AKATSUKI Study
- Conditions
- Congenital hemophilia A with FVIII inhibitors
- Registration Number
- JPRN-jRCTs041200037
- Lead Sponsor
- Matsushita Tadashi
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 15
Patients must meet all of the following criteria for study entry:
1) Informed Consent Form signed by the patient or the patient's legally authorized representative. Pediatric patients who are capable: Informed Assent Form signed by the patient
2) Patient or caregiver: Willing and able to comply with all study procedures (including filling out questionnaires on bleeds/drugs used)
3) Diagnosed with congenital hemophilia A and meets either of the following criteria:
a. Will start ITI after study enrollment and has positive FVIII inhibitor titer ( >= 0.6 BU/mL) as evidenced by most recent titer results within 8 weeks before enrollment
b. Is already undergoing ITI at study enrollment and has not yet met partial success as evidenced by most recent titer results within 8 weeks before enrollment
Patients who meet any of the following criteria will be excluded from study entry:
1) Inherited or acquired bleeding disorder other than congenital hemophilia A
2) Previous (within the last 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or high risk for thromboembolic diseases(e.g. protein S deficiency)
3) At high risk for thrombotic microangiopathy (TMA) in the opinion of the investigator based on previous or familial history of TMA (e.g., thrombotic thrombocytopenic purpura [TTP], atypical hemolytic uremic syndrome [aHUS])
4) Participating or planning to participate in other intervention trials
5) Otherwise unsuitable for study participation in the opinion of the investigator
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To comprehensively evaluate the following safety of emicizumab under and after ITI in patients with congenital hemophilia A with FVIII inhibitors.<br>- Adverse event (Mainly thromboembolic events)<br>- Abnormal laboratory values
- Secondary Outcome Measures
Name Time Method - Number of bleeds over time requiring treatment with coagulation factor products<br>- Number of patients meeting partial success of responsiveness to ITI therapy<br>- Time to achieve negative FVIII inhibitor titers and partial success in patients starting ITI after study enrollment<br>- FVIII inhibitor titers under and after ITI therapy <br>- Patient health-related quality of life (HR-QoL) as measured by Hemophilia-Specific Quality of Life Short Form (Haemo-QoL-SF) and caregiver HR-QoL as measured by Inhibitor-Specific Quality of Life with Aspects of Caregiver Burden (Adapted INHIB-QoL) scores