Cognitive Function in Rett Syndrome During Trofinetide Treatment

Part of paid clinical trials in Minneapolis, Minnesota.

Sponsor
University of Minnesota
Study ID
NCT06705816
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
18 Years - 99 Years
Healthy Volunteers
Not accepted

Interventions

  • None interventional — OTHER
    This is an observational study

Study Details

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.

Key Dates

Start date
Dec 1, 2024
Status verified
Jun 2025
Primary completion
Jun 30, 2026
Completion
Jun 30, 2026

Study Design

Enrollment
20 participants (estimated)

Arms

  • Arm: Study group
    Patients with Rett Syndrome who are interested in Trofinetide treatment

Primary Outcome Measure

Cognitive Function in Rett Syndrome During Trofinetide Treatment [ Time Frame: baseline, 6weeks to 12 months ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of MinnesotaMinneapolisMinnesota55414
Breanne Byiers

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