A Study to Learn About How Changing Therapy From Emicizumab to Marstacimab Affects People With the Severe Hemophilia A.

Launched by PFIZER · Nov 21, 2024

Keywords

ClinConnect Summary

This clinical trial is studying how switching from a medication called emicizumab to a new treatment called marstacimab affects people with severe hemophilia A, a bleeding disorder that makes it hard for blood to clot. The goal is to understand how safe marstacimab is, how the body processes it, and how well it works for patients who have been on emicizumab for at least six months. To participate in this study, you need to be a male aged 12 to 75 years old, weigh at least 35 kilograms, and have been diagnosed with severe hemophilia A, either with or without inhibitors (which are antibodies that can interfere with treatment).

If you join the study, you will receive marstacimab injections under the skin once a week for four months, starting after a short break from emicizumab. The study will last up to six months, and patients can continue to use their usual treatments for bleeding while participating. It’s important to note that the study is not yet recruiting participants, and there are some health conditions that may exclude you from joining. This research will help guide doctors and patients in making informed decisions about changing treatments in the future.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • 1. Male and 12 to \<75 years of age with a minimum body weight of 35 kg at the time of signing the informed consent.
• • 2. Diagnosis of severe hemophilia A (FVIII activity \<1%) with or without inhibitors.
• • 3. On emicizumab therapy at a standard clinical dose for ≥6 months.
• Exclusion Criteria:
• • 1. Previous or current treatment for or history of coronary artery diseases, venous or arterial thrombosis, or ischemic disease.
• • 2. Any medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory abnormality that may increase the risk of study participation or, in the investigator's judgment, make the participant inappropriate for the study.
• • 3. Known hemostatic defect other than hemophilia A.
• • 4. Current use of any prohibited concomitant medication(s) or unwillingness or inability to use a required concomitant medication(s).
• • 5. Previous administration of an investigational product (drug or vaccine) within 30 days or 5 half-lives preceding the first dose of study intervention used in this study (whichever is longer). Participation in studies of other investigational products (drug or vaccine) at any time during participation in this study.
• • 6. Platelet count \<100,000/μl or hemoglobin \<10 g/dL.
• • 7. Clinically significant renal or hepatic function abnormality based on laboratory results at screening, or known kidney or liver disease.
• • 8. CD4 cell count ≤200/μl if HIV positive.
• • 9. Screening 12-lead ECG that demonstrates clinically significant abnormalities that, in the opinion of the investigator, may affect participant safety or interpretation of study results.
• • 10. Known planned surgical procedure.
• • 11. Hypersensitivity or allergic reaction to hamster protein or other components of the study intervention.
• • 12. Investigator site staff directly involved in the conduct of the study and their family members, site staff otherwise supervised by the investigator, and sponsor and sponsor-delegate employees directly involved in the conduct of the study and their family members.