Characteristics of Prader-Willi Syndrome and Early-onset Morbid Obesity
|ClinicalTrials.gov Identifier: NCT00375089|
Recruitment Status : Completed
First Posted : September 12, 2006
Last Update Posted : September 22, 2014
|Condition or disease||Intervention/treatment|
|Prader-Willi Syndrome Obesity||Other: Group 1 Other: Group 2|
PWS is a complex neurobehavioral syndrome. Clinical features include obesity, increased appetite, low muscle tone, cognitive impairment, distinct behavioral features, hypogonadism, and neonatal failure-to-thrive. It is the most commonly recognized genetic cause of obesity; however, many obese children do not in fact have PWS. These individuals are therefore diagnosed with EMO, a condition that shares features with PWS. The development of new advances and strategies for treating PWS and EMO requires a thorough understanding of the conditions at both the clinical and molecular levels. One goal of this study is to collect long-term data on individuals with PWS and EMO in order to gain a better understanding of the natural progression of the conditions, from the neonatal period well into adulthood. Specific to PWS, this study will establish a genotype-phenotype correlation among the different sub-types and will evaluate the effects of growth hormone treatment on disease progression. Lastly, the study will compare PWS with EMO in terms of clinical features and genetic basis.
Participation in this natural history study will entail an initial evaluation, followed by yearly study visits until the age of 3 and then every 2 years thereafter. Each study visit will last between 3 and 4 hours, and will include a physical exam (including a DEXA scan to determine body composition), psychological testing, an interview with the study physician, and an evaluation of the participant's diet history. In addition, blood tests will be completed for genetic testing and photos will be taken to evaluate disease progression. Cognitive and behavioral assessments will also be conducted and will last between 10 and 30 minutes.
|Study Type :||Observational|
|Actual Enrollment :||392 participants|
|Official Title:||Prader-Willi Syndrome and Early-onset Morbid Obesity Natural History Clinical Protocol|
|Study Start Date :||September 2006|
|Actual Primary Completion Date :||January 2014|
|Actual Study Completion Date :||January 2014|
Individuals with Prader-Willi syndrome.
Other: Group 1
Individuals with Prader-Willi syndrome. Monitoring every 6 months.
Individuals with Early-onset Morbid Obesity
Other: Group 2
Individuals with Early-onset Morbid Obesity.
Other Name: PWS
- Phenotypic assessments of participants [ Time Frame: until end of study ]phenotypic assessments will include cognitive level, behavioral analysis, physical features including body measurements and composition, co-morbidities (skin picking, psychiatric history, seizures, autistic behavior) medications required, and further comparison with the underlying molecular diagnosis.
- longitudinal pattern of progression [ Time Frame: until end of study ]assessment of cognition, behavior and body composition. In addition the age that growth hormone treatment began in the PWS participants will be correlated with physical features, body composition, cognition, behavior, developmental milestones, pubertal issues, and the onset of nutritional phases.
Biospecimen Retention: Samples With DNA
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00375089
|United States, California|
|University of California at Irvine|
|Orange, California, United States, 92868|
|United States, Florida|
|University of Florida|
|Gainesville, Florida, United States, 32610-0296|
|United States, Kansas|
|Kansas University Medical Center|
|Kansas City, Kansas, United States, 66160|
|United States, Tennessee|
|Vanderbilt University Medical Center|
|Nashville, Tennessee, United States, 37201|
|Study Chair:||Arthur Beaudet, MD||Baylor College of Medicine|