Overview

This trial is active, not recruiting.

Condition hemophilia
Treatment coagulation factor viia (recombinant)
Phase phase 3
Sponsor LFB USA, Inc.
Collaborator Laboratoire français de Fractionnement et de Biotechnologies
Start date August 2016
End date May 2017
Trial size 12 participants
Trial identifier NCT02548143, LFB-FVIIa-008-14

Summary

The purpose of this study, PerSept 3, is to evaluate LR769 for the prevention of excessive bleeding and achievement of hemostasis in congenital hemophilia A or B patients who have inhibitors to Factor VIII or Factor IX , are aged 6 months to 75 years, inclusive; and who are undergoing elective surgical or other invasive procedures. Administration of LR769 will be performed just prior to surgery/procedure and will be repeated during and after the surgery/procedure to achieve and maintain adequate hemostasis as determined by the investigator's judgment.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Initial Treatment: Patients who meet all eligibility criteria will receive an initial intravenous (IV) bolus dose of 75 - 200 µg/kg of LR769 (dose must be divisible by 25) before the surgical incision or start of the invasive procedure. The initial dose will be selected by the investigator in conjunction with the surgeon/practitioner. The initial dose of LR769 will be followed by repeated administration of 75 µg/kg of LR769 every 2 hours (±5 minutes) for the first 48 hours after completion of the procedure. The minimum duration of LR769 treatment for major procedures will be 5 days, and for minor procedures 2 days except for certain procedures that may not require this duration of treatment
coagulation factor viia (recombinant)
LR769

Primary Outcomes

Measure
Percentage of surgical or other invasive procedures with a "good" or "excellent" response to LR769 treatment
time frame: 48 (±4) hours after the last administration of LR769

Secondary Outcomes

Measure
Percentages of success as defined as the combination of "good" and "excellent" responses by the investigator or designee
time frame: Immediately post operative to 48(±4) after the last administration of LR769 average time 5 weeks
Percentages of "poor," "moderate," "good," and "excellent" response by the investigator or designee
time frame: Immediately post operative to 48(±4) after the last administration of LR769 average time 5 weeks
Percentages of success as defined as the combination of "good" and "excellent" responses by the surgeon /practitioner
time frame: Intraoperative period
Percentages of "poor," "moderate," "good," and "excellent" response by the surgeon/practitioner
time frame: Intraoperative period
Intraoperative blood loss
time frame: Intraoperative period
Number of events requiring transfusion
time frame: Intraoperative period to 48 (±4) hours after last administration of LR769 average time 5 weeks
Changes in hemoglobin
time frame: Intraoperative period to 48 (±4) hours after last administration of LR769 average time 5 weeks
Amount of LR769 used
time frame: Intraoperative period to last dose of LR769 average time 5 weeks
Number and type of bleeding episodes at the surgical site
time frame: Intraoperative period to 48 (±4) hours after last administration of LR769 average time 5 weeks
Number of surgical interventions/re-explorations for bleeding episodes
time frame: Intraoperative period to 48 (±4) hours after last administration of LR769 average time 5 weeks

Eligibility Criteria

Male participants from 6 months up to 75 years old.

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. a positive inhibitor test Bethesda Unit (BU) ≥5 (as confirmed at screening by the institutional lab), OR 2. a BU <5 but expected to have a high anamnestic response to FVIII or FIX, as demonstrated from the patient's medical history, precluding the use of FVIII or FIX products to treat or prevent bleeding, OR 3. a BU <5 but expected to be refractory to increased dosing of FVIII or FIX, as demonstrated from the patient's medical history, precluding the use of FVIII or FIX products to treat or prevent bleeding episodes 3. be ≥6 months to ≤75 years of age; different age restrictions may apply per local regulation and ethical considerations (enrollment of children <12 years of age will not begin until after review of data from the PERSEPT 2 study by the DMC) 4. be scheduled for an elective surgical or other invasive procedure 5. be capable of understanding and willing to comply with the conditions of the protocol OR in the case of a patient <18 years of age, parent(s)/legal guardian(s) must be capable of understanding and willing to comply with the conditions of the protocol 6. have read, understood, and provided written Informed Consent (patient and/or parent(s)/legal guardian(s) if the patient is <18 years of age) or Assent, if applicable 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 (ie, the patient should not be receiving systemic immunosuppressive medication; cluster of differentiation 4 (CD4) 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 allergy or hypersensitivity to rabbits 6. have a platelet count<100,000/µL 7. have received an investigational drug within 30 days of the planned first LR769 administration , or is expected to receive such drug during participation in this study (with the exception of patients who are or were participating in another LR769 study, eg, a study assessing the treatment of bleeding episodes with LR769) 8. 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) 9. have a history of arterial and/or venous thromboembolic events (such as myocardial infarction, ischemic strokes, transient ischemic attacks, deep venous thrombosis (DVT) or pulmonary embolism (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 10. have an active malignancy (those with non-melanoma skin cancer are allowed) 11. 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 trial participation or trial outcome (eg, a history of non-responsiveness to bypassing products)

Additional Information

Official title A Phase 3 Study of the Safety and Efficacy of Coagulation Factor VIIa (Recombinant) for the Prevention of Excessive Bleeding in Congenital Hemophilia A or B Patients With Inhibitors to Factor VIII or IX Undergoing Elective Surgery or Other Invasive Procedures (PERSEPT 3)
Principal investigator Miguel Escobar, MD
Description This study is an international, multicenter, single-arm, Phase 3 study. Patients aged 6 months to 75 years, inclusive, who have congenital hemophilia A or B with inhibitors to Factor VIII or Factor IX and who are scheduled for an elective surgical or other invasive procedure will be enrolled. Both major and minor surgical or other invasive procedures are allowed in the study. Initial Treatment: Regardless of procedure, patients who meet all eligibility criteria will receive an initial intravenous (IV) bolus dose of 75 - 200 µg/kg of LR769 administered within ≤2 minutes before the surgical incision or start of the invasive procedure. The initial dose will be selected by the investigator in conjunction with the surgeon/practitioner based on the complexity of the procedure, the number of tissue planes to be traversed, the patient's previous responses to bypassing agents, and other factors deemed relevant by the investigator. Administration will be repeated no more frequently than every 2 hours (±5 minutes) during and after surgery or invasive procedure. Treatment for Major Surgical Procedures: The initial dose of LR769 will be followed by repeated administration of 75 µg/kg of LR769 every 2 hours (±5 minutes) for the first 48 hours after completion of the procedure. The minimum duration of LR769 treatment for major procedures will be 5 days and dosing frequency will be followed according to the guidelines specified in the protocol. Treatment for Minor Surgical or Other Invasive Procedures: The initial dose of LR769 will be followed by repeated administration of 75 µg/kg of LR769 every 2 hours (±5 minutes) for the first 48 hours. The minimum duration of LR769 infusion for minor procedures will be 2 days, except for certain procedures that may not require this duration of treatment to achieve hemostasis as noted below.
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by LFB USA, Inc..