Oral Ifetroban for Duchenne Muscular Dystrophy

(DMD Trial)

Recruiting at 9 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

48 Participants Needed

Trial Summary

What is the purpose of this trial?

Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss of ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, and inevitably premature death of affected young men in their late twenties. DMD is the most common fatal genetic disorder diagnosed in childhood. It affects approximately 1 in every 3,500 live male births across all races and cultures, and results in 20,000 new cases each year worldwide.Significant advances in respiratory care have unmasked CM as the leading cause of death. As there are yet no specific cardiac treatments to extend life, the current study aims to address this unmet medical need using a new therapeutic strategy for patients with DMD. Funding Source - FDA OOPD

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.

How is the drug Ifetroban unique in treating Duchenne Muscular Dystrophy?

Ifetroban is unique because it is an oral medication specifically being studied for Duchenne Muscular Dystrophy, a condition with limited treatment options. Unlike other treatments, Ifetroban is known for its potential to target specific pathways involved in muscle damage and inflammation, which may offer a novel approach to managing this condition. ^{1 2 3 4 5}

Research Team

Larry Markham, MD

Principal Investigator

Riley Children's Hospital

Eligibility Criteria

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.

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

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

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

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

Treatment Details

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.

Participant Groups

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+

References

1. United States pubmed.ncbi.nlm.nih.gov

Efficacy of irbesartan, a receptor selective antagonist of angiotensin II, in reducing portal hypertension. [2019]

2. Germany pubmed.ncbi.nlm.nih.gov

Comparative pharmacokinetics of isosorbide nitrates after repeated doses of sustained release isosorbide dinitrate. [2016]

3. Netherlands pubmed.ncbi.nlm.nih.gov

[Effects of isosorbide dinitrate on coronary and systemic circulation in children: comparison with dipyridamole]. [2013]

4. Netherlands pubmed.ncbi.nlm.nih.gov

Effects of long-term Irbesartan in reducing portal pressure in cirrhotic patients: comparison with propranolol in a randomised controlled study. [2019]

5. Germany pubmed.ncbi.nlm.nih.gov

[Comparative study of the bioavailability and pharmacokinetics of isosorbide dinitrate formulations in retard form and in standard preparations by determination of isosorbide-5-mononitrate]. [2016]