Clinical trials: Prader Willis Syndrome
Prader-Willi Syndrome
Mitochondrial Complex I Dysfunction in PWS
Prader-Willi Syndrome (PWS) is characterized by profound infantile hypotonia, growth delay, cognitive impairment, muscle weakness and exercise intolerance.
- Trial name
- NCT03831425
- Testing
- Coenzyme Q10
- Where
- Canada
- Sponsor
- The Hospital for Sick Children
Prader-Willi Syndrome
Effect of Intranasal Oxytocin on Dysphagia in Children and Adolescents With Prader-Willi Syndrome
This phase 2B is designed to test the effectiveness of intranasal Oxytocin on Prader Willi Syndrome (PWS). This is a prospective, multicentre, randomised, double-blind, Phase 2B clinical study planned to include around 24 PWS…
- Trial name
- DYSMOT (NCT05298085)
- Testing
- Oxytocin nasal spray
- Where
- 4 sites in France
- Volunteers
- 25 people
- Sponsor
- University Hospital, Toulouse
Hyperphagia in Prader-Willi Syndrome
OLE Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi Syndrome
To evaluate long-term safety and tolerability of carbetocin nasal spray (3.2 mg TID) in subjects with PWS
- Trial name
- NCT06420297
- Testing
- Carbetocin
- Where
- 30 sites in United States, Canada and 4 more
- Volunteers
- 168 people
- Sponsor
- ACADIA Pharmaceuticals Inc.
Prader-Willi Syndrome, Self-Injurious Behavior
Treatment of Self-Injurious Behavior in Individuals With Prader-Willi Syndrome
Prader-Willi syndrome (PWS) is a genetic disorder usually caused by the deletion of a specific gene. One of the symptoms of PWS is self-injurious behavior (SIB); a common form of SIB in PWS patients is skin picking.
- Trial name
- NCT00065923
- Testing
- Topiramate
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
22q11 Deletion Syndrome, DiGeorge Syndrome
SNP-based Microdeletion and Aneuploidy RegisTry (SMART)
This multi-center prospective observational study is designed to track birth outcomes and perinatal correlates to the Panorama prenatal screening test in the general population among ten thousand women who present clinically and…
- Conditions
- 22q11 Deletion Syndrome, DiGeorge Syndrome, Trisomy 21, Trisomy 18
- Trial name
- SMART (NCT02381457)
- Where
- 21 sites in United States, Australia and 4 more
- Volunteers
- 20,960 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Prader-Willi Syndrome
Observational Study of VYKAT™ XR in Patients With Prader-Willi Syndrome
Prospective observational study developed to generate more evidence of the safety profile, clinical characteristics and outcomes of patients with PWS treated with VYKAT XR.
- Trial name
- NCT07450664
- Testing
- VYKAT XR
- Where
- 25 sites in United States
- Volunteers
- 200 people
- Sponsor
- Soleno Therapeutics, Inc.
Prader-Willi Syndrome, Aggression
Guanfacine Extended Release for the Reduction of Aggression and Self-injurious Behavior Associated With Prader-Willi Syndrome
This is a placebo-controlled clinical trial to assess whether Guanfacine Extended Release (GXR) reduces aggression and self injurious behavior in individuals with Prader Willi Syndrome (PWS).
- Conditions
- Prader-Willi Syndrome, Aggression, Self-Injurious Behavior, Pathologic Processes
- Trial name
- PWS-GXR (NCT05657860)
- Testing
- Guanfacine Extended Release
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Maimonides Medical Center
Prader-Willi Syndrome
Study of Tesomet With Open-label Extension in Subjects With Prader-Willi Syndrome
This study will evaluate the safety and efficacy of Tesomet (tesofensine + metoprolol) in subjects with PWS.
- Trial name
- PWS (NCT05198362)
- Testing
- Tesomet
- Where
- United States
- Sponsor
- Saniona
Prader-Willi Syndrome
A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension
The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Prader Willi syndrome (PWS) ages 6 to 65 years.
- Trial name
- NCT04257929
- Testing
- Pitolisant oral tablets
- Where
- 13 sites in United States
- Volunteers
- 65 people
- Sponsor
- Harmony Biosciences Management, Inc.
Prader-Willi Syndrome
Dose Clinical Trial of Guanfacine Extended Release for the Reduction of Aggression and Self-injuries Behavior Associated With Prader-Willi Syndrome
This is a placebo-controlled clinical trial to assess the utility of Guanfacine Extended Release (GXR) in the management of patients with Prader Willi Syndrome (PWS) who have significant aggression or self-injury.
- Trial name
- NCT04066088
- Testing
- Guanfacine extended release (GXR)
- Where
- United States
- Sponsor
- NYU Langone Health