ClinicalTrials.gov record NCT06705816

Cognitive Function in Rett Syndrome During Trofinetide Treatment

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

Cognitive Function in Rett Syndrome During Trofinetide Treatment

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
Age18 years old to 99 years old
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is an observational study that tests non-invasive eye-tracking and physiological measures before and after starting trofinetide in people with Rett syndrome who seek trofinetide through the Gillette Children's RTT clinic. The study uses two pre-treatment assessments and a post-treatment assessment (about 4 weeks after starting treatment), with optional later follow-up visits. Measures include attention, eye movements, learning tasks, and heart-rate measures collected over time.

What it is studying. The study is testing whether remote eye-tracking tasks and simple autonomic (heart-rate) measures detect changes in attention, oculomotor (eye movement) function, learning, and autonomic function during trofinetide treatment. It also examines whether these measures are stable over time or sensitive to clinical changes in people with Rett syndrome.

What the researchers hope to learn. To determine whether eye-tracking and psychophysiological measures can track changes over time in attention, eye movements, learning, and autonomic function in Rett syndrome during trofinetide treatment. To evaluate specific outcome measures such as saccade latency, gaze-fixation duration, heart rate variability, and heart rate suppression for future use in research and outcome measurement.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Other
  • None interventional

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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:
  • Provision of signed and dated informed consent form by the individual\'s parent/legal guardian
  • Stated willingness to comply with all study procedures and availability for the duration of the study
  • Documented diagnosis of Rett syndrome
  • 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.
  • Participant's current pharmacological treatment regimen has been stable for at least 4 weeks.
  • Seeking prescription for trofinetide through the Gillette Children's Rett syndrome clinic

Exclusion criteria

  • Diagnosis of a progressive medical or neurological condition that in the opinion of the investigator would interfere with the conduct of the study.
  • Current clinically significant systemic illness that is likely to result in the deterioration of the participant\'s condition during the study.
  • Participants taking any other investigational drug currently or within the past 30 days.
  • Known, uncorrected visual impairment that would limit the ability to view images during eye-tracking tasks.
  • Severe behavioral problems (i.e., aggression, property destruction, extreme hyperactivity) that would interfere with participation in study activities.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants complete non-invasive testing of attention, eye movements (eye-tracking), learning, and autonomic activity (for example heart-rate measures). Assessments include two pre-treatment visits and a post-treatment assessment about 4 weeks after starting trofinetide, with optional additional follow-up(s) for families who return for standard-of-care visits or travel for research-only visits up to later time points. Tasks involve watching images while eye movements and heart-rate are recorded; the study is observational (trofinetide is being prescribed through clinic physicians, not provided by the study). Participants must be available for scheduled assessments and willing to comply with study procedures.

How the study is structured. Observational, within-person (cohort) study. Participants complete two assessments before starting trofinetide and at least one assessment after starting the drug (about 4 weeks later); families may opt into additional follow-up visits. No experimental treatment is assigned by the study — it observes people who are beginning trofinetide through their clinic care.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Rett syndrome

Clinical trials20 trials listed for Rett syndromeEvery trial the registry lists for Rett syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Rett syndrome researchLatest monthly update: September 2026. Includes: Seizure add-on drug. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Dec 1, 2024
Primary completion
June 2027 (estimated)
Study completion
June 2027 (estimated)
Target enrollment
20 participants (estimated)
Last updated on registry
Jun 18, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
University of Minnesota

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

  • University of MinnesotaRecruiting

    Minneapolis, Minnesota, 55414 · United States

    • Breanne Byiers

Terms used on this page · explanations by MedicalUpdates.ai

outcome measures
Something the study measures to judge its results, such as a symptom score, a blood test or survival. Outcomes are grouped as primary, secondary and sometimes exploratory.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
informed consent form
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
legal guardian
A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
investigational drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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