Safety and Efficacy of FVIIa to Prevent Excessive Bleeding in Hemophilia Patients With Inhibitors Undergoing Major Surgery

NCT05695391 | Terminated Phase 3 Results FDA Drug

• 1 other identifier: F7TG2202
• Study Type: interventional
• Target: 2
• Locations: 6 countries
• Sites: 15

Brief Summary

This is an interventional, prospective, international, multicenter, single-arm, Phase 3, and sequential efficacy and safety study in adolescents and adults with congenital hemophilia A or B with inhibitors to factor VIII (FVIII) or factor IX (FIX) undergoing elective major surgical procedures.

Conditions

Hemophilia

Outcome Measures

Primary Outcomes (1)

• Eptacog Beta (Activated) (EB) Effectiveness Based on Protocol's Aggregate Score

  Successfully treated major procedures defined by a good or excellent global hemostatic response, based on an aggregate score from 3 timepoints (intra-operative, 24h post-operative, 120h post-operative), each individual score depending on comparison between actual and preliminary-communicated expected blood loss and transfusions for a similar patient without bleeding disorder undergoing the same surgical procedure, but also on the use or not of IMP extra-doses or of rescue therapy.

  From surgical wound closure until 120 hours after surgical wound closure

Study Arms (1)

Coagulation Factor VIIa (Recombinant)

EXPERIMENTAL

Biological: Coagulation Factor VIIa (Recombinant)

Interventions

Coagulation Factor VIIa (Recombinant) BIOLOGICAL

Eptacog Beta (activated) (EB)

Coagulation Factor VIIa (Recombinant)

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Each patient must meet the following criteria to be enrolled in this study:
• be male with a diagnosis of congenital hemophilia A or B of any severity
• have one of the following:
• current positive inhibitor test BU ≥5 (as confirmed at screening by the institutional lab) or history of high-responding inhibitors (BU ≥5) not further successfully treated by Immune Tolerance Induction OR
• a condition precluding the use of FVIII or FIX products to treat or prevent bleeding such as a previous anamnestic response after exposure to factor concentrates or a previous failure to respond to FVIII or FIX concentrates
• be ≥12 years to ≤65 years of age on the day of informed consent
• be scheduled for an elective major surgical procedure as defined in the study protocol (see Table ''Definitions for the specific purpose of Study F7TG2202'')
• have Hb ≥ 12 g/dL
• be capable of understanding and willing to comply with the conditions of the protocol OR in the case of a patient under the age of legal majority, parent(s)/legal guardian(s) must be capable of understanding and willing to comply with the conditions of the protocol
• have read, understood, and provided written informed consent (patient or parent(s)/legal guardian(s) if the patient is minor according to local regulation) and, where applicable according to local regulation, patient's assent if the patient is minor

You may not qualify if:

• Patients who meet any of the following criteria will be excluded from the study.
• have any coagulation disorder other than hemophilia A or B
• be immunosuppressed (i.e. the patient should not be receiving systemic immunosuppressive medication; CD4+ cell counts at screening should be \>200/μL)
• known intolerance to EB or any of its excipients
• currently receiving immune tolerance induction (ITI) therapy
• have a known or suspected allergy or hypersensitivity to rabbits or rabbit proteins
• have platelet count \<100,000/μL
• have received an investigational drug within 30 days or within 5 half-lives of that investigational drug, whichever is longer, of the planned first EB administration, or be expected to receive such drug during participation in this study. Patients who have received fitusiran in a clinical study may not participate in this clinical study for 6 months since the last dose and if they have an antithrombin III level not in the normal range at screening.
• for patients using emicizumab, have received during the last 6 months or currently receiving a maintenance dosing regimen of emicizumab different from the indicated one ± 10% of approved dose), i.e. different from 1.5 mg/kg once weekly (±10%), 3 mg/kg (±10%) every two weeks or 6 mg/kg (±10%) every four weeks
• for patients using emicizumab, currently be any plans, or notes in the patient's medical records that would suggest the need to increase or decrease emicizumab dosing due to antidrug antibodies (ADAs), reduced PK, or coagulation/safety-related issues (e.g. lack of response, or potential/actual thromboembolic concerns, etc)
• have a clinically relevant hepatic (aspartate aminotransferase \[AST\] and/or alanine aminotransferase \[ALT\] \>3 times the upper limit of normal \[ULN\]) and/or renal impairment (creatinine \>2 times the ULN)
• have a history of arterial and/or venous thromboembolic events (such as myocardial infarction, ischemic strokes, transient ischemic attacks, DVT, or PE) within 2 years prior to the planned first dose of LR769, uncontrolled arrhythmia, or current New York Heart Association (NYHA) functional classification score of stages II - IV
• have an active malignancy (those with non-melanoma skin cancer are allowed)
• have any life-threatening disease or other disease or condition which, according to the investigator's judgment, could imply a potential hazard to the patient, or interfere with the study participation or study outcome (e.g. chronic, unmanaged hepatitis infection)
• be using aspirin, non-steroidal anti-inflammatory drugs (NSAIDS), herbs, natural medications, or other drugs with platelet inhibitory properties within one week prior to surgery and for the duration of treatment with EB

Sponsors & Collaborators

• Laboratoire français de Fractionnement et de Biotechnologies: lead

Study Sites (15)

Orthopaedic Institute for Children - Orthopaedic Hemophilia Treatment Center

Los Angeles, California, 90007, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

Tulane Univertsity School of Medecine

New Orleans, Louisiana, 70112, United States

Location

M Health Fairview Center for bleeding and Clotting disorders

Minneapolis, Minnesota, 55455, United States

Location

University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

Hospital Queen Elisabeth - Kota Kinabalu

Kota Kinabalu, Sabah Province, 88586, Malaysia

Location

Hospital Ampang

Ampang, Selangor Province, 68000, Malaysia

Location

Hospital Universitario Dr. José Eleuterio González de Nuevo León

Monterrey, Nuevo León, 64460, Mexico

Location

Charlotte Maxeke Johannesburg Academic Hospital

Johannesburg, Gauteng, 2196, South Africa

Location

Chiang Mai University

Chiang Mai, 50200, Thailand

Location

Maharaj Nakorn Chiangmai Hospital, Chiangmai University

Chiang Mai, 50200, Thailand

Location

Acibadem Adana Hospital

Seyhan, Adana, 01130, Turkey (Türkiye)

Location

Hacettepe Üniversitesi Rektörlüğü Sihhiye

Altındağ, Ankara, 06230, Turkey (Türkiye)

Location

Istanbul Üniversitesi Onkoloji Enstitusu

Fatih, Istanbul, 34093, Turkey (Türkiye)

Location

Karadeniz Teknik Üniversitesi

Trabzon, Trabzon, 61080, Turkey (Türkiye)

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor VII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Enzyme Precursors; Enzymes and Coenzymes; Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Limitations and Caveats

The study was prematurely discontinued due to persistent difficulties in recruiting patients.There was no safety issue or concern. As the study was terminated early, no conclusions can be drawn from this study regarding the efficacy of Eptacog Beta (activated).

Results Point of Contact

• Title: Eric CARBONNELLE, PhD
• Organization: LFB Biotechnologies

carbonnellee@lfb.fr

+33 169827010

Study Officials

• Miguel ESCOBAR, MD

  The University of Texas Health Science Center, Houston

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2022
• First Posted: January 25, 2023
• Study Start: June 7, 2024
• Primary Completion: May 28, 2025
• Study Completion: July 7, 2025
• Last Updated: July 31, 2026
• Results First Posted: July 31, 2026

Record last verified: 2026-07

Locations

US (5); ME (1); ZA (1); TH (2); MY (2); TU (4)