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

Pfizer

Status and phase: Enrolling; Phase 3

Conditions: Hemophilia B; Hemophilia A

Treatments: Drug: PF-06741086

Study type: Interventional

Funder types: Industry

Identifiers

NCT05145127

B7841007

PHASE 3 ANTI-TFPI OLE (Other Identifier)

2022-500470-33-00 (Registry Identifier)

Details and patient eligibility

About

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.

Enrollment: 145 estimated patients

Sex: Male

Ages: 1 to 74 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• All participants will have a minimum body weight as defined by parent studies
• Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests, and other study procedures.
• Participants have successfully completed participation in parent studies, defined as did not require "Early Termination"

Exclusion criteria

• Previous or current treatment for or history of coronary artery disease, venous or arterial thrombosis (CTCAE Grade >3), or ischemic disease (except catheter-associated thrombosis)
• Abnormal renal function as defined by eGFR <30 mL.min/1.73 m(2)
• Known planned surgical procedure during the planned study period
• Unstable hepatic function as determined by the Investigator clinical assessment and review of the participant's most recent laboratory results, which would make the participant inappropriate for the study
• For participants known to be HIV+, worsening disease status as determined by the Investigator clinical assessment and review of participant's most recent laboratory results, to include recent locally available CD4 count (if available), which would make the participant inappropriate for the study
• Regular, concomitant therapy with immunomodulatory drugs (eg, IVIG, and routine systemic corticosteroids, rituximab)
• Ongoing or planned use of immune tolerance induction or prophylaxis with FVIII or FIX replacement during the study
• Participation in other study involving investigational drug(s) or investigational vaccine(s) within 30 days or 5 half-lives prior to or during study participation, with the exception of participation in parent studies
• Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the Investigator, and their respective family members

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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145 participants in 1 patient group

PF-06741086

Experimental group

Description:

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:

Drug: PF-06741086