrome | Phase 2 | Withdrawn | The Hospital for Sick Children | 2023-06-01 | 2024-06-18 | |
| Understanding the Role of Gut Microbiota in Hyperphagia in Prader-Willi Syndrome NBT-NM108 - Prader-Willi Syndrome, Obesity | Phase 2 | Completed | Rutgers, The State University of New Jersey | 12 | 2023-01-06 | 2024-07-16 |
| Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome RGH-706, Placebo - Prader-Willi Syndrome | Phase 2 | Completed | Gedeon Richter Plc. | 39 | 2022-09-22 | 2024-06-03 |
| A Study of Oral ARD-101 in Patients With Prader-Willi Syndrome ARD-101 - Prader-Willi Syndrome | Phase 2 | Completed | Aardvark Therapeutics, Inc. | 19 | 2022-05-27 | 2026-09-08 |
| A Study to Assess RAD011 (Cannabidiol Oral Solution) for the Treatment of Participants With Prader-Willi Syndrome RAD011, Placebo - Prader-Willi Syndrome | Phase 2 / Phase 3 | Terminated | Radius Pharmaceuticals, Inc. | 4 | 2022-04-13 | 2023-10-19 |
| 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 |
| Study of Tesomet With Open-label Extension in Subjects With Prader-Willi Syndrome Placebo, Tesomet - Prader-Willi Syndrome | Phase 2 | Withdrawn | Saniona | 2021-12-28 | 2022-12-13 | |
| A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension Pitolisant oral tablets, Placebo oral tablet - Prader-Willi Syndrome | Phase 2 | Completed | Harmony Biosciences Management, Inc. | 65 | 2020-12-09 | 2026-05-12 |
| CBDV vs Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS) CBDV Compound, Placebo - Prader-Willi Syndrome | Phase 2 | Terminated | Eric Hollander | 6 | 2020-11-23 | 2026-05-07 |
| tVNS in Children With Prader-Willi Syndrome tVNS - Prader-Willi Syndrome, Social Behavior | Phase 1 / Phase 2 | Withdrawn | Stanford University | 2020-07-01 | 2021-05-06 | |
| Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome Livoletide, Placebo - Prader-Willi Syndrome, Hyperphagia | Phase 2 / Phase 3 | Terminated | Millendo Therapeutics SAS | 158 | 2019-03-25 | 2021-02-17 |
| A Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Participants With Prader-Willi Syndrome Cannabidiol Oral Solution - Prader-Willi Syndrome | Phase 2 | Terminated | Radius Pharmaceuticals, Inc. | 2 | 2018-09-06 | 2023-06-22 |
| Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome Cannabidiol, Placebo - Prader-Willi Syndrome | Phase 2 | Terminated | Radius Pharmaceuticals, Inc. | 7 | 2018-05-09 | 2023-08-01 |
| Intranasal Oxytocin vs. Placebo for the Treatment of Hyperphagia in Prader-Willi Syndrome Intranasal Oxytocin (IN-OXT), Matched Placebo - Prader-Willi Syndrome, Hyperphagia | Phase 2 | Completed | Eric Hollander | 32 | 2018-04-11 | 2025-02-07 |
| A Study of GLWL-01 in Patients With Prader-Willi Syndrome GLWL-01, Placebo - Prader-Willi Syndrome | Phase 2 | Completed | GLWL Research Inc. | 19 | 2018-02-20 | 2020-03-27 |
Showing 20 of 31. Sign in to see all of them.
Hyperphagia (6) · Obesity (6) · Social Behavior (2) · Anxiety Disorders (1) · Compulsive Behavior (1) · Disease (1) · Hoarding (1) · Obsessive-Compulsive Disorder (1) · Sexual Infantilism (1)
Abnormalities, Multiple (31) · Chromosome Disorders (31) · Congenital Abnormalities (31) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (31) · Genetic Diseases, Inborn (31) · Imprinting Disorders (31) · Intellectual Disability (31) · Nervous System Diseases (31) · Neurobehavioral Manifestations (31) · Neurologic Manifestations (31) · Nutrition Disorders (31) · Nutritional and Metabolic Diseases (31) · Overnutrition (31) · Overweight (31) · Obesity (25)
Oxytocin (7) · Cannabidiol (2) · Sodium Chloride (2) · setmelanotide (2) · CKD732 (1) · Cognitive Behavioral Therapy (1) · Metoprolol (1) · Tesofensine (1) · cannabidivarin (1) · coenzyme Q10 (1) · cyclic des-acyl ghrelin (6-13) (1) · cyclo-L-glycyl-L-2-allylproline (1) · pitolisant (1)
University Hospital, Toulouse (5) · Radius Pharmaceuticals, Inc. (3) · Rhythm Pharmaceuticals, Inc. (3) · Eric Hollander (2) · Essentialis, Inc. (2) · Aardvark Therapeutics, Inc. (1) · Bright Minds Biosciences Pty Ltd (1) · ConSynance Therapeutics (1) · GLWL Research Inc. (1) · Gedeon Richter Plc. (1) · Harmony Biosciences Management, Inc. (1) · Millendo Therapeutics SAS (1) · Montefiore Medical Center (1) · Neuren Pharmaceuticals Limited (1) · Rutgers, The State University of New Jersey (1)
United States (22) · France (7) · Australia (2) · Canada (2) · Italy (2) · Spain (2) · Belgium (1) · Czechia (1) · Netherlands (1) · United Kingdom (1)
Eric Hollander, MD (3) · Ahmed Elkashef, MD (2) · David Meeker (2) · Jennifer Miller, MD (2) · David Meeker, MD (1) · Eric Storch, Ph.D. (1) · Ingrid Tein, MD (1) · Jordan Press (1) · Shawn McCandless, MD (1) · Sophie Guillaume, MS (1)
PMID 21702900, PMID 26994593, PMID 28100688, PMID 28823614, PMID 33232306, PMID 35213714, PMID 40605372