Auditory EEG and Behavioral Assessments in Individuals With Rett Syndrome

ConditionRett Syndrome
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age2+
SponsorUniversity of Rochester

About this trial

This observational research study is designed to learn more about how the brain processes sound in individuals with Rett syndrome. Participants will complete a noninvasive electroencephalogram (EEG) recording while listening to simple sounds through headphones. In addition, parents or caregivers will complete questionnaires and an interview about the participant's symptoms, communication abilities, daily functioning and overall health. The information collected from this study may help researchers better understand brain activity patterns and clinical features associated with Rett syndrome and support future research efforts.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

Have a clinical diagnosis of classic Rett syndrome

Have a pathogenic MECP2 genetic variant confirmed via genetic testing

Are past the clinical regression stage

Are within the eligible age range for the study (2 years or older)

Disqualifiers

Experiences hearing impairment or hearing loss

Presence of medical conditions that would prevent safe participation in EEG recording

Inability to tolerate EEG procedures

Any condition that in the opinion of the investigator would interfere with study participation or data quality

Trial population

The study population includes individuals diagnosed with Rett syndrome and age and sex-matched controls who are participating in an observational research study focused on brain activity and clinical characteristics. Rett syndrome participants will undergo a noninvasive EEG recording and their parent or caregiver will complete questionnaires and an interview related to symptoms, communication abilities, daily functioning and overall health. Control participants will also complete a noninvasive EEG recording and they or their parent will complete a demographics questionnaire.

Trial design

Design model

Case-control

Time perspective

Cross-sectional

Treatments tested in this trial

  • Electroencephalogram (EEG) Recording

    Procedure/Surgery

    Participants will undergo a noninvasive electroencephalogram (EEG) recording to measure brain activity in response to auditory stimuli. During the procedure, participants will listen to simple sounds through headphones while brain signals are recorded. No behavioral responses are required.

Treatment groups

60 Participants
are divided into 2 treatment groups
Group A: Rett Syndrome Participants1 intervention
Group B: Age- and sex-matched controls1 intervention

Trial outcomes

Primary outcomes

1

Mean Auditory Evoked Response Amplitude Measured by EEG

Auditory evoked brain responses will be assessed using noninvasive electroencephalogram (EEG) recordings while participants listen to simple sounds through headphones. The mean amplitude of auditory evoked responses will be derived from EEG recordings to describe auditory processing in individuals with Rett syndrome.

Time frame
Assessed at a single study visit (baseline, up to 1 day)
2

Mean Auditory Evoked Response Latency Measured by EEG

Auditory evoked brain responses will be assessed using noninvasive electroencephalogram (EEG) recordings while participants listen to simple sounds through headphones. The mean latency of auditory evoked responses will be derived from EEG recordings to describe auditory processing in individuals with Rett syndrome.

Time frame
Assessed at a single study visit (baseline, up to 1 day)

Secondary outcomes

1

Total Score on the Rett Syndrome Severity Scale

Overall disease severity will be assessed using the Rett Syndrome Severity Scale. The total severity score will be used to describe clinical severity in individuals with Rett syndrome. The scale may be completed by a clinician or trained member of the research team.

Time frame
Assessed at a single study visit (baseline, up to 1 day)
2

Adaptive Behavior Composite Score on the Vineland Adaptive Behavior Scales

Adaptive behavior will be evaluated through a structured caregiver interview using the Vineland Adaptive Behavior Scales. The composite score will be used to describe daily living skills, communication and social functioning.

Time frame
Assessed at a single study visit (baseline, up to 1 day)
3

Total Observer-Reported Communication Ability (ORCA) Score

Communication abilities will be assessed using a caregiver-reported questionnaire called the ORCA which evaluates expressive and receptive communication skills. ORCA total scores will be used to describe communication ability in individuals with Rett syndrome.

Time frame
Assessed at a single study visit (baseline, up to 1 day)
4

Total Score on the Rett Syndrome Behavior Questionnaire

The Rett Syndrome Behavior Questionnaire is a caregiver-reported instrument assessing behavioral and clinical symptoms associated with Rett syndrome. The total questionnaire score will be used to describe symptom severity among participants enrolled in the study.

Time frame
Assessed at a single study visit (baseline, up to 1 day)

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.