# PAR-18-953 (ASCOT-DMD)

> **NIH NIH R21** · VIRGINIA COMMONWEALTH UNIVERSITY · 2021 · $256,214

## Abstract

Project Summary
The premise of this application is informed by the strategic priorities of the Muscular Dystrophy
Coordinating Committee (MDCC) of the National Institutes of Health (NIH), and from our clinical
and research experience on cognition in Duchenne Muscular Dystrophy (DMD). Cognitive health
is one of the key determinants of quality-of-life (QoL) in DMD. The improvement in life-expectancy,
along with the exciting pace of drug development and drug approval in this rare disease space
has provided a backdrop to be more inclusive of treating cognitive symptoms in DMD. The
preclinical success can be more rapidly translated to clinical trials by establishing sensitive and
robust performance-outcome (PerfO) measures of cognition. In this application, we bring a team
science approach, leveraging the research expertise of Drs. Thangarajh, Adams and Kaat to
investigate the strength of the NIH Toolbox Cognition Battery (NIHTB-CB), as a performance-
outcome measure for cognition in DMD. The NIHTB-CB permits continuity of measurement, fulfills
the NIH mandate for scientific rigor and reproducibility, and is scalable for multi-site clinical trials,
unlike gold-standard neuropsychological tests which cannot be applied with high fidelity in clinical
trial settings. The specific aims of the proposal are to establish the (i) test-retest reliability and
practice effects of the NIHTB-CB, (ii) to use patient-reported outcome measures (PROMs) to
identify participants’ self-reported function, QoL, and symptom burden, and (iii) to longitudinally
assess cognitive development using NIHTB-CB. This prospective, longitudinal study will assess
46 participants with NIHTB-CB, cognition-specific PROMs and standardized rating scales. The
NIHTB-CB will be administered at baseline, 3 weeks (± 1 week), and at 1 year, to investigate the
test-retest reliability and practice effects, an attribute that is critical for FDA’s approval for any
PerfO measure. Innovation: This proposal is the first to investigate whether the NIHTB-CB holds
promise as a PerfO measure of cognition in DMD. We bring a team science approach to improve
the cognitive research footprint in DMD. The completion of our study will provide the scientific
rationale to adopt the NIH Toolbox as a PerfO measure of cognition in clinical trials in DMD.

## Key facts

- **NIH application ID:** 10292233
- **Project number:** 1R21TR004007-01A1
- **Recipient organization:** VIRGINIA COMMONWEALTH UNIVERSITY
- **Principal Investigator:** Mathula Thangarajh
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $256,214
- **Award type:** 1
- **Project period:** 2021-08-15 → 2023-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10292233

## Citation

> US National Institutes of Health, RePORTER application 10292233, PAR-18-953 (ASCOT-DMD) (1R21TR004007-01A1). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10292233. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*