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

Stopped early · Phase 3

Conditions studied: Hemophilia

In brief

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.

Key facts

Study ID: NCT05695391
Run by: Laboratoire français de Fractionnement et de Biotechnologies
People needed: 2
Starts: 2024-06-07
Expected to finish: 2025-07-07
Last updated by the study team: 2026-07-31

Who can join

Age: 12 and older, up to 65. Sex: male. Healthy volunteers: not accepted.

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
have active gastric or duodenal ulcer disease
have received a FVII- or FVIIa-containing product (either plasma derived or recombinant) within 24 hours prior to administration of EB
have a contraindication to antifibrinolytics
have planned combined major surgeries at the same time or have already been enrolled and treated for a previous elective major surgery in the same SCOPE HIM study
be administered pharmacologic thromboprophylaxis within 5 half-lives of that medication before surgery or for the duration of treatment with EB -

Where it is running

Orthopaedic Institute for Children - Orthopaedic Hemophilia Treatment Center — Los Angeles, California, United States
Children's Healthcare of Atlanta — Atlanta, Georgia, United States
Tulane Univertsity School of Medecine — New Orleans, Louisiana, United States
M Health Fairview Center for bleeding and Clotting disorders — Minneapolis, Minnesota, United States
University of Texas Health Science Center at Houston — Houston, Texas, United States
Hospital Queen Elisabeth - Kota Kinabalu — Kota Kinabalu, Sabah Province, Malaysia
Hospital Ampang — Ampang, Selangor Province, Malaysia
Hospital Universitario Dr. José Eleuterio González de Nuevo León — Monterrey, Nuevo León, Mexico
Charlotte Maxeke Johannesburg Academic Hospital — Johannesburg, Gauteng, South Africa
Chiang Mai University — Chiang Mai, Thailand
Maharaj Nakorn Chiangmai Hospital, Chiangmai University — Chiang Mai, Thailand
Acibadem Adana Hospital — Seyhan, Adana, Turkey (Türkiye)
Hacettepe Üniversitesi Rektörlüğü Sihhiye — Altındağ, Ankara, Turkey (Türkiye)
Istanbul Üniversitesi Onkoloji Enstitusu — Fatih, Istanbul, Turkey (Türkiye)
Karadeniz Teknik Üniversitesi — Trabzon, Trabzon, Turkey (Türkiye)

Full record on ClinicalTrials.gov

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