Last updated 32 days ago
A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
100 patients around the world
Available in Brazil
Pfizer
100Patients around the world
This study is for people with
Hemophilia
Hemophilia b
Hemophilia a
Requirements for the patient
To 17 Years
Male
Medical requirements
- Male participants of appropriate age and required minimum weightParticipants aged 12 to 17 years must be at least 25 kgs at time of consent.Participants aged 6 to 11 years must be at least 19 kgs at time of consent.Minimum weight requirement for participants aged 1 to 5 years is to be determined.Participants with a diagnosis of severe hemophilia A or moderately severe to severe hemophilia BParticipants must have at least 1 year of diary and/or medical records available in which exogenous FVIII or FIX replacement or bypass agent infusions and hemophilic bleeding episodes were consistently documented over the 12 months prior to the time of consent.Non-Inhibitor Cohort must also meet the following criteria:No current detectable inhibitor and no documented history of inhibitors in the 5 years prior to consentMust have at least 50 exposure days to FVIII/FIX replacement productsMust be at least 80% compliant with a stable routine prophylaxis regimen with FVIII/FIX replacement products, for at least 12 months prior to consentInhibitor Cohort must also meet the following criteria:Documentation of current high titer inhibitor (≥5 BU/mL); or current low titer inhibitor (<5 BU/mL) refractory to FVIII or FIX replacement and with FVIII or FIX recovery <60% of expected within previous 12 months prior to the time of consentParticipants who have documented inhibitors while on factor-replacement therapy but who do not meet the high quantitative inhibitor criteria described in the prior bullet at the time of screening (eg, participant with a previously documented high-titer inhibitor ≥5 BU/mL) and whose condition precludes re-challenge with FVIII or FIX replacement may be considered for eligibility on a case-by-case basis with discussion and agreement from the Pfizer medical monitor.Hemophilia A participants with on-demand treatment regimen with ≥12 bleeding episodes or hemophilia B participants with on-demand treatment regimen with ≥8 bleeding episodes (spontaneous or traumatic) necessitating treatment with bypass factor in the 12 months prior to informed consentParticipants must be on an on-demand bypass treatment regimen during the 12 months prior to informed consent
- Known coronary artery, thrombotic, or ischemic disease, including congenital or acquired thrombophilic disease such as Anti-thrombin III, Factor V Leiden mutation, prothrombin 20210 mutation, protein C activity, protein S activity and antiphospholipid syndrome.Known planned surgical procedure during the planned study periodKnown hemostatic defect other than hemophilia A or BAbnormal hematology, renal or hepatic function laboratory results at screeningOther acute or chronic medical or psychiatric condition that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigatorIndividuals with known allergic reaction or hypersensitivity to hamster protein or other components of the study interventionCurrent routine prophylaxis with bypassing agent, non-coagulation non-factor replacement therapy (eg, emicizumab), or any previous treatment with a gene therapy product for treatment of hemophiliaParticipants with inhibitors who are being treated using a prophylaxis treatment regimen with a bypass agent, and, participants who have previously received non-factor-based hemophilia therapy (eg, fitusiran, concizumab, emicizumab) will be considered on a case-by-case basis, only after discussion and agreement between the investigator and the Pfizer medical monitorOngoing or planned use of ITI, or prophylaxis with FVIII or FIX replacement at any time after initiation of treatment with study interventionParticipation in other studies involving investigational drug(s) or investigational vaccine(s) within 30 days (or as determined by local requirements) or 5 half-lives prior to study entry or during study participationPrevious exposure to marstacimab during participation in other marstacimab clinical studiesCD4 cell count ≤200/uL if HIV-positiveInvestigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, and sponsor and sponsor delegate employees directly involved in the conduct of the study and their family members
StudyBASIS KIDS
SponsorPfizer
Conditions
Hemophilia bHemophilia a
Requirements
To 17 YearsMale
Unique study IDclinicaltrials.gov
NCT05611801
