PF-06741086 Long-term Treatment in Severe Hemophilia

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03363321
• Date of registration: 30/11/2017
• Prospective Registration: Yes
• Primary sponsor: Pfizer
• Public title: PF-06741086 Long-term Treatment in Severe Hemophilia
• Scientific title: A MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS PF-06741086 IN SUBJECTS WITH SEVERE HEMOPHILIA
• Date of first enrolment: May 30, 2018
• Target sample size: 20
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03363321
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Prevention. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Brazil, Chile, Croatia, France, Poland, South Africa, Spain, Switzerland, United States

Contacts

• Name: Pfizer CT.gov Call Center
• Affiliation: Pfizer

Key inclusion & exclusion criteria

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



Age minimum: 12 Years
Age maximum: 74 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Hemophilia A or B

Intervention(s)

Biological: PF-06741086

Primary Outcome(s)

Percentage of subejct with infusion site reactions [Time Frame: Day 1 to Day 393]

Number of subjects with change from baseline in electrocardiogram (ECG) parameters [Time Frame: Day 1 to Day 29]

Percentage of subjects with injection site reactions [Time Frame: Day 1 to Day 393]

Frequency, severity and causal relationship of treatment emergent adverse events (TEAEs) and withdrawals due to TEAEs [Time Frame: Day 1 up to Day 393]

Number of subjects with changes from baseline in vital signs [Time Frame: Day 1 to Day 393]

Percentage of subjects with laboratory abnormalities [Time Frame: Day 1 to Day 393]

Secondary Outcome(s)

Number of treatments for breakthrough bleeding episodes [Time Frame: Day 1 to Day 393]

Number of bleeding episodes [Time Frame: Day 1 to Day 393]

Secondary ID(s)

B7841003

2017-001255-31

Source(s) of Monetary Support

Please refer to primary and secondary sponsors