Safety and Tolerability of VGB-R04 in Patients With Haemophilia B
Study Details
- Hemophilia B
- Inclusion Criteria:
- Male ≥18 years and ≤75years of age;
- Confirmed diagnosis of hemophilia B (baseline FIX activity ≤ 2% of normal or documented history of FIX activity ≤2%);
- At least 100 days exposure history to FIX;
- Currently receiving FIX Prophylaxis therapy or on-demand treatment to prevent bleeding;
- Have acceptable laboratory values:
- Hemoglobin ≥110 g/L;
- Platelets ≥100×10'9 cells/L;
- AST, ALT, alkaline phosphatase ≤2×upper limit of normal (ULN) at the testing laboratory;
- Bilirubin ≤3× ULN ;
- Creatinine ≤1.5× ULN.
- No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein;
- Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences;
- Able to provide informed consent and comply with the requirements of the study.
- Exclusion Criteria:
- Have significant underlying liver disease within the past 6 months prior to or at Screening, including but not limited to:
- Preexisting diagnosis of portal hypertension;
- Splenomegaly;
- Encephalopathy;
- Reduction of serum albumin;
- Evidence of significant liver fibrosis;
- Have anti-VGB-R04 neutralizing antibody titers ≥1:5;
- Evidence of severe infection disease, i.e., human immunodeficiency virus (HIV) infection, syphilis, tuberculosis, etc.;
- Evidence of active hepatitis B virus infection (HBV-DNA \>103 IU/ml) or hepatitis C virus infection (HCV antigen and HCV-RNA positive);
- Evidence of malignant tumours or those with a previous history of malignant tumours;
- Have a history of chronic infection or other chronic diseases that the Investigator considers to constitute an unacceptable risk;
- Any immunodeficiency;
- Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational drug within the last 4 weeks;
- Have used glucocorticoids, immunosuppressive drugs, or antipsychotics within the last 3 months;
- Previous history of hypersensitivity or allergic reaction to any FIX products or any immunoglobulin;
- Unable or unwilling to comply with the schedule of visits and study assessments described in the clinical protocol;
- Any concurrent clinically significant major disease or any other condition that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study.
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.