# Determining minimal clinically important differences for neurodevelopmental outcome measures in Angelman syndrome

> **NIH NIH R03** · RESEARCH TRIANGLE INSTITUTE · 2021 · $90,132

## Abstract

Project Summary/Abstract
Angelman syndrome (AS) is a rare neurogenetic condition that results in a host of clinical traits,
including severe developmental delays and extremely limited functional abilities. Therapeutic
development efforts have increased substantially in the last several years, with seven
pharmaceutical companies currently conducting or preparing Phase 1 trials. As a result, there is
a critical need to improve the utility of core clinical outcome measures so that they can be
deployed effectively in future clinical trials in AS. The overall goal of this study is to use existing
data on over 800 administrations of two core instruments, the Bayley Scales of Infant
Development and the Vineland Adaptive Behavior Scales, to establish meaningful change
thresholds for these two measures for individuals with AS. To achieve this goal, we will calculate
for each measure (a) distribution-based minimal clinically important difference (MCID), and (b)
anchor-based MCID. The anchor-based MCID aligns well with the U.S. Food and Drug
Administration’s (FDA’s) guidance on establishing “meaningful within-patient change” (see
https://www.fda.gov/media/132505/download), yet even the FDA recognizes that empirical data
are necessary to contextualize changes on Clinical Outcome Assessments. The distribution-
based MCID approach will provide this “reality check” on the anchor-based approach, ensuring
that thresholds for change are both statistically significant and clinically meaningful.

## Key facts

- **NIH application ID:** 10186591
- **Project number:** 1R03HD105507-01
- **Recipient organization:** RESEARCH TRIANGLE INSTITUTE
- **Principal Investigator:** ANJALI SADHWANI
- **Activity code:** R03 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $90,132
- **Award type:** 1
- **Project period:** 2021-05-01 → 2023-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10186591

## Citation

> US National Institutes of Health, RePORTER application 10186591, Determining minimal clinically important differences for neurodevelopmental outcome measures in Angelman syndrome (1R03HD105507-01). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10186591. Licensed CC0.

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