Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors

Condition(s) studied

Investigational drug(s) / intervention(s)

PF-06741086: 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.

Study summary

Study B7841007 is an open-label extension study to assess the long-term safety, tolerability, and efficacy of prophylaxis treatment with marstacimab in participants who did not require "Early Termination" from the Phase 3 Study B7841005 and from the Phase 3 Study B7841008.

Study B7841005: approximately 145 adolescent and adult participants 12 to \<75 years of age with severe hemophilia A or moderately severe to severe hemophilia B (defined as FVIII activity \<1% or FIX activity ≤2%, respectively) with or without inhibitors are expected to be enrolled in Study B7841005 during which they will receive prophylaxis (defined as treatment by SC injection of marstacimab).

Study B7841008: this is an ongoing Phase 3, open-label study in pediatric participants \<18 years of age with severe hemophilia A (FVIII Coagulation Factor Activity \<1%) or moderately severe to severe hemophilia B (FIX Coagulation Factor Activity ≤2%). A sequential approach will be used in enrolling at least 100 pediatric participants, at least 20 of which will be aged ≥12 to \<18 years and at least 80 participants will be aged ≥1 to \<12 years. At the start of study B7841008, the dosing and data available in adolescent and adult participants in Study B7841005 supported the initiation of B7841008 study in participants aged ≥12 to \<18 years. Subsequently, additional safety and efficacy data from adolescent participants in Study B7841005 became available for benefit/risk assessment in support of dosing participants aged ≥6 to \<12 years. Based on the positive benefit/risk assessment conducted by both internal Pfizer review and eDMC review, dosing of the ≥6 to \<12 years age group was initiated in June 2023 in B7841008 Study. Data from participants ≥6 years from B7841008 Study and Study B7841005 will support the dosing of participants aged ≥1 to \<6 years.

All participants will be provided the prefilled pen (PFP) for administration of marstacimab in the study. Use of the prefilled syringe (PFS) will be permitted at the investigator's discretion for those participants who have difficulty with administration of the PFP. Additionally, participants will be provided the PFS for use in this study in countries where the PFS is anticipated to be the only presentation available commercially. An optional, open-label, single arm, substudy using the PFP was completed in the first 23 participants rolled over from Study B7841005 who agreed to participate in the substudy.

Eligibility

Primary outcome measure(s)

• Number of subject reporting Adverse Events — Baseline up to 7 years
• Number of subjects reporting Serious Adverse Events — Baseline up to 7 years
• Incidence and severity of thrombotic events — Baseline up to 7 years
• Incidence and severity of thrombotic microangiopathy — Baseline up to 7 years
• Number of subjects reporting Disseminated intravascular coagulalopathy/consumption coagulopathy — Baseline up to 7 years
• Incidence of clinically significant persistent NAb against marstacimab — Baseline up to 7 years
• Incidence and severity of injection site reaction — Baseline up to 7 years
• Clinically significant changes in vital signs from baseline — Baseline up to 7 years
• Incidence of clinically significant laboratory value abnormalities — Baseline up to 7 years
• Incidence of severe hypersensitivity and anaphylactic reactions — Baseline up to 7 years

Trial sites (75)

+ 35 more sites — see the full list on the official registry below.

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