A global registry for caregivers of individuals with Rett syndrome
Official title: The Rett Syndrome Global Registry
Run by Rett Syndrome Research Trust · NCT04900493 · Data updated 2026-11-12
This study is creating a platform for caregivers of people with Rett syndrome to share their experiences and track care strategies to help improve understanding and treatment of the condition.
Quick facts
| Study type | Observational |
|---|---|
| Sex | All |
| Enrollment | 5000 (estimated) |
| Sponsor | Rett Syndrome Research Trust Academic / other |
| Locations | 1 site (Trumbull, Connecticut) |
| Status | Recruiting |
| Started | January 31, 2022 |
| Main results expected | June 30, 2031 |
| Study ends | June 30, 2031 |
| Registry record updated | February 17, 2026 |
| Trial ID | NCT04900493 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates for this registry include caregivers of individuals diagnosed with Rett syndrome or those with a mutation in the MECP2 gene.
✕Probably not a fit
Patients with genetic mutations inconsistent with Rett syndrome or those diagnosed with MECP2 Duplication Syndrome may not benefit from this registry.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
The Rett Syndrome Global Registry is a remote, caregiver-reported platform designed to collect and analyze data about the experiences of those caring for individuals with Rett syndrome. Caregivers can track symptoms, care strategies, and access aggregate data to enhance their understanding and management of the condition. The registry aims to support research and therapeutic development by providing qualified researchers with de-identified data to improve outcomes for those affected by Rett syndrome. This initiative seeks to consolidate medical records and facilitate better communication between caregivers and healthcare providers.
Why it matters
Potential benefit: If successful, this registry could lead to improved care strategies and therapeutic developments for individuals with Rett syndrome.
How similar studies have gone: Other similar registries have shown success in enhancing understanding and treatment of rare disorders, indicating a promising approach.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Parent/caregiver must be willing and able to provide written informed consent electronically prior to entering data into the registry. 2. Rett individuals of any age, living or deceased, must have a diagnosis of Rett syndrome and/or have a mutation in MECP2. Exclusion Criteria: 1. Individuals who have a genetic mutation that is inconsistent with Rett syndrome or who have a different disorder. 2. Individuals with MECP2 Duplication Syndrome
Where it's running
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Rett Syndrome Research Trust
Trumbull, Connecticut, United States Recruiting
Study contacts
- Principal investigator: Jana von Hehn, PhD — Rett Syndrome Research Trust
- Study coordinator: Jana von Hehn, PhD
- Email: support@rettglobalregistry.org
- Phone: 203-445-0041
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Auditory EEG and behavioral testing for people with Rett syndrome
This project will record noninvasive EEG while people with Rett syndrome and matched controls listen to simple sounds to see how their brains process sound.
-
Fenfluramine HCl for Rett Syndrome in a Phase 3 study
This study tests whether fenfluramine hydrochloride helps people with Rett syndrome when compared to a placebo.
-
Daily oral vorinostat for girls and young women with Rett syndrome
This trial will try daily oral vorinostat at two escalating dose levels, using a four-week placebo baseline and an N-of-1 design so each participant serves as her own control, to see if the drug is safe and shows clinical or molecular benefit in girls and young women with typical Rett syndrome.
-
Efficacy of Bionetide for treating Rett syndrome in females
This study is testing if an oral medication called Bionetide can help improve behavior and communication in girls and young women aged 5 to 20 with Rett syndrome.
-
Assessing cognitive function in Rett syndrome patients receiving trofinetide treatment
This study is testing how well eye-tracking tasks can measure cognitive abilities in people with Rett syndrome who are starting treatment with trofinetide.
-
Identifying biomarkers in Rett Syndrome
This study is trying to find specific brain signals in girls with Rett Syndrome to see how they differ from girls without the condition and how these signals relate to the severity of their symptoms over time.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.