15 completed, 2 active not recruiting, 1 recruiting, 1 unknown. 20 Phase 1, 10 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 20 | Completed 15, Active not recruiting 2, Recruiting 1, Unknown 1, Withdrawn 1 |
| Phase 2 | 10 | Completed 6, Active not recruiting 2, Recruiting 1, Withdrawn 1 |
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 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 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 |
| A Pharmacokinetic and Safety Study of CSTI-500 in Subjects With Prader-Willi Syndrome CSTI-500 - Prader-Willi Syndrome | Phase 1 | Completed | ConSynance Therapeutics | 10 | 2022-11-14 | 2023-08-09 |
| 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 Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants RO7248824 - Angelman Syndrome | Phase 1 | Completed | Hoffmann-La Roche | 21 | 2021-04-29 | 2022-09-08 |
| A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS) RO7248824 - Angelman Syndrome | Phase 1 | Completed | Hoffmann-La Roche | 74 | 2020-08-19 | 2025-08-24 |
| tVNS in Children With Prader-Willi Syndrome tVNS - Prader-Willi Syndrome, Social Behavior | Phase 1 / Phase 2 | Withdrawn | Stanford University | 2020-07-01 | 2021-05-06 | |
| A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome GTX-102 - Angelman Syndrome | Phase 1 / Phase 2 | Completed | Ultragenyx Pharmaceutical Inc | 74 | 2020-02-24 | 2026-01-09 |
| Intranasal Oxytocin for Infants With Prader-Willi Syndrome Oxytocin, Placebo - Prader-Willi Syndrome | Phase 1 / Phase 2 | Completed | University of Florida | 15 | 2017-08-01 | 2020-03-26 |
| Single Dose Pharmacokinetic (PK) Study OV101 - Angelman Syndrome, Fragile X Syndrome | Phase 1 | Completed | Ovid Therapeutics Inc. | 12 | 2017-04-03 | 2017-11-30 |
| Oxytocin Trial in Prader-Willi Syndrome Intranasal oxytocin, Placebo - Prader Willi Syndrome | Phase 1 | Completed | University of Florida | 24 | 2015-03-01 | 2018-04-05 |
| Parents Resources for Decreasing the Incidence of Change Triggered Temper Outbursts Visual Scheduling, signalling change - Child Behavior | Phase 1 | Completed | Queen's University, Belfast | 80 | 2015-01-01 | 2020-02-17 |
| Deep Brain Stimulation for the Treatment of Obesity in Patients With Prader-Willi Syndrome Deep Brain Stimulation - Prader-Willi Syndrome | Phase 1 | Unknown | University of Sao Paulo General Hospital | 6 | 2014-10-01 | 2015-12-16 |
| Clinical Study of Diazoxide Choline Controlled-Release Tablet (DCCR) in Patients With Prader-Willi Syndrome DCCR, Placebo - Prader-Willi Syndrome | Phase 1 / Phase 2 | Completed | Essentialis, Inc. | 13 | 2014-04-01 | 2016-09-01 |
| Evaluation of Tolerance, Suckling and Food Intake After Repeated Nasals Administrations of Oxytocin in PWS Infants oxytocin - Prader Willi Syndrome | Phase 1 / Phase 2 | Completed | University Hospital, Toulouse | 18 | 2013-05-01 | 2024-02-21 |
| Assessment of the Pharmacokinetics of Circadin® in Children With Neurodevelopmental Disorders and Sleep Disturbances Circadin 2 mg, Circadin 10 mg - Sleep Problems | Phase 1 | Completed | Neurim Pharmaceuticals Ltd. | 16 | 2013-03-01 | 2014-04-04 |
| Bioequivalence of Two Somatropin Products (Norditropin® Versus Genotropin®) in Healthy Adult Volunteers somatropin - Genetic Disorder, Prader-Willi Syndrome | Phase 1 | Completed | Novo Nordisk A/S | 30 | 2011-07-14 | 2017-02-24 |
| Tolerance of Intranasal Administration of OT in Prader-Willi Newborn Babies Oxytocin - Prader-Willi Syndrome | Phase 1 / Phase 2 | Completed | University Hospital, Toulouse | 5 | 2011-07-01 | 2017-02-23 |
| Study on Tolerability of Levodopa/Carbidopa in Children With Angelman Syndrome Levodopa/Carbidopa (4:1) - Angelman Syndrome | Phase 1 | Completed | Boston Children's Hospital | 16 | 2009-01-01 | 2016-11-21 |
| Behavioral Treatment of Obsessive-Compulsive Symptoms in Youth With Prader-Willi Syndrome: A Pilot Project Cognitive-behavioral therapy, Wait-list control - Prader Willi Syndrome, Obsessive-compulsive Disorder | Phase 1 / Phase 2 | Completed | University of South Florida | 24 | 2008-09-01 | 2011-08-08 |
Prader-Willi Syndrome (12) · Angelman Syndrome (8) · Fragile X Syndrome (2) · Obesity (2) · Autism Spectrum Disorder (1) · Autistic Disorder (1) · Child Behavior (1) · Compulsive Behavior (1) · Disease (1) · Down Syndrome (1) · Genetic Diseases, Inborn (1) · Growth Disorders (1) · Hoarding (1) · Intellectual Disability (1) · Obsessive-Compulsive Disorder (1)
Abnormalities, Multiple (20) · Chromosome Disorders (20) · Congenital Abnormalities (20) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (20) · Nervous System Diseases (20) · Genetic Diseases, Inborn (19) · Neurobehavioral Manifestations (13) · Neurologic Manifestations (13) · Intellectual Disability (12) · Nutrition Disorders (12) · Nutritional and Metabolic Diseases (12) · Overnutrition (12) · Overweight (12) · Obesity (10)
Oxytocin (4) · Carbidopa (1) · Cognitive Behavioral Therapy (1) · Deep Brain Stimulation (1) · Human Growth Hormone (1) · Levodopa (1) · Melatonin (1) · Sodium Chloride (1) · carbidopa, levodopa drug combination (1) · gaboxadol (1)
Hoffmann-La Roche (2) · University Hospital, Toulouse (2) · University of Florida (2) · Boston Children's Hospital (1) · ConSynance Therapeutics (1) · Essentialis, Inc. (1) · Ionis Pharmaceuticals, Inc. (1) · MavriX Bio, LLC (1) · Neurim Pharmaceuticals Ltd. (1) · Novo Nordisk A/S (1) · Ovid Therapeutics Inc. (1) · Queen's University, Belfast (1) · Rhythm Pharmaceuticals, Inc. (1) · Stanford University (1) · Ultragenyx Pharmaceutical Inc (1)
United States (13) · France (4) · Netherlands (3) · United Kingdom (3) · Australia (2) · Israel (2) · Italy (2) · Spain (2) · Brazil (1) · Canada (1) · Germany (1)
David Meeker (1) · Durval Damiani (1) · Eric Storch, Ph.D. (1) · Italo Biaggioni, MD (1) · Jennifer L Miller, MD (1) · Jennifer Miller, MD (1) · Kate Woodcock, PhD (1) · Virginia Kimonis, MD (1) · Wen-Hann Tan, BMBS (1)
PMID 28100688, PMID 28823614, PMID 33232306, PMID 36947593, PMID 40646322
2019 (1)