Hemophilia A With InhibitorNCT04805801
The Safety of Emicizumab SC Injection in Korean Hemophilia A Patients With/Without FVIII Inhibitors
An observational study of Hemophilia A With Inhibitor and Hemophilia A Without Inhibitor, run by JW Pharmaceutical at 1 site in South Korea.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia A With Inhibitor, Hemophilia A Without Inhibitor
- What is being tested?
- Emicizumab subcutaneous injection
- Type of treatment
- Injection or infusion
- How many people
- 17 participants
- Where
- 1 siteSouth Korea
- Study dates
- 28 Aug 2019to Feb 2025
- How long will I take part?
- 6 Months
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: 1 .Hemophilia A patients with FVIII inhibitors 2. Severe Hemophilia A patients without FVIII inhibitors
Who may be able to join
- Patients with Hemophilia A (congenital factor VIII deficiency) who will receive Hemlibra SC inj. according to medical decision by investigator
- Signed informed consent form
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
South Korea1 site, 1 recruiting
- Recruiting
Yonsei University Severance Hospital
Seoul
Chuhl-Joo Lyu, Prof, +82222282060, CJ@yuhs.ac
About this study
Official title: Observational Study on the Safety of Hemlibra Subcutaneous Injection in Korean Hemophilia A Patients With/Without FVIII Inhibitors
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.
Read the detailed description
Target subject :
- Patients with Hemophilia A with FVIII inhibitors
- Patients with severe Hemophilia A without FVIII inhibitors.
- Being tested
- Emicizumab subcutaneous injection
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Emicizumab subcutaneous injectionDrug
Also called Emicizumab concentration, FVIII inhibitor titer, FVIII Activity lab test
Safety and efficacy and PK, FVIII inhibitor titers will be evaluated by follow-up observation.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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 Receives: Drug: Emicizumab subcutaneous injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: for 24weeks
Secondary outcomes (1)
Annualized Bleeding episode Rate (ABR)
The number of bleeds will be also annualized for each patient using the following formula
Measured: for 24weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2021
- Study start28 Aug 2019
- Primary completionJul 2024 (estimated)
- Study completionFeb 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia A With InhibitorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 17 participants (estimated)
- Target duration
- 6 Months
Sponsor and investigators
- Lead sponsor
- JW Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chuhl Joo Lyu, Prof, Principal investigator, Severance Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04805801
- Sponsor's protocol ID
- JWP-HEM-401
- Status last verified
- Jul 2021
- First submitted
- 17 Mar 2021
- Last updated
- 21 Jul 2021
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational Study on the Safety of Hemlibra Subcutaneous Injection in Korean Hemophilia A Patients With/Without FVIII Inhibitors
- ClinicalTrials.gov ID
- NCT04805801
- Acronym / sponsor ID
- JWP-HEM-401
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 17 participants (estimated)
- Lead sponsor
- JW PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 28 Aug 2019 (actual)
- Primary completion
- Jul 2024 (estimated)
- Study completion
- Feb 2025 (estimated)
- First posted
- 18 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 :
- Patients with Hemophilia A with FVIII inhibitors
- Patients with severe Hemophilia A without FVIII inhibitors.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Target follow-up
- 6 Months
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Hemophilia A with FVIII inhibitors | – | Drug: Emicizumab subcutaneous injectionHemophilia 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 | – | Drug: Emicizumab subcutaneous injectionHemophilia 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 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- 1 .Hemophilia A patients with FVIII inhibitors 2. Severe Hemophilia A patients without FVIII inhibitors
Key inclusion criteria (2 listed)
- Patients with Hemophilia A (congenital factor VIII deficiency) who will receive Hemlibra SC inj. according to medical decision by investigator
- Signed informed consent form
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Emicizumab subcutaneous injectionAlso: Emicizumab concentration, FVIII inhibitor titer, FVIII Activity lab testSafety and efficacy and PK, FVIII inhibitor titers will be evaluated by follow-up observation. | Drug | Hemophilia A with FVIII inhibitors, Hemophilia A without FVIII inhibitors |
Primary outcome
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.
1 secondary outcome
Annualized Bleeding episode Rate (ABR)
Time frame: for 24weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jul 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Chuhl Joo Lyu, Prof principal investigator
Severance Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A With Inhibitor and Hemophilia A Without Inhibitor, run by JW Pharmaceutical at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 21 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Chuhl Joo Lyu, Prof (+82222282060, CJ@yuhs.ac), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse events (time frame: for 24weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jul 2021.