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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04805801
Recruitment Status : Recruiting
First Posted : March 18, 2021
Last Update Posted : July 21, 2021
Sponsor:
Information provided by (Responsible Party):
JW Pharmaceutical

Study Description
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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.

Condition or disease Intervention/treatment
Hemophilia A With Inhibitor Hemophilia A Without Inhibitor Drug: Emicizumab subcutaneous injection

Detailed Description:

Target subject :

  1. Patients with Hemophilia A with FVIII inhibitors
  2. Patients with severe Hemophilia A without FVIII inhibitors.
Study Design
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Study Type : Observational [Patient Registry]
Estimated Enrollment : 17 participants
Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration: 6 Months
Official Title: Observational Study on the Safety of Hemlibra Subcutaneous Injection in Korean Hemophilia A Patients With/Without FVIII Inhibitors
Actual Study Start Date : August 28, 2019
Estimated Primary Completion Date : July 2024
Estimated Study Completion Date : February 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia
Drug Information available for: Emicizumab

Groups and Cohorts
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Group/Cohort Intervention/treatment
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
Safety and efficacy and PK, FVIII inhibitor titers will be evaluated by follow-up observation.
Other Name: Emicizumab concentration, FVIII inhibitor titer, FVIII Activity lab test
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
Safety and efficacy and PK, FVIII inhibitor titers will be evaluated by follow-up observation.
Other Name: Emicizumab concentration, FVIII inhibitor titer, FVIII Activity lab test


Outcome Measures
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Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. Annualized Bleeding episode Rate (ABR) [ Time Frame: for 24weeks ]
    The number of bleeds will be also annualized for each patient using the following formula


Biospecimen Retention:   Samples Without DNA
Emicizumab Concentration Factor FVIII Inhibitor FVIII Activity

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
1 .Hemophilia A patients with FVIII inhibitors 2. Severe Hemophilia A patients without FVIII inhibitors
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04805801


Contacts
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Contact: Chuhl Joo Lyu, Prof +82222282060 CJ@yuhs.ac
Contact: YoungShil Park, Prof. +8224407174 pysmd@khnmc.or.kr

Locations
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Korea, Republic of
Yonsei University Severance Hospital Recruiting
Seoul, Korea, Republic of
Contact: Chuhl-Joo Lyu, Prof    +82222282060    CJ@yuhs.ac   
Sponsors and Collaborators
JW Pharmaceutical
Investigators
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Principal Investigator: Chuhl Joo Lyu, Prof Severance Hospital
More Information
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Responsible Party: JW Pharmaceutical
ClinicalTrials.gov Identifier: NCT04805801    
Other Study ID Numbers: JWP-HEM-401
First Posted: March 18, 2021    Key Record Dates
Last Update Posted: July 21, 2021
Last Verified: July 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
 Hemorrhagic Disorders
Genetic Diseases, Inborn
Antibodies, Bispecific
Immunologic Factors
Physiological Effects of Drugs