Eptacog Beta for Glanzmann's Thrombasthenia

(STRENGTH Trial)

Overseen By Karen Zimowski, MD

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Emory University

Must not be taking: Aspirin, NSAIDs, Anticoagulants, others

Disqualifiers: Thrombocytopenia, Active malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 4

6 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new drug, eptacog beta, to treat and prevent severe bleeding in individuals with Glanzmann Thrombasthenia, a rare bleeding disorder. The study aims to evaluate the drug's effectiveness and safety compared to usual treatments like platelet transfusions. Participants will use their current treatments for the first three months, then switch to eptacog beta for six months to assess its impact on bleeding control. Individuals with Glanzmann Thrombasthenia who frequently experience severe bleeding and can manage daily tasks in English may be suitable candidates. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group.

Do I have to stop taking my current medications for the trial?

Yes, you will need to stop using aspirin, NSAIDs, herbs, natural medications, or other drugs that affect platelets, as well as anticoagulant agents, for the duration of the study.

Is there any evidence suggesting that eptacog beta is likely to be safe for humans?

Research has shown that eptacog beta (EB) is generally safe for people with bleeding disorders. In studies, patients who took eptacog beta did not encounter any unexpected safety issues. Most participants found the treatment safe, and it was well-tolerated at various doses.

Eptacog beta did not cause any serious side effects, suggesting it could be a safer option for managing bleeding episodes in conditions like Glanzmann's Thrombasthenia. However, since the FDA has not yet approved eptacog beta for this specific condition, ongoing trials are crucial to confirm its safety and effectiveness. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Glanzmann's Thrombasthenia?

Most treatments for Glanzmann's Thrombasthenia, like platelet transfusions and recombinant activated factor VII, aim to manage bleeding episodes as they occur. However, Eptacog Beta stands out due to its potential for both on-demand use and routine administration for bleed prophylaxis. Researchers are excited because Eptacog Beta works by specifically enhancing the clotting process using a recombinant factor that mimics the natural clotting mechanism more closely. This innovative approach could mean fewer bleeding events and improved quality of life for patients.

What evidence suggests that eptacog beta might be an effective treatment for Glanzmann's Thrombasthenia?

Research shows that eptacog beta may help treat bleeding in people with Glanzmann's Thrombasthenia, a rare bleeding disorder. Studies have found that similar treatments using recombinant activated factor VII, a protein that aids blood clotting, were 100% effective for moderate bleeds and about 82% effective for severe ones. This suggests that eptacog beta might also effectively control bleeding episodes for this condition, although the FDA has not yet approved it for this use. In this trial, participants will initially receive standard-of-care on-demand therapy for acute bleeding. They will then use eptacog beta on-demand to treat acute bleeding. The trial aims to confirm these early results and learn more about how well eptacog beta can prevent and manage bleeding episodes. ^{1 2 4 6 7}

Who Is on the Research Team?

Karen Zimowski, MD

Principal Investigator

Emory University

Are You a Good Fit for This Trial?

This trial is for individuals with Glanzmann Thrombasthenia, a rare bleeding disorder. Participants will initially manage bleeds with standard treatments and then switch to the study drug, eptacog beta (EB), for serious bleeding events. They must be willing to visit their hematologist 4-5 times over 9 months and keep a diary of bleeds.

Inclusion Criteria

My kidneys are working well.

I have Glanzmann thrombasthenia.

Severe bleeding phenotype

Exclusion Criteria

Thrombocytopenia (platelet count < 100k)

I am taking blood thinners for the study period.

I have had a blood clot in the last 2 years.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Non-interventional Phase

Participants receive standard-of-care on-demand therapy for acute bleeding events

3 months

Approximately 1-2 visits with hematologist

Interventional On-demand Phase

Participants use eptacog beta to treat serious bleeding events

6 months

Approximately 3 visits with hematologist

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Optional Extension Phase

Participants receive routine intravenous infusions of the study drug for bleed prophylaxis

6 months

What Are the Treatments Tested in This Trial?

Interventions

• Eptacog Beta

Trial Overview Eptacog beta (EB) is being tested for treating and preventing acute bleeding episodes in Glanzmann Thrombasthenia patients. The trial includes an initial observation period followed by treatment with EB, plus an optional phase for routine prevention using EB infusions.

How Is the Trial Designed?

Group I: Eptacog beta Experimental Treatment 1 Intervention

For the initial 3 months, participants will be in a non-interventional phase and receive standard-of-care on-demand therapy for acute bleeding at their hematologist's discretion. Following this non-interventional phase, they will use eptacog beta on-demand to treat acute bleeding for 6 months. Throughout the study, they will log bleeding events and management in a diary. Hemostatic efficacy after treatment will be assessed using a 4-point hemostasis efficacy scale. Participants will have the option to re-consent for an exploratory phase, adding 6 months to their time on the study (total of 15 months). For those entering the optional extension phase, eptacog beta will be routinely administered for bleed prophylaxis. The treating hematologist will determine the best method/location for infusions. The number of bleeding events over time will be compared between the prophylaxis phase and the on-demand phase.

Who Is Running the Clinical Trial?

Emory University

Lead Sponsor

Trials

1,735

Recruited

2,605,000+

Laboratoire Français du Fractionnement et des Biotechnologies (LFB BIOTECHNOLOGIES)

Collaborator

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07136857

Eptacog Beta in Glanzmann's (HeT_LFB-Strength- ... This study is evaluating an investigational drug, eptacog beta (EB), for the treatment and prevention of acute bleeding episodes in people with Glanzmann ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11780185/

Efficacy and safety of recombinant activated factor VII in ... Overall treatment efficacy was 100.0% for moderate bleeds whatever the strategy and 81.6% for severe bleeds, rFVIIa + AF or rFVIIa + PLT ± AF ...

3. withpower.com withpower.com/trial/phase-2-thrombasthenia-7-2025-62eeb

Eptacog Beta for Glanzmann's Thrombasthenia This study is evaluating an investigational drug, eptacog beta (EB), for the treatment and prevention of acute bleeding episodes in people with Glanzmann ...

4. link.springer.com link.springer.com/article/10.1007/s00277-023-05287-2

Current status and future prospects of activated ... The rate of success in six patients who underwent minor procedures was 100% and that in six patients who underwent major procedures was 66.7%; ...

5. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/full/10.1002/ajh.27602

2025 Clinical Trials Update on Hemophilia, VWD, and ... Hypersensitivity and loss of efficacy within first 4 exposure days, Bayer. Eptacog beta (Sevenfact). Recombinant FVIIa. Phase 3, NCT05695391 ...

6. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0268960X15300060

Safety update on the use of recombinant activated factor VII ... Overall, rFVIIa has shown a consistently favourable safety profile, with no unexpected safety concerns, in all approved indications. No confirmed cases of ...

7. researchgate.net researchgate.net/publication/320245704_Safety_and_dose-dependency_of_eptacog_beta_activated_in_a_dose_escalation_study_of_non-bleeding_congenital_haemophilia_A_or_B_patients_with_or_without_inhibitors

Safety and dose‐dependency of eptacog beta (activated) ... Results: Administration of rhFVIIa at all doses was well tolerated. Pharmacokinetic analyses showed that peak FVIIa plasma levels (Cmax ) were approximately ...