Phase 2 N=41 Randomized Quadruple-blind Treatment

Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy Cardiomyopathy · Cardiomyopathy, Dilated

Bottom Line

View on ClinicalTrials.gov: NCT03340675 ↗

Enrolled (actual)

Serious AEs

17.1%

Results posted

Jan 2026

Primary outcome: Primary: Incidence of Treatment-Emergent Adverse Events — 14; 9; 10 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Ifetroban (Drug); Placebo (Drug)
Age: Pediatric, Adult, Older Adult · 7+ yrs
Sex: Male
Sponsor: Cumberland Pharmaceuticals
Primary completion: Mar 2024

Outcome Measures

Outcome	Result	p-value
PRIMARY Incidence of Treatment-Emergent Adverse Events	14; 9; 10	—
SECONDARY Pharmacokinetics Area Under the Curve	3196; 761; 45116; 13789	—
SECONDARY Pharmacokinetics Maximum Serum Concentration (Cmax)	1560; 272; 9787; 2276	—
SECONDARY Pharmacokinetics Time to Reach Cmax (Tmax) Concentration	0.71; 1.09; 2.81; 1.89	—
SECONDARY Pharmacokinetics Plasma Terminal Half-life Concentration	12.96; 8.99	—
SECONDARY Change From Baseline in Left Ventricular Ejection Fraction	1.8; 0; -1.5	—
SECONDARY Change From Baseline in FEV1	-0.05; -0.11; 0.01	—
SECONDARY Change From Baseline in Percent Predicted FEV1	-1.92; -7.89; -3.29	—
SECONDARY Change From Baseline in FVC	-0.08; -0.07; -0.08	—
SECONDARY Change From Baseline in Percent Predicted FVC	-3.31; -6.11; -8.14	—
SECONDARY Change From Baseline in Maximal Expiratory Pressure	-1.69; 2.00; -16.50	—
SECONDARY Change From Baseline in Maximal Inspiratory Pressure	-6.00; -9.13; 1.17	—
SECONDARY Change From Baseline in Peak Expiratory Flow Rate	0.09; -0.19; 0.51	—
SECONDARY Change From Baseline in Percent Predicted Peak Expiratory Flow Rate	-3.58; -3.83; 2.40	—
SECONDARY Change From Baseline in Quality-of-life	-0.06; 4.39; -4.03; -7.62; 0.43; -11.4	—

Summary

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

Eligibility Criteria

Inclusion criteria

Males 7 years of age and older with the diagnosis of DMD, defined as phenotype consistent with DMD and either positive genotype, first degree relative with positive genotype, or confirmatory muscle biopsy.
Stable dose of oral corticosteroids for at least 8 weeks or has not received corticosteroids for at least 30 days.
Stable cardiac function defined as change in left ventricular ejection fraction (LVEF) of 5% following the baseline CMR as measured by a subsequent CMR at the same center. Should this occur, changes in cardiac medications are allowed on the study.

a. Late-stage cohort: Subjects are eligible for the late-stage cohort if the subject has: i. LVEF 35%-45% by cine cardiac magnetic resonance imaging (MRI) or echocardiography or ii. historically documented LVEF 35%-45% by cine cardiac magnetic resonance imaging (MRI) or echocardiography and if their baseline MRI is less than 50%.

Subjects aged 18 years and older, informed consent obtained directly. For subjects ages 7-17 years old (yo), both assent from the subject and permission from a parent or guardian.

Exclusion criteria

Clinically significant illness other than DMD
Clinically significant laboratory abnormality not associated with DMD
Major surgery within six weeks prior to the first dose of study drug, or planned surgery during this study which would interfere with the ability to perform study procedures
Require antiarrhythmic therapy and/or initiation of diuretic therapy for management of acute heart failure in the last 6 months
A LVEF of < 35% by Cardiac Magnetic Resonance Imaging (CMR) and/or fractional shortening of < 15% based on echocardiography (ECHO) during screening
A known bleeding disorder or has received anticoagulant treatment within 2 weeks of study entry
Allergy to gadolinium contrast or known renal insufficiency defined as abnormal cystatin C or creatinine above the upper limit of normal for age. The male serum reference ranges as follows:
Age 7-9 years - 0.2-0.6 mg/dL
Age 10-11 years - 0.3-0.7 mg/dL
Age 12-13 years - 0.4-0.8 mg/dL
Age 14-15 years - 0.5-0.9 mg/dL
Age 16 years or older - 0.8-1.3 mg/dL
Non-MR compatible implants (e.g. neurostimulator, automatic implantable cardioverter-defibrillator [AICD])
Subjects who participated in a therapeutic clinical trial within 30 days or five half-lives (whichever is longer) of study entry
Any other condition that could interfere with the subject's participation

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03340675). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.