CANADE Study

Not Applicable

• Conditions: Congenital hemophilia A without factor VIII inhibitor

• Registration Number: JPRN-jRCTs031210222
• Lead Sponsor: Bingo Masato

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-08-04
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 100

Inclusion Criteria

1)Inclusion criteria common to all patients (Group A, Group B, Group C)
- Male, agedover 20
- Patients with congenital severe hemophilia A without factor VIII inhibitors at the time of enrollment
(Severe: endogenous factor VIII activity <1%, inhibitor negative: inhibitor titer <0.6 BU / mL)
- Patients with a history of factor VIII administration at least 50 exposure days during bleeding or with routine replacement therapy
- Patients or the patient's legally authorized representative who have received sufficient explanation about this study and have voluntarily given their own consent in writing to participate in the study.
- Patients who can comply with the requirements and procedures for this study
2) Additional inclusion criteria in the continuous emicizumab administration group (group A)
- Patients receiving emicizumab continuously according to the dosing regimen described in the package insert for at least 12 weeks prior to enrollment
3) Additional inclusion criteria in the newly introduced emicizumab group (group B)
- In the treatment selection between the doctor and the patient, there are problems in the treatment of factor VIII preparations such as difficulty in home intravenous injection and frequent injections to stop bleeding, and emicizumab is an effective solution. Patients who are expected and wish to receive emicizumab.
Although there are no absolute criteria or conditions for the introduction of emicizumab, the Japanese Society for Thrombosis and Hemostasis, Guidelines for Hemostasis Treatment for Hemostasis Patients 2019 Supplement 10) states that the convenience of subcutaneous injection is an advantage. Cases, cases with difficulty in intravenous injection and risk of bleeding, cases with low activity, etc. may be good indications, and factor VIII rather than emicizumab in cases with high activity or target joints. These are fully referred to.
4) Additional inclusion criteria in the factor VIII administration group (Group C)
- Patients receiving when bleeding or regular replacement therapy with factor VIII
- Patients who are not scheduled to introduce emicizumab for the next year at the time of enrollment

Exclusion Criteria

- Patients who have difficulty in visiting the hospital for the regular visits, during bleeding, or surgery / surgical procedures
- Patients diagnosed with bleeding disorders other than congenital hemophilia A
- Patients with abnormal liver function or platelet count at the time of screening (PT-INR> 1.2, AST / ALT level is 5 times or greater the upper limit of normal of the implementing medical institution, platelet count <100,000 / micro L)
- Patients with extremely difficult blood sampling procedure
- Patients who are judged by the principal investigator and/or the investigator to be inappropriate for the study

Study & Design

• Study Type: Interventional
• Study Design: Not specified