Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy (DMD)
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ClinicalTrials.gov Identifier: NCT03340675 |
Recruitment Status :
Recruiting
First Posted : November 13, 2017
Last Update Posted : August 2, 2022
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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
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Duchenne Muscular Dystrophy Cardiomyopathy Cardiomyopathy, Dilated | Drug: Ifetroban Drug: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 48 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Intervention Model Description: | Randomized, placebo-controlled, double-blind, dose-ranging |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Masking Description: | Each individual bottle is labelled with a unique numeric code that identifies the contents to the unblinded sponsor representative. |
Primary Purpose: | Treatment |
Official 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 |
Actual Study Start Date : | October 19, 2020 |
Estimated Primary Completion Date : | August 31, 2024 |
Estimated Study Completion Date : | August 31, 2024 |

Arm | Intervention/treatment |
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Experimental: Oral Ifetroban - Low Dose
Weight based, once daily oral ifetroban
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Drug: Ifetroban
Weight based, once daily oral ifetroban |
Experimental: Oral Ifetroban - High Dose
Weight based, once daily oral ifetroban
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Drug: Ifetroban
Weight based, once daily oral ifetroban |
Placebo Comparator: Placebos
Matching Placebo
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Drug: Placebo
Matching oral placebo |
- Incidence of Treatment-Emergent Adverse Events (safety & tolerability) [ Time Frame: Baseline through 12 months ]Percentage of subjects with one or more treatment emergent adverse event
- Pharmacokinetics Area under the curve [ Time Frame: Day 0 and Day 7 ]Measurements of Area under the curve concentration of ifetroban and its acyl glucuronide metabolite
- Pharmacokinetics maximum serum concentration (Cmax) [ Time Frame: Day 0 and Day 7 ]Measurements of maximum serum concentration (Cmax) of ifetroban and its acyl glucuronide metabolite
- Pharmacokinetics time to reach Cmax (Tmax) concentration [ Time Frame: Day 0 and Day 7 ]Measurement of time to reach Cmax (Tmax) concentration of ifetroban and its acyl glucuronide metabolite
- Pharmacokinetics plasma terminal half-life concentration [ Time Frame: Day 0 and Day 7 ]Measurement of plasma terminal half-life concentration of ifetroban and its acyl glucuronide metabolite
- Change from baseline in left ventricular ejection fraction [ Time Frame: Baseline through 12 months ]There should be no change in left ventricular ejection fraction. Patients with DMD have a decline.
- Change from baseline in pulmonary function [ Time Frame: Baseline through 12 months ]Change from baseline in forced expiratory volume in 1 second
- Change from baseline in quality-of-life [ Time Frame: Baseline through 12 months ]The 23 items Pediatric Quality of Life Inventory (PedQL) questionnaire measures these core dimensions of health as delineated by the World Health Organization, as well as role (school) functioning. Items measured include 1) Physical Functioning (8 items), 2) Emotional Functioning (5 items), 3) Social Functioning (5 items), and 4) School Functioning (5 items). Each item is measured on a 5 point Likert scale with 0 indicating never and 4 indicating almost always.The Likert scores are reversed scored and linearly transform to a 0-100 scale with 0=100, 1-75, 2=50, 5=25, and 4=0. A higher score indicates better health-related quality of life.

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Ages Eligible for Study: | 7 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
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 < 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.
- 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
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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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03340675
Contact: Ines M Macias-Perez, PhD | 6159795778 | imaciasperez@cumberlandpharma.com | |
Contact: Ingrid Anderson, PhD, CCRP | 615-255-0068 ext 250 | ianderson@cumberlandpharma.com |
United States, Arkansas | |
Arkansas Children's Hospital | Recruiting |
Little Rock, Arkansas, United States, 72202 | |
Contact: Annette Guy 501-364-3380 GuyEA@archildrens.org | |
Principal Investigator: Markus Renno, MD | |
United States, California | |
Mattel Children's Hospital | Recruiting |
Los Angeles, California, United States, 90095 | |
Contact: Emilie Douine-Barthelemy 310-991-2674 EDouine@mednet.ucla.edu | |
United States, District of Columbia | |
Children's National Hospital | Recruiting |
Washington, District of Columbia, United States, 20010 | |
Contact: Robert Lowndes 301-512-3337 rlowndes@childrensnational.org | |
United States, Georgia | |
Children's Healthcare of Atlanta | Recruiting |
Atlanta, Georgia, United States, 30341 | |
Contact: Melissa Burnett 404-785-0381 melissa.burnett@choa.org | |
United States, Illinois | |
Lurie Children's Hospital | Recruiting |
Chicago, Illinois, United States, 60611 | |
Contact: Kathleen Van't Hof kvanthof@luriechildrens.org | |
Principal Investigator: Katheryn Gambetta, MD | |
United States, Indiana | |
Riley Children's Hospital | Recruiting |
Indianapolis, Indiana, United States, 46202 | |
Contact: Jennifer Howell 317-278-7818 jemrich@iu.edu | |
United States, Maryland | |
Kennedy Krieger Institute | Recruiting |
Baltimore, Maryland, United States, 21205 | |
Contact: Genila Bibat, MD bibat@kennedykrieger.org | |
Contact: Sharanya Suresh suresh@kennedyKrieger.org | |
Principal Investigator: Doris Leung, MD | |
United States, Missouri | |
Saint. Louis Children's Hospital | Recruiting |
Saint Louis, Missouri, United States, 63110 | |
Contact: Jessie Schwieterman, PT, CCRP j.schwieterman@wustl.edu | |
Principal Investigator: Craig Zaidman, MD | |
United States, Ohio | |
Cincinnati Children's Hospital Medical Center | Recruiting |
Cincinnati, Ohio, United States, 45229 | |
Contact: Kenneth Mucheck Kenneth.Mucheck@cchmc.org | |
Contact: Remesha Palmer Remesha.Palmer@cchmc.org | |
Principal Investigator: Chet Villa, MD | |
United States, Tennessee | |
Monroe Carrell Jr. Children's Hospital at Vanderbilt | Recruiting |
Nashville, Tennessee, United States, 37232 | |
Contact: Kim Crum, RN, BS 615-936-2497 kimberly.crum@vumc.org | |
Principal Investigator: Jonathan H Soslow, MD |
Principal Investigator: | Larry Markham, MD | Riley Children's Hospital |
Responsible Party: | Cumberland Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT03340675 |
Other Study ID Numbers: |
CPI-IFE-007 FD-R-6371 ( Other Grant/Funding Number: FDA Orphan Products Development ) |
First Posted: | November 13, 2017 Key Record Dates |
Last Update Posted: | August 2, 2022 |
Last Verified: | July 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Muscular Dystrophies Muscular Dystrophy, Duchenne Cardiomyopathies Cardiomyopathy, Dilated Heart Diseases Cardiovascular Diseases Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Cardiomegaly Laminopathies Ifetroban Platelet Aggregation Inhibitors |