Safety and Efficacy Study of NGGT003 in Hemophilia A Patients
Study Details
- Hemophilia A
- Drug: NGGT003
- Inclusion Criteria:
- Voluntarily sign the informed consent form;
- Male, age ≥18 years old;
- Diagnosed with hemophilia A according to the "Guidelines for Diagnosis and Treatment of Hemophilia A (2022 Edition)", and the endogenous FVIII activity level was \<1 IU/dL (\<1%);
- The exposure days (EDs) of treatment with any recombinant or plasma-derived FVIII product were ≥150 days;
- Anti-AAV neutralizing antibody titer ≤1:5, binding antibody titer ≤1:100;
- Bleeding events and/or FVIII product injections have occurred within 12 weeks before screening;
- No history of allergy to FVIII products;
- FVIII inhibitor titer﹤0.6BU/mL;
- Commitment to use other drugs during the study requires the consent of the investigator;
- Willing and able to comply with study procedures and requirements;
- Willing to use effective contraceptive methods within 52 weeks after administration.
- Exclusion Criteria:
- Positive for hepatitis B surface antigen, hepatitis C, human immunodeficiency virus (HIV)，syphilis test;
- Clinically significant abnormalities in liver function test: alanine aminotransferase (ALT) \>1.5 × upper limit of normal (ULN) and/or aspartate aminotransferase (AST) \>1.5× ULN;TBil）\>1.5×ULN；Serum creatinine (Scr) \>1.5×ULN; hemoglobin \<110g/L, platelets \<10e9/L;
- History of being positive for FVIII inhibitors;
- Have other bleeding factors except hemophilia;
- Plan major surgery within 52 weeks;
- Have contraindications to glucocorticoid, including but not limited to allergy to glucocorticoids, epilepsy, new unhealed fractures, in trauma repair period, uncontrolled infection, severe osteoporosis, etc, which assessed and determined by the investigators;
- History of allergy to human albumin;
- Have serious diseases or active infections in cardiovascular, respiratory, digestive tract, endocrine, renal, blood, nervous, mental and other systems before screening;
- With hepatitis, cirrhosis, liver cancer or other major liver diseases;
- History of malignant tumors;
- Abnormal and clinical significant vital signs, physical examination, laboratory examination or other related examination results during the screen, which are not suitable for trial according to the investigator;
- Previous gene therapy treatment;
- Participation in any other clinical trial before the screening and have taken medication within four weeks or five half-lives of the study drug;
- Any other condition that may not be appropriate for the study in the opinion of the investigator.
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.