Development of the ORCA Communication Measure for Rett Syndrome

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Brief Title

Development of the ORCA Communication Measure for Rett Syndrome

Official Title

Validation of the Observer-Reported Communication Ability (ORCA) Measure in Rett Syndrome

Brief Summary

      This measurement validation study will use qualitative and quantitative methodology to
      evaluate the Observer-Reported Communication Ability Measure (ORCA), to appropriately capture
      communication abilities in individuals with Rett syndrome. The ORCA Measure is a
      caregiver-reported questionnaire that collects caregiver observations of their child's
      communication abilities including expressive, receptive and pragmatic communication types.
      Caregivers will participate via phone interviews and online surveys. Approximately 270
      participants will be enrolled.
    

Detailed Description

      This study will develop the Observer-Reported Communication Ability Measure (ORCA),
      originally validated for Angelman syndrome, to appropriately capture communication abilities
      in individuals with Rett syndrome. The ORCA Measure is a caregiver-reported questionnaire
      that collects caregiver observations of their child's communication abilities including
      expressive, receptive and pragmatic communication. The goal of the study is to generate a
      validated tool to comprehensively assess communication in Rett syndrome and create an
      important endpoint for use in clinical trials.

      In phase 1, a hybrid approach to concept elicitation and cognitive testing will occur with
      caregivers to assess the existing ORCA measure and evaluate ORCA content validity.
      Approximately 20 parents/caregivers of individuals with Rett syndrome will participate in
      qualitative in-depth interviews. The ORCA measure, instructions, recall period, and response
      choices will be evaluated.

      In phase 2, psychometric properties including reliability, floor/ceiling effects, construct
      validity, and test-retest will be evaluated with approximately 250 caregiver-parents of
      individuals with Rett syndrome. Caregivers will also complete additional questionnaires to
      allow comparison between the ORCA measure and existing measures. Analysts will use a variety
      of statistical methods to evaluate the psychometric properties of the ORCA measure in Rett
      syndrome.
    


Study Type

Observational


Primary Outcome

Observer-Reported Communication Ability Measure (ORCA)


Condition

Rett Syndrome


Study Arms / Comparison Groups

 Phase 1: Qualitative Interviews
Description:  Approximately 20 parents/caregivers will discuss typical communication abilities of their loved one and complete the ORCA measure for determination of its content validity through a hybrid approach of concept elicitation and cognitive testing.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

270

Start Date

July 2, 2021

Completion Date

March 2022

Primary Completion Date

March 2022

Eligibility Criteria

        Inclusion Criteria:

          -  Parent or caregiver at least 18 years of age, with a female child, age 2 or older,
             diagnosed with Rett syndrome confirmed by a disease-causing MECP2 genetic mutation.

          -  Fluency in the English language

          -  Currently lives with the child with Rett syndrome

          -  Ability and willingness to participate in all study activities.

          -  Access to a telephone and/or access to a device with internet access that is capable
             of supporting electronic survey completion

        Exclusion Criteria:

          -  Parent or caregiver with clinically significant condition or situation that makes them
             not be suitable for the study, in the opinion of research staff.

          -  Parent or caregiver of a child with Rett syndrome who also has another diagnosis in
             addition to Rett syndrome.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Contacts

Bryce B Reeve, PhD, 203-445-0041, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04920110

Organization ID

ORCA-101-RSRT


Responsible Party

Sponsor

Study Sponsor

Rett Syndrome Research Trust

Collaborators

 Duke University

Study Sponsor

Bryce B Reeve, PhD, Principal Investigator, Duke University


Verification Date

September 2021