189 Participants Needed

PF-06741086 for Hemophilia

Recruiting at 84 trial locations
PC
Overseen ByPfizer CT.gov Call Center
Age: Any Age
Sex: Male
Trial Phase: Phase 3
Sponsor: Pfizer
Must be taking: FVIII/FIX replacement
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop taking your current medications. However, it mentions that participants should not be on certain therapies like regular immunomodulatory drugs or prophylaxis with bypassing agents during the study. It's best to discuss your specific medications with the study team.

How is the drug PF-06741086 different from other hemophilia treatments?

PF-06741086 is unique because it is a monoclonal antibody that targets the tissue factor pathway inhibitor (TFPI), which is different from traditional treatments that replace missing clotting factors. This approach helps initiate clotting through the extrinsic pathway, offering a novel mechanism for managing hemophilia.12345

What is the purpose of this trial?

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.

Research Team

PC

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Eligibility Criteria

Adults and teens with severe Hemophilia A or moderately to severely severe Hemophilia B, weighing at least 35 kg. They must have a history of compliance with their current treatment regimen, no inhibitors for non-inhibitor cohort, or specific inhibitor criteria for the inhibitor cohort. Exclusions include planned surgeries, other hemostatic defects, abnormal organ function, certain drug therapies including gene therapy for hemophilia.

Inclusion Criteria

Participants who are enrolled into the Non-Inhibitor Cohort must also meet the following criteria:
I, or someone legally allowed to make decisions for me, can sign the consent form.
I've needed infusions for bleeding 6+ times in the last 6 months and will continue treatment.
See 8 more

Exclusion Criteria

My liver condition is stable.
I am using or plan to use immune tolerance therapy or clotting factor replacements during the study.
Screening ECG that demonstrates clinically relevant abnormalities that may affect participant safety or interpretation of study results.
See 13 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Observation

Participants continue their current hemophilia regimen to establish a baseline

6 months

Treatment

Participants receive PF-06741086 once weekly subcutaneous administration

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • PF-06741086
Trial Overview PF-06741086 is being tested as a potential new treatment option for Hemophilia A and B. It's given once weekly through subcutaneous injection and may offer benefits over existing treatments by working differently than factor replacement products and being effective even in the presence of inhibitors.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: PF-06741086Experimental Treatment1 Intervention
Participants will be assigned to treatment with PF-06741086 after a 6 month Observation Phase on their current hemophilia regimen.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Pfizer

Lead Sponsor

Trials
4,712
Recruited
50,980,000+
Known For
Vaccine Innovations
Top Products
Viagra, Zoloft, Lipitor, Prevnar 13

Albert Bourla

Pfizer

Chief Executive Officer since 2019

PhD in Biotechnology of Reproduction, Aristotle University of Thessaloniki

Patrizia Cavazzoni profile image

Patrizia Cavazzoni

Pfizer

Chief Medical Officer

MD from McGill University

Findings from Research

Advancements in hemophilia treatment over the past 50 years have led to the availability of safe and effective factors VIII and IX, as well as integrated care models, but challenges remain in completely preventing joint bleeding and deterioration.
New recombinant clotting factors with extended half-lives may enhance treatment adherence and improve clinical outcomes by allowing for more convenient infusion schedules and better management of individual patient needs.
New therapies for hemophilia.Pipe, SW.[2018]
BAY 94-9027 (damoctocog alfa pegol) demonstrated long-term safety in previously treated pediatric patients with severe hemophilia A, with no development of FVIII inhibitors or anti-PEG antibodies over a median treatment duration of 5 years.
The treatment significantly reduced the annualized bleeding rate (ABR), with a median ABR of 1.5 for patients under 6 years and 1.9 for those aged 6-12 years, and a spontaneous ABR of 0.0 in the last year of treatment, indicating effective bleed prevention.
PROTECT VIII kids extension study: Long-term safety and efficacy of BAY 94-9027 (damoctocog alfa pegol) in children with severe haemophilia A.Mancuso, ME., Biss, T., Fischer, K., et al.[2021]

References

Aptamer ARC19499 mediates a procoagulant hemostatic effect by inhibiting tissue factor pathway inhibitor. [2021]
Integrated analysis of safety data from 12 clinical interventional studies of plasma- and albumin-free recombinant factor VIII (rAHF-PFM) in haemophilia A. [2015]
A first-in-human study of the safety, tolerability, pharmacokinetics and pharmacodynamics of PF-06741086, an anti-tissue factor pathway inhibitor mAb, in healthy volunteers. [2023]
New therapies for hemophilia. [2018]
PROTECT VIII kids extension study: Long-term safety and efficacy of BAY 94-9027 (damoctocog alfa pegol) in children with severe haemophilia A. [2021]
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