Actively Recruiting

Phase 2
All Genders
NCT07136857

Eptacog Beta in Glanzmann's (HeT_LFB-Strength-Study_FID531)

Led by Emory University · Updated on 2025-12-01

6

Participants Needed

1

Research Sites

60 weeks

Total Duration

On this page

Sponsors

E

Emory University

Lead Sponsor

L

Laboratoire français de Fractionnement et de Biotechnologies

Collaborating Sponsor

AI-Summary

What this Trial Is About

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 diary documenting bleeding episodes and treatments, and to answer questions about how bleeding affects their daily life. Blood samples will be collected to monitor their condition and any potential side effects of the study drug. At the end of the main study, participants will have the option to enter an optional extension phase, where they will receive routine intravenous infusions of the study drug 2 to 3 times per week for 6 months to help prevent future bleeding episodes and complications.

CONDITIONS

Official Title

Eptacog Beta in Glanzmann's (HeT_LFB-Strength-Study_FID531)

Who Can Participate

All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Adult or pediatric persons with inherited Glanzmann thrombasthenia
  • Severe bleeding phenotype
  • Adequate hepatic function
  • Adequate renal function
  • Adults (18 years or older) or caregiver for minors or those with impaired decision-making have provided written informed consent
  • Ability to speak, read, and understand English
Not Eligible

You will not qualify if you...

  • Platelet count less than 100,000 (thrombocytopenia)
  • Acquired Glanzmann thrombasthenia due to autoimmune disease, malignancy, or medication
  • Other inherited or acquired bleeding disorders besides Glanzmann thrombasthenia
  • History of blood clots (venous or arterial) within 2 years before enrollment
  • Active cancer
  • Known or suspected allergy to rabbits, rabbit proteins, recombinant factor VIIa, or any study drug components
  • Use of investigational drugs within 30 days or 5 half-lives before the study
  • Use of aspirin, NSAIDs, herbs, or other platelet inhibitors during the study
  • Use of anticoagulant drugs during the study
  • Any life-threatening disease or condition that may affect safety or study participation
  • Use of systemic immunomodulators at enrollment or planned during the study

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Arthur M. Blank Hospital | Children's Healthcare of Atlanta

Atlanta, Georgia, United States, 30329

Actively Recruiting

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Research Team

K

Karen Zimowski, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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