A MULTIPLE DOSE STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF SUBCUTANEOUS AND/OR INTRAVENOUS PF-06741086 IN SUBJECTS WITH SEVERE HEMOPHILIA
- Conditions
- Hemophilia A or BMedDRA version: 20.0 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 100000004850MedDRA version: 20.0 Level: LLT Classification code 10060612 Term: Hemophilia A System Organ Class: 100000004850MedDRA version: 20.0 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX) System Organ Class: 100000004850Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2016-001885-27-HR
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 24
Subjects must meet all of the following inclusion criteria to be eligible for enrollment in the study:
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 of age.
3. Body Mass Index (BMI) = 17.5 and = 30.5 kg/m2 and total body weight =50 and =100 kg.
4. Diagnosis of severe hemophilia A or B (Factor VIII or Factor IX activity = 1%), with no known Factor VIII or Factor IX inhibitors during the 12-month period prior to Screening.
5.Patients with an episodic (on demand) treatment regimen prior to Screening, who are willing and able to washout from Factor VIII (for at least 72 hours) or Factor IX (for at least 96 hours) replacement therapy prior to Screening laboratory assessments of factor activity and have no plans to institute prophylactic factor treatment during the study period.
6. Had at least 6 acute bleeding episodes (spontaneous/traumatic) during the 6-month period prior to Screening. Surgical bleeding episodes do not apply to this criterion.
7. If receiving therapy for human immunodeficiency virus (HIV) or active hepatitis infection, have stable disease and be on a stable regimen at the time of study entry (ie, stable dosing for at least 3 months before consent).
8. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
9. If with partners currently pregnant or if able to father children, agree to use a highly effective method of contraception throughout the study and for least 28 days after the last dose of investigational product.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 24
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
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.
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. Detectable or documented history of inhibitors (= 0.6 Bethesda Units [BU]) against Factor VIII or Factor IX during the 12-month period prior to Screening.
6. Currently receiving treatment for acute bleeding episodes with rFVIIa or activated prothrombin complex concentrate (eg Factor Eight Inhibitor Bypass Agent [FEIBA]).
7. Regular, concomitant therapy with immunomodulating drugs (eg, intravenous immunoglobulin [IVIG], and routine systemic corticosteroids).
8. Abnormal renal or hepatic function as defined by the following laboratory results at Screening:
• Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels > 3 times the upper limit of normal (ULN).
• Total bilirubin level > 2 mg/dL (> 35 umol/L).
• Serum albumin < the lower limit of normal (LLN).
• Serum creatinine level > 1.25 times the ULN.
9. Abnormal hematology values as defined by the following laboratory results at Screening:
• Platelet count < 100,000 /µL.
• Fibrinogen level < LLN.
• Hemoglobin level < 10 gm/dL.
10. Abnormal coagulation activity as defined by the following laboratory results at Screening:
• Prothrombin time (PT) > 1.25 times the ULN.
11. CD4 cell count = 200 /uL.
12. Known hypersensitivity or allergic reaction to hamster protein.
13. Known sensitivity to heparin or heparin-induced thrombocytopenia.
14. Investigational 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 directly involved in the conduct of the study.
15. Participation in other studies involving investigational drug(s) within 30 days (or as determined by the local requirement, whichever is longer) or 5 half-lives prior to study entry and/or during study participation.
16. Other severe acute or chronic medical or psychiatric condition 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.
17. With partners currently pregnant or able to father children 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.
18. Had major surgery, as judged by the investigator, within 3 months prior to the study or have elective surgery planned during the study.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method