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NCT03196323 · ClinicalTrials.gov registry record

Development of a Behavioral Outcome Measure for Rett Syndrome (RettBe)

A clinical trial, sponsored by University of South Florida.

Completed
Registry status
146
Enrollment target

NCT03196323: Completed study, sponsored by University of South Florida.

NCT03196323 is a clinical trial that has completed, run by University of South Florida. The registered enrollment target is 146 participants. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT03196323 has completed, sponsored by University of South Florida.

COMPLETED
Registry status
146 participants
Enrollment target

Study Summary

The overall purpose of this study is to develop a broad-based (i.e., multiple domains) behavioral outcome measure for children between the ages of 3-18 years with Rett syndrome (RTT). The innovative approach of this proposal consists of integrating the process of developing a behavioral questionnaire to an ongoing large-scale data collection project. The Natural History Study of Rett Syndrome and Related Disorders (RTT5211) is a project that collects data on diverse aspects of the clinical evolution of individuals with RTT and related disorders. This project will serve as the basis for recruitment of subjects and it will also provide key demographic and clinical data for cohort characterization and for determining clinical relevance of the instrument (RettBe). An initial 100-subject cohort will allow for the testing with one rater of RettBe 1.0, a 50-item questionnaire formed from existing measures, a panel of clinicians and behavioral experts in RTT, and a focus group of parents and caregivers of children with RTT. Scores on RettBe 1.0 will be statistically analyzed to determine their psychometric properties, including its content validity. Items that do not meet psychometric standards (e.g., ceiling effect) will be eliminated. Additional items will be added if the parental survey attached to RettBe 1.0 or clinician input suggests so. The resulting modified assessment, called RettBe 2.0, will be administered to a larger (validation) cohort of 300 participants. RettBe 2.0 will also be subjected to analysis of psychometric properties. RettBe 2.0 will also be administered to two raters per subject, in order to determine inter-rater reliability. In addition, these raters will be completing other behavioral and clinical measures for further evaluating the validity of RettBe 2.0 as well as for determining its clinical and functional significance. Finally, the investigators will obtain input from a panel of clinicians (site PIs and their designated clinicians) about cont

Primary Outcome

Scores from RettBe 1.0, a 50-item questionnaire will be analyzed for psychometric properties, including content validity. RettBe 1.0 scores will be subjected to factor analysis to ensure domain-appropriate balance and structure.

Trial Details

FieldValue
Enrollment Target 146 participants
Start Date 2017-06-01
Est. Completion 2020-09-24
University of South Florida

252 total trials

What the finished NCT03196323 record still lists

NCT03196323 is an observational study that tracks outcomes without assigning an intervention. The registered 146 participants enrollment target is mid-sized for trials with a published cap.

The record links to 0 conditions, and to 0 interventions.

NCT03196323 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT03196323 about?

NCT03196323 is a clinical study titled "Development of a Behavioral Outcome Measure for Rett Syndrome (RettBe)". The overall purpose of this study is to develop a broad-based (i.e., multiple domains) behavioral outcome measure for children between the ages of 3-18 years with Rett syndrome (RTT). The innovative approach of this proposal consists of integrating the process of developing a behavioral questionnair...

What is the current status of trial NCT03196323?

This trial is currently completed. The enrollment target is 146 participants. The study started on 2017-06-01. Estimated completion is 2020-09-24.

Who is sponsoring clinical trial NCT03196323?

This trial is sponsored by University of South Florida, which has 252 total clinical trials registered on ClinicalTrials.gov.

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Source: ClinicalTrials.gov NCT03196323, the US trial registry maintained by the National Library of Medicine. NCT03196323 (mid enrollment · none site footprint · completed) retrieved and formatted by PlainTrial, see methodology.