Phase 3 Clinical Project of Pegylated Recombinant Human Coagulation Factor VIII-Fc Fusion Protein

Study Identifier:
SS-117-III01
ClinicalTrials.gov Identifier:
NCT06142552
EudraCT Identifier:
N/A
Recruiting

Study Details

Medical Condition
  • Hemophilia A, Severe
Study Drug
  • Drug: FRSW117
Date
Dec 2023 - Jan 2026
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Male
Age: 12 - 65 Years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • inclusion Criteria：
  • 12≤ age ≤65 year-old men;
  • Patients with clinically confirmed severe hemophilia A, i.e. at screening (central laboratory testing) or previous medical records confirm: FⅧ activity \< 1%；
  • Previous documented treatment with any recombinant and/or blood-derived coagulation factor Ⅷ products or cryoprecipitation products and dosed ≥150 exposure days (EDs≥150)
  • Normal prothrombin time (PT) or International Normalized Ratio (INR)\<1.3
  • Bleeding events were recorded in detail for at least 6 months prior to screening（Participants in the on demand /PPX group were required to have at least 6 episodes of spontaneous bleeding within 6 months）
  • Fully understand and know about this study and sign informed consent to participate in the clinical study voluntarily, subject and/or their guardian can cooperate with them for bleeding treatment at home, and have the ability to complete all study procedures
  • Exclusion Criteria:
  • Known or suspected allergy to the investigational drug or its excipients, including mouse or hamster proteins;
  • Hypersensitivity or anaphylaxis after FⅧ or IgG2 injection in the past;
  • FⅧ inhibitor positive (≥0.6 BU/mL) during the screening period, or have a history of FⅧ inhibitor positive in the past, or a family history of FⅧ inhibitor positive;
  • Von Willebrand factor (vWF) antigen test results were lower than the lower limit of normal value;
  • Severe anemia at the screening stage (hemoglobin \< 60 g/L);
  • Platelet count during screening period \< 100×109 /L;
  • Abnormal liver function:
  • .Alanine aminotransferase (ALT), or aspartate aminotransferase (AST) \>3 times upper limit of normal (ULN); or Serum total bilirubin (TBIL) \>1.5x ULN;
  • Patients with abnormal renal function:
  • Creatinine clearance (Ccr) \<50 ml/min (according to Cockcroft and Gault formula); orSerum creatinine (Cr) \>1.5x ULN;
  • People with active hepatitis C, that is, hepatitis C virus (HCV) antibody positive and HCV RNA positive; Or anti-treponema pallidum specific antibody (TPHA) positive; Or positive for antibodies against the human immunodeficiency virus (HIV);
  • Patients with coagulation dysfunction other than hemophilia A;
  • Have a medical condition that may increase the risk of bleeding;
  • A history of drug or alcohol abuse;
  • Have a known mental disorder that may affect trial compliance;
  • Patients who have received transfusions of blood or blood components within 4 weeks prior to screening;
  • Participants who had participated in other clinical trials within 1 month before screening;
  • Use of any anticoagulant or antiplatelet drugs, off-label maximum dose of non-steroidal anti-inflammatory drugs (NSAID) within 7 days prior to screening; Or patients who need to be treated with anticoagulant or antiplatelet drugs or off-label maximum doses of SAID during clinical trials;
  • Severe cardiovascular and cerebrovascular disease or major thromboembolic events, such as stroke, myocardial infarction, unstable angina, congestive heart failure (New York Heart Association \[NYHA\] grade ≥ III), and severe arrhythmias (including QTc interphase \> 480 ms, corrected by Fridericia formula), uncontrolled hypertension (systolic ≥ 160 mmHg or diastolic ≥100 mmHg), deep vein thrombosis, etc.
  • Study patients who had used emesezumab within 6 months prior to first administration of the drug;
  • Patients who had used monoclonal antibody therapy, Fc fusion protein products (except FRSW107 and FRSW117), PEG products (except FRSW117), or intravenous immunoglobulin infusion within 3 months before the first administration of the investigational drug;
  • Study patients who underwent major surgery within 3 months prior to initial drug administration (major surgery is defined in 6.2.3 Perioperative management);
  • Study patients who have used FⅧ preparation of any standard half-life (e.g., Bycoch, Coproch, Biinidin, Renjie, NoL, Antaine, etc.) within 3 days or 5 half-lives prior to first administration of the drug (taking the elderly); Patients who have used any other extended half-life preparation FⅧ within 4 days or 5 half-lives prior to first dosing (for the elderly);
  • Study patients with fever, severe active bacterial or viral infection, and allergies within 2 weeks before the first administration of the drug;
  • Systemic immunomodulators (such as glucocorticoids \[\> 10 mg/ day equivalent dose of prednisone\], alpha-interferon, immunoglobulin, cyclophosphamide, cyclosporin, etc.) used within 14 days prior to the first administration of the study drug or planned during the study period were allowed to be inhaled, nasal spray, or topical corticosteroids;
  • Those who had been vaccinated within 4 weeks prior to initial administration of the study drug; Or who plan to be vaccinated during PK blood collection (only for subjects in the PK subgroup);
  • Plan to have a child or sperm donation during the entire trial period and within 3 months after the last dose, or do not want to use effective physical contraception (such as condoms, diaphragms, Iuds, etc.);
  • Have other serious medical conditions that the researchers said could not benefit from them
  • 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
Beijing tongren hospital,CMU
Beijing, China
Status
Recruiting
Location
XiangYa Hospital CentralSouth University
Changsha, China
Status
Recruiting
Location
The Second Affiliated Hospital of Chongqing Medical University
Chongqing, China
Status
Not yet recruiting
Location
Fujian Medical University Union Hospital
Fuzhou, China
Status
Recruiting
Location
Nanfang Hospital of Southern Medical University
Guangzhou, China
Status
Recruiting
Location
The First Affiliated Hospital，Zhejiang University School of Medicine
Hangzhou, China
Status
Not yet recruiting
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