CTRI/2023/02/049471
Not yet recruiting
Phase 3
An Open-Label Study in Pediatric (Below 18 Years of Age), Severe Hemophilia A Participants (Coagulation Factor Activity Less than 1%) With or Without Inhibitors or Moderately Severe to Severe Hemophilia B Participants (Coagulation Factor Activity â?¤2%) With or Without Inhibitors Comparing 12 Months of Historical Standard Treatment to Marstacimab Prophylaxis
Overview
- Phase
- Phase 3
- Intervention
- Not specified
- Conditions
- Health Condition 1: D67- Hereditary factor IX deficiencyHealth Condition 2: D66- Hereditary factor VIII deficiency
- Sponsor
- Pfizer Inc.
- Status
- Not yet recruiting
- Last Updated
- 3 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\.Male participants of appropriate age and required minimum weight
- •2\. Participants aged 12 to 17 years must be atleast 25 kgs at time of consent.
- •3\. Participants aged 6 to 11 years must be at least 19 kgs at time of consent.
- •4\. Minimum weight requirement for participants aged 1 to 5 years is to be determined.
- •5\. Participants with a diagnosis of severe hemophilia A or moderately severe to severe hemophilia B
- •6\. 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.
- •7\. Participants who are enrolled into the Non\-Inhibitor Cohort must also meet the following criteria:
- •(a). No current detectable inhibitor and no documented history of inhibitors in the 5 years prior to consent
- •(b). Must have at least 50 exposure days to FVIII/FIX replacement products
- •(c). Must be at least 80% compliant with a stable routine prophylaxis regimen with FVIII/FIX replacement products, for at least 12 months prior to consent.
Exclusion Criteria
- •1\. Known coronary artery, thrombotic, or ischemic disease
- •2\. Known planned surgical procedure during the planned study period
- •3\. Known hemostatic defect other than hemophilia A or B
- •4\. Abnormal hematology, renal or hepatic function laboratory results at screening
- •5\. 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
- •6\. Individuals with known allergic reaction or hypersensitivity to hamster protein or other components of the study intervention
- •7\. 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
- •8\. 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
- •9\. Ongoing or planned use of ITI, or prophylaxis with FVIII or FIX replacement at any time after initiation of treatment with study intervention.
- •10\. Participation 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 participation.
Outcomes
Primary Outcomes
Not specified
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