Prader-Willi Syndrome Clinical Trial
— RDCRNOfficial title:
Sleep Abnormalities in Rare Genetic Disorders: Angelman Syndrome, Rett Syndrome, and Prader Willi
Verified date | January 2016 |
Source | Baylor College of Medicine |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Institutional Review Board |
Study type | Observational |
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 Syndrome or Prader-Willi Syndrome. These individuals will serve as control
subjects.
The study will use questionnaires designed to identify sleep disorders and how they affect
behavior and quality of life.
The principal goals of this study are:
1. To see how common sleep disorders are in individuals with Angelman Syndrome, Rett
Syndrome or Prader-Willi Syndrome;
2. To see how sleep disorders affect behavior in these individuals;
3. To see whether sleep disorders and related behavior problems improve or worsen with
age;
4. To see how specific disease conditions relate to sleep disorders and how bad the sleep
disorders are;
5. To develop new treatment options to improve quality of life and behavior issues; and
6. To evaluate current treatment options to improve sleep problems in these individuals.
Status | Completed |
Enrollment | 804 |
Est. completion date | July 2013 |
Est. primary completion date | July 2013 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | Both |
Age group | 1 Year to 18 Years |
Eligibility |
Inclusion Criteria: Eligible Inclusion Criteria - Subjects 1. Enrollment in a RDCRN consortium registry for either AS, RTT or PWS. 2. Have a clinical diagnosis of AS, RTT or PWS, or be a normal sibling of an individual with AS, RTT or PWS who is enrolled in the study. 3. Be between 0 to18 years of age inclusive. 4. Be English-speaking (study questionnaires will only be available in English). Inclusion Criteria - Controls 1. Must have a sibling with either AS, RTT or PWS enrolled in the study. 2. Must not have a diagnosis of any neurological disorder. 3. Be between 0 to18 years of age inclusive 4. Be English-speaking (study questionnaires will only be available in English). Exclusion Criteria: Exclusion Criteria - Subjects 1. No clinical diagnosis of AS, RTT, or PWS. 2. Diagnosis of a severe genetic disorder in addition to AS, RTT, or PWS. 3. Be over 18 years of age inclusive. Exclusion Criteria - Controls 1. Diagnosis of a neurological disorder. 2. Diagnosis of a severe genetic disorder. 3. Be over 19 years of age inclusive. |
Observational Model: Case Control, Time Perspective: Prospective
Country | Name | City | State |
---|---|---|---|
United States | University of Alabama at Birmingham | Birmingham | Alabama |
United States | Children's Hospital Boston | Boston | Massachusetts |
United States | University of Florida College of Medicine | Gainesville | Florida |
United States | Greenwood Genetic Center | Greenwood | South Carolina |
United States | Baylor College of Medicine | Houston | Texas |
United States | University of California, Irvine Medical Center | Irvine | California |
United States | Kansas University Medical Center | Kansas City | Kansas |
United States | Vanderbilt University | Nashville | Tennessee |
United States | Rady Children's Hospital | San Diego | California |
Lead Sponsor | Collaborator |
---|---|
Baylor College of Medicine | Children's Hospital Boston, Greenwood Genetic Center, University of Alabama at Birmingham, University of California, Irvine, University of California, San Diego, University of Florida, University of Kansas Medical Center, Vanderbilt University |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change in sleep behavior as measured by the child's sleep habits questionnaire (CSHQ) for Rett Syndrome, Angleman and control group | Change from Baseline sleep behaviors at 24 months | No | |
Secondary | Pediatric Sleep Questionnaire (PSQ) - Sleep Disordered Breathing Subscale | Change from Baseline sleep behaviors at 24 months | No | |
Secondary | Child's Sleep Habits Questionnaire (CSHQ) (ages 0-19) | Change from Baseline sleep behaviors at 24 months | No | |
Secondary | Pediatric Daytime Sleepiness Scale (PDSS) (ages 6-19) | Change from Baseline sleep behaviors at 24 months | No | |
Secondary | Cleveland Adolescent Sleepiness Questionnaire (CASQ) (ages 6-19) | Change from Baseline sleep behaviors at 24 months | No | |
Secondary | Narcolepsy Questionnaire (ages 0-19) | Change from Baseline sleep behaviors at 24 months | No | |
Secondary | Unique Questionnaire (ages 0-19) | Change from Baseline sleep behaviors at 24 months | No |
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