The Safety of Emicizumab SC Injection in Korean Hemophilia A Patients With/Without FVIII Inhibitors

Overview

To evaluate Safety and efficacy and pharmacokinetics, FVIII Inhibitor titers of Hemlibra subcutaneous injection (SC inj.) in Korean Hemophilia A patients with/without FVIII Inhibitors.

Full Title of Study: “Observational Study on the Safety of Hemlibra Subcutaneous Injection in Korean Hemophilia A Patients With/Without FVIII Inhibitors”

Study Type

  • Study Type: Observational [Patient Registry]
  • Study Design
    • Time Perspective: Other
  • Study Primary Completion Date: July 2024

Detailed Description

Target subject : 1. Patients with Hemophilia A with FVIII inhibitors 2. Patients with severe Hemophilia A without FVIII inhibitors.

Interventions

  • Drug: Emicizumab subcutaneous injection
    • Safety and efficacy and PK, FVIII inhibitor titers will be evaluated by follow-up observation.

Arms, Groups and Cohorts

  • Hemophilia A with FVIII inhibitors
    • Hemophilia A patients with FVIII inhibitors will administer Emicizumab at a loading dose of 3 mg/kg/week SC for the first 4 weeks of treatment followed by 1.5 mg/kg QW SC or 3mg/kg Q2W or 6mg/kg Q4W
  • Hemophilia A without FVIII inhibitors
    • Hemophilia A patients without FVIII inhibitors will administer Emicizumab at a loading dose of 3 mg/kg/week SC for the first 4 weeks of treatment followed by 1.5 mg/kg QW SC or 3mg/kg Q2W or 6mg/kg Q4W

Clinical Trial Outcome Measures

Primary Measures

  • Adverse events
    • Time Frame: for 24weeks
    • AEs will be described for AEs and Adverse Drug Reactions (ADRs), Adverse Events of Special Interest (AESIs), other important AEs. Severity, outcome, causal relationship with study drug, etc. of AEs will be analyzed.

Secondary Measures

  • Annualized Bleeding episode Rate (ABR)
    • Time Frame: for 24weeks
    • The number of bleeds will be also annualized for each patient using the following formula

Participating in This Clinical Trial

Inclusion Criteria

1. Patients with Hemophilia A (congenital factor VIII deficiency) who will receive Hemlibra SC inj. according to medical decision by investigator 2. Signed informed consent form Exclusion Criteria:

1. Subjects who are hypersensitive to Emicizumab 2. Subjects who are hypersensitive to mouse or hamster protein 3. Subjects with myocardial infarction or the history who are prohibited from administering L-Arginine containing drug 4. Subjects who participated in other clinical trials within a month before enrollment (or the first administration of Hemlibra SC inj.) (Unable to participate in other clinical trials related to Hemophilia A treatment even after enrollment) 5. Subjects who the investigator deems inappropriate for the study. 6. Moderate/Mild for Hemophilia A patients without FVIII inhibitors

Gender Eligibility: All

Minimum Age: N/A

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • JW Pharmaceutical
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Chuhl Joo Lyu, Prof, Principal Investigator, Severance Hospital
  • Overall Contact(s)
    • Chuhl Joo Lyu, Prof, +82222282060, CJ@yuhs.ac

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