NCT04900493 · Rett Syndrome Research Trust
The Rett Syndrome Global Registry
What this study is about
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.
View original scientific description
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.
Primary outcome measures
Frequency of genetic mutation types and clinical diagnoses.
Time frame: 1 year
Measured by data obtained from genetic reports and caregiver-reported clinical diagnoses of enrolled patients.
Caregiver report of developmental milestone achievement over time.
Time frame: 5 years
Measured by the percent of individuals who have achieved developmental milestones between 1 and 4 times per year.
Caregiver report of symptom burden and development history over time.
Time frame: 5 years
Measured by the percent of individuals who report symptoms and their intervention requirements between 1 and 4 times per year.
Caregiver report of composition and frequency of co-morbidities over time.
Time frame: 5 years
Measured by the type and number of non-Rett medical conditions between 1 and 4 times per year.
Caregiver report of the composition and frequency of medication and over-the-counter treatments over time.
Time frame: 5 years
Measured by the percent of individuals receiving these care strategies by symptom between 1 and 4 times per year.
Caregiver report of the composition and frequency of physician specialty utilization and care received at Rett Clinics over time.
Time frame: 5 years
Measured by the type and number of physician specialties used to manage symptoms and the number of individuals who receive care at a Rett clinic between 1 and 2 times per year.
Caregiver report of the composition of the barriers to clinical trial participation over time.
Time frame: 5 years
Measured by the type and number of reasons given for individuals not able or willing to participate in clinical trials between 1 and 2 times per year.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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.
Exclusion criteria
- Individuals who have a genetic mutation that is inconsistent with Rett syndrome or who have a different disorder.
- Individuals with MECP2 Duplication Syndrome
Where
- Trumbull, Connecticut
Collaborators
Baylor College of Medicine, Vanderbilt University Medical Center, Children's Hospital of Philadelphia, Rush University, Boston Children's Hospital, RTI International
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Feb 17, 2026 · Source of record for eligibility and locations