# A Ph2 Randomized, Placebo-controlled, Multiple Dose Study to Determine the Safety, Pharmacokinetics and Efficacy of Oral Ifetroban in Subjects with Duchenne Musculary Dystrophy IND136895 (10/10/2018)

> **NIH FDA R01** · CUMBERLAND PHARMACEUTICALS, INC. · 2020 · $97,112

## Abstract

PROJECT SUMMARY/ABSTRACT
Duchenne muscular dystrophy (DMD) is a rare and fatal disease affecting approximately 1 in every 3,500 live
male births across all races and cultures with no cure. DMD results in loss of ambulation, respiratory failure,
cardiomyopathy (CM), and premature death. CM is now the leading cause of death in DMD. Limited
ambulation in DMD patients often masks typical symptoms of heart failure, allowing unchecked CM disease
progression. Without aggressive screening in DMD, the first manifestation of CM can be irreversible heart
failure or sudden cardiac death. Our group and others have shown activation of the thromboxane prostanoid
receptor (TPr) located on platelets, immune cells, smooth muscle and cardiomyocytes contributes to
deleterious effects in the heart. Further, TPr blockade with the potent and selective TPr antagonist, ifetroban,
prevents cardiac fibrosis, improves cardiac function and mortality in multiple animal models of DMD CM.
Isoprostanes (IP) activate the TPr and are elevated in DMD. Not only are they the most reliable marker for
assessing oxidative stress, IP are mediators of vascular dysfunction. They perpetuate the inflammatory
response to oxidant injury, which may play a key role in DMD CM pathogenesis. Our overall goal is to
determine the safety and efficacy of ifetroban to support its approval for the treatment of DMD CM, a rare
condition where no current therapy exists thereby directly serving the Orphan Products Development’s
mission. The proposed clinical study aims to determine the safety, pharmacokinetics and efficacy of oral
ifetroban in patients with DMD CM. In addition to quantitative cardiac and respiratory outcomes, the
measurable effect of oral ifetroban on muscle strength, daily activity and quality-of-life will be investigated in
DMD patients. This randomized, placebo-controlled, multicenter phase 2 trial is designed to test the central
hypothesis that TPr signaling promotes cardiac inflammation and drives IP overproduction contributing to DMD
CM, thereby perpetuating the inflammatory response which leads to cardiac fibrosis and vascular dysfunction.
Aim 1 will determine the safety and efficacy of ifetroban, an oral TPr antagonist, as treatment for DMD
CM. A network of pediatric cardiologists with DMD and cardiac imaging expertise at the largest DMD centers in
the country will expedite enrollment for this rare disease. The same imaging equipment and standardized
cardiac imaging protocol will be used to compare cardiac imaging data across centers. Aim 2 will determine
the effect of TPr blockade on protein expression and CMR structures. Specimens and imaging will be
used to determine the effects of TPr blockade on skeletal muscle and CM disease progression. Successful
completion of this phase 2 trial will help support the approval of ifetroban for treatment of DMD CM. Study
outcomes will provide the necessary information for design of the pivotal phase 3 study to have the greatest
impact on reduci...

## Key facts

- **NIH application ID:** 10246611
- **Project number:** 3R01FD006371-01A1S1
- **Recipient organization:** CUMBERLAND PHARMACEUTICALS, INC.
- **Principal Investigator:** Ines Macias-Perez
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2020
- **Award amount:** $97,112
- **Award type:** 3
- **Project period:** 2020-09-01 → 2022-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10246611

## Citation

> US National Institutes of Health, RePORTER application 10246611, A Ph2 Randomized, Placebo-controlled, Multiple Dose Study to Determine the Safety, Pharmacokinetics and Efficacy of Oral Ifetroban in Subjects with Duchenne Musculary Dystrophy IND136895 (10/10/2018) (3R01FD006371-01A1S1). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10246611. Licensed CC0.

---

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