Cognitive Function in Rett Syndrome During Trofinetide Treatment

Launched by UNIVERSITY OF MINNESOTA · Nov 22, 2024

Keywords

ClinConnect Summary

This clinical trial is studying how a new treatment called trofinetide affects cognitive functions in individuals with Rett syndrome, a condition that leads to severe intellectual and developmental disabilities. Researchers want to find better ways to measure attention and learning in these individuals using non-invasive methods like eye-tracking, which can help assess cognitive abilities without being influenced by difficulties with movement or communication. The trial will involve assessments before and after starting treatment to see if there are any changes in these cognitive areas.

To participate, individuals must be starting trofinetide treatment through the Gillette Children's Rett syndrome clinic and have a confirmed diagnosis of Rett syndrome. They should show no signs of developmental regression in the past six months and have had a stable medication regimen for at least four weeks. Participants will undergo a few assessments over the course of the study, and families interested in additional follow-up can choose to participate in extra visits. This trial is currently recruiting participants of all ages who meet these criteria, and it aims to provide valuable insights into how trofinetide treatment can impact cognitive function in those with Rett syndrome.

Gender

ALL

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.