Rett SyndromeRett-ORCANCT04920110
Development of the ORCA Communication Measure for Rett Syndrome
An observational study of Rett Syndrome, run by Rett Syndrome Research Trust at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Rett Syndrome
- How many people
- 272 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Jul 2021to 10 Feb 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
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 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Parents/caregivers of individuals with Rett syndrome confirmed by an MECP2 genetic mutation, who are fluent in English.
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Validation of the Observer-Reported Communication Ability (ORCA) Measure in Rett Syndrome
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.
Read the 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.
- Conditions
- Rett Syndrome
- Keywords
- ORCA, Communication, Duke, MECP2, RSRT
- Medical subject headings
- Rett Syndrome, Communication
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Observer-Reported Communication Ability Measure (ORCA)
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.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2021
- Study start2 Jul 2021
- Primary completion10 Feb 2022
- Study completion10 Feb 2022
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
- 272 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rett Syndrome Research Trust
- Sponsor type
- University, hospital or other
- Collaborators
- Duke University
- Responsible party
- Sponsor
- Investigators
- Bryce B Reeve, PhD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04920110
- Sponsor's protocol ID
- ORCA-101-RSRT
- Acronym
- Rett-ORCA
- Status last verified
- Apr 2022
- First submitted
- 2 Jun 2021
- Last updated
- 22 Apr 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Validation of the Observer-Reported Communication Ability (ORCA) Measure in Rett Syndrome
- ClinicalTrials.gov ID
- NCT04920110
- Acronym / sponsor ID
- Rett-ORCA / ORCA-101-RSRT
- Conditions
- Rett Syndrome
- Keywords
- ORCA, Communication, Duke, MECP2, RSRT
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 272 participants (actual)
- Lead sponsor
- Rett Syndrome Research TrustUniversity, hospital or other
- Collaborators
- Duke University
- Study start
- 2 Jul 2021 (actual)
- Primary completion
- 10 Feb 2022 (actual)
- Study completion
- 10 Feb 2022 (actual)
- First posted
- 9 Jun 2021 (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.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Parents/caregivers of individuals with Rett syndrome confirmed by an MECP2 genetic mutation, who are fluent in English.
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2022.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Bryce B Reeve, PhD principal investigator
Duke University
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 Rett Syndrome Research Trust 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 5 inclusion and 2 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: finished, as of the sponsor's last update on 22 Apr 2022. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Observer-Reported Communication Ability Measure (ORCA) (time frame: 1 year). 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 22 Apr 2022.