# REFINING METRICS OF FOOD BEHAVIOR IN SMITH-MAGENIS SYNDROME

> **NIH NIH R21** · BAYLOR COLLEGE OF MEDICINE · 2022 · $225,420

## Abstract

SUMMARY
Individuals with Smith-Magenis Syndrome (SMS) commonly experience excessive weight gain which leads to
obesity, and current clinical trials are underway to treat obesity in this population. The exact causes
exacerbating weight gain in this population are not known, as not all patients suffer from obesity. In addition to
cognitive impairments, adults and children with SMS also suffer from anxiety, behavioral disturbances, and
sleep disorder. How each of these comorbidities contribute to the presence or absence of food-related
behaviors, such as preoccupation with food, emotional outbursts around food availability, and sneaking/hiding
food, in SMS is unknown. Our previous data show that there is a discrepancy between measures of satiety in
currently available questionnaires and the need for caregivers to lock away food. These findings indicate that
the current measures do not adequately assess the satiety and food-related behavior concerns for the SMS
population. Since these questionnaires were not created for the SMS population, it is likely they are missing
important features necessary to fully measure the food-related behavioral phenotype in SMS. Without an
accurate measurement of food-related behaviors in this population, strong clinical trials searching for an impact
on obesity and its comorbidities in the SMS population will lack important clinical outcomes. In this proposal,
we aim to clinically validate a clinical measure of food-related behaviors specific to the SMS population
for use in clinical trials targeted to address obesity, hyperphagia, and food-related concerns.
Approach: The FDA has previously modified an existing questionnaire for Prader-Willi Syndrome (the
Hyperphagia Questionnaire) for use in a clinical trial (HQ-CT) using focus groups and expert review. We will
use a similar approach. We will use focus groups of caregivers of individuals with SMS to understand how
environmental controls around food and behavior alter answers on the Food-Related Problems Questionnaire
(FRPQ) and the Hyperphagia Questionnaire for Clinical Trials (HQ-CT), currently used as primary endpoint
measures for Prader-Willi Syndrome clinical trials. Additionally, we will seek to uncover other important food-
related behaviors that may not be captured on these measures. Using this information, combined with our
expertise in SMS and obesity, we will create a modified measure for food-related behaviors targeted to the
SMS population: the SMS-FRBQ. We will then perform subsequent testing and validation for use in future
clinical trials. Translation and validation of the SMS-FRBQ in Spanish will expand opportunities for patient
enrollment. Summary: Successful completion of this project will result in a measure of food-related
behaviors specific to the SMS population (SMS-FRBQ) which is necessary to adequately assess the
efficacy and impact of therapeutic intervention trials aimed at treating weight gain or its contributing
factors in this complex rare...

## Key facts

- **NIH application ID:** 10519747
- **Project number:** 1R21HD106345-01A1
- **Recipient organization:** BAYLOR COLLEGE OF MEDICINE
- **Principal Investigator:** Sarah H. ELSEA
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $225,420
- **Award type:** 1
- **Project period:** 2022-08-26 → 2024-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10519747

## Citation

> US National Institutes of Health, RePORTER application 10519747, REFINING METRICS OF FOOD BEHAVIOR IN SMITH-MAGENIS SYNDROME (1R21HD106345-01A1). Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/grant/nih/10519747. Licensed CC0.

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