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

NCT07136857 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: STUDY000094812025P010364
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

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

Keywords

Severe Bleeding; Prevention of Bleeding; Eptacog beta (EB)

Outcome Measures

Primary Outcomes (2)

• Proportion of bleeding events successfully treated within 24 hours of the first Eptacog Beta (EB) 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.

  24 hours after the first EB dose administration

• Number of bleeding events over the study period

  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.

  6 months following the start of the interventional prophylaxis phase

Secondary Outcomes (15)

• Number of treatment-Emergent Adverse Events and Clinically Significant Laboratory Abnormalities

  6 months following the start of EB administration for each study phase

• EuroQol five-dimensional questionnaire for youth (EQ-5D-Y) score for participants 4-15 years of age.

  Baseline, 9 and 15 months after baseline

• EuroQol five-dimensional 5 Level (EQ-5D-5L) score for participants ≥16 years

  Baseline, 9 and 15 months after baseline

• Patient Reported Outcomes Measurement Information System (PROMIS) fatigue short form for adults

  Baseline, 9 and 15 months after baseline

• PROMIS fatigue short form (pediatric version)

  Baseline, 9 and 15 months after baseline

Study Arms (1)

Eptacog beta

EXPERIMENTAL

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.

Drug: EPTACOG BETA

Interventions

EPTACOG BETA DRUG

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.

Also known as: SEVENTFACT®; EB

Eptacog beta

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Emory University: lead
• Laboratoire Français du Fractionnement et des Biotechnologies (LFB BIOTECHNOLOGIES): collaborator

Study Sites (1)

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

Atlanta, Georgia, 30329, United States

RECRUITING

MeSH Terms

Conditions

Thrombasthenia; Hemorrhage

Interventions

recombinant FVIIa

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Karen Zimowski, MD

  Emory University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Karen Zimowski, MD

CONTACT

404-785-1200; kzimows@emory.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: August 15, 2025
• First Posted: August 22, 2025
• Study Start: October 2, 2025
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: December 1, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)