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Monoclonal Antibodies

Marstacimab for Hemophilia

Phase 3

Recruiting

Research Sponsored by Pfizer

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

All participants will have a minimum body weight of 35 kg

Must not have

Ongoing or planned use of immune tolerance induction or prophylaxis with FVIII or FIX replacement during the study

Previous or current treatment for or history of coronary artery disease, venous or arterial thrombosis (CTCAE Grade >3), or ischemic disease (except catherter-associated thrombosis)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline up to 7 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is an extension of a previous study to assess the long-term safety and efficacy of a prophylactic treatment for hemophilia A and B. Approximately 145 participants will receive injections of the marstacimab drug once weekly for the duration of the study.

Who is the study for?

This trial is for adolescents and adults aged 12 to <75 with severe hemophilia A or B, weighing at least 35 kg, who completed a prior study without early termination. It's not for those planning surgery, with unstable HIV or liver function, abnormal kidney function, using certain immune treatments, or having a history of significant heart or blood vessel problems.

What is being tested?

The trial tests marstacimab (PF-06741086), given as a weekly subcutaneous injection to prevent bleeding in hemophilia patients. Participants from an earlier phase will continue treatment to assess long-term safety and effectiveness. An optional substudy uses a pre-filled pen for administration.

What are the potential side effects?

While specific side effects are not listed here, common risks may include irritation at the injection site, potential allergic reactions to the drug components, and general discomfort associated with subcutaneous injections.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I weigh at least 35 kg.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently using or plan to use immune tolerance therapy for my condition.

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I have had serious heart or blood vessel problems.

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I have a surgery scheduled during the study period.

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My kidney function is significantly reduced.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline up to 7 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline up to 7 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to 7 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Annualized rate of bleeding episodes

Change in joints measured by the HJHS

Changes in EQ-5D questionnaire data

+1 more

Side effects data

From 2020 Phase 2 trial • 20 Patients • NCT03363321

40%

Haemarthrosis

20%

Tonsillitis

20%

Disorientation

20%

Contusion

20%

Arthralgia

20%

Skull fracture

20%

Inflammation

20%

Cerebral haemorrhage

20%

Generalised tonic-clonic seizure

20%

Injection site haematoma

20%

Injection site reaction

100%

80%

60%

40%

20%

Study treatment Arm

Cohort 1: 300mg - 300mg Non-Inhibitor

Cohort 2: 300mg Loading + 150mg - 300mg Loading + 150mg Non-Inhibitor

Cohort 4: 300mg - 300mg Inhibitor

Cohort 3: 450mg - 300mg Loading + 150mg Non-Inhibitor

Cohort 5: De Novo 300mg Loading + 150mg Inhibitors

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: PF-06741086Experimental Treatment1 Intervention

For participants aged ≥12 years 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. For participants aged ≥6 to \<12 years is marstacimab 150 mg SC for initial loading dose followed by 75 mg SC QW. 150 mg sc qw is prescribed for participants who meet dose escalation criteria.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

PF-06741086

2021

Completed Phase 2

~100

0 / 5Eligibility criteria met