The Rett Syndrome Global Registry

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Brief Title

The Rett Syndrome Global Registry

Official Title

The Rett Syndrome Global Registry

Brief Summary

      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.
    

Detailed Description

      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.
    


Study Type

Observational [Patient Registry]


Primary Outcome

Frequency of genetic mutation types and clinical diagnoses.

Secondary Outcome

 Caregiver report of the frequencies of the level of effectiveness for therapies, diets and equipment use over time.

Condition

Rett Syndrome



Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

5000

Start Date

January 31, 2022

Completion Date

June 30, 2031

Primary Completion Date

June 30, 2031

Eligibility 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
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Jana von Hehn, PhD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04900493

Organization ID

Rett-Registry


Responsible Party

Sponsor

Study Sponsor

Rett Syndrome Research Trust

Collaborators

 Baylor College of Medicine

Study Sponsor

Jana von Hehn, PhD, Principal Investigator, Rett Syndrome Research Trust


Verification Date

April 2022