Actively Recruiting

Age: 18Years - 99Years
All Genders
ID06705816

Cognitive Function in Rett Syndrome During Trofinetide Treatment

Led by University of Minnesota · Updated on 2025-06-12

20

Participants Needed

1

Research Sites

N/A

Total Duration

On this page

AI-Summary

What this Trial Is About

Researchers are exploring cognitive functions in individuals with Rett Syndrome (RTT), a severe intellectual and developmental disability, using eye-tracking tasks. These tasks help assess attention and learning while minimizing the impact of motor and verbal difficulties common in RTT. The study aims to understand if these measures change over time or with health status changes, especially during treatment with the recently FDA-approved drug trofinetide. This observational study follows a within-subject design involving participants initiating trofinetide treatment. It includes two pre-treatment assessments and one post-treatment assessment at 4 weeks, with optional follow-up visits up to 12 months later. The non-invasive measures include eye-tracking tasks, heart rate variability, and autonomic function evaluations to monitor changes in attention, oculomotor control, learning, and heart function. Participants will undergo assessments at baseline, after 6 weeks, and up to 12 months, including measurements of eye movement gaze fixations, heart rate variability, and heart rate suppression. Families interested in further follow-up can take part in additional visits aligned with standard care or research-only appointments. The study monitors cognitive and physiological responses during trofinetide treatment to better understand its impact over time.

CONDITIONS

Brief Title

Cognitive Function in Rett Syndrome During Trofinetide Treatment

Who Can Participate

Age: 18Years - 99Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Individuals initiating trofinetide treatment for Rett syndrome through physicians at the Gillette Children's RTT clinic
  • Signed and dated informed consent by parent or legal guardian
  • Willingness to comply with all study procedures and available for study duration
  • Documented diagnosis of Rett syndrome
  • No active developmental regression in ambulation, hand function, or communication/social skills in the past 6 months
  • Stable current medication regimen for at least 4 weeks
  • Seeking trofinetide prescription through the Gillette Children's Rett syndrome clinic
Not Eligible

You will not qualify if you...

  • Diagnosis of a progressive medical or neurological condition interfering with study conduct
  • Current significant systemic illness likely to worsen during the study
  • Use of any other investigational drug currently or within past 30 days
  • Known uncorrected visual impairment limiting ability to view eye-tracking images
  • Severe behavioral problems such as aggression, property destruction, or extreme hyperactivity interfering with study participation

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

Monitoring

Duration - 6 weeks to 12 months

Participants with Rett Syndrome who are starting trofinetide treatment are observed to assess cognitive function and other health measures over time.

Visits at baseline and multiple follow-ups between 6 weeks and 12 months

Trial Site Locations

Total: 1 location

1

University of Minnesota

Minneapolis, Minnesota, United States, 55414

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Research Team

B

Breanne Byiers

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

1

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