Severe Hemophilia A or B, with or without inhibitors to Factor VIII (FVIII) or Factor IX (FIX). MedDRA version: 20.0 Level: LLT Classification code 10053751 Term: Hemophilia A with anti factor VIII System Organ Class: 100000004850 MedDRA version: 20.1 Level: LLT Classification code 10053754 Term: Hemophilia B without inhibitors System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10053753 Term: Hemophilia A without inhibitors System Organ Class: 100000004850 M
Conditions
Interventions
Product Name: PF-06741086 100 mg/ml
Product Code: PF-06741086
Pharmaceutical Form: Solution for injection
INN or Proposed INN: PF-06741086
Other descriptive name: PF-06741086
Concentration unit: mg
Sponsors
Pfizer Inc., 235 East 42nd Street, New York, NY 10017
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: All subjects must meet the following inclusion criteria to be eligible for enrollment into the study. Subjects continuing from B7841002 within 30 days of the B7841003 Day 1 visit do not require screening procedures to confirm eligibility: 1.Evidence of a personally signed and dated informed consent document indicating that the subject (or a legally acceptable representative/parent(s)/legal guardian) has been informed of all pertinent aspects of the study. 2.Males =18 and =65 years) yes F.1.3.1 Number of subjects for this age range 3
Exclusion criteria
Exclusion criteria: Subjects with any of the following characteristics/conditions will not be included in the study: 1.Females. 2.Known coronary artery, thrombotic, or ischemic disease, or known severe, uncontrolled hypercholesterolemia. 3.Known hemostatic defect other than hemophilia A or B. 4.ATIII, Protein C, or Protein S deficiency, Factor V Leiden, Prothrombin 20210 mutation, or other known pro thrombotic condition. 5.Regular, concomitant therapy with immunomodulating drugs (eg, intravenous immunoglobulin [IVIG], and routine systemic corticosteroids). 6.Currently receiving treatment for acute bleeding episodes with APCC (eg, Factor Eight Inhibitor Bypass Agent [FEIBA]) and cannot substitute treatment with rFVIIa at a dose level of approximately 90 µg/kg for the duration of the study. 7.Abnormal renal or hepatic function as defined by the following laboratory results at any time prior to Day 1: •Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels >3 times the upper limit of normal (ULN). •Total bilirubin level >2 mg/dL (>35 µmol/L). •Serum albumin 1.25 times the ULN. 8.Abnormal hematology values as defined by the following laboratory results at any time prior to Day 1: •Platelet count 1.25 times the ULN. 10.CD4 cell count =200/µL. 11.Known hypersensitivity (eg. latex allergy) or allergic reaction to hamster protein. 12.Known sensitivity to heparin or heparin induced thrombocytopenia. 13.Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or subjects who are Pfizer employees, including their family members, directly involved in the conduct of the study. 14.Participation in other studies involving investigational drug(s) (excluding PF 06741086) within 30 days prior to study entry and/or during study participation. 15.Other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal ideation or behavior or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study. 16.Fertile male subjects who are unwilling or unable to use a highly effective method of contraception as outlined in this protocol for the duration of the study and for at least 28 days after the last dose of investigational product. 17.Had major surgery, as judged by the investigator, within 3 months prior to the study or have elective surgery planned during the study (applies only to de novo subjects).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: •To determine the safety and tolerability of long term treatment (up to 365 days) with PF 06741086 in severe hemophilia A and B subjects with or without inhibitors to FVIII or FIX.;Secondary Objective: •To determine the efficacy of long term treatment with PF 06741086 in severe hemophilia A and B subjects with or without inhibitors to FVIII or FIX. ;Primary end point(s): •Frequency, severity and causal relationship of treatment emergent adverse events (TEAEs); Day 1 up to Day 393. •Frequency and magnitude of abnormal laboratory findings (including hematology, chemistry, urinalysis); Day 1 up to Day 393. •Changes from baseline in vital sign (blood pressure, pulse rate, temperature and respiration rate) measurements and physical examinations; Day 1 up to Day 393 and ECG Day 1 to Day 29 in de novo subjects only. •Frequency, severity and casual relationship of infusion and injection site reactions; Day 1 up to Day 393.;Timepoint(s) of evaluation of this end point: Timepoints are all listed in the list of primary endpoints. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •Frequency and annualized rate of bleeding episodes; Day 1 up to Day 393. •Frequency of rescue (FVIII, or FIX) therapy for treatment of breakthrough bleeding episodes. ;Timepoint(s) of evaluation of this end point: Timepoints are all listed in the list of secondary endpoints. | — |
Countries
Brazil, Bulgaria, Chile, Croatia, France, Poland, South Africa, Spain, Switzerland, United States
Contacts
Public ContactClinical Trials.gov Call center
Pfizer Inc.
Outcome results
None listed