ClinicalTrials.gov record NCT04900493

The Rett Syndrome Global Registry

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

The Rett Syndrome Global Registry

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

The Rett Syndrome Global Registry is a fully remote, caregiver-reported registry for people with Rett syndrome. Families can enter and track symptoms, care strategies, and consolidate medical records in a central, secure database. Caregivers may opt in to allow de-identified, aggregate data to be shared with researchers and therapeutic developers.

What it is studying. The registry gathers caregiver-reported information and consolidated medical records to learn how Rett syndrome presents and is managed over time, including symptoms, comorbidities, medications, and care strategies. It creates de-identified datasets that researchers and therapeutic developers can request for research and clinical development planning.

What the researchers hope to learn. Increase understanding of Rett syndrome progression, symptom burden, comorbidities, and care strategies; provide de-identified datasets to researchers and therapeutic developers; assist clinical trial planning, endpoint selection, and regulatory filings.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

The Rett Syndrome Global Registry is a fully remote, global, caregiver-reported registry intended to meet the needs of caregivers, clinicians and researchers, and therapeutic developers with the goal to increase our understanding of this rare disorder, support better outcomes for those with Rett syndrome, and facilitate improved therapeutic development. The Rett Global Registry allows families to provide data about their experience with Rett syndrome to improve their loved one's care while contributing to research. Participants may opt in to track and graph symptoms and care strategies over time to support day-to-day care. Participants may also access aggregate data to see similarities and differences in care strategies and consolidate their personal information in a central location. Participants may opt-in to complete medical record consolidation and summary that is centrally accessible, able to be shared with care providers, and utilized for research. The registry database is designed and maintained to clinical trial standards and supports research and therapeutic development while meeting or exceeding federal privacy and confidentiality requirements. These datasets including caregiver-reported Rett syndrome progression, quality of life, at home day-to-day data, and consolidated medical records from office visits or hospital stays, provides unique and previously unused sources of information important for improving our understanding of Rett syndrome, allow additional avenues of research, and support therapeutic development. Specifically, the registry is intended to assist with clinical development planning, trial design, trial endpoints, and regulatory filings.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participate remotely by entering caregiver-reported information into the online registry and using built-in tracking and graphing tools. Caregivers may be asked to report symptoms, developmental milestones, treatments, co‑existing conditions, and health care use periodically (the record notes data entries about 1–4 times per year for many measures, and some items 1–2 times per year) over a target period of up to 5 years. Participants can opt in to consolidate and upload medical records for a central summary that can be shared with providers and used for research. Participants may also access aggregate, de‑identified registry data to see comparisons of care strategies.

How the study is structured. A fully remote, family-based observational registry in which caregivers enter data online. Participants may opt in to track symptoms over time and to upload or consolidate medical records. The registry is cross-sectional in design with a target duration of 5 years and stores de-identified aggregate data for approved research use.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Rett syndrome

Clinical trials20 trials listed for Rett syndromeEvery trial the registry lists for Rett syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Rett syndrome researchLatest monthly update: September 2026. Includes: Seizure add-on drug. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jan 31, 2022
Primary completion
Jun 30, 2031 (estimated)
Study completion
Jun 30, 2031 (estimated)
Target enrollment
5000 participants (estimated)
Last updated on registry
Feb 17, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Rett Syndrome Research Trust
Collaborators
  • Baylor College of Medicine
  • Vanderbilt University Medical Center
  • Children's Hospital of Philadelphia
  • Rush University
  • Boston Children's Hospital
  • RTI International

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

Registry
An organised collection of information about people with a particular condition, gathered over time for research. Joining a registry usually means sharing medical information, not receiving a treatment.
written informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
clinical trial
A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
endpoints
Something the study measures to judge its results, such as a symptom score, a blood test or survival. Outcomes are grouped as primary, secondary and sometimes exploratory.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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