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Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW (RDCRN)

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ClinicalTrials.gov Identifier: NCT02670694
Recruitment Status : Completed
First Posted : February 2, 2016
Last Update Posted : February 2, 2016
Sponsor:
Collaborators:
Boston Children’s Hospital
University of California, San Diego
Greenwood Genetic Center
University of Florida
Vanderbilt University
University of Kansas Medical Center
University of California, Irvine
University of Alabama at Birmingham
Information provided by (Responsible Party):
Daniel Glaze, Baylor College of Medicine

Study Description
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Brief Summary:

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.

Condition or disease
Rett Syndrome Prader-Willi Syndrome Angelman Syndrome Sleep Problems

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Study Design
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Study Type : Observational
Actual Enrollment : 804 participants
Observational Model: Case Control
Time Perspective: Prospective
Official Title: Sleep Abnormalities in Rare Genetic Disorders: Angelman Syndrome, Rett Syndrome, and Prader Willi
Study Start Date : June 2011
Actual Primary Completion Date : July 2013
Actual Study Completion Date : July 2013

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort
Rett Syndrome
Children and adolescents, age between 0-19 years, with clinical diagnosis of Rett Syndrome; currently enrolled in the Rare Disease Clinical Research Network registry.
Angelman's Syndrome
Children and adolescents, age between 0-19 years, with clinical diagnosis of Angelman's Syndrome; currently enrolled in the Rare Disease Clinical Research Network registry.
Prader-Willi Syndrome
Children and adolescents, age between 0-19 years, with clinical diagnosis of Prader-Willi Syndrome; currently enrolled in the Rare Disease Clinical Research Network registry.
Control
Siblings of RTT, AS and PW subjects will serve as control subjects.


Outcome Measures
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Primary Outcome Measures :
  1. Change in sleep behavior as measured by the child's sleep habits questionnaire (CSHQ) for Rett Syndrome, Angleman and control group [ Time Frame: Change from Baseline sleep behaviors at 24 months ]

Secondary Outcome Measures :
  1. Pediatric Sleep Questionnaire (PSQ) - Sleep Disordered Breathing Subscale [ Time Frame: Change from Baseline sleep behaviors at 24 months ]
  2. Child's Sleep Habits Questionnaire (CSHQ) (ages 0-19) [ Time Frame: Change from Baseline sleep behaviors at 24 months ]
  3. Pediatric Daytime Sleepiness Scale (PDSS) (ages 6-19) [ Time Frame: Change from Baseline sleep behaviors at 24 months ]
  4. Cleveland Adolescent Sleepiness Questionnaire (CASQ) (ages 6-19) [ Time Frame: Change from Baseline sleep behaviors at 24 months ]
  5. Narcolepsy Questionnaire (ages 0-19) [ Time Frame: Change from Baseline sleep behaviors at 24 months ]
  6. Unique Questionnaire (ages 0-19) [ Time Frame: Change from Baseline sleep behaviors at 24 months ]

Eligibility Criteria
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Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
This research study will investigate sleep behavior in the rare neurological disorders Angelman Syndrome (AS), Rett Syndrome (RTT) and Prader-Willi Syndrome (PWS).
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02670694


Locations
United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
United States, California
University of California, Irvine Medical Center
Irvine, California, United States, 92868
Rady Children's Hospital
San Diego, California, United States, 92123
United States, Florida
University of Florida College of Medicine
Gainesville, Florida, United States, 32601
United States, Kansas
Kansas University Medical Center
Kansas City, Kansas, United States, 66160
United States, Massachusetts
Children's Hospital Boston
Boston, Massachusetts, United States, 02115
United States, South Carolina
Greenwood Genetic Center
Greenwood, South Carolina, United States, 29646
United States, Tennessee
Vanderbilt University
Nashville, Tennessee, United States, 37240
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
Sponsors and Collaborators
Baylor College of Medicine
Boston Children’s Hospital
University of California, San Diego
Greenwood Genetic Center
University of Florida
Vanderbilt University
University of Kansas Medical Center
University of California, Irvine
University of Alabama at Birmingham
Investigators
Study Chair: Daniel Glaze, MD Baylor College of Medicine
Study Director: Alan Percy, MD University of Alabama at Birmingham
Principal Investigator: Sanjeev Kothare, MD Harvard Medical School, Children's Hospital Boston
More Information
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Responsible Party: Daniel Glaze, Principal Investigator, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT02670694     History of Changes
Other Study ID Numbers: H-26535
First Posted: February 2, 2016    Key Record Dates
Last Update Posted: February 2, 2016
Last Verified: January 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Daniel Glaze, Baylor College of Medicine:
Rett Syndrome
Prader-Willi Syndrome
Angelman Syndrome
Sleep Problems

Additional relevant MeSH terms:
Angelman Syndrome
Syndrome
Prader-Willi Syndrome
Rett Syndrome
Genetic Diseases, Inborn
Dyssomnias
Sleep Wake Disorders
Parasomnias
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
 Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Obesity
Overnutrition
Nutrition Disorders
Mental Retardation, X-Linked
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Mental Disorders
Signs and Symptoms
Movement Disorders
Central Nervous System Diseases