Clinical trials: Prader Labhart Willi Syndrome, open to volunteers
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.
Prader-Willi Syndrome
The Intervention of Obesity in Children With Prader-Willi Syndrome Using Prebiotics and Probiotics
Prader-Willi syndrome (PWS) is a rare genetic disease, with hyperappetite and severe obesity. At present, there is no effective drugs and interventions to help control the appetite of PWS patients.
- Trial name
- NCT05791604
- Testing
- probiotics including B. lactis B420, B. lactis HN019, B. animalis Bb-12, L. rhamnosus GG, Prebiotics with galactomannan and oligofructose
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Children's Hospital of Fudan University
Silver Russell Syndrome, Beckwith-Wiedemann Syndrome
IDMet (RaDiCo Cohort) (RaDiCo-IDMet)
The goal of this observational study is to describe the natural history of imprinting disorders (IDs) according to their metabolic profile in all patients (adults and children) affected with an ID regardless of the severity of…
- Conditions
- Silver Russell Syndrome, Beckwith-Wiedemann Syndrome, Transient Neonatal Diabetes Mellitus, Angelman Syndrome
- Trial name
- IDMet (NCT05945576)
- Where
- 20 sites in France
- Volunteers
- 2,000 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
Prader-Willi Syndrome
Register of Patients With Prader-Willi Syndrome
Prader-Willi Syndrome (PWS) is a rare syndrome with a prevalence of 15 to 20 000 at birth. PWS represents a large fraction of mental retardation syndromes due to a genetic cause and the most frequent cause of genetic obesity.
- Trial name
- NCT02829684
- Testing
- Data collection
- Where
- France
- Volunteers
- 500 people
- Sponsor
- University Hospital, Toulouse
Prader-Willi Syndrome
A 2-Part Study to Assess Efficacy, Safety and Tolerability of BMB-101 for the Treatment of Patients With Prader-Willi Syndrome.
The goal of this clinical trial is to evaluate the safety and effects of a new drug called BMB-101 in people with Prader-Willi Syndrome (PWS).
- Trial name
- NCT07266324
- Testing
- BMB-101
- Where
- 2 sites in Australia
- Volunteers
- 16 people
- Sponsor
- Bright Minds Biosciences Pty Ltd
Prader-Willi Syndrome
Regulating Together for Prader-Willi Syndrome: A Group Behavioral Therapy for Emotion Dysregulation
The goal of this study is to help teens with Prader-Willi Syndrome (PWS) and their families learn practical strategies for managing issues like irritability, meltdowns, and anxiety.
- Trial name
- RT-PWS (NCT07607730)
- Testing
- Regulating Together group therapy
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's Mercy Hospital Kansas City
Prader-Willi Syndrome
Autistic Symptomatology and Sensory Profile in Children With Prader-Willi Syndrome
Prader-Willi Syndrome (PWS) is a rare neurodevelopmental disorder stemming from genetic damage in the 15q11-q13 region, leading to hypothalamic dysfunction.
- Trial name
- CASSPER (NCT06877715)
- Testing
- psychological and sensorial tests
- Where
- France
- Volunteers
- 75 people
- Sponsor
- University Hospital, Toulouse
Prader-Willi Syndrome, Hypothalamic Obesity
Tirzepatide in PWS, HO and GNSO
This research study is comparing the effectiveness of a weight loss medication called Tirzepatide in young adults with Prader-Willi Syndrome and/or hypothalamic obesity, as compared to young adults with obesity that is unrelated…
- Conditions
- Prader-Willi Syndrome, Hypothalamic Obesity, Obesity/Therapy
- Trial name
- NCT06901245
- Testing
- Tirzepatide
- Where
- 3 sites in United States
- Volunteers
- 36 people
- Sponsor
- Grace Kim
Intellectual Disability, Neurodevelopmental Disorders
Physical Activity and Community EmPOWERment Project
Purpose: Conduct a wait-list randomized controlled trial (RCT) of an inclusive physical activity program called PACE for adults with intellectual disability (ID) who are not yet showing signs of Alzheimer's Disease…
- Conditions
- Intellectual Disability, Neurodevelopmental Disorders, Autism Spectrum Disorder, Down Syndrome
- Trial name
- PACE (NCT06740162)
- Testing
- PACE Program
- Where
- 2 sites in United States
- Volunteers
- 376 people, healthy volunteers welcome
- Sponsor
- University of North Carolina, Chapel Hill
Prader-Willi Syndrome, PWS-like Syndrome
GROWing Up With Rare GENEtic Syndromes
Introduction Rare complex syndromes Patients with complex genetic syndromes, by definition, have combined medical problems affecting multiple organ systems, and intellectual disability is often part of the syndrome.
- Conditions
- Prader-Willi Syndrome, PWS-like Syndrome, Silver Russel Syndrome, Congenital Hypopituitarism
- Trial name
- GROW UR GENES (NCT04463316)
- Testing
- Retrospective file studies
- Where
- Netherlands
- Volunteers
- 600 people
- Sponsor
- dr. Laura C. G. de Graaff-Herder
Prader-Willi Syndrome
Brain Olfactory Pathways in Prader-Willi Syndrome
Studying the cerebral activity of children with Prader-Willi Syndrom (PWS) when the study propose to them nasal activations.
- Trial name
- PRADOL (NCT07006207)
- Testing
- olfactory stimulation, functional MRI
- Where
- France
- Volunteers
- 30 people
- Sponsor
- University Hospital, Toulouse
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
Cerebellar TMS and Satiety in Prader-Willi Syndrome
This study uses a noninvasive technique called transcranial magnetic stimulation (TMS) to study hyperphagia and satiety in Prader-Willi syndrome.
- Trial name
- NCT05938543
- Testing
- Repetitive Transcranial Magnetic Stimulation (rTMS)
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Brigham and Women's Hospital
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.
Rare Diseases, Amyloidosis
Institutional Registry of Rare Diseases
The goal of this observational study is to create a single macro registry system with data collection on common clinical features, grouping the different rare diseases (RD).
- Conditions
- Rare Diseases, Amyloidosis, Sarcoidosis, Phacomatosis
- Trial name
- NCT06573723
- Where
- Argentina
- Volunteers
- 380 people
- Sponsor
- Hospital Italiano de Buenos Aires
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.
Obstructive Sleep Apnea (OSA), Orofacial Myofunctional Disorders
Oromyofunctional Therapy: a Rehabilitation Program for OSA in Children With Down Syndrome and Prader-Willi Syndrome
Obstructive sleep apnea (OSA) is a prevalent medical condition with important implications for overall health and quality of life in both children. Therefore, it is important to treat OSA early and effectively.
- Trial name
- OROFIT-P (NCT07122505)
- Testing
- Orofacial myofunctional therapy
- Where
- Belgium
- Volunteers
- 60 people
- Sponsor
- University Ghent
Prader-Willi Syndrome
A Study of CSTI-500 in Patients With Prader-Willi Syndrome
This is a proof-of-concept, open-label, dose-escalation study to evaluate the safety, tolerability, pharmacokinetics, and efficacy of CSTI-500 in participants with genetically confirmed Prader-Willi Syndrome (PWS) who are 13 to…
- Trial name
- NCT07348601
- Testing
- CSTI-500
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- ConSynance Therapeutics
Fragile X Syndrome, Angelman Syndrome
Parent and Infant Inter(X)Action Intervention (PIXI)
The objective is to develop and test, through an iterative process, an intervention to address and support the development of infants with a confirmed diagnosis of a neurogenetic disorder with associated developmental delays or…
- Conditions
- Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome
- Trial name
- NCT03836300
- Testing
- Parent-Infant Inter(X)action Intervention (PIXI)
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- RTI International