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Recruiting NCT05432349

Rett Syndrome Registry

— Observational Rett Syndrome Rett Syndrome, Atypical Genetic Disease Genetic Diseases, X-Linked

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: Rett Syndrome, Rett Syndrome, Atypical, Genetic Disease, Genetic Diseases, X-Linked. Basic parameters: 0 years — 99 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Rett Syndrome Real World Data Observational Registry

Overview

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.

Primary outcome measures

  • Natural History [Time frame: 5 years]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

United States · 19 centers
  • University of Alabama — Birmingham
  • Children's Hospital Los Angeles — Los Angeles
  • UCSF Benioff Children's Hospital — Oakland
  • Rady Children's Hospital - San Diego — San Diego
  • Children's Hospital Colorado — Denver
  • Nicklaus Children's Hospital — Miami
  • Rush University Medical Center — Chicago
  • Kennedy Krieger Institute — Baltimore
  • … and 11 more centers

Identifiers

NCT: NCT05432349 · Pro00060206

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗