A Phase 1b Study to Assess the Safety, Tolerability, PK and PD of MG1113 in Hemophilia Patient

Korábbi kezelés

• Treatment history due to symptoms of fever within 28 days of IP administration or any surgery planned during the study period
• Received immune tolerance induction within 30 days prior to administration of the investigational product
• Received emicizumab within 30 days prior to administration of the investigational product
• Currently using systemic immunomodulator treatment (e.g., Corticosteroid*, IVIG, interferon or rituximab)
  • *High-dose corticosteroids (it is allowed to administer corticosteroid equivalent to up to 20mg/kg daily based on Prednisolone, but if a dose of more is continuously administered in excess of 14 days, it is considered as high dose, such case is excluded from this study. However, inhaled, intranasal, and topical administration of corticosteroids is allowed irrespective of the dose.)
• Participated in another clinical trial within 30 days of investigational product administration

Más betegség

• Congenital or acquired anticoagulant disorders other than hemophilia A or B, or conditions of other diseases that increase the risk of bleeding or thrombus (e.g., autoimmune disease)
• Be at risk of venous thromboembolism or thrombotic microangiopathy per investigator's judgment or have related medical history or family history
• Be at risk of cardia and/or coronary disease per investigator's judgment or have related medical history or family history
• Risk factors for venous or arterial disease (e.g., uncontrolled hypertension, uncontrolled diabetes)
• Known or suspected hypersensitivity to the IP or its components
• Clinically significant active chronic disease

Laborértékek

• Any of the following results from laboratory tests:
  • AST(sGOT) or AST(sGPT) > 3 x UNL
  • Total bilirubin > 2 mg/mL
  • Hb < 9.0 g/dL
  • Absolute Neutrophil Count < 1500 /μL
  • Platelet count < 10\^5 /μL
  • Have hepatitis B (HBs Ag positive) or C (anti-HCV positive), or have HIV positive test result If the anti-HCV antibody test is positive, the positive hepatitis virus result must be confirmed by a quantitative HCV RNA test
  • Serum Creatinine > 1.5 x Upper limit of normal (ULN)
• Determined to be ineligible to participate in the study per investigator's judgment due to other reasons including the laboratory test results

Egyéb

• Subjects who refuse the following wash-out times of Factor VIII, Factor IX, and bypassing agent from the time of first IP administration (Factor VIII: 72 hrs, Factor IX: 96 hrs, Bypassing agent e.g., rFVIIa or aPCC: 96 hrs)

• Male severe hemophilia A or B patients (FVIII or FIX activity <1%) aged 19-60 years (both inclusive) at screening
  • Patients without inhibitors against FVIII or FIX (having difficulty in their Self-injection of current standard treatment regimen) OR
  • Patients with inhibitors who has a positive inhibitor result of confirmed human factor VIII or IX with an inhibitor titer(≥ 0.6 BU) and failed after ITI treatment or not undergoing ITI
• ≥50 kg in weight with calculated BMI between 18.5 and 29.9 kg/m\^2 (BMI = (Weight [kg])/(height [m])\^2)
• Documentation of ≥4 bleeding episodes (any type or location of bleeds, treated or not) within 6 months prior to screening
• Agree to use medically acceptable adequate dual contraceptive methods (condom, vasectomy, spermicide, oral contraceptives, intrauterine device, and complete sexual abstinence, etc.) and not to donate sperm until 60 days after administration of the investigational product
• Voluntarily decided to participate in the study and provided written consent to follow precautions after receiving a detailed explanation on this study and fully understanding the information