Nervous System Diseases Clinical Trial
— RSROfficial title:
Rett Syndrome Real World Data Observational Registry
NCT number | NCT05432349 |
Other study ID # | Pro00060206 |
Secondary ID | |
Status | Recruiting |
Phase | |
First received | |
Last updated | |
Start date | August 2, 2022 |
Est. completion date | July 2028 |
The Rett Syndrome Registry is a longitudinal observational study of individuals with MECP2 mutations and a diagnosis of Rett syndrome. Designed together with the IRSF Rett Syndrome Center of Excellence Network medical directors, this study collects data on the signs and symptoms of Rett syndrome as reported by the Rett syndrome experts and by the caregivers of individuals with Rett syndrome. This study will be used to develop consensus based guidelines for the care of your loved ones with Rett syndrome and to facilitate the development of better clinical trials and other aspects of the drug development path for Rett syndrome.
Status | Recruiting |
Enrollment | 3000 |
Est. completion date | July 2028 |
Est. primary completion date | July 2027 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 0 Years to 99 Years |
Eligibility | Inclusion Criteria: - Male or female with a pathologic loss of function alteration of MECP2 Exclusion Criteria: - Male or female with a gain of function alteration of MECP2, including those with MEPC2 duplication or triplication |
Country | Name | City | State |
---|---|---|---|
United States | Kennedy Krieger Institute | Baltimore | Maryland |
United States | University of Alabama | Birmingham | Alabama |
United States | Boston Children's Hospital | Boston | Massachusetts |
United States | The Children's Hospital at Montefiore | Bronx | New York |
United States | The University of North Carolina at Chapel Hill | Chapel Hill | North Carolina |
United States | Rush University Medical Center | Chicago | Illinois |
United States | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio |
United States | Nationwide Children's Hospital | Columbus | Ohio |
United States | Children's Health | Dallas | Texas |
United States | Children's Hospital Colorado | Denver | Colorado |
United States | Greenwood Genetic Center | Greenwood | South Carolina |
United States | Texas Children's Hospital | Houston | Texas |
United States | Children's Hospital Los Angeles | Los Angeles | California |
United States | Vanderbilt Kennedy Center | Nashville | Tennessee |
United States | UCSF Benioff Children's Hospital | Oakland | California |
United States | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
United States | Washington University in St. Louis | Saint Louis | Missouri |
United States | Gillette Children's Specialty Healthcare | Saint Paul | Minnesota |
Lead Sponsor | Collaborator |
---|---|
International Rett Syndrome Foundation | Baylor College of Medicine, Boston Children's Hospital, Children's Health UTSW, Children's Hospital Colorado, Children's Hospital of Philadelphia, Gillette Children's Specialty Healthcare, Greenwood Genetic Center, Hive Networks, Hugo W. Moser Research Institute at Kennedy Krieger, Inc., Montefiore Medical Center, Rush University, St. Louis Children's Hospital, UCSF Benioff Children's Hospital Oakland, University of Alabama at Birmingham, Vanderbilt University Medical Center |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Natural History | To longitudinally evaluate the natural history of patients with mutations on the MECP2 gene, estimating and defining their clinical spectrum (e.g. disease course and complications of disease). | 5 years |
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