Recruiting

Phase 2

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

Sponsor:

Emory University

Code:

NCT07136857

Conditions

Glanzmann Thrombasthenia

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Interventions

EPTACOG BETA

Study Details

Brief summary:

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

Glanzmann Thrombasthenia

Study ID

NCT07136857

Start date

Oct 2, 2025

Status verified date

Nov, 2025

Completion date

Dec, 2026

Anticipated

Primary completion date

Dec, 2026

Anticipated

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

6 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Eptacog beta

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.

Interventions

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 measure

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 ]
Number of bleeding events over the study period [ Time Frame: 6 months following the start of the interventional prophylaxis phase ]

Central Contacts and Locations

Central contacts

Karen Zimowski, MD

404-785-1200 kzimows@emory.edu

Locations

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

Recruiting

Atlanta, Georgia, United States, 30329

More Information

Sponsor

Emory University

Last update posted

Dec 1, 2025

Last verified

Nov, 2025

Keywords

Severe Bleeding
Prevention of Bleeding
Eptacog beta (EB)

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Emory University on 2025-12-01.