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

NCT04805801 | Unknown FDA Drug

• 1 other identifier: JWP-HEM-401
• Study Type: observational
• Target: 17
• Locations: 1 country
• Sites: 1

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.

Conditions

Hemophilia A With Inhibitor; Hemophilia A Without Inhibitor

Outcome Measures

Primary Outcomes (1)

• Adverse events

  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.

  for 24weeks

Secondary Outcomes (1)

• Annualized Bleeding episode Rate (ABR)

  for 24weeks

Study Arms (2)

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

Drug: Emicizumab subcutaneous injection

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

Drug: Emicizumab subcutaneous injection

Interventions

Emicizumab subcutaneous injection DRUG

Safety and efficacy and PK, FVIII inhibitor titers will be evaluated by follow-up observation.

Also known as: Emicizumab concentration, FVIII inhibitor titer, FVIII Activity lab test

Hemophilia A with FVIII inhibitors; Hemophilia A without FVIII inhibitors

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

1 .Hemophilia A patients with FVIII inhibitors 2. Severe Hemophilia A patients without FVIII inhibitors

You may qualify if:

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

You may not qualify if:

• Subjects who are hypersensitive to Emicizumab
• Subjects who are hypersensitive to mouse or hamster protein
• Subjects with myocardial infarction or the history who are prohibited from administering L-Arginine containing drug
• 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)
• Subjects who the investigator deems inappropriate for the study.
• Moderate/Mild for Hemophilia A patients without FVIII inhibitors

Sponsors & Collaborators

• JW Pharmaceutical: lead

Study Sites (1)

Yonsei University Severance Hospital

Seoul, South Korea

RECRUITING

Biospecimen

Retention: SAMPLES WITHOUT DNA

Emicizumab Concentration Factor FVIII Inhibitor FVIII Activity

Study Officials

• Chuhl Joo Lyu, Prof

  Severance Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Chuhl Joo Lyu, Prof

CONTACT

+82222282060; CJ@yuhs.ac

YoungShil Park, Prof.

CONTACT

+8224407174; pysmd@khnmc.or.kr

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Target Duration: 6 Months
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 17, 2021
• First Posted: March 18, 2021
• Study Start: August 28, 2019
• Primary Completion: July 1, 2024
• Study Completion: February 1, 2025
• Last Updated: July 21, 2021

Record last verified: 2021-07

Locations

KR (1)