Trial record 4 of 16 for:    angelman syndrome

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

This study has been completed.
Sponsor:
Collaborators:
Children's Hospital Boston
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
ClinicalTrials.gov Identifier:
NCT02670694
First received: November 6, 2015
Last updated: February 1, 2016
Last verified: January 2016
  Purpose

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
Rett Syndrome
Prader-Willi Syndrome
Angelman Syndrome
Sleep Problems

Study Type: Observational
Study Design: Observational Model: Case Control
Time Perspective: Prospective
Official Title: Sleep Abnormalities in Rare Genetic Disorders: Angelman Syndrome, Rett Syndrome, and Prader Willi

Resource links provided by NLM:


Further study details as provided by Baylor College of Medicine:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Pediatric Sleep Questionnaire (PSQ) - Sleep Disordered Breathing Subscale [ Time Frame: Change from Baseline sleep behaviors at 24 months ] [ Designated as safety issue: No ]
  • Child's Sleep Habits Questionnaire (CSHQ) (ages 0-19) [ Time Frame: Change from Baseline sleep behaviors at 24 months ] [ Designated as safety issue: No ]
  • Pediatric Daytime Sleepiness Scale (PDSS) (ages 6-19) [ Time Frame: Change from Baseline sleep behaviors at 24 months ] [ Designated as safety issue: No ]
  • Cleveland Adolescent Sleepiness Questionnaire (CASQ) (ages 6-19) [ Time Frame: Change from Baseline sleep behaviors at 24 months ] [ Designated as safety issue: No ]
  • Narcolepsy Questionnaire (ages 0-19) [ Time Frame: Change from Baseline sleep behaviors at 24 months ] [ Designated as safety issue: No ]
  • Unique Questionnaire (ages 0-19) [ Time Frame: Change from Baseline sleep behaviors at 24 months ] [ Designated as safety issue: No ]

Enrollment: 804
Study Start Date: June 2011
Study Completion Date: July 2013
Primary Completion Date: July 2013 (Final data collection date for primary outcome measure)
Groups/Cohorts
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.

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   1 Year to 18 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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
Children's Hospital Boston
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

No publications provided

Responsible Party: Daniel Glaze, Principal Investigator, Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT02670694     History of Changes
Other Study ID Numbers: H-26535 
Study First Received: November 6, 2015
Last Updated: February 1, 2016
Health Authority: United States: Institutional Review Board

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

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

ClinicalTrials.gov processed this record on February 04, 2016