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
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- Pfizer Inc.
- 入组人数
- 15
- 试验地点
- 2
- 主要终点
- 1. Incidence of marstacimab- elated adverse events
研究概览
简要总结
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. A 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 can be with or without inhibitors 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 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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Other
- 盲法
- None
入排标准
- 年龄范围
- 12.00 Year(s) 至 74.00 Year(s)（—）
- 性别
- Male
入选标准
- •Male and 12 to less than 75 years of age with a minimum body weight of 35 kg at the time of signing the informed consent.
- •Diagnosis of severe hemophilia A (FVIII activity less than 1%) without inhibitors.
- •On emicizumab therapy at a standard clinical dose for more than or equal to 6 months.
排除标准
- •Previous or current treatment for or history of coronary artery diseases, venous or arterial thrombosis, or ischemic disease.
- •Any medical or psychiatric condition including recent or active suicidal ideation or 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.
- •Known hemostatic defect other than hemophilia A
- •Current use of any prohibited concomitant medications or unwillingness or inability to use a required concomitant medication
- •Previous administration of an investigational product, drug or vaccine within 30 days or 5 halflives preceding the first dose of study intervention used in this study, whichever is longer.
- •Participation in studies of other investigational products at any time during participation in this study
- •Platelet count less than 100,000per microL or hemoglobin less than 10 g per dL
- •Clinically significant renal or hepatic function abnormality based on laboratory results at screening,or known kidney or liver disease.
- •CD4 cell countless than or equal to 200per microL if HIV positive.
- •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
- •Known planned surgical procedure
- •Hypersensitivity or allergic reaction to hamster protein or other components of the study intervention
- •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.
结局指标
主要结局
1. Incidence of marstacimab- elated adverse events
时间窗: 1. Incidence of marstacimab- elated adverse events - Approximately 178 days: from the time the participant provides informed consent through and | including a minimum of 28 calendar days after last dose of study treatment. | 2. Incidence of marstacimab-related serious AEs - Approximately 178 days: from the time the participant provides informed consent through and including a minimum of 28 calendar days after last dose of study treatment.
2. Incidence of marstacimab-related serious AEs
时间窗: 1. Incidence of marstacimab- elated adverse events - Approximately 178 days: from the time the participant provides informed consent through and | including a minimum of 28 calendar days after last dose of study treatment. | 2. Incidence of marstacimab-related serious AEs - Approximately 178 days: from the time the participant provides informed consent through and including a minimum of 28 calendar days after last dose of study treatment.
次要结局
- Change from baseline in tissue factor pathway inhibitor (TFPI).(Up to day 135)
- Change from baseline in prothrombin fragment 1+2 (PF 1+2)(Up to day 135)
- Change from baseline in D-dimer(Upto Day 135)
- Change from baseline in TGA (thrombin generation assay).(Upto Day 135)
- Incidence of Anti-Drug Antibody (ADA)(Upto Day 135)
- Incidence of clinically significant persistent neutralizing antibodies (NAb)(Upto Day 135)
- Plasma Concentration Versus Time of Marstacimab (Listings)(Up to day 135)
- Maximum Observed Plasma Concentration (Cmax) of Marstacimab(Up to day 135)
- Average Plasma Concentration (Cavg) of Marstacimab(Up to day 135)
- Minimum Observed Plasma Concentration (Cmin)(Up to day 135)