The Rett Syndrome Global Registry
ConditionRett Syndrome
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorRett Syndrome Research Trust
About this trial
The Rett Global Registry is a fully remote, global, caregiver-reported registry to collect information about caring for a loved one with Rett syndrome. In addition, caregivers have the ability to track and graph their loved one's symptoms and care strategies over time, store information for central access, and opt-in to complete medical record consolidation and summary. Qualified researchers and therapeutic developers may request access to de-identified aggregate information to further Rett research, or assist with clinical development planning to facilitate and expedite more effective clinical trials.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Parent/caregiver must be willing and able to provide written informed consent electronically prior to entering data into the registry.
Rett individuals of any age, living or deceased, must have a diagnosis of Rett syndrome and/or have a mutation in MECP2.
Disqualifiers
Individuals who have a genetic mutation that is inconsistent with Rett syndrome or who have a different disorder.
Individuals with MECP2 Duplication Syndrome
Trial population
Individuals of all ages diagnosed with Rett Syndrome and/or have a mutation in MECP2. MECP2 Duplication syndrome individuals are not included and will be directed to the MECP2 Duplication registries.
Trial design
Design model
Family-based
Time perspective
Cross-sectional
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
Frequency of genetic mutation types and clinical diagnoses.
Measured by data obtained from genetic reports and caregiver-reported clinical diagnoses of enrolled patients.
Time frame
1 year2
Caregiver report of developmental milestone achievement over time.
Measured by the percent of individuals who have achieved developmental milestones between 1 and 4 times per year.
Time frame
5 years3
Caregiver report of symptom burden and development history over time.
Measured by the percent of individuals who report symptoms and their intervention requirements between 1 and 4 times per year.
Time frame
5 years4
Caregiver report of composition and frequency of co-morbidities over time.
Measured by the type and number of non-Rett medical conditions between 1 and 4 times per year.
Time frame
5 yearsSecondary outcomes
1
Caregiver report of the frequencies of the level of effectiveness for therapies, diets and equipment use over time.
Measured by percent of patients perceiving these care strategies as successful between 1 and 2 times per year.
Time frame
5 years2
Caregiver report of the frequencies of the level of effectiveness, degree of side effect severity, and other symptom impacts of medications and over-the-counter treatments over time.
Measured by percent of patients receiving medications or over-the-counter treatments and the percent perceiving these care strategies as successful, with side effects, and impact on other symptoms between 1 and 4 times per year.
Time frame
5 years3
Caregiver report of the percent of individuals requiring emergency care and unplanned hospital admissions over time.
Measured by the number of patients requiring emergency care and hospital admissions at least 1 time per year.
Time frame
5 years4
Percent of individuals using registry features, including tracking, medical record consolidation, central storage, and family connections over time.
Measured by the number of individuals using these features between 1 and 4 times per year.
Time frame
5 yearsOther outcomes
1
Number of research studies conducted through the registry for the purpose of collecting new data over time.
Measured by the number of studies initiated at least 1 time per year.
Time frame
5 years2
Number of research studies conducted with existing datasets for the purpose of mining registry data over time.
Measured by the number of studies initiated at least 1 time per year.
Time frame
5 yearsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Rett Syndrome Research Trust
Lead sponsor
Baylor College of Medicine
Collaborator
Vanderbilt University Medical Center
Collaborator
Children's Hospital of Philadelphia
Collaborator
Rush University
Collaborator
Boston Children's Hospital
Collaborator
RTI International
Collaborator
Source ClinicalTrials.gov