Study of Efficacy and Safety of FRSW107 in Pediatric Patients With Severe Hemophilia A
Will Be Recruiting
Study Details
Medical Condition
- Hemophilia A, Severe
Study Drug
- Drug: FRSW107
Date
Dec 2026 - Dec 2026
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Male
Age: 1 Year - 12 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- Children \<12 years old, male;
- Weight \>10kg;
- clinically confirmed patients with severe hemophilia A (defined as confirmation at the time of screening or previous medical records: coagulation factor VI activity \<1%);
- Treated patients, that is, those who had previously received EVI treatment and met the following criteria: \<6 years old patients who had been treated with coagulation factor VI for \>50 exposure days (EDs250), and \< 26 years old patients who had been treated with coagulation factor VI for \>150 exposure days(\> 150);
- Normal prothrombin time (PT) or International normalized ratio (INR) \<1.3;
- At least 6 months of treatment and detailed records of bleeding events before screening;
- The subject's legally authorized representative (i.e. guardian) fully understands and knows about this study and signs the informed consent. Children with the ability to give informed consent (≥8 years old) should be informed and sign the informed consent voluntarily;
Exclusion Criteria
- 1\. People who have been allergic to any component of EVI preparation (including but not limited to mouse or hamster protein or virus vaccine, gene recombination preparation containing mouse or hamster protein, etc.); Those who have had serious adverse reactions to previous vaccine injections or have not recovered from mild to moderate adverse reactions to vaccine injections; 2.Patients with hypersensitivity or anaphylaxis after injection of coagulation factor VI or Fc fusion protein products; 3. Positive factor VI inhibitor at screening (20.6 BU/mL), or previous history of factor VI inhibitor, or family history of inhibitor; 4.the screening results of von Willebrand factor (vWE) antigen were lower than the lower limit of normal value; 5. Severe anemia (hemoglobin \<60g/L) at the time of screening; 6. Platelet count \<100×10⁹ during screening /L; 7.abnormal liver function: alanine aminotransferase (ALT), or aspartate aminotransferase (AST) \>3 times the upper limit of normal (ULN); Serum bilirubin (TBIL\>3× ULN; 8. Patients with abnormal renal function: serum creatinine (SCr) \>1.5×ULN or according toCreatinine clearance calculated by Cockcroft-Gault formula \< 60 mL/min (CTCAE Level 1); 9. hepatitis B virus surface antigen (HBsAg), hepatitis C virus (HCV) antibody, anti-human immunodeficiency virus antibody (Anti-HIV) and anti-treponema pallidum specific antibody (Anti-TP) test has one or more positive; 10. Patients with coagulation dysfunction other than hemophilia A; 11,.have other medical conditions that may increase the risk of bleeding or blood clots; 12. Have a known mental disorder that may affect trial compliance; 13. Patients who have used EV preparations of any standard half-life (e.g., Bekochi, Koyuki, Biinstop, Renjie, etc.) within 3 days or 5 half-lives prior to the first dose; Patients who have used any other half-life extension FVI preparations within 4 days or 5 half-lives prior to dosing (older at the time of retrieval); 14. Patients who have used emesezumab within 6 months prior to first dosing; 15. Severe cardiovascular and cerebrovascular disease, such as cerebral arteritis, moyamoya disease, stroke, viral myocarditis, endocarditis, endocardial fibroplasia, severe arrhythmia, congestive heart failure (New York Heart Association grade \> III), uncontrolled hypertension, thromboembolic disease, and uncontrolled diabetes, occurred within 6 months prior to the first medication; 16. Patients who had used monoclonal antibody therapy, Fc fusion protein products, or intravenous immunoglobulin within 3 months before the first dose; 17.those who underwent major surgical procedures and transfusions of blood or blood components within 4 weeks prior to initial dosing, or who plan to undergo elective surgery (other than minor surgery such as tooth extraction) during the study treatment period; Those who underwent major surgical procedures and transfusions of blood or blood components within 4 weeks prior to initial dosing, or who plan to undergo elective surgery during the study treatment period; 18.patients with fever, active infection, allergies (such as allergic rhinitis, allergic asthma, allergic dermatitis, etc.) within 2 weeks prior to the first dose； 19.people with immune deficiency diseases or autoimmune diseases such as systemic lupus erythematosus, or have a history of organ transplantation or stem cell transplantation; Systemic immunomodulators (such as corticosteroids (\>10mg/ day equivalent dose of prednisone), alpha-interferon, immunoglobulin, cyclophosphamide, cyclosporin, etc.) used within 14 days prior to the first administration or planned during the study period were allowed to use inhaled, nasal, ocular, intraarticular or topical corticosteroids; 20.patients who were treated with any anticoagulation (other than heparin sealing treatment) or platelet aggregation inhibitors within 7 days prior to initial administration or who required anticoagulation (other than heparin sealing treatment) or platelet aggregation inhibitors during study therapy; 21. Participants who have participated in other clinical trials within 1 month before screening; 22.had other serious medical conditions from which the researchers did not believe they could benefit; He suffered from severe skin disease, which interfered with the observation of local injection reaction.
- 23\. Subjects deemed unsuitable by other investigators.
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.
Trial Locations
Location
Status
Location
Children's Hospital Affiliated to Chongqing Medical University
Chongqing, China
Status
N/A
Location
Guangzhou Women and Children Medical Center
Guangzhou, China
Status
N/A
Location
Nanfang Hospital, Southern Medical University
Guangzhou, China
Status
N/A
Location
Affiliated Hospital of Guizhou Medical University
Guizhou, China
Status
N/A
Location
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, China
Status
N/A
Location
Anhui Children's Hospital
Hefei, China
Status
N/A
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