Eptacog beta to treat and prevent serious bleeding in Glanzmann thrombasthenia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• ATHNdataset registry for people with bleeding and clotting disorders

  This registry collects health, treatment, and patient-reported information from people with bleeding and clotting disorders seen at ATHN-affiliated centers to help improve care and outcomes.

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• Using videomicroscopy to predict bleeding risk in patients with bleeding disorders

  This study is testing if a new way to look at tiny blood vessels under the tongue can help predict bleeding risks in adults with von Willebrand disease and Glanzmann thrombasthenia.

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• Understanding inherited blood disorders and their genetic basis

  This study is trying to understand the genetics behind inherited blood disorders like Glanzmann thrombasthenia by looking at the medical histories and blood tests of both healthy volunteers and patients.

  Recruiting Rockefeller University · New York, New York · NCT00230165

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.