An Open-Label Study in Pediatric (<18 Years of Age), Severe Hemophilia A Participants (Coagulation Factor Activity <1%) With or Without Inhibitors or Moderately Severe to Severe Hemophilia B Participants (Coagulation Factor Activity<= 2%) With or Without Inhibitors Comparing 12 Months of Historical Standard Treatment to Marstacimab Prophylaxis - BASIS KIDS

Kawai Norisuke0 sites100 target enrollmentMarch 14, 2023

Overview

Phase: Phase 3
Intervention: Not specified
Conditions: Not specified
Sponsor: Kawai Norisuke
Enrollment: 100
Status: Recruiting
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

March 14, 2023

End Date

TBD

Last Updated

2 years ago

Study Type

Interventional

Sex

Male

Investigators

Sponsor

Kawai Norisuke

Eligibility Criteria

Inclusion Criteria

•Inclusion Criteria:
•\*Male participants of appropriate age and required minimum weight
•\*Participants 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 B
•\*Participants 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.
•Participants who are enrolled into the 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 consent
•\*Must have at least 50 exposure days to FVIII/FIX replacement products

Exclusion Criteria

•Exclusion Criteria:
•\*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 period
•\*Known hemostatic defect other than hemophilia A or B
•\*Abnormal hematology, renal or hepatic function laboratory results at screening
•\*Other 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 investigator
•\*Individuals with known allergic reaction or hypersensitivity to hamster protein or other components of the study intervention
•\*Current 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 hemophilia
•\*Participants 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 monitor
•\*Ongoing or planned use of ITI, or prophylaxis with FVIII or FIX replacement at any time after initiation of treatment with study intervention