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Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy (DMD)

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ClinicalTrials.gov Identifier: NCT03340675
Recruitment Status : Not yet recruiting
First Posted : November 13, 2017
Last Update Posted : November 14, 2017
Sponsor:
Collaborator:
Information provided by (Responsible Party):

Study Description
Brief Summary:
This is an open-label, multicenter, dose-ranging study to determine the safety, pharmacokinetics and efficacy of two dose ranges of oral ifetroban in subjects with DMD. DMD patients who meet the inclusion criteria and none of the exclusion criteria will receive oral ifetroban once daily for 12 months.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Diseases Drug: Oral Ifetroban Phase 2

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 32 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: Dose-ranging study, enrollment onto low dose followed by high dose
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Multicenter, Dose-Ranging Study to Determine the Safety, Pharmacokinetics and Efficacy of Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
Anticipated Study Start Date : March 2018
Estimated Primary Completion Date : April 2019
Estimated Study Completion Date : July 2019


Arms and Interventions

Arm Intervention/treatment
Experimental: Oral Ifetroban - Low Dose
Weight based, once daily oral ifetroban
Drug: Oral Ifetroban
Weight-based dosing
Experimental: Oral Ifetroban - High Dose
Weight based, once daily oral ifetroban
Drug: Oral Ifetroban
Weight-based dosing


Outcome Measures

Primary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: Baseline through 12 months ]
    number of subjects with one or more treatment emergent adverse event


Secondary Outcome Measures :
  1. Peak plasma concentration of ifetroban and its acyl glucuronide metabolite after administration [Pharmacokinetics] [ Time Frame: Day 0 (first dose) and at Month 6 (steady state) ]
    Peak plasma concentration of ifetroban and its acyl glucuronide metabolite after administration.

  2. Change from baseline in myocardial strain (a sensitive measure of heart function using cardiac MRI) [Efficacy] [ Time Frame: Baseline and 12 months ]
    There should be no change in myocardial strain. Patients with DMD are likely to have an increase in myocardial strain over time (2% or more in 12 months)

  3. Change from baseline in forced expiratory volume in 1 second (FEV1) [Pulmonary function] [ Time Frame: Baseline and 12 months ]
    a low FEV1 indicates poor pulmonary function; normal values for FEV1 vary from person to person. Each person has their own predicted FEV1 value.

  4. Pediatric Quality of Life (PedQL) Inventory including Neuromuscular module [Quality of life] [ Time Frame: Baseline and 12 months ]
    The 23 items PedQL measure 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.


Eligibility Criteria

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Ages Eligible for Study:   7 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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 12 weeks or has not received corticosteroids for at least 12 weeks.
  • Stable cardiac function defined as change in left ventricular ejection fraction of < 15% and no heart failure admission over the last 12 months; left ventricular ejection fraction 35% or greater by cine cardiac MRI (CMR) 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 ifetroban without change in dose. Aldosterone receptor antagonists (eg. Spironolactone or eplerenone) allowed if started 12 months or greater from first dose of ifetroban. No changes throughout the study allowed.
  • Use of contraceptives for sexually active males throughout the study
  • Subjects aged 18 years and older, informed consent obtained directly. For subjects ages 7-17 years, 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 and/or antidiuretic therapy for heart failure
  • A left ventricular ejection fraction (EF) of < 35% by CMR and/or fractional shortening of < 15% based on echocardiography (ECHO) during screening
  • Concurrent use of nitrates, alpha-adrenergic receptor blockers, or phosphodiesterase inhibitors
  • A known bleeding disorder or has received chronic anticoagulant treatment within 2 weeks of study entry
  • Allergy to gadolinium contrast or known renal insufficiency
  • Non-MR compatible implants (e.g. neurostimulator, AICD)
  • Any other condition that could interfere with the subject's participation
Contacts and Locations

Information from the National Library of Medicine

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


Contacts
Contact: Ines M Macias-Perez, PhD 6159795778 imaciasperez@cumberlandpharma.com
Contact: Byron Kaelin, RN 6156274121 bkaelin@cumberlandpharma.com

Sponsors and Collaborators
Cumberland Pharmaceuticals
Vanderbilt University Medical Center
Investigators
Principal Investigator: Larry Markham, MD Vanderbilt University School of Medicine
More Information

Responsible Party: Cumberland Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03340675     History of Changes
Other Study ID Numbers: CPI-IFE-007
First Posted: November 13, 2017    Key Record Dates
Last Update Posted: November 14, 2017
Last Verified: November 2017
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

Additional relevant MeSH terms:
Muscular Diseases
Muscular Disorders, Atrophic
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Ifetroban
Platelet Aggregation Inhibitors