23 completed, 6 terminated, 5 withdrawn, 3 active not recruiting. 41 Phase 2, 10 Phase 1, 5 Phase 3.
Follow Imprinting Disorders: new trials by email Literature Review of Imprinting Disorders trials Research Report on Imprinting 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 |
|---|---|---|---|---|---|---|
| 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 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 Setmelanotide in Patients With Prader-Willi Syndrome Setmelanotide - Prader-Willi Syndrome, Obesity | Phase 2 | Active not recruiting | Rhythm Pharmaceuticals, Inc. | 18 | 2025-03-04 | 2026-11-13 |
| 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| An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001) NNZ-2591 - Prader-Willi Syndrome | Phase 2 | Withdrawn | Neuren Pharmaceuticals Limited | 2023-09-01 | 2024-12-09 | |
| A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype. Alogabat - Angelman Syndrome | Phase 2 | Completed | Hoffmann-La Roche | 48 | 2023-07-27 | 2025-12-04 |
| 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 |
| An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome NNZ-2591 - Angelman Syndrome | Phase 2 | Completed | Neuren Pharmaceuticals Limited | 17 | 2022-07-12 | 2025-01-31 |
| 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 | |
| HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome ION582 - Angelman Syndrome | Phase 1 / Phase 2 | Active not recruiting | Ionis Pharmaceuticals, Inc. | 71 | 2021-12-22 | 2026-08-20 |
| 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 |
Showing 20 of 41. Sign in to see all of them.
Prader-Willi Syndrome (31) · Angelman Syndrome (10) · Hyperphagia (6) · Obesity (6) · Social Behavior (2) · Anxiety Disorders (1) · Autism Spectrum Disorder (1) · Autistic Disorder (1) · Compulsive Behavior (1) · Disease (1) · Hoarding (1) · Mitochondrial Diseases (1) · Obsessive-Compulsive Disorder (1) · Rare Diseases (1) · Rett Syndrome (1)
Abnormalities, Multiple (41) · Chromosome Disorders (41) · Congenital Abnormalities (41) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (41) · Genetic Diseases, Inborn (41) · Nervous System Diseases (41) · Intellectual Disability (32) · Neurobehavioral Manifestations (32) · Neurologic Manifestations (32) · Nutritional and Metabolic Diseases (32) · Nutrition Disorders (31) · Overnutrition (31) · Overweight (31) · Obesity (25)
Oxytocin (7) · Cannabidiol (2) · Sodium Chloride (2) · cyclo-L-glycyl-L-2-allylproline (2) · setmelanotide (2) · CKD732 (1) · Cognitive Behavioral Therapy (1) · Levodopa (1) · Metoprolol (1) · Minocycline (1) · Tesofensine (1) · alpha-tocotrienol quinone (1) · cannabidivarin (1) · carbidopa, levodopa drug combination (1) · coenzyme Q10 (1)
University Hospital, Toulouse (5) · Radius Pharmaceuticals, Inc. (3) · Rhythm Pharmaceuticals, Inc. (3) · Eric Hollander (2) · Essentialis, Inc. (2) · Neuren Pharmaceuticals Limited (2) · Ultragenyx Pharmaceutical Inc (2) · Aardvark Therapeutics, Inc. (1) · Bright Minds Biosciences Pty Ltd (1) · ConSynance Therapeutics (1) · Edison Pharmaceuticals Inc (1) · GLWL Research Inc. (1) · Gedeon Richter Plc. (1) · Harmony Biosciences Management, Inc. (1) · Healx AI (1)
United States (29) · France (11) · Australia (6) · Italy (5) · Spain (5) · Israel (4) · United Kingdom (4) · Canada (3) · Germany (2) · Argentina (1) · Belgium (1) · Brazil (1) · Czechia (1) · Netherlands (1) · Portugal (1)
Eric Hollander, MD (3) · Ahmed Elkashef, MD (2) · David Meeker (2) · Jennifer Miller, MD (2) · Amit Rakhit, MD, MBA (1) · Anne Slavotinek, MD (1) · Carlos A. Bacino, MD (1) · Cary Fu, MD (1) · David Meeker, MD (1) · Eric Storch, Ph.D. (1) · Ingrid Tein, MD (1) · James Shaw (1)
PMID 21702900, PMID 26994593, PMID 28100688, PMID 28823614, PMID 30126448, PMID 33232306, PMID 33443117, PMID 35213714, PMID 36947593, PMID 40605372