# Evaluating the psychometric properties of three clinical trial outcome measures for Rett syndrome

> **NIH NIH R21** · UNIVERSITY OF MINNESOTA · 2020 · $179,154

## Abstract

The long term goal of this project is to establish psychometrically credible outcome measures
for RTT clinical trials based on the relevant to core and supporting diagnostic domains. To
achieve this end, the short-term goal of this project is to establish the psychometric properties
for three non-invasive, objective clinical assessment measures of clinically-relevant symptoms.
The project will achieve this objective by evaluating internal and test-retest reliability and
preliminary convergent validity evidence for an objective, non-invasive method of evaluating
peripheral vasomotor function using portable infrared thermography (IRT; Aim 1), an objective
measure of sleep patterns using in-home wrist-based actigraphy (ACT; Aim 2), and an
objectively scored assessment of gain/loss of tactile sensitivity in response to a standardized set
of calibrated tactile stimuli (mQST; Aim 3). Based on our pilot work, in which we have
documented the feasibility of all three measures, we anticipate that all three measures will show
adequate internal reliability, and that test-retest reliability will vary between variables derived
from each method. Completion of the proposed project will lead to optimized quantification
methods for each of the proposed measures, and empirical estimates of reliability, which can be
used as a foundation for power analyses for future clinical trials in RTT for interventions with
anticipated effects on sleep, autonomic function, and/or somatosensory function.

## Key facts

- **NIH application ID:** 9987348
- **Project number:** 5R21HD101075-02
- **Recipient organization:** UNIVERSITY OF MINNESOTA
- **Principal Investigator:** Breanne J Byiers
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $179,154
- **Award type:** 5
- **Project period:** 2019-08-01 → 2023-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9987348

## Citation

> US National Institutes of Health, RePORTER application 9987348, Evaluating the psychometric properties of three clinical trial outcome measures for Rett syndrome (5R21HD101075-02). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/9987348. Licensed CC0.

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