PF-06741086 for Hemophilia

No longer recruiting at 103 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment, PF-06741086, for individuals with hemophilia A or B. It aims to determine if this weekly injection under the skin can reduce bleeding episodes more effectively than existing treatments. The trial is particularly significant for those who have difficulty with regular vein access. Individuals with severe hemophilia A or moderately severe to severe hemophilia B, who frequently experience bleeding episodes, might be suitable candidates. Participants should not have certain heart conditions or liver issues. As a Phase 3 trial, this treatment represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking therapy.

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.

Is there any evidence suggesting that PF-06741086 is likely to be safe for humans?

Research has shown that PF-06741086, also known as marstacimab, is generally safe for people. In earlier studies, healthy volunteers received single doses and tolerated the treatment well without serious side effects. Further research involving individuals with hemophilia showed similar results. Participants tolerated the treatment well, and no deaths occurred. The safety data from these studies supports further investigation of PF-06741086 as a potential treatment for hemophilia. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for hemophilia?

PF-06741086 is unique because it offers a new approach to treating hemophilia with a potentially more targeted mechanism. Unlike current standard treatments that often rely on regular infusions of clotting factors, PF-06741086 is designed to work differently by targeting specific pathways in the blood that help control bleeding. Researchers are excited about this treatment because it could reduce the frequency of bleeding episodes and provide a longer-lasting effect, potentially improving the quality of life for individuals living with hemophilia.

What evidence suggests that PF-06741086 might be an effective treatment for hemophilia?

Research has shown that PF-06741086, also known as marstacimab, could be beneficial in treating hemophilia A and B. Studies have found it significantly reduces bleeding compared to standard treatments. This medication is a monoclonal antibody that targets a protein to improve blood clotting. Unlike treatments that replace missing factors in the blood, marstacimab remains effective even for patients who have developed resistance to those treatments. It is also convenient, requiring only a once-weekly injection under the skin, which may help patients adhere to their treatment plan. Participants in this trial will receive PF-06741086 after a 6-month observation phase on their current hemophilia regimen. ^{2 3 5 6 7}

Who Is on the Research Team?

Pfizer CT.gov Call Center

Principal Investigator

Pfizer

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Published Research Related to This Trial

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]

Citations

1. ashpublications.org ashpublications.org/blood/article/142/Supplement%201/285/501845/Efficacy-and-Safety-of-the-Anti-Tissue-Factor

Efficacy and Safety of the Anti-Tissue Factor Pathway Inhibitor ... Marstacimab (PF-06741086) is a monoclonal antibody targeted to the tissue factor pathway inhibitor (TFPI) protein to improve hemostasis via the ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT03938792

NCT03938792 | Study of the Efficacy and Safety PF ... Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT03363321

PF-06741086 Long-term Treatment in Severe Hemophilia This study is designed to evaluate the safety, tolerability and efficacy of long-term treatment with PF-06741086 in subjects with severe hemophilia who ...

4. pfizer.com pfizer.com/news/press-release/press-release-detail/pfizer-announces-positive-marstacimab-results-pivotal-phase

Pfizer Announces Positive Marstacimab Results from ... Study demonstrates statistically significant and clinically relevant reduction in annualized bleeding rate compared to prophylaxis and ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12205756/

Marstacimab for the Treatment of Hemophilia A or B - PMC Marstacimab, the first FDA-approved anti-TFPI antibody for prophylaxis in hemophilia A and B without inhibitors, addresses key unmet needs.

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/29908043/

A first-in-human study of the safety, tolerability ... Single doses of PF-06741086 were evaluated in a phase 1 study in healthy volunteers. Data from this study support further investigation of PF-06741086 in ...

7. sciencedirect.com sciencedirect.com/science/article/pii/S1538783622023492

A first‐in‐human study of the safety, tolerability ... Single doses of PF‐06741086 at multiple dose levels were safe and well tolerated in a healthy adult male population.