Development of the ORCA Communication Measure for Rett Syndrome (Rett-ORCA)

• ClinicalTrials.gov Identifier: NCT04920110
• Recruitment Status: Completed
• First Posted: June 9, 2021
• Last Update Posted: April 22, 2022
• Sponsor: Rett Syndrome Research Trust
• Collaborator: Duke University
• Information provided by (Responsible Party): Rett Syndrome Research Trust

Tracking Information

• First Submitted Date: June 2, 2021
• First Posted Date: June 9, 2021
• Last Update Posted Date: April 22, 2022
• Actual Study Start Date: July 2, 2021
• Actual Primary Completion Date: February 10, 2022 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: June 8, 2021)

Observer-Reported Communication Ability Measure (ORCA) [ Time Frame: 1 year ]

Using qualitative and quantitative methodology the existing ORCA measure will be evaluated for use in Rett syndrome and modified if needed. The ORCA measure produces a single score that is an estimate of an individual's overall level of communication ability. Higher ORCA scores reflect greater communication ability; the mastery of expressive, receptive, and pragmatic types of communication and higher vocabularies for verbal words and symbols on assistive devices. The ORCA T-score range is from 26.82 to 83.24.

Original Primary Outcome Measures (submitted: June 7, 2021)

Observer-Reported Communication Ability Measure (ORCA) [ Time Frame: 1 year ]

Using qualitative and quantitative methodology the existing ORCA measure will be evaluated for use in Rett syndrome and modified if needed

• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Parents/caregivers of individuals with Rett syndrome confirmed by an MECP2 genetic mutation, who are fluent in English.
• Condition: Rett Syndrome
• Intervention: Not Provided

Study Groups/Cohorts

• Phase 1: Qualitative Interviews
  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.
• Phase 2: Cross-Sectional Assessment of Psychometric Properties
  Approximately 250 parents/caregivers will complete the ORCA measure and additional measures to determine its psychometric properties including reliability, floor/ceiling effects and construct validity.

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: April 20, 2022): 272
• Original Estimated Enrollment (submitted: June 7, 2021): 270
• Actual Study Completion Date: February 10, 2022
• Actual Primary Completion Date: February 10, 2022 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT04920110
• Other Study ID Numbers: ORCA-101-RSRT
• Has Data Monitoring Committee: Not Provided

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Rett Syndrome Research Trust
• Original Responsible Party: Same as current
• Current Study Sponsor: Rett Syndrome Research Trust
• Original Study Sponsor: Same as current
• Collaborators: Duke University

Investigators

• Principal Investigator: Bryce B Reeve, PhD; Duke University

• PRS Account: Rett Syndrome Research Trust
• Verification Date: April 2022