# Pilot Trial of Vamorolone for the Treatment of Becker Muscular Dystrophy

> **NIH NIH R21** · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · 2021 · $130,941

## Abstract

Project Summary
Novel treatments are desperately needed by patients with Becker muscular dystrophy (BMD), and anti-
inflammatory therapies hold great promise for treating patients with this condition. However, the slow
progressive nature of the disease, small numbers of patients, variable onset and clinical presentation, and lack
of biomarkers presents a serious barrier to drug development in BMD. A first-in-class steroid, vamorolone, has
shown promise for the treatment of young boys with Duchenne muscular dystrophy and is in a randomized,
blinded and controlled, confirmatory study for this indication. In this project, we propose a 24-week pilot trial of
vamorolone 6 mg/kg/day in 30 ambulatory patients with BMD at 2 sites, evaluating safety and tolerability.
Biomarkers, creatine kinase, macrophage derived chemokine, and microRNA 146a, and the timed 10 meter
run/walk test will also be evaluated throughout the trial, to assess the usefulness of these potential treatment
responsive biomarkers to this anti-inflammatory drug in BMD patients. If vamorolone is demonstrated to be
safe and tolerable in this study, a controlled study of vamorolone treatment for BMD patients will be planned.
Evaluation of pharmacodynamic biomarkers in this short trial serves to provide objective evidence for drug
mechanism of action; if there is expected change in this pilot trial, these biomarkers would be further studied in
the next phase trial.

## Key facts

- **NIH application ID:** 10215387
- **Project number:** 5R21AR076660-02
- **Recipient organization:** UNIVERSITY OF PITTSBURGH AT PITTSBURGH
- **Principal Investigator:** Paula R Clemens
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $130,941
- **Award type:** 5
- **Project period:** 2020-07-13 → 2024-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10215387

## Citation

> US National Institutes of Health, RePORTER application 10215387, Pilot Trial of Vamorolone for the Treatment of Becker Muscular Dystrophy (5R21AR076660-02). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10215387. Licensed CC0.

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