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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04920110
Recruitment Status : Completed
First Posted : June 9, 2021
Last Update Posted : April 22, 2022
Sponsor:
Collaborator:
Duke University
Information provided by (Responsible Party):
Rett Syndrome Research Trust

Study Description
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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.

Condition or disease
Rett Syndrome

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 Design
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Study Type : Observational
Actual Enrollment : 272 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Validation of the Observer-Reported Communication Ability (ORCA) Measure in Rett Syndrome
Actual Study Start Date : July 2, 2021
Actual Primary Completion Date : February 10, 2022
Actual Study Completion Date : February 10, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rett Syndrome

Groups and Cohorts
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Group/Cohort
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.


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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.
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04920110


Locations
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United States, North Carolina
Duke University
Durham, North Carolina, United States, 27701
Sponsors and Collaborators
Rett Syndrome Research Trust
Duke University
Investigators
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Principal Investigator: Bryce B Reeve, PhD Duke University
More Information
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Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
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Responsible Party: Rett Syndrome Research Trust
ClinicalTrials.gov Identifier: NCT04920110    
Other Study ID Numbers: ORCA-101-RSRT
First Posted: June 9, 2021    Key Record Dates
Last Update Posted: April 22, 2022
Last Verified: April 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Rett Syndrome Research Trust:
ORCA
Communication
Duke
MECP2
RSRT
Additional relevant MeSH terms:
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Rett Syndrome
Syndrome
Disease
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
 Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System