Eptacog beta to treat and prevent serious bleeding in Glanzmann thrombasthenia
Study To Assess Response to Eptacog Beta iN Patients With Glanzmann THromboasthenia (STRENGTH )
PHASE2 · Emory University · NCT07136857
This will test whether eptacog beta can stop acute bleeds and help prevent serious bleeding in people with Glanzmann thrombasthenia who have a severe bleeding history.
Quick facts
| Phase | PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 6 (estimated) |
| Sex | All |
| Sponsor | Emory University (other) |
| Locations | 1 site (Atlanta, Georgia) |
| Trial ID | NCT07136857 on ClinicalTrials.gov |
What this trial studies
This Phase 2 protocol enrolls six adults or children with Glanzmann thrombasthenia who meet criteria for a severe bleeding phenotype. The first three months are non-interventional, with participants using their usual on-demand therapy while keeping a diary and providing retrospective and prospective bleeding data. After that, participants enter a six-month on-demand phase where eptacog beta (75 mcg/kg) may be used for breakthrough serious bleeds, with treatment frequency and duration guided by the treating physician. Hemostatic efficacy will be rated using a 4-point score and safety and bleeding patterns will be monitored over approximately 9 months with 4–5 hematology visits.
Who should consider this trial
Good fit: Ideal candidates are English-speaking adults or children with inherited Glanzmann thrombasthenia who have a severe bleeding phenotype (for example ≥2 treated bleeds in the past year, prior hospitalization for bleeding, severe anemia, or need for prophylactic therapy) and adequate liver and kidney function.
Not a fit: Patients with platelet counts under 100,000, other inherited or acquired bleeding disorders, recent (within 2 years) venous or arterial thrombosis, active malignancy, or known/suspected hypersensitivity to rabbit proteins are unlikely to benefit or are excluded.
Why it matters
Potential benefit: If successful, eptacog beta could offer an effective on-demand option to control serious bleeds and reduce bleeding-related hospitalizations for people with severe Glanzmann thrombasthenia.
How similar studies have performed: Products in the same class (recombinant activated factor VII) have been used successfully in Glanzmann thrombasthenia, but eptacog beta is a newer formulation with limited prior data in this condition.
Eligibility criteria
Show full inclusion / exclusion criteria
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 thrombasthenia * Have a history of venous or arterial thrombotic event within 2 years of study enrollment * Active malignancy * Known or suspected hypersensitivity to rabbits, rabbit protein, other forms of rFVIIa, or to any of the EB excipients * Have received an investigational drug within 30 days or within 5 half-lives of that investigational drug (whichever is longer) or are expected to receive such a drug during participation in this study * Be using aspirin, non-steroidal anti-inflammatory drugs (NSAIDS), herbs, natural medications, or other drugs with platelet inhibitor properties for the duration of the study * Be using or administered anticoagulant agents for the duration of the study * Have any life-threatening disease or other disease or condition which, according to the investigator's judgement, could imply a potential hazard to the patient, or interfere with the study participation or study outcome * Use of systemic immunomodulators at enrollment or planned use during the study
Where this trial is running
Atlanta, Georgia
- Arthur M. Blank Hospital | Children's Healthcare of Atlanta — Atlanta, Georgia, United States (RECRUITING)
Study contacts
- Principal investigator: Karen Zimowski, MD — Emory University
- Study coordinator: Karen Zimowski, MD
- Email: kzimows@emory.edu
- Phone: 404-785-1200
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Glanzmann Thrombasthenia, Severe Bleeding, Prevention of Bleeding, Eptacog beta