Clinical trials: Prader Willi Syndrome
Microdeletion Syndromes, Trisomy 21
Development of Non-invasive Prenatal Test for Microdeletion and Other Genetic Syndromes Based on Cell Free DNA
The purpose of this study is to collect blood from families with a child who has been diagnosed with a chromosomal disorder including microdeletions in order to further develop a non-invasive prenatal screening test based on…
- Conditions
- Microdeletion Syndromes, Trisomy 21, Trisomy 18, Trisomy 13
- Trial name
- Microdel Triad (NCT02109770)
- Where
- 2 sites in United States
- Volunteers
- 216 people
- Sponsor
- Natera, Inc.
Prader-Willi Syndrome, Social Behavior
tVNS in Children With Prader-Willi Syndrome
The purpose of the current project is to test the impact of transcutaneous vagus nerve stimulation (tVNS) on social ability in children with Prader-Willi Syndrome (PWS).
- Trial name
- NCT04396470
- Testing
- tVNS
- Sponsor
- Stanford University
Prader-Willi Syndrome
Evaluation of the Psychological Profile of Adult Patients With Prader-Willi Syndrome
The primary aim of the present study is to investigate the psychological profile of adult patients with PWS and the fatigue experienced by family members.
- Trial name
- PROPSICOPWS (NCT06295315)
- Where
- Italy
- Volunteers
- 36 people
- Sponsor
- Istituto Auxologico Italiano
Prader-Willi Syndrome
Cortisol Activity in Patients With Prader-Willi Syndrome and Healthy Controls
The purpose of the study is to find out if people with Prader-Willi syndrome have a difference in the protein which changes inactive cortisone to the active stress hormone cortisol.
- Trial name
- NCT00932932
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Children's Mercy Hospital Kansas City
Prader-Willi Syndrome
A Study of Pitolisant in Patients With Prader-Willi Syndrome
This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global clinical study to assess the efficacy and safety of pitolisant in patients living with Prader-Willi syndrome.
- Trial name
- NCT06366464
- Testing
- Pitolisant tablet
- Where
- 57 sites in United States, Australia and 11 more
- Volunteers
- 134 people
- Sponsor
- Harmony Biosciences Management, Inc.
Hyperphagia in Prader-Willi Syndrome
Treatment of Hyperphagia Behavioral Symptoms in Children and Adults Diagnosed With Prader-Willi Syndrome
The purpose of this study is to evaluate the safety and effectiveness of intranasal FE 992097 in children and adults with Prader-Willi Syndrome.
- Trial name
- NCT01968187
- Testing
- FE 992097
- Where
- 3 sites in United States
- Volunteers
- 38 people
- Sponsor
- Ferring Pharmaceuticals
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
Hypothalamic Obesity, Prader-Willi Syndrome
A Study of RM-718 in Healthy Subjects and Patients With MC4R Pathway Impairment
The purpose of this study is to evaluate the safety, tolerability, and PK of RM-718 in healthy subjects with obesity and in patients with MC4R Pathway Impairment
- Conditions
- Hypothalamic Obesity, Prader-Willi Syndrome, PWS
- Trial name
- NCT06239116
- Testing
- Part C: RM-718, Part D: RM-718
- Where
- 7 sites in United States
- Volunteers
- 111 people, healthy volunteers welcome
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Sleep Apnea, Central, Prader-Willi Syndrome
Oxygen Versus Medical Air for Treatment of CSA in Prader Will Syndrome
The aim of this study is to determine if treatment with Medical Air (21% oxygen in room air) compared to supplemental oxygen (100% oxygen) will lead to similar improvements in the central apnea-hypopnea index (CAHI) for infants…
- Trial name
- NCT03031626
- Testing
- Medical Air vs Oxygen
- Where
- Canada
- Volunteers
- 9 people
- Sponsor
- The Hospital for Sick Children
Prader-Willi Syndrome
Study of the Efficacy of Topiramate in Patients With Prader Willi Syndrome Over 8 Weeks
There is no specific treatment for core symptoms of PWS. Regarding behavioral and psychiatric symptoms (hyperphagia, imulsivity and self-mutilations), one of the only drug options consists in antipsychotics, that are not…
- Trial name
- TOPRADER (NCT02810483)
- Testing
- Topiramate
- Where
- France
- Volunteers
- 69 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Prader-Willi Syndrome
Intranasal Oxytocin for Infants With Prader-Willi Syndrome
The purpose of this study is to compare the change in suck and swallow competency from baseline to morning of day 6 with intranasal oxytocin spray vs placebo in infants/children with Prader-Willi Syndrome who are in nutritional…
- Trial name
- NCT03245762
- Testing
- Oxytocin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- University of Florida
Prader-Willi Syndrome
A Study of Pitolisant in Participants With Prader-Willi Syndrome
The purpose of this study is to assess safety of pitolisant in eligible participants with Prader-Willi syndrome (PWS) who completed the End of Treatment (EOT) visit in a parent study (HBS-101-CL-002 [Open Label Extension (OLE)]…
- Trial name
- NCT07219485
- Testing
- Pitolisant
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Harmony Biosciences Management, Inc.
Prader-Willi Syndrome
A Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Participants With Prader-Willi Syndrome
The objective of this study is to assess the long-term safety and tolerability of Cannabidiol Oral Solution (CBD) in participants with Prader-Willi Syndrome.
- Trial name
- NCT03458416
- Testing
- Cannabidiol Oral Solution
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Radius Pharmaceuticals, Inc.
Silver Russell Syndrome, Prader-Willi Syndrome
Aromatase Inhibitor in Bone Maturation, Children With Silver Russell or Prader-Willi Syndrome
There is currently no drug with pediatric marketing authorization capable of limiting the advance in bone maturation of children with aggressive adrenarche.
- Trial name
- ANASILPRA (NCT01520467)
- Testing
- Anastrozole
- Where
- France
- Volunteers
- 27 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Prader-Willi Syndrome
Effects of Transcutaneous Vagus Nerve Stimulation on Emotion Regulation and Executive Functioning in Prader-Willi Syndrome
The STIM-PRADER study aims to assess the effectiveness of auricular vagal neuromodulation therapy (aVNT) on emotional, behavioral, and cognitive domains impaired in Prader-Willi Syndrome (PWS).
- Trial name
- STIM-PRADER (NCT06720571)
- Testing
- Auricular vagal neuromodulation therapy (aVNT)
- Where
- 4 sites in France
- Volunteers
- 24 people
- Sponsor
- University of Bordeaux
Prader-Willi Syndrome
Follow-up of Prader Willi Syndrome Infants Treated by Oxytocin and Comparison With Not-treated Infants.
The objective of this study is to collect data on tolerance and effects of early treatment with oxytocin in children with Prader Willi Syndrome aged from 3 to 4 years and to compare these infants with not treated age-matched…
- Trial name
- OT2SUITE (NCT03081832)
- Testing
- Oxytocin, Control
- Where
- France
- Volunteers
- 34 people
- Sponsor
- University Hospital, Toulouse
Prader-Willi Syndrome
Oxytocin Intranasal Administrations in Children With Prader-Willi Syndrome Aged From 3 to 12 Years
Positive results in preclinical and clinical studies in adults and infants with Prader-Willi syndrome lead investigators to set up a new study in children with Prader-Willi syndrome.
- Trial name
- Oxyjeune (NCT03114371)
- Testing
- Oxytocin
- Where
- France
- Volunteers
- 40 people
- Sponsor
- University Hospital, Toulouse
Rett Syndrome, Prader-Willi Syndrome
Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW
This study will investigate sleep behavior in subjects with Angelman Syndrome, Rett Syndrome or Prader-Willi Syndrome. The study will also investigate sleep behavior in healthy siblings of subjects with Angelman Syndrome, Rett…
- Conditions
- Rett Syndrome, Prader-Willi Syndrome, Angelman Syndrome, Sleep Problems
- Trial name
- RDCRN (NCT02670694)
- Where
- 9 sites in United States
- Volunteers
- 804 people, healthy volunteers welcome
- Sponsor
- Baylor College of Medicine
Mental Disorder, Fetus Disorder
Diagnosis of Prader-Willi Syndrome and Angelman Syndrome
In a retrospective study, data were assessed from cases regarding PWS/AS that underwent molecular diagnosis at the National Chen-Kung University Hospital, Tainan, Taiwan, between January 2001 and December 2014.
- Trial name
- NCT04155944
- Where
- Taiwan
- Volunteers
- 60 people
- Sponsor
- National Cheng-Kung University Hospital
Prader Willi Syndrome
Post Exercise Irisin Levels in PWS Patients
Post exercise irisin levels in PWS patients Obesity, short stature, hypogonadism, hypotonia and impaired cognition are the major clinical features of Prader-Willi syndrome (PWS), a complex neurogenetic disorder due to lack of…
- Trial name
- NCT02728544
- Testing
- exercise and blood tests
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Shaare Zedek Medical Center