Assessing cognitive function in Rett syndrome patients receiving trofinetide treatment

Cognitive Function in Rett Syndrome During 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.

Quick facts

• Study type: Observational
• Enrollment: 20 (estimated)
• Ages: 18 Years to 99 Years
• Sex: All
• Sponsor: University of Minnesota (Academic / other)
• Locations: 1 site (Minneapolis, Minnesota)
• Trial ID: NCT06705816 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates are individuals diagnosed with Rett syndrome who are starting trofinetide treatment and meet the study's eligibility criteria.

Not a fit: Patients experiencing active developmental regression or those not seeking trofinetide treatment may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could enhance the understanding of cognitive function in Rett syndrome and improve assessment methods for this population.

How similar studies have performed: While eye-tracking tasks have been explored in Rett syndrome, this study's longitudinal approach is relatively novel and untested.

Eligibility criteria

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 this trial is running

Minneapolis, Minnesota

• University of Minnesota — Minneapolis, Minnesota, United States (Recruiting)

Study contacts

• Study coordinator: Breanne Byiers

Conditions Rett Syndrome