Assessing cognitive function in Rett syndrome patients receiving trofinetide treatment

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study evaluates cognitive functions in individuals with Rett syndrome using remote eye-tracking tasks. These tasks are designed to minimize the impact of motor and verbal deficits, providing a more accurate assessment of cognitive abilities. Participants will be those initiating trofinetide treatment at the Gillette Children's RTT clinic, and the study aims to determine the stability and sensitivity of eye-tracking measures over time. The study will include individuals of all ages diagnosed with Rett syndrome who meet specific eligibility criteria.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Any individuals who are initiating trofinetide treatment for RTT through the physicians associated with the Gillette Children's RTT clinic will be potentially eligible for participation, regardless of age. To enroll, participants must meet the following criteria:

  1. Provision of signed and dated informed consent form by the individual\'s parent/legal guardian
  2. Stated willingness to comply with all study procedures and availability for the duration of the study
  3. Documented diagnosis of Rett syndrome
  4. Participant is not showing active signs of developmental regression, defined as: no loss or degradation of ambulation within the past 6 months; no loss or degradation of hand function within the past 6 months; and no loss or degradation of verbal or non-verbal communication or social skills in the past 6 months.
  5. Participant's current pharmacological treatment regimen has been stable for at least 4 weeks.
  6. Seeking prescription for trofinetide through the Gillette Children's Rett syndrome clinic

Exclusion Criteria:

1. Diagnosis of a progressive medical or neurological condition that in the opinion of the investigator would interfere with the conduct of the study.
2. Current clinically significant systemic illness that is likely to result in the deterioration of the participant\'s condition during the study.
3. Participants taking any other investigational drug currently or within the past 30 days.
4. Known, uncorrected visual impairment that would limit the ability to view images during eye-tracking tasks.
5. Severe behavioral problems (i.e., aggression, property destruction, extreme hyperactivity) that would interfere with participation in study activities.

Where it's running

• University of Minnesota
  Minneapolis, Minnesota, United States Recruiting

Study contacts

• Principal investigator: Breanne Byiers — University of Minnesota
• Study coordinator: Breanne Byiers
• Email: byier001@umn.edu
• Phone: 612-626-7892

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Efficacy of Bionetide for treating Rett syndrome in females

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• Gene editing to correct common MECP2 mutations in Rett syndrome

  This project will test CRISPR/Cas9 delivered with AAV to correct common MECP2 mutations in females with Rett syndrome using lab and animal work.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.