Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 18 March 2024
• Main ID: NCT03340675
• Date of registration: 03/11/2017
• Prospective Registration: Yes
• Primary sponsor: Cumberland Pharmaceuticals
• Public title: Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy DMD
• Scientific title: A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study With an Open-Label Extension to Determine the Safety, Pharmacokinetics and Efficacy of Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
• Date of first enrolment: October 19, 2020
• Target sample size: 48
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03340675
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Larry Markham, MD
• Affiliation: Riley Children's Hospital

• Name: Ines M Macias-Perez, PhD

Key inclusion & exclusion criteria

Inclusion criteria:

1. 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.

2. Stable dose of oral corticosteroids for at least 8 weeks or has not received
corticosteroids for at least 30 days.

3. Stable cardiac function defined as change in left ventricular ejection fraction (LVEF)
of < 15% and no heart failure admission over the last 12 months; LVEF 35% or greater
by cine cardiac magnetic resonance imaging (MRI) or echocardiography; myocardial
damage in one or more left ventricular segments evident by late gadolinium enhancement
allowed; concurrent angiotensin-converting enzyme inhibitors (ACEI), beta-blocker (BB)
or angiotensin receptor blocker (ARB) therapy allowed (selection of which dictated by
clinical care) if started three months or greater from first dose of IMP without
change in dose. Aldosterone receptor antagonists (eg. Spironolactone or eplerenone)
allowed if started 12 months or greater from first dose of Investigational Medicinal
Product (IMP). No changes throughout the study allowed, except in the event of a
decline in left ventricular ejection fraction (LVEF) >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%.

4. 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:

1. Clinically significant illness other than DMD

2. Clinically significant laboratory abnormality not associated with DMD

3. 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

4. Require antiarrhythmic therapy and/or initiation of diuretic therapy for management of
acute heart failure in the last 6 months

5. A LVEF of < 35% by Cardiac Magnetic Resonance Imaging (CMR) and/or fractional
shortening of < 15% based on echocardiography (ECHO) during screening

6. A known bleeding disorder or has received anticoagulant treatment within 2 weeks of
study entry

7. 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

8. Non-MR compatible implants (e.g. neurostimulator, automatic implantable
cardioverter-defibrillator [AICD])

9. Subjects who participated in a therapeutic clinical trial within 30 days or five
half-lives (whichever is longer) of study entry

10. Any other condition that could interfere with the subject's participation



Age minimum: 7 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Cardiomyopathy, Dilated

Duchenne Muscular Dystrophy Cardiomyopathy

Intervention(s)

Drug: Placebo

Drug: Ifetroban

Primary Outcome(s)

Incidence of Treatment-Emergent Adverse Events (safety & tolerability) [Time Frame: Baseline through 12 months]

Secondary Outcome(s)

Change from baseline in pulmonary function [Time Frame: Baseline through 12 months]

Change from baseline in quality-of-life [Time Frame: Baseline through 12 months]

Pharmacokinetics plasma terminal half-life concentration [Time Frame: Day 0 and Day 7]

Change from baseline in left ventricular ejection fraction [Time Frame: Baseline through 12 months]

Pharmacokinetics maximum serum concentration (Cmax) [Time Frame: Day 0 and Day 7]

Pharmacokinetics Area under the curve [Time Frame: Day 0 and Day 7]

Pharmacokinetics time to reach Cmax (Tmax) concentration [Time Frame: Day 0 and Day 7]

Secondary ID(s)

FD-R-6371

CPI-IFE-007

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Vanderbilt University Medical Center