💬In plain words
What the trial is, and whether it could suit you
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 visits with their hematologist over the 9-month study period. They will also be asked to complete a di
Sign in to get the eligibility rules as simple questions to take to your doctor.
📋 Eligibility rules (as published)
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
Arthur M. Blank Hospital | Children's Healthcare of Atlanta
Atlanta, United States
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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