A PHASE 1B OPEN-LABEL NON-RANDOMIZED STUDY TO ASSESS THE SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF MARSTACIMAB TREATMENT FOLLOWING THE DISCONTINUATION OF EMICIZUMAB THERAPY IN ADOLESCENT AND ADULT PARTICIPANTS WITH SEVERE HEMOPHILIA A WITHOUT INHIBITORS.
Pfizer
Summary
The purpose of the study is to learn about safety, how the body processes marstacimab and how it works in patients with severe hemophilia A without inhibitors. Hemophilia A is rare bleeding disorder where the blood doesn't clot normally. This causes a person to bleed a lot, even from a small cut. These patients who are on emicizumab medicine for routine prophylaxis for at least 6 months, and desire to switch to marstacimab medicine. Inhibitors are antibodies that the immune system develops because it sees the infused clotting factor as a foreign substance that needs to be destroyed. Antibodies are proteins that eat up the activated factor before it has time to stop the bleeding. Prophylaxis are preventive medicines. This study is seeking for participants: * with severe Hemophilia A withouth inhibitors who are on emicizumab treatment for at least 6 months. * must be 12 to less than 75 years old * must have a body weight of at least 35 kilograms. The results from this study will serve as a guide to doctors and their hemophilia A patients who will change their medicines in the real-world clinical setting. Patients who can take part in the study will receive marstacimab medicine as weekly injections under the skin of 150 milligrams for 4 months. Study treatment with marstacimab will be initiated no earlier than 14 days after last dose of emicizumab. The study can last up to 6 months. The sponsor will provide marstacimab. Patients will continue their usual treatment with the infused clotting factor for their bleeds when taking part in the study. Roll-over into an optional study treatment extension period will be available to participants who wish to continue prophylaxis with marstacimab in countries where it is not commercially available.
Description
This is a non-randomized open-label study in approximately 10-15 adolescent and adult participants between ages 12 to \<75 years with severe hemophilia A (defined as FVIII activity \<1%) without inhibitors who are currently on treatment with emicizumab for ≥6 months. The study is designed to assess the safety and PK/PD during a 4-month treatment period with marstacimab 150 mg SC QW after a minimum 14-day wash-out period following discontinuation of emicizumab. Assessment of safety will be conducted by summarizing AEs and SAEs reported during the study, from the time each participant provides i…
Eligibility
- Age range
- 12–74 years
- Sex
- Male
- Healthy volunteers
- No
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%) 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…
Interventions
- DrugMARSTACIMAB
Marstacimab 150 mg administered subcutaneous (SC) once weekly (QW)
Locations (4)
- Orthopaedic Institute for Children/Orthopaedic Hemophilia Treatment CenterLos Angeles, California
- Nirmal Hospital Pvt Ltd.Surat, Gujarat
- K J Somaiya Hospital & Research CentreMumbai, Maharashtra
- Ege University Faculty of Medicine Dean's OfficeBornova, İzmir