Actively Recruiting
Study To Assess Response to Eptacog Beta in Patients With Glanzmann Thromboasthenia (STRENGTH)
Led by Emory University · Updated on 2025-12-01
6
Participants Needed
1
Research Sites
N/A
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
Researchers are evaluating eptacog beta (EB), an investigational drug not yet approved by the FDA, for treating and preventing acute bleeding episodes in people with Glanzmann Thrombasthenia, a rare inherited bleeding disorder. This Phase 2 study includes participants with severe bleeding symptoms and aims to assess EB's effectiveness and safety in managing serious bleeding events and preventing future bleeds during an optional extension phase. The study begins with a 3-month non-interventional phase where participants use their standard treatments for bleeding events while recording these episodes. Afterward, for 6 months, participants will use eptacog beta on-demand to treat breakthrough bleeding, with doses given intravenously at 75 mcg/kg every 3 hours as needed. Participants may then opt into a 6-month extension phase where EB is given routinely 2 to 3 times per week to prevent bleeding complications. Participants will visit their hematologist about 4 to 5 times over the 9-month study and keep a diary logging bleeding events and treatments. They will complete questionnaires on how bleeding affects their daily life. Blood samples will be collected to monitor their condition and potential side effects. The study will evaluate how effectively EB controls bleeding within 24 hours and compare bleeding rates between the on-demand and prophylaxis phases.
CONDITIONS
Brief Title
Eptacog Beta in Glanzmann's (HeT_LFB-Strength-Study_FID531)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adult or pediatric persons diagnosed with inherited Glanzmann thrombasthenia
- Severe bleeding phenotype defined by multiple treated bleeding events or hospitalizations
- Adequate liver (hepatic) function
- Adequate kidney (renal) function
- Adults (18 years or older) or caregivers for minors provide informed consent and participants provide assent if applicable
- Ability to speak, read, and understand English
You will not qualify if you...
- Platelet count below 100,000 (thrombocytopenia)
- Acquired Glanzmann thrombasthenia due to autoimmune disease, cancer, or medication
- Other inherited or acquired bleeding disorders besides Glanzmann thrombasthenia
- History of venous or arterial blood clots within 2 years prior to enrollment
- Active cancer
- Known or suspected allergy to rabbits, rabbit proteins, related drugs, or any component of eptacog beta
- Recent or planned use of investigational drugs
- Use of aspirin, NSAIDs, herbs, natural medications, or platelet inhibitors during the study
- Use of anticoagulant medications during the study
- Life-threatening diseases or other conditions that might interfere with 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
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 3 months
Participants receive standard-of-care on-demand therapy for acute bleeding events and complete a diary logging these events and their management.
Participants are monitored as per standard care with diary entries for bleeding events
Duration - 6 months
Participants use eptacog beta on-demand to treat acute bleeding episodes, logging bleeding events and management while assessing hemostatic efficacy.
Participants receive infusions as needed and complete assessments at varying time points following dosing
Duration - 6 months
Participants may continue eptacog beta prophylaxis with routine infusions to prevent bleeding events, with bleeding events monitored over time.
Infusions administered routinely with ongoing monitoring of bleeding events
Trial Site Locations
Total: 1 location
1
Arthur M. Blank Hospital | Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30329
Actively Recruiting
Research Team
K
Karen Zimowski, MD
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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