A Phase 3 Study of the Safety and Efficacy of Coagulation Factor VIIa (Recombinant) for the Prevention of Excessive Bleeding in Patients With Congenital Hemophilia A or B With Inhibitors to Factor VIII or IX Undergoing Elective Major Surgical Procedures SCOPE HIM

NCT ID: NCT05695391

Last Updated: 2025-07-09

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 2 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-06-07
• Study Completion Date: 2025-06-01

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Coagulation Factor VIIa (Recombinant)

Group Type: EXPERIMENTAL

Coagulation Factor VIIa (Recombinant)

Intervention Type: BIOLOGICAL

LR769

Interventions

Coagulation Factor VIIa (Recombinant)

LR769

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

Each patient must meet the following criteria to be enrolled in this study:

1. be male with a diagnosis of congenital hemophilia A or B of any severity

2. have one of the following:

1. 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

2. 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

3. be ≥12 years to ≤65 years of age on the day of informed consent

4. be scheduled for an elective major surgical procedure as defined in the study protocol (see Table ''Definitions for the specific purpose of Study F7TG2202'')

5. have Hb ≥ 12 g/dL

6. 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

7. 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 -

Exclusion Criteria

Patients who meet any of the following criteria will be excluded from the study.

1. have any coagulation disorder other than hemophilia A or B

2. be immunosuppressed (i.e. the patient should not be receiving systemic immunosuppressive medication; CD4+ cell counts at screening should be >200/μL)

3. known intolerance to LR769 or any of its excipients

4. currently receiving immune tolerance induction (ITI) therapy

5. have a known or suspected allergy or hypersensitivity to rabbits or rabbit proteins

6. have platelet count <100,000/μL

7. have received an investigational drug within 30 days or within 5 half-lives of that investigational drug, whichever is longer, of the planned first LR769 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.

8. 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

9. 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)

10. 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)

11. 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

12. have an active malignancy (those with non-melanoma skin cancer are allowed)

13. 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)

14. 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 LR769

15. have active gastric or duodenal ulcer disease

16. have received a FVII- or FVIIa-containing product (either plasma derived or recombinant) within 24 hours prior to administration of LR769

17. have a contraindication to antifibrinolytics

18. 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

19. be administered pharmacologic thromboprophylaxis within 5 half-lives of that medication before surgery or for the duration of treatment with LR769 -

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Laboratoire français de Fractionnement et de Biotechnologies

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Miguel ESCOBAR, MD

Role: PRINCIPAL_INVESTIGATOR

The University of Texas Health Science Center, Houston

Locations

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)

Countries

United States; Malaysia; Mexico; South Africa; Thailand; Turkey (Türkiye)

Other Identifiers

F7TG2202

Identifier Type: -

Identifier Source: org_study_id