Clinical trials: Intellectual Disabilities Nutritional
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.
Adrenoleukodystrophy, Adrenomyeloneuropathy
Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
This study is a national, non-randomized, open-label, multi-site with minimal risk study in adult with adrenomyeloneuropathy (AMN), childhood and adult subjects with cerebral ALD (cALD), juvenile/adult metachromatic…
- Conditions
- Adrenoleukodystrophy, Adrenomyeloneuropathy, Metachromatic Leukodystrophy, Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia
- Trial name
- MATRIX (NCT04925349)
- Testing
- Blood sample collection
- Where
- 2 sites in France
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Metabolic Syndrome X, Down Syndrome
Frequency of Metabolic Syndrome in Down Syndrome Patients
The aim of this study is to assess the frequency of metabolic syndrome in Down syndrome patients because the prevalence of diabetes mellitus and obesity is higher in individuals with Down syndrome than in the general population.
- Trial name
- NCT00864461
- Where
- Mexico
- Volunteers
- 96 people, healthy volunteers welcome
- Sponsor
- Universidad de Guanajuato
Angelman Syndrome, Prader-Willi Syndrome
Development of a Newborn Screening Assay for Angelman Syndrome and Prader-Willi Syndrome
The overall purpose of this project is to establish the capability of screening for Angelman syndrome (AS) and Prader-Willi syndrome (PWS) in public health newborn screening (NBS) programs, with an aim of developing and…
- Trial name
- NCT05783791
- Testing
- Newborn Screening Assay
- Where
- United States
- Volunteers
- 11 people, healthy volunteers welcome
- Sponsor
- University of Wisconsin, Madison
Intellectual Disability, Exercise
Effects of Guided Exercise on Functional Performance and Independence in Adults With Intellectual Disability
Adults with intellectual disabilities (ID) have significantly lower levels of fitness compared to the general population. The health benefits of enhanced muscular strength in the general population are well established.
- Conditions
- Intellectual Disability, Exercise, Obesity
- Trial name
- NCT04616638
- Testing
- Resistance Training for Empowerment (RT-POWER), A traditional RT intervention
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- University of Delaware
Hypotonia-Cystinuria Syndrome, Muscle Hypotonia
PREPL in Health and Disease
Evaluation of PREPL activity in healthy controls and known or possible PREPL deficient patients
- Conditions
- Hypotonia-Cystinuria Syndrome, Muscle Hypotonia, Healthy Volunteers, Dwarfism, Growth Hormone Deficiency
- Trial name
- PHD (NCT02263781)
- Testing
- Blood draw, muscle biopsy
- Where
- Belgium
- Volunteers
- 125 people, healthy volunteers welcome
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Vision Impairment, Hearing Impairment
Health Intervention for Adolescents With Intellectual Disability
People with intellectual disability die five to twenty years earlier than the general population. They also experience high levels of unrecognised disease and receive inadequate levels of health promotion or screening.
- Conditions
- Vision Impairment, Hearing Impairment, Obesity
- Trial name
- Ask (NCT00519311)
- Testing
- Health Intervention Package
- Where
- Australia
- Volunteers
- 732 people, healthy volunteers welcome
- Sponsor
- The University of Queensland
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
Down Syndrome
Parent Supported Weight Reduction in Down Syndrome
The purpose of this study is to determine whether a nutrition and physical activity education program for families of overweight or obese adolescents with Down syndrome is more effective when behavioral lifestyle change…
- Trial name
- NCT01256112
- Testing
- Parent Supported Behavioral Intervention, Nutrition/Activity Education (NAE)
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Prader-Willi Syndrome
CBDV vs Placebo in Children and Adults up to Age 30 With Prader-Willi Syndrome (PWS)
This study aims to examine the feasibility and safety of cannabidivarin (CBDV) as a treatment for children and young adults with PWS.
- Trial name
- NCT03848481
- Testing
- CBDV Compound
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Eric Hollander
Aggression, Intellectual Disability
Reducing Aggression Among People With an Intellectual Disability
The study is an randomised controlled trial (RCT) to test whether supplementation of vitamins, minerals and n-3 fatty acids may reduce aggressive behavior in people with intellectual disabilities.
- Trial name
- PSYVB (NCT03212092)
- Testing
- Multivitamin, mineral & n-3 FA
- Where
- 7 sites in Netherlands
- Volunteers
- 137 people, healthy volunteers welcome
- Sponsor
- Leiden University Medical Center
Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension
Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)
The study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose…
- Conditions
- Coronavirus Infection (COVID-19), Pulmonary Arterial Hypertension, Urinary Tract Infections in Children, Hypertension
- Trial name
- POPS or POP02 (NCT04278404)
- Testing
- The POP02 study is collecting bodily fluid samples (i.e., whole blood, effluent samples) of children prescribed the following drugs of interest per standard of care:
- Where
- 52 sites in United States, Canada
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Duke University
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
Child Behavior
Parents Resources for Decreasing the Incidence of Change Triggered Temper Outbursts
'PREDICTORS' (Parents Resources for Decreasing the Incidence of Change Triggered Temper Outbursts) aims to evaluate web-based training packages for caregivers of children who show frequent temper outbursts following changes to…
- Trial name
- PREDICTORS (NCT02567357)
- Testing
- Visual Scheduling, signalling change
- Where
- United Kingdom
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Queen's University, Belfast
Obesity
A Family-Based Weight Loss Intervention for Youth With Intellectual Disability
The purpose of this randomized controlled trial is twofold. First, to examine the efficacy of a 24-session, 6-month family-based behavioral intervention (FBBI) - as compared to a waitlist FBBI group, which later receives the same…
- Trial name
- HealthU (NCT02033642)
- Testing
- Family Based Behavioral Intervention, Maintenance
- Where
- United States
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- University of Massachusetts, Boston
Prader Willi Syndrome, Adrenal Insufficiency
Diagnosis of Central Adrenal Insufficiency in Patients With Prader-Willi Syndrome
The purpose of this study is to determine presence of central adrenal insufficiency in children with Prader Willi Syndrome using low dose (1 mcg) ACTH stimulation test compared to results of overnight metyrapone test.
- Trial name
- NCT02368379
- Testing
- Low dose (1 mcg) ACTH stimulation test, Overnight metyrapone test
- Where
- United States
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- Nationwide Children's Hospital
Prader-Willi Syndrome, Obesity
Understanding the Role of Gut Microbiota in Hyperphagia in Prader-Willi Syndrome
This study aims to use a high-fiber supplementation, an intervention known to create shifts in the gut microbiota towards a healthier structure, to explore the relationship between gut microbiota, appetite control and feeding…
- Trial name
- PWSGUT (NCT05541003)
- Testing
- NBT-NM108
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Rutgers, The State University of New Jersey
Diabetes Mellitus Type 2, Prader Willi Syndrome
Liraglutide Use in Prader-Willi Syndrome
Liraglutide is a glucagon like peptide -1 (GLP-1) agonist, which is approved for use in patients with type 2 diabetes. Studies of liraglutide have shown an appetite suppressive effect and has been associated with weight loss in…
- Trial name
- NCT01542242
- Testing
- Liraglutide
- Where
- Canada
- Volunteers
- 1 people, healthy volunteers welcome
- Sponsor
- Vancouver General Hospital
Prader Willi Syndrome, Obesity
Correlation of Hyperghrelinemia With Carotid Artery Intima-Media Thickness in Children With Prader-Willi Syndrome
Prader-Willi syndrome (PWS) is a genetic disorder characterized by childhood-onset obesity and endocrine dysfunction that leads to cardiovascular disability and early death within the first 3 decades of life.To assess the…
- Trial name
- NCT00474643
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Samsung Medical Center
Prader-Willi Syndrome
PWS Outcomes Assessment Study
This is a longitudinal study during which qualitative interviews will be conducted with caregivers of Soleno C601/C602 study subjects.
- Trial name
- NCT04102839
- Testing
- Interview
- Where
- United States
- Volunteers
- 59 people, healthy volunteers welcome
- Sponsor
- Soleno Therapeutics, Inc.