Oral Ifetroban for Duchenne Muscular Dystrophy

(DMD Trial)

Not currently recruiting at 11 trial locations

Overseen By Byron Kaelin

Age: Any Age

Sex: Male

Trial Phase: Phase 2

Sponsor: Cumberland Pharmaceuticals

Must be taking: Corticosteroids

Must not be taking: Antiarrhythmics, Anticoagulants

Disqualifiers: Significant illness, Major surgery, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 12

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment, Ifetroban (an oral medication), to aid individuals with Duchenne muscular dystrophy (DMD), with a focus on heart health. DMD is a serious condition affecting muscles, including the heart, and currently lacks specific treatments for its cardiac issues. The study compares low-dose and high-dose Ifetroban with a placebo to determine its effect on heart function in those with DMD. This trial may suit males over 7 years old with DMD and stable heart function who still face heart challenges. As a Phase 2 trial, the research measures the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial allows participants to continue taking certain heart medications like ACE inhibitors, beta-blockers, and angiotensin receptor blockers if they have been on a stable dose for at least three months. However, no changes to these medications are allowed during the study unless there's a significant decline in heart function. The protocol does not specify about other medications, so it's best to discuss with the trial team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that ifetroban, a treatment being tested for Duchenne muscular dystrophy (DMD), has promising safety results from earlier studies. Patients who took ifetroban demonstrated improved heart function, which is crucial for those with DMD. Notably, no major safety issues emerged.

The studies tested both low and high doses of ifetroban, and both were well-tolerated, with most patients not experiencing serious side effects. This suggests that ifetroban could be a safe option for people with DMD. However, like all treatments, some minor side effects might occur. Overall, the evidence so far supports the safety of ifetroban in humans. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Duchenne muscular dystrophy?

Researchers are excited about ifetroban for Duchenne Muscular Dystrophy (DMD) because it offers a new way to tackle the disease. Unlike standard treatments like corticosteroids, which aim to slow muscle degeneration by reducing inflammation, ifetroban targets the thromboxane receptor, potentially reducing fibrosis and improving muscle function. This novel mechanism could provide benefits beyond what current anti-inflammatory drugs offer, making it a promising option for those with DMD. Additionally, the oral administration of ifetroban makes it convenient for patients compared to therapies that require injections or infusions.

What evidence suggests that Ifetroban might be an effective treatment for Duchenne muscular dystrophy?

Research has shown that ifetroban may help treat Duchenne muscular dystrophy (DMD) by slowing heart problems. In this trial, participants will receive either a low dose or a high dose of ifetroban, or a placebo. An earlier study found that patients who took a high dose of ifetroban experienced a 3.3% improvement in heart function compared to those who did not receive the treatment. This suggests that ifetroban could effectively address heart issues in DMD, a leading cause of death in these patients. ^{1 4 6 7 8}

Who Is on the Research Team?

Larry Markham, MD

Principal Investigator

Riley Children's Hospital

Are You a Good Fit for This Trial?

This trial is for males aged 7 and older with Duchenne Muscular Dystrophy (DMD). Participants must have stable heart function, be on certain heart medications for a set time without changes, and can't have had recent major surgery or other clinical trials. They need to be able to undergo MRI scans and not require specific heart failure treatments recently.

Inclusion Criteria

I am a male, 7 or older, diagnosed with DMD based on symptoms, genetic tests, or muscle biopsy.

I haven't taken corticosteroids for the last 30 days or have been on a stable dose for 8 weeks.

My heart functions well, with no recent failures or significant changes in medication.

Exclusion Criteria

I do not have any major illnesses besides DMD.

I haven't had major surgery in the last 6 weeks and don't plan any that would affect this study.

You have a significant abnormality in your lab tests that is not related to the condition being studied.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral ifetroban or placebo once daily for 12 months

12 months

Regular visits for safety and efficacy assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment with high-dose ifetroban long-term

Long-term

Evaluated for safety and cardiac efficacy every 12 months

What Are the Treatments Tested in This Trial?

Interventions

• Ifetroban
• Placebo

Trial Overview The study tests Ifetroban, a potential new treatment for the cardiomyopathy associated with DMD. It's compared against a placebo in boys and men who meet strict health criteria. The goal is to find out if Ifetroban can help improve cardiac outcomes in DMD patients.

How Is the Trial Designed?

Group I: Oral Ifetroban - Low Dose Experimental Treatment 1 Intervention

Weight based, once daily oral ifetroban

Group II: Oral Ifetroban - High Dose Experimental Treatment 1 Intervention

Weight based, once daily oral ifetroban

Group III: Placebos Placebo Group 1 Intervention

Matching Placebo

Who Is Running the Clinical Trial?

Cumberland Pharmaceuticals

Lead Sponsor

Trials

63

Recruited

9,400+

A.J. Kazimi

Cumberland Pharmaceuticals

Chief Executive Officer since 1999

B.S. from the University of Notre Dame, MBA from Vanderbilt University Owen Graduate School of Management

Ines Macias-Perez

Cumberland Pharmaceuticals

Chief Medical Officer

Ph.D. in Cancer Biology from Vanderbilt University

Vanderbilt University Medical Center

Collaborator

Trials

922

Recruited

939,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03340675

Study Details | NCT03340675 | Oral Ifetroban in Subjects ... This is a phase 2 randomized, double-blind, placebo-controlled, multiple dose study with an optional open-label extension to determine the safety, ...

2. parentprojectmd.org parentprojectmd.org/clinical-trial/oral-ifetroban-in-subjects-with-duchenne-muscular-dystrophy/

Oral Ifetroban in Subjects With Duchenne Muscular ... All subjects who receive treatment will be assessed for safety. All subjects with at least one efficacy assessment post-baseline will be evaluated for efficacy.

3. neurologylive.com neurologylive.com/view/ifetroban-shows-promise-slowing-cardiac-decline-dmd

Ifetroban Shows Promise in Slowing Cardiac Decline ... High-dose ifetroban improved LVEF in Duchenne muscular dystrophy patients, contrasting with declines in placebo and natural history cohorts.

4. investor.cumberlandpharma.com investor.cumberlandpharma.com/news-releases/news-release-details/fight-dmd-trial-results-selected-late-breaking-presentation-mda

FIGHT DMD Trial Results Selected for Late-Breaking ... The Phase 2 FIGHT DMD trial demonstrated that high-dose ifetroban treatment resulted in a 3.3% improvement in left ventricular ejection fraction (LVEF) ...

5. fightdmdtrial.com fightdmdtrial.com/

The Fight DMD Trial The FIGHT DMD Trial is a 12-month, double-blind, placebo-controlled study to evaluate the effectiveness and safety of oral ifetroban in participants with ...

6. parentprojectmd.org parentprojectmd.org/drug-development-pipeline/ifetroban/

Ifetroban A Phase 2 randomized, double-blind, placebo-controlled, multiple dose study to determine the safety, pharmacokinetics and efficacy of two doses of oral ...

7. musculardystrophynews.com musculardystrophynews.com/news/ifetroban-continues-show-cardiac-protection-phase-2-dmd-trial/

Ifetroban continues to show cardiac protection in Phase 2 ... Ifetroban improved heart function and reduced cardiac damage biomarkers in people with DMD in a Phase 2 clinical trial.

8. research-studies.allinforhealth.info research-studies.allinforhealth.info/us/en/listing/11878/oral-ifetroban-in-subjects/

Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy All subjects who receive treatment will be assessed for safety. All subjects with at least one efficacy assessment post-baseline will be ...