Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy (DMD)
J
John Parent, MD
Primary Investigator
Enrolling By Invitation
7 years or above
Male
Phase
2
48 participants needed
1 Location
Brief description of study
What is the purpose of this study?
Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss ofbulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, an dvitably premature death of affected young men in their late twenties. DMD is the mostgenetic 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. This is a randomized, double-blind, placebo-controlled, multiple dose study with an open-label extension to determine the safety, pharmacokinetics and efficacy of low or high doses of oral ifetroban compared to placebo in subjects with DMD.
THIS STUDY IS ENROLLING BY INVITATION ONLY
Detailed description of study
What will happen during the study?
DMD patients who meet the inclusion criteria and none of the exclusion criteria will receive oral ifetroban or placebo once daily for 12 months. Subjects will be enrolled into one of three treatment groups, low-dose ifetroban, high-dose ifetroban or placebo. Each dose level will be evaluated by eight subjects with early stage (LVEF > 45%) DMD-associated cardiomyopathy and eight subjects with more advanced stage (LVEF 35-45%) cardiac disease as there may be differences in the treatment effect based on cardiac involvement. Each subject treated will be evaluated for first-dose and steady-state exposure PK. 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. Blood and urine will be collected for standard and novel cardiac biomarkers. Target enrollment met for early-stage subjects (LVEF > 45%); study closed to enrollment prior to meeting target enrollment in the late-stage cohort (LVEF 35-45%). All subjects who complete the 12-month study are eligible to enter an optional open label extension period consisting of treatment with high-dose ifetroban only. Subjects in the open label extension will be evaluated for safety and cardiac efficacy every 12 months.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Duchenne Muscular Dystrophy Cardiomyopathy, Riley
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Age: 7 years or above
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Gender: Male
Inclusion criteria:
- Males 7 years of age and older with the diagnosis of DMD, defined as phenotypewith DMD and either positive genotype, first degree relative with positivegenotype, or confirmatory muscle biopsy.
- Stable dose of oral corticosteroids for at least 8 weeks or has not receivedds for at least 30 days.
- Stable cardiac function defined as change in left ventricular ejection fraction (LVEF)< 15% and no heart failure admission over the last 12 months; LVEF 35% or greaterby cine cardiac magnetic resonance imaging (MRI) or echocardiography; myocardialdamage in one or more left ventricular segments evident by late gadolinium enhancementwed; concurrent angiotensin-converting enzyme inhibitors (ACEI), beta-blocker (BB)giotensin receptor blocker (ARB) therapy allowed (selection of which dictated by) if started three months or greater from first dose of IMP withouthange in dose. Aldosterone receptor antagonists (eg. Spironolactone or eplerenone)wed if started 12 months or greater from first dose of Investigational MedicinalProduct (IMP). No changes throughout the study allowed, except in the event of adecline in left ventricular ejection fraction (LVEF) >5% following the baseline CMR asured by a subsequent CMR at the same center. Should this occur, changes in cardiacdications are allowed on the study.
- Subjects aged 18 years and older, informed consent obtained directly. For subjectsges 7-17 years old (yo), both assent from the subject and permission from a parent orguardian.
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 plannedurgery during this study which would interfere with the ability to perform studydures
- Require antiarrhythmic therapy and/or initiation of diuretic therapy for management ofute heart failure in the last 6 months
- A LVEF of < 35% by Cardiac Magnetic Resonance Imaging (CMR) and/or fractionalhortening of < 15% based on echocardiography (ECHO) during screening
- A known bleeding disorder or has received anticoagulant treatment within 2 weeks ofudy entry
- Allergy to gadolinium contrast or known renal insufficiency defined as abnormalystatin C or creatinine above the upper limit of normal for age. The male serumges 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 fivehalf-lives (whichever is longer) of study entry
- Any other condition that could interfere with the subject's participation
Updated on
19 May 2025.
Study ID: PCRD-CUMBERLAND-CPI-IFE-007, 1910414014
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