Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors (NCT05145127)
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: To join this study, participants generally need to meet the listed inclusion criteria (3 items) and avoid the listed exclusions (10 items).
Location: University of Iowa — Iowa City, Iowa
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- 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
🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
📍 All Participating Trial Locations (75 Sites)
- University of Iowa — Iowa City, Iowa
- Northwell Health HTC — New Hyde Park, New York
- The Feinstein Institutes for Medical Research — Philadelphia, Pennsylvania
- Washington Institute for Coagulation d/b/a WACBD — Seattle, Washington
- Arbesu Hematología — Mendoza,
- Royal Children's Hospital — Melbourne, Victoria
- HEMOES — Vitória, Espírito Santo
- Stollery Children's Hospital — Edmonton, Alberta
- Hamilton Health Sciences - McMaster University Medical Centre — Hamilton, Ontario
- Hamilton Health Sciences - McMaster University Medical Centre — Hamilton, Ontario
- Nanfang Hospital, Southern Medical University — Guangzhou, Guangdong
- The Affiliated Hospital of Guizhou Medical University — Guiyang, Guizhou
- Tongji Hospital, Tongji Medical College of Huazhong University of Science and Technology — Wuhan, Hubei
- Jiangxi Provincial People's Hospital — Nanchang, Jiangxi
- Jiangxi Provincial People's Hospital — Nanchang, Jiangxi
- Institute of hematology&blood disease hospital — Tianjin, Tianjin Municipality
- Beijing Children's Hospital, Capital Medical University — Beijing,
- Klinicki bolnicki centar Zagreb — Zagreb,
- Aarhus Universitetshospital, Skejby — Aarhus, Central Jutland
- Hôpital Universitaire Necker Enfants Malades — Paris,
- The University of Hong Kong (HKU)-Queen Mary Hospital (QMH) — Hong Kong,
- Prince of Wales Hospital — Hong Kong,
- Queen Mary Hospital — Hong Kong,
- The Chinese University of Hong Kong — Ma Liu Shui,
- Nirmal Hospital Pvt Ltd. — Surat, Gjuarat
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.