The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients.
Study Details
- Hemophilia B
- Inclusion Criteria:
- Male ≥18 years and ≤65years of age;
- Confirmed diagnosis of hemophilia B, and endogenous FIX ≤2%:
- Have had ≥100 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX protein products;
- The subject had at least 3 or more bleeding events and/or chronic hemophilia arthritis in one or more joints in the previous 1 year requiring treatment with FIX agents;
- Agree to use reliable barrier contraception and prohibition of sperm donation until 52 weeks after the administration of ZS801.
- Subjects voluntarily participate and are fully informed, fully understand the research and can comply with the requirements of the research protocol, are willing to complete the research as planned, and voluntarily cooperate with the provision of biological samples for testing.
- Exclusion Criteria:
- Hypersensitivity to any component of the study drug (including immunosuppressants) or a condition that can not use;
- Inability to tolerate immunosuppressants or steroid drugs;
- Have FIX inhibitor as assessed by laboratory; or documented history of FIX inhibitor;
- Who have a history or are currently suffering from any of the following serious clinical diseases:
- History of malignancy or current presence of any malignancy;
- Have active autoimmune disease;
- Severe heart disease, including angina pectoris, myocardial infarction, heart failure, clinically significant congenital heart disease, heart valve disease, arrhythmia and atrioventricular block, etc.;
- Have underlying liver disease or history of liver disease (such as portal hypertension, ascites, splenomegaly, esophageal varices, hepatic encephalopathy or hepatic fibrosis);
- Have HBsAg positive or HCV-Ab positive, or are currently receiving hepatitis B or hepatitis C antiviral therapy;
- Diabetes mellitus that is poorly controlled after drug treatment;
- Uncontrolled hypertension or hypotension;
- laboratory values:
- Hemoglobin\<110g/L;
- Platelets\<100×10\^9/L;
- aspartate aminotransferase, Alanine transaminase, alkaline phosphatase\>2×ULN;
- Total bilirubin\>1.5×ULN;
- Creatinine\>ULN;
- Albumin\
- HIV antibody positive or Treponema pallidum antibody positive.
- Have AAV5 capsid neutralizing antibody titers \>1:640;
- Those who have received clinical trials of gene therapy before screening, or have used FIX clinical trial drugs within 1 month, or participated in other drug/device clinical trials within 3 months, or plan to participate in other clinical trials during this study;
- Those who have planned surgery within 52 weeks after the infusion;
- Those who lost more than 400 mL of blood within 3 months before screening;
- Those with epilepsy, history of mental illness (such as schizophrenia, depression, mania or anxiety) or obvious mental disorder, incapacitated or incapacitated by other reasons;
- Patients with a history of drug abuse or alcoholism;
- Investigators believe that subjects have poor compliance or are expected to be less likely to complete follow-up;
- There are clinically significant diseases or other reasons that the researcher and/or collaborators consider unsuitable to participate in this researcher.
Protocol Summary
This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.