Rett SyndromeNCT06705816
Cognitive Function in Rett Syndrome During Trofinetide Treatment
An observational study of Rett Syndrome, run by University of Minnesota at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Rett Syndrome
- What is being tested?
- None interventional
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Dec 2024to Jun 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 99 years
- All sexes
- Adult, Older adult
Study population: 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.
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Minnesota
Minneapolis, Minnesota, 55414
Breanne Byiers
About this study
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
- Rett Syndrome
- Being tested
- None interventional
- Medical subject headings
- Rett Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
None interventionalOther
This is an observational study
| Study group | Role | What they receive |
|---|---|---|
| Study group | Patients with Rett Syndrome who are interested in Trofinetide treatment Receives: Other: None interventional |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cognitive Function in Rett Syndrome During Trofinetide Treatment
change in saccade latencies during the 'statistical learning' paradigm
Measured: 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.
Measured: baseline, 6weeks to 12 months
Secondary outcomes (2)
Heart rate variability
Change in baseline heart rate variability during sustained attention during eye-tracking tasks.
Measured: baseline, 6weeks to 12 months
Heart rate suppression
Change in baseline heart rate suppression during sustained attention during eye-tracking tasks.
Measured: baseline, 6weeks to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2024
- Study start1 Dec 2024
- Primary completionJun 2027 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rett SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Breanne Byiers, Principal investigator, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06705816
- Sponsor's protocol ID
- 33488
- Status last verified
- Jun 2026
- First submitted
- 11 Nov 2024
- Last updated
- 18 Jun 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cognitive Function in Rett Syndrome During Trofinetide Treatment
- ClinicalTrials.gov ID
- NCT06705816
- Acronym / sponsor ID
- 33488
- Conditions
- Rett Syndrome
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- University of MinnesotaUniversity, hospital or other
- Study start
- 1 Dec 2024 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 26 Nov 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Study group | – | Other: None interventionalPatients with Rett Syndrome who are interested in Trofinetide treatment |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- 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.
Key inclusion criteria (7 listed)
- 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:
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| None interventionalThis is an observational study | Other | Study group |
Primary outcome
Cognitive Function in Rett Syndrome During Trofinetide Treatment
Time frame: baseline, 6weeks to 12 months
change in saccade latencies during the 'statistical learning' paradigm
Eye movement gaze fixations
Time frame: baseline, 6weeks to 12 months
change in duration of gaze fixation to the correct image during the 'Looking While Listening' paradigm.
2 secondary outcomes
Heart rate variability
Time frame: baseline, 6weeks to 12 months
Heart rate suppression
Time frame: baseline, 6weeks to 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Breanne Byiers principal investigator
University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Rett Syndrome, run by University of Minnesota at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 18 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Breanne Byiers (612-626-7892, byier001@umn.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cognitive Function in Rett Syndrome During Trofinetide Treatment (time frame: baseline, 6weeks to 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jun 2026.