NCT03938792

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

An Open-Label Study in Adolescent and Adult Severe (Coagulation Factor Activity <1%) Hemophilia A Participants With or Without Inhibitors or Moderately Severe to Severe Hemophilia B Participants (Coagulation Factor Activity ≤2%) With or Without Inhibitors Comparing Standard Treatment to PF-06741086 Prophylaxis

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 189 (actual)
• Sponsor: Pfizer · Industry
• Sex: Male
• Age: 12 Years – 74 Years
• Healthy volunteers: Not accepted

Summary

Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently than factor replacement products and will work in the presence of inhibitors. The potential for once weekly (QW) subcutaneous (SC) administration provides for treatment options in the absence of reliable vascular access, increased convenience and may enable better compliance. Combined, these qualities should result in a reduction of bleeding episodes.

Conditions

• Hemophilia A
• Hemophilia B

Interventions

Type	Name	Description
DRUG	PF-06741086	300 milligrams(mg) subcutaneous (sc) loading dose followed by 150 mg sq once weekly (qw). 300 mg sc qw is prescribed for participants who meet dose escalation criteria.

Timeline

• Start date: 2020-03-09
• Primary completion: 2025-04-29
• Completion: 2025-04-29
• First posted: 2019-05-06
• Last updated: 2025-06-10

Locations

62 sites across 20 countries: United States, Bulgaria, Canada, China, Croatia, France, Hong Kong, India, Italy, Japan, Mexico, Oman, Russia, Saudi Arabia, Serbia, South Africa, South Korea, Spain, Taiwan, Turkey (Türkiye)

Regulatory

• FDA-regulated drug study