48 Participants Needed

Oral Ifetroban for Duchenne Muscular Dystrophy

(DMD Trial)

Recruiting at 9 trial locations
BK
IM
IA
BK
Overseen ByByron Kaelin
Age: Any Age
Sex: Male
Trial Phase: Phase 2
Sponsor: Cumberland Pharmaceuticals
Must be taking: Corticosteroids
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

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.12345

Who Is on the Research Team?

LM

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.
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.
See 7 more

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?
3Treatment groups
Experimental Treatment
Placebo Group
Group I: Oral Ifetroban - Low DoseExperimental Treatment1 Intervention
Weight based, once daily oral ifetroban
Group II: Oral Ifetroban - High DoseExperimental Treatment1 Intervention
Weight based, once daily oral ifetroban
Group III: PlacebosPlacebo Group1 Intervention
Matching Placebo

Find a Clinic Near You

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

Efficacy of irbesartan, a receptor selective antagonist of angiotensin II, in reducing portal hypertension. [2019]
Comparative pharmacokinetics of isosorbide nitrates after repeated doses of sustained release isosorbide dinitrate. [2016]
[Effects of isosorbide dinitrate on coronary and systemic circulation in children: comparison with dipyridamole]. [2013]
Effects of long-term Irbesartan in reducing portal pressure in cirrhotic patients: comparison with propranolol in a randomised controlled study. [2019]
[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]
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