Eptacog Beta in Glanzmann's (HeT_LFB-Strength-Study_FID531)
This study is evaluating an investigational drug, eptacog beta (EB), for the treatment and prevention of acute bleeding episodes in people with Glanzmann Thrombasthenia, a rare inherited bleeding disorder. Eptacog beta (EB) is not currently approved by the U.S. Food and Drug Administration (FDA) for this condition. The
| Condition(s) | Glanzmann Thrombasthenia |
|---|---|
| Status | Recruiting |
| Phase | Phase 2 |
| Study type | Interventional |
| Summary | This study is evaluating an investigational drug, eptacog beta (EB), for the treatment and prevention of acute bleeding episodes in people with Glanzmann Thrombasthenia, a rare inherited bleeding disorder. Eptacog beta (EB) is not currently approved by the U.S. Food and Drug Administration (FDA) for this condition. The study will assess the effectiveness and safety of eptacog beta (EB) when used to treat serious bleeding events, and in an optional phase, when used routinely to prevent bleeding. During the first three (3) months, participants will manage any bleeding episodes with their standard treatment (e.g., factor products or platelet transfusions). After this initial period, they will use the study drug to treat serious bleeding events. Participants will have approximately 4 to 5 visi |
| Who can participate | Inclusion Criteria: * Adult or Pediatric persons with inherited Glanzmann thrombasthenia (see diagnostic criteria below) * Severe bleeding phenotype * Adequate hepatic function * Adequate renal function * Adults subject (≥18 years of age) or caregiver (parent or legally authorized representative) for minor subjects, subjects with cognitive impairment, or subjects with impaired decision-making capacity have provided written informed consent, and the participant has given consent/assent (if applicable) * Ability to speak, read, and understand the English language Exclusion Criteria: * Thrombocytopenia (platelet count \< 100k) * Acquired Glanzmann thrombasthenia secondary to autoimmune disease, malignancy, or medication * Inherited or acquired bleeding diathesis other than Glanzmann thrombast |
| Sex | All |
| Lead sponsor | Emory University |
| Locations | Atlanta, Georgia, United States |
| Start date | 2025-10-02 |
| NCT ID | NCT07136857 |
| Official listing | https://clinicaltrials.gov/study/NCT07136857 |