Clinical trials: Prader Willis Syndrome
Prader-Willi Syndrome
An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)
A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Prader-Willi Syndrome.
- Trial name
- PWS-001 (NCT05879614)
- Testing
- NNZ-2591
- Where
- 4 sites in United States
- Sponsor
- Neuren Pharmaceuticals Limited
Prader-Willi Syndrome
Growth Hormone in Children Under 2 Years With Prader-Willi in Hospital of Sabadell
The PWS is a genetic disease with intellectual disabilities associated with multiple manifestations in other body systems. It is characterized by hypothalamic-pituitary abnormalities with severe hypotonia during the early years…
- Trial name
- NCT02205450
- Testing
- Recombinant Somatropin
- Where
- Spain
- Volunteers
- 16 people
- Sponsor
- Corporacion Parc Tauli
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
Prader-Willi Syndrome, Obesity
Plasma Adiponectin Level and Sleep Structures in Children With Prader-Willi Syndrome
Context: Adiponectin is an adipose tissue-derived hormone with an insulin sensitizing effect, and has been related to obstructive sleep apnea syndrome.
- Trial name
- NCT01622751
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- Samsung Medical Center
Sleep Disorder, Hyperphagia
Effect of Deep Propioceptive Stimulation in Prader-Willi Syndrome
The main objective is to study the effect of deep propioceptive stimulation with a heavy blanket for 2 weeks compared with a placebo blanket on sleep quality and behavior in patients with PWS
- Conditions
- Sleep Disorder, Hyperphagia, Behavior Disorders
- Trial name
- NCT05044000
- Testing
- Deep propioceptive stimulation
- Where
- Spain
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Corporacion Parc Tauli
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
Genetic Disorder, Prader-Willi Syndrome
Bioequivalence of Two Somatropin Products (Norditropin® Versus Genotropin®) in Healthy Adult Volunteers
This trial is conducted in United States of America (USA). The aim of this trial is to examine the bioequivalence of Norditropin® versus Genotropin® in healthy adult volunteers.
- Conditions
- Genetic Disorder, Prader-Willi Syndrome, Growth Disorder, Idiopathic Short Stature
- Trial name
- NCT01401244
- Testing
- somatropin
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Novo Nordisk A/S
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, Sarcopenia
Ultrasound to Assess Sarcopenia in Prader Willi Syndrome
The aim of the study is to examine the clinical applicability of ultrasound as a diagnostic tool for sarcopenia in individuals with PWS by investigating the association between ultrasound-derived measurements, DXA-derived…
- Conditions
- Prader-Willi Syndrome, Sarcopenia, Metabolic Syndrome
- Trial name
- PWS (NCT06448871)
- Testing
- Ultrasound assessment of the right lower extremity muscles, Dual-energy X-ray absorptiometry (DXA) scan
- Where
- Taiwan
- Volunteers
- 49 people
- Sponsor
- Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical Foundation
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
GH in Adults With PWS, Effect on Hypotonia Evaluated by Functional MRI, Relationship With Strength and Body Composition
Prader-Willi syndrome (PWS) is a genetic disorder associated with growth hormone (GH) deficiency, central hypotonia and hyperphagia that leads to life-threatening obesity.
- Trial name
- NCT03616509
- Testing
- Growth hormone
- Where
- Spain
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Corporacion Parc Tauli
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
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
Prader-Willi Syndrome
Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome
The primary objective of this study is to assess the efficacy of cannabidiol oral solution on hyperphagia-related behavior in patients with Prader-Willi Syndrome (PWS).
- Trial name
- NCT02844933
- Testing
- Cannabidiol
- Where
- 7 sites in United States
- Volunteers
- 7 people
- Sponsor
- Radius Pharmaceuticals, Inc.
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.
Prader-Willi Syndrome
A Clinical Evaluation of Non-Invasive Vagus Nerve Stimulation for Temper Outbursts in People With PWS
The goal of the VNS4PWS clinical study is to test the efficacy, safety, and acceptability of transcutaneous vagus nerve stimulation (tVNS) treatment in people with PWS.
- Trial name
- VNS4PWS (NCT06144645)
- Testing
- tVNS, intermittent stimulation, tVNS, continuous stimulation
- Where
- 18 sites in United States
- Volunteers
- 102 people
- Sponsor
- Foundation for Prader-Willi Research
Obesity, Over-weight
An Efficacy, Safety, and Pharmacokinetics Study of Beloranib in Obese Subjects With Prader-Willi Syndrome
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics for certain doses of beloranib in obese subjects with Prader-Willi Syndrome.
- Conditions
- Obesity, Over-weight, Prader-Willi Syndrome
- Trial name
- NCT01818921
- Testing
- ZGN-440 sterile diluent, 1.2 mg ZGN-440 for injectable suspension, 1.8 mg ZGN-440 for injectable suspension
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Zafgen, Inc.
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
Weight Change, Body
Weight Change in PWS Over Six Months
This is a completely text-messaging based study and all data will be collected via surveys administered on a mobile phone. For this study, we will gather and analyze data on changes in body weight over a 6-month interval in…
- Trial name
- NCT03585244
- Testing
- weekly weight
- Where
- United States
- Volunteers
- 180 people
- Sponsor
- Foundation for Prader-Willi Research
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