Actively Recruiting
Rett Syndrome Real World Data Observational Registry
Led by International Rett Syndrome Foundation · Updated on 2023-01-11
3000
Participants Needed
18
Research Sites
52 weeks
Total Duration
On this page
Sponsors
I
International Rett Syndrome Foundation
Lead Sponsor
B
Boston Children's Hospital
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are conducting a long-term observational study called the Rett Syndrome Registry to gather detailed information about individuals diagnosed with Rett syndrome who have MECP2 mutations. This study aims to collect data from Rett syndrome experts and caregivers about the signs and symptoms experienced by those with this condition. The goal is to help develop standardized care guidelines and support the creation of better clinical trials and drug development focused on Rett syndrome. This study does not involve treatments or drugs but focuses on observing and recording real-world data over time. Participants include males and females with a loss of function mutation in the MECP2 gene. The study collects information continuously to understand the natural history of Rett syndrome over a 5-year period. Participants and their caregivers will provide data about symptoms and experiences related to Rett syndrome. This may involve regular assessments and reporting to help researchers monitor changes and progression. The study measures the natural history of the condition and aims to improve future care and research. Participation may last up to five years, with ongoing data collection and observation.
CONDITIONS
Brief Title
Rett Syndrome Registry
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female with a pathologic loss of function alteration of MECP2
You will not qualify if you...
- Male or female with a gain of function alteration of MECP2, including those with MEPC2 duplication or triplication
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 5 years
Participants are observed over time to collect natural history data related to Rett Syndrome.
Trial Site Locations
Total: 18 locations
1
University of Alabama
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Not Yet Recruiting
3
UCSF Benioff Children's Hospital
Oakland, California, United States, 94609
Actively Recruiting
4
Children's Hospital Colorado
Denver, Colorado, United States, 80045
Actively Recruiting
5
Rush University Medical Center
Chicago, Illinois, United States, 60612
Actively Recruiting
6
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
Actively Recruiting
7
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
8
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States, 55101
Actively Recruiting
9
Washington University in St. Louis
St Louis, Missouri, United States, 63110
Actively Recruiting
10
The Children's Hospital at Montefiore
The Bronx, New York, United States, 10467
Actively Recruiting
11
The University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27517
Not Yet Recruiting
12
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Not Yet Recruiting
13
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Not Yet Recruiting
14
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
15
Greenwood Genetic Center
Greenwood, South Carolina, United States, 29646
Actively Recruiting
16
Vanderbilt Kennedy Center
Nashville, Tennessee, United States, 37232
Actively Recruiting
17
Children's Health
Dallas, Texas, United States, 75207
Actively Recruiting
18
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
D
Dominique C. Pichard, MD
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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