An observational study in Rett Syndrome, sponsored by University of Minnesota. Recruiting at 1 site in United States. Open to participants aged 18 Years to 99 Years. Per ClinicalTrials.gov, last updated 2026-06-18.
Sponsored by University of Minnesota · Observational
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.
98 studies on the registry are indexed under Rett Syndrome; 20 are open to participants now.
This study's planned enrollment of 20 is below the median of 98 across 32 observational studies indexed under Rett Syndrome.
Browse Rett Syndrome studies →University of Minnesota is the lead sponsor of 1,184 studies on the registry; 195 are open to participants now.
Of its 132 completed or terminated interventional studies of FDA-regulated products, 91 (69%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
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:
Exclusion Criteria:
Patients with Rett Syndrome who are interested in Trofinetide treatment
Other: None interventional
This is an observational study
Cognitive Function in Rett Syndrome During Trofinetide Treatment
change in saccade latencies during the 'statistical learning' paradigm
Time frame: baseline, 6weeks to 12 months
Eye movement gaze fixations
change in duration of gaze fixation to the correct image during the 'Looking While Listening' paradigm.
Time frame: baseline, 6weeks to 12 months
Heart rate variability
Change in baseline heart rate variability during sustained attention during eye-tracking tasks.
Time frame: baseline, 6weeks to 12 months
Heart rate suppression
Change in baseline heart rate suppression during sustained attention during eye-tracking tasks.
Time frame: baseline, 6weeks to 12 months
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
University of Minnesota