PF-06741086 Long-term Treatment in Severe Hemophilia

Pfizer

Status and phase: Completed; Phase 2

Conditions: Hemophilia A or B

Treatments: Biological: PF-06741086

Study type: Interventional

Funder types: Industry

Identifiers

NCT03363321

2017-001255-31 (EudraCT Number)

B7841003

Details and patient eligibility

About

This study is designed to evaluate the safety, tolerability and efficacy of long-term treatment with PF-06741086 in subjects with severe hemophilia who participated in the 3-month Phase 1b/2 B7841002 study. Additionally, de novo subjects will be recruited into this study.

Enrollment: 20 patients

Sex: Male

Ages: 12 to 74 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Severe hemophilia A or B (Factor VIII or Factor IX activity ≤ 1%)
• Subjects enrolled as Factor VIII or Factor IX inhibitor patients must have a positive inhibitor test result (above the upper limit of normal) at the local laboratory and must receive a bypass agent as primary treatment for bleeding episodes.
• Episodic (on-demand) treatment regimen prior to screening
• At least 6 acute bleeding episodes during the 6-month period prior to screening

Exclusion criteria

• Known coronary artery, thrombotic, or ischemic disease
• Concomitant treatment with activated prothrombin complex concentrate

Trial design

Primary purpose: Prevention

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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20 participants in 6 patient groups

PF-06741086 (Cohort 1)

Experimental group

Treatment:

Biological: PF-06741086

PF-06741086 (Cohort 2)

Experimental group

Treatment:

Biological: PF-06741086

PF-06741086 (Cohort 3)

Experimental group

Treatment:

Biological: PF-06741086

PF-06741086 (Cohort 4)

Experimental group

Treatment:

Biological: PF-06741086

PF-06741086 (Cohort 5)

Experimental group

Treatment:

Biological: PF-06741086

PF-06741086 (Cohort 6)

Experimental group

Treatment:

Biological: PF-06741086