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

Recruiting

• Conditions: Hemophilia A With Inhibitor; Hemophilia A Without Inhibitor
• Interventions: Drug: Emicizumab subcutaneous injection

• Registration Number: NCT04805801
• Lead Sponsor: JW Pharmaceutical

Brief Summary

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.

Detailed Description

Target subject :

1. Patients with Hemophilia A with FVIII inhibitors

2. Patients with severe Hemophilia A without FVIII inhibitors.

Study Timeline

• Study Start: 2019-08-28
• Study First Submitted: 2021-03-17
• Study First Submitted QC: 2021-03-17
• Study First Posted: 2021-03-18
• Status Verified: 2021-07
• Last Update Submitted: 2021-07-15
• Last Update Posted: 2021-07-21
• Primary Completion: 2024-07
• Study Completion: 2025-02

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 17

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Hemophilia A with FVIII inhibitors	Emicizumab subcutaneous injection	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	Emicizumab subcutaneous injection	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

Primary Outcome Measures

Name	Time	Method
Adverse events	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 Outcome Measures

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

Trial Locations

Locations (1)

Yonsei University Severance Hospital; 🇰🇷 Seoul, Korea, Republic of