Actively Recruiting
Cognitive Function in Rett Syndrome During Trofinetide Treatment
Led by University of Minnesota · Updated on 2025-06-12
20
Participants Needed
1
Research Sites
78 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Assessing cognitive functions among individuals with severe intellectual and developmental disabilities (IDD), including RTT, is often challenging due to floor effects of many standardized assessment batteries in this population. In addition, deficits in motor function and verbal ability may obscure certain abilities in this population when using standard IQ measures. Remote eye-tracking tasks have been proposed as an alternative approach for assessing cognitive functions among individuals with severe IDD, because eye-tracking tasks can be designed to minimize the influence of gross motor and receptive language deficits on performance. Although several types of eye-tracking tasks have been evaluated in RTT, most have been implemented only at a single time-point. As a result, it is unclear whether these measures are stable over time, or sensitive to developmental changes or alterations to health status that occur in RTT (e.g., developmental regression, development of seizures, change in medication, etc.). With the recent FDA approval of trofinetide for the treatment of RTT, we have a novel opportunity to test the sensitivity of eye-tracking and other psychophysiological measures to treatment changes. Anecdotally, parents and clinicians have reported improvements in attention and alertness during trofinetide treatment, but currently available outcome measures do not capture these types of effects. Therefore, we propose to conduct a pilot trial of changes in measures of attention, oculomotor function, learning, and autonomic function, all collected using non-invasive measures, during trofinetide treatment. This is an observational within-subject design with a 4-week post-treatment assessment compared to two pre-treatment assessments. Additional optional follow-up assessments will be performed with families who are interested and returning for standard-of-care visits to Gillette or who are willing to travel for a research-only visit.
CONDITIONS
Official Title
Cognitive Function in Rett Syndrome During Trofinetide Treatment
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Individuals initiating trofinetide treatment for Rett syndrome through the Gillette Children's RTT clinic
- Signed informed consent by the parent or legal guardian
- Willingness to comply with all study procedures and availability for study duration
- Documented diagnosis of Rett syndrome
- No active developmental regression in the past 6 months (no loss or worsening of walking, hand function, communication, or social skills)
- Stable pharmacological treatment regimen for at least 4 weeks
You will not qualify if you...
- Diagnosis of a progressive medical or neurological condition that could interfere with the study
- Current clinically significant systemic illness likely to worsen during the study
- Use of any other investigational drug currently or within the past 30 days
- Known, uncorrected visual impairment limiting ability to view images during eye-tracking
- Severe behavioral problems such as aggression, property destruction, or extreme hyperactivity interfering with participation
AI-Screening
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Trial Site Locations
Total: 1 location
1
University of Minnesota
Minneapolis, Minnesota, United States, 55414
Actively Recruiting
Research Team
B
Breanne Byiers
CONTACT
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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