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Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
Phase 2 trial testing Ifetroban in Duchenne Muscular Dystrophy Cardiomyopathy in 46 participants. Completed in 23 January 2026.
| Lead sponsor | Cumberland Pharmaceuticals |
|---|---|
| Phase | Phase 2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | quadruple |
| Primary purpose | treatment |
| Enrollment | 46 |
| Start date | 19 October 2020 |
| Primary completion | 6 March 2024 |
| Estimated completion | 23 January 2026 |
| Sites | 10 locations across United States |
Cumberland Pharmaceuticals — full company profile →
7 and older, male only, with Duchenne Muscular Dystrophy Cardiomyopathy or Cardiomyopathy, Dilated. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percentage of subjects with one or more treatment emergent adverse event
| Group | Value | 95% CI |
|---|---|---|
| High Dose Ifetroban | 14 | |
| Low Dose Ifetroban | 9 | |
| Placebo | 10 |
Plasma ifetroban and its acyl glucuronide metabolite were measured pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7. Area under the curve until the last measurement was calculated for the plasma concentration versus time curves for ifetroban and its acyl glucuronide metabolite after administration of assigned oral ifetroban dose
| Group | Value | 95% CI |
|---|---|---|
| High Dose Ifetroban | 3196 | ± 2485 |
| Low Dose Ifetroban | 761 | ± 585 |
| Group | Value | 95% CI |
|---|---|---|
| High Dose Ifetroban | 45116 | ± 30211 |
| Low Dose Ifetroban | 13789 | ± 9700 |
Plasma ifetroban and its acyl glucuronide metabolite were measured pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7 to determine the maximum plasma concentration of ifetroban and its acyl glucuronide metabolite after administration of assigned oral ifetroban dose
| Group | Value | 95% CI |
|---|---|---|
| High Dose Ifetroban | 1560 | ± 2028 |
| Low Dose Ifetroban | 272 | ± 300 |
| Group | Value | 95% CI |
|---|---|---|
| High Dose Ifetroban | 9787 | ± 7148 |
| Low Dose Ifetroban | 2276 | ± 1955 |
Plasma ifetroban and its acyl glucuronide metabolite were measured pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7 to determine the time to maximum plasma concentration of ifetroban and its acyl glucuronide metabolite following administration of assigned oral ifetroban dose
| Group | Value | 95% CI |
|---|---|---|
| High Dose Ifetroban | 0.71 | ± 0.24 |
| Low Dose Ifetroban | 1.09 | ± 1.2 |
| Group | Value | 95% CI |
|---|---|---|
| High Dose Ifetroban | 2.81 | ± 6.43 |
| Low Dose Ifetroban | 1.89 | ± 2.47 |
Plasma ifetroban were measured pre-dose and at 0.5, 1.0, 4.0, 8.0 and 24.0 h post dose on Day 0 and pre-dose and 0.5 h post-dose on Day 7. The terminal elimination half-life was calculated from the plasma concentration versus time curves for ifetroban following administration of assigned oral ifetroban dose
| Group | Value | 95% CI |
|---|---|---|
| High Dose Ifetroban | 12.96 | ± 5.73 |
| Low Dose Ifetroban | 8.99 | ± 4.6 |
Change from baseline at month 12 in left ventricular ejection fraction
| Group | Value | 95% CI |
|---|---|---|
| High Dose Ifetroban | 1.8 | ± 5.44 |
| Low Dose Ifetroban | 0 | ± 5.16 |
| Placebo | -1.5 | ± 3.27 |
Change from baseline at month 12 in Forced Expiratory Volume in 1 second
| Group | Value | 95% CI |
|---|---|---|
| High Dose Ifetroban | -0.05 | ± 0.233 |
| Low Dose Ifetroban | -0.11 | ± 0.294 |
| Placebo | 0.01 | ± 0.169 |
Change from baseline at month 12 in percent predicted Forced Expiratory Volume in 1 second
| Group | Value | 95% CI |
|---|---|---|
| High Dose Ifetroban | -1.92 | ± 10.67 |
| Low Dose Ifetroban | -7.89 | ± 9.226 |
| Placebo | -3.29 | ± 6.775 |
Change from baseline at month 12 in Forced Vital Capacity
| Group | Value | 95% CI |
|---|---|---|
| High Dose Ifetroban | -0.08 | ± 0.291 |
| Low Dose Ifetroban | -0.07 | ± 0.378 |
| Placebo | -0.08 | ± 0.291 |
Change from baseline at month 12 in percent predicted Forced Vital Capacity
| Group | Value | 95% CI |
|---|---|---|
| High Dose Ifetroban | -3.31 | ± 10.491 |
| Low Dose Ifetroban | -6.11 | ± 11.050 |
| Placebo | -8.14 | ± 15.700 |
Change from baseline at month 12 in maximal expiratory pressure
| Group | Value | 95% CI |
|---|---|---|
| High Dose Ifetroban | -1.69 | ± 8.625 |
| Low Dose Ifetroban | 2.00 | ± 16.466 |
| Placebo | -16.50 | ± 25.774 |
Change from baseline at month 12 in maximal inspiratory pressure
| Group | Value | 95% CI |
|---|---|---|
| High Dose Ifetroban | -6.00 | ± 7.000 |
| Low Dose Ifetroban | -9.13 | ± 8.935 |
| Placebo | 1.17 | ± 20.14 |
Time frame: From initiation of treatment through study completion, up to 1 year. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | High Dose Ifetroban | Low Dose Ifetroban | Placebo |
|---|---|---|---|---|
| Pneumonia | Infections and infestations | — | — | — |
| Cerebrovascular accident | Nervous system disorders | — | — | — |
| Oesophageal obstruction | Gastrointestinal disorders | — | — | — |
| Hypervolaemia | Metabolism and nutrition disorders | — | — | — |
| Femur fracture | Injury, poisoning and procedural complications | — | — | — |
| Dehydration | Metabolism and nutrition disorders | — | — | — |
| Metabolic acidosis | Metabolism and nutrition disorders | — | — | — |
| Fall | Injury, poisoning and procedural complications | — | — | — |
| Hip fracture | Injury, poisoning and procedural complications | — | — | — |
| Reaction | System | High Dose Ifetroban | Low Dose Ifetroban | Placebo |
|---|---|---|---|---|
| COVID-19 | Infections and infestations | — | — | — |
| Nausea | Gastrointestinal disorders | — | — | — |
| Vomiting | Gastrointestinal disorders | — | — | — |
| Headache | Nervous system disorders | — | — | — |
| Diarrhoea | Gastrointestinal disorders | — | — | — |
| Abdominal pain | Gastrointestinal disorders | — | — | — |
| Nasopharyngitis | Infections and infestations | — | — | — |
| Pneumonia | Infections and infestations | — | — | — |
| Contusion | Injury, poisoning and procedural complications | — | — | — |
| Fall | Injury, poisoning and procedural complications | — | — | — |
| Back pain | Musculoskeletal and connective tissue disorders | — | — | — |
| Muscle contracture | Musculoskeletal and connective tissue disorders | — | — | — |
| Dizziness | Nervous system disorders | — | — | — |
| Ejection fraction decreased | Investigations | — | — | — |
| Rash | Skin and subcutaneous tissue disorders | — | — | — |
| Delayed puberty | Endocrine disorders | — | — | — |
| Constipation | Gastrointestinal disorders | — | — | — |
| Abdominal pain upper | Gastrointestinal disorders | — | — | — |
| Oesophageal obstruction | Gastrointestinal disorders | — | — | — |
| Foot fracture | Injury, poisoning and procedural complications | — | — | — |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | — | — | — |
| Anxiety | Psychiatric disorders | — | — | — |
| Fatigue | General disorders | — | — | — |
Most-reported serious reactions: Pneumonia, Cerebrovascular accident, Oesophageal obstruction, Hypervolaemia, Femur fracture, Dehydration, Metabolic acidosis, Fall.
Data from ClinicalTrials.gov NCT03340675 adverse events section.
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
8 peer-reviewed publications reference this trial (live from Europe PMC):
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