NCT07136857 · Emory University
Eptacog Beta in Glanzmann's (HeT_LFB-Strength-Study_FID531)
(STRENGTH)
What this study is about
This study is evaluating an experimental 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.
View original scientific description
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.
Interventions
DRUG
EPTACOG BETA
Eptacog beta (SEVENFACT®; EB) is a lyophilized powder in single-use vials (1, 2, or 5 mg) of coagulation factor VIIa (recombinant)-jncw. It is reconstituted with sterile water (provided in the kit) and administered intravenously. SEVENFACT® may be administered by a healthcare provider (HCP) at the study site or home, or by a trained participant/caregiver. Participants will be monitored in a healthcare facility for 60 minutes after the first dose for hypersensitivity.
Primary outcome measures
Proportion of bleeding events successfully treated within 24 hours of the first Eptacog Beta (EB) administration
Time frame: 24 hours after the first EB dose administration
Proportion of acute bleeding events successfully treated with EB within 24 hours of the first EB administration based on an "excellent or good" response on a 4-point hemostatic efficacy scale and without rebleeding before 36 hours. Hemostatic Efficacy Scale: None = No effect; bleeding unchanged or worsened; continued treatment needed. Moderate = Some effect (e.g., pain decreased, bleeding signs improved) but bleeding continued; further treatment needed. Good = Bleeding symptoms are largely reduced but not completely resolved; no further EB infusions are needed. Excellent = Full relief of bleeding symptoms; no additional EB infusion required.
Number of bleeding events over the study period
Time frame: 6 months following the start of the interventional prophylaxis phase
The number of bleeding events occurring while receiving routine EB prophylaxis will be compared with the number of bleeding events that occurred during on-demand therapy. \*This outcome applies only to the optional interventional prophylaxis phase.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
- Atlanta, Georgia
Collaborators
Laboratoire français de Fractionnement et de Biotechnologies
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Dec 1, 2025 · Source of record for eligibility and locations