PF-06741086 for Hemophilia

Recruiting at 84 trial locations

Overseen By Pfizer CT.gov Call Center

Age: Any Age

Sex: Male

Trial Phase: Phase 3

Sponsor: Pfizer

Must be taking: FVIII/FIX replacement

Must not be taking: Immunomodulatory drugs

Disqualifiers: Coronary artery disease, Thrombosis, others

No Placebo Group All 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 Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

189 Participants Needed

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. ^{1 2 3 4 5}

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

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.

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.

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

Group I: PF-06741086 Experimental Treatment 1 Intervention

Participants will be assigned to treatment with PF-06741086 after a 6 month Observation Phase on their current hemophilia regimen.

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

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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

1. United States pubmed.ncbi.nlm.nih.gov

Aptamer ARC19499 mediates a procoagulant hemostatic effect by inhibiting tissue factor pathway inhibitor. [2021]

2. England pubmed.ncbi.nlm.nih.gov

Integrated analysis of safety data from 12 clinical interventional studies of plasma- and albumin-free recombinant factor VIII (rAHF-PFM) in haemophilia A. [2015]

3. England pubmed.ncbi.nlm.nih.gov

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]

4. United States pubmed.ncbi.nlm.nih.gov

New therapies for hemophilia. [2018]

5. England pubmed.ncbi.nlm.nih.gov

PROTECT VIII kids extension study: Long-term safety and efficacy of BAY 94-9027 (damoctocog alfa pegol) in children with severe haemophilia A. [2021]