TERMINATED

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

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2024-06-07

Study Completion:2025-06-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:TERMINATED

Study ID

NCT05695391

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:12 Years to 65 Years

Sexes Eligible for Study:MALE

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
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 -	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 -

Inclusion Criteria

Exclusion Criteria

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 -

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 -

Contacts and Locations

Principal Investigator

Miguel ESCOBAR, MD

PRINCIPAL_INVESTIGATOR

The University of Texas Health Science Center, Houston

Study Locations (Sites)

Orthopaedic Institute for Children - Orthopaedic Hemophilia Treatment Center

Los Angeles, California, 90007

United States

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322

United States

Tulane Univertsity School of Medecine

New Orleans, Louisiana, 70112

United States

M Health Fairview Center for bleeding and Clotting disorders

Minneapolis, Minnesota, 55455

United States

University of Texas Health Science Center at Houston

Houston, Texas, 77030

United States

Collaborators and Investigators

Sponsor: Laboratoire français de Fractionnement et de Biotechnologies

Miguel ESCOBAR, MD, PRINCIPAL_INVESTIGATOR, The University of Texas Health Science Center, Houston

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-06-07

Study Completion Date2025-06-01

Study Record Updates

Study Start Date2024-06-07

Study Completion Date2025-06-01

Terms related to this study

Additional Relevant MeSH Terms

Hemophilia