A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B

Phase 3

• Conditions: Health Condition 1: D67- Hereditary factor IX deficiencyHealth Condition 2: D66- Hereditary factor VIII deficiency

• Registration Number: CTRI/2023/02/049471
• Lead Sponsor: Pfizer Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 6 March 2023

Recruitment & Eligibility

• Status: ot Yet Recruiting
• Sex: Not specified
• Target Recruitment: 0

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.

8. Participants who are enrolled into the Inhibitor Cohort must also meet the following criteria:

(a). 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 consent

(b). Participants 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.

(c). 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 consent.

(d). Participants must be on an on-demand bypass treatment regimen during the 12 months prior to informed 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.

11. Previous exposure to marstacimab during participation in other marstacimab clinical studies

12. CD4 cell count <=200/uL if HIV-positive

13. Investigator 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

Study & Design

• Study Type: Interventional
• Study Design: Not specified