The FIGHT DMD Trial - An FDA Sponsored Study Aimed to Impact the Cardiomyopathy associated with DMD
Larry W. Markham, MD
Professor, Pediatrics and Medicine
Indiana University School of Medicine
Chief, Division of Pediatric Cardiology
Riley Hospital for Children at IU Health
June 30, 2023
Disclosures
No direct financial conflicts or disclosures
PI for the clinical trial
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Ifetroban
Ifetroban prevents fibrosis & inflammation by blocking thromboxane receptor signaling
Ifetroban increased survival & cardiac output in severe pre-clinical mouse DMD models
Safety is well established with nearly 1,400 clinical trial participants dosed in nearly 30 clinical studies
A dime-size, once daily, oral medication taken in a fasted state
IFETROBAN
Phase 2 Objective: Evaluate efficacy, safety and PK of ifetroban in DMD through daily, oral ifetroban/placebo for 12 months;
2/3 of subjects randomized to ifetroban
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FIGHT DMD
Trial Design
Early-stage Cohort
Enrollment closed
Late-stage Cohort
Study Timeline
February 2023
October
Early-stage
Goal: Complete
2020
cohort
late-stage cohort
First patient
enrollment
enrollment by
enrolled
completed!
end of 2023
July 2022
May 2023
Initial safety
Interim safety
analysis
and efficacy
completed
analysis
underway
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Baseline Characteristics:
First 25 patients who completed 6 months of treatment
Treatment Group
High-Dose*
Low-Dose*
Placebo
Ifetroban
Ifetroban
Number of patients
9
8
8
Ambulatory, N (%)
Yes
2 (22)
3 (38)
3 (38)
No
7 (78)
5 (62)
5 (62)
Ventilator Support, N (%)
Yes
2 (22)
2 (25)
0
No
7 (78)
6 (75)
8 (100)
Stage of DMD, N (%)
Early Stage (LVEF >45%)
6 (67)
7 (88)
8 (100)
Late Stage (LVEF 35-45%)
3 (33)
1 (12)
0
Background Therapy, N (%)
No Background Therapy
2 (22)
1 (13)
1 (13)
Steroids
4 (45)
5 (62)
4 (50)
Steroids + ASO
3 (33)
2 (25)
3 (37)
*Low dose ifetroban: < 35 kg 50 mg/day, > 35 kg 100 mg/day
High dose ifetroban: < 35 kg 150 mg/day, > 35 kg 300 mg/day
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Cumberland Pharmaceuticals Inc. published this content on 25 July 2023 and is solely responsible for the information contained therein. Distributed by Public, unedited and unaltered, on 25 July 2023 15:54:28 UTC.
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