Congenital hemophilia A with FVIII inhibitors
Conditions
Interventions
Any type of FVIII concentrate can be used for ITI but the dosing regimen must be 50 IU/kg three times a week. However, when using extended half-life FVIII concentrates, a dosing frequency of twice a w
Sponsors
Suzuki Nobuaki
Nishi Kazuhiko
Chugai Pharmaceutical Co., Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 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
Exclusion criteria
Exclusion criteria: 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To comprehensively evaluate the following safety of emicizumab under and after ITI in patients with congenital hemophilia A with FVIII inhibitors. - Adverse event (Mainly thromboembolic events) - Abnormal laboratory values | — |
Secondary
| Measure | Time frame |
|---|---|
| - Number of bleeds over time requiring treatment with coagulation factor products - Number of patients meeting partial success of responsiveness to ITI therapy - Time to achieve negative FVIII inhibitor titers and partial success in patients starting ITI after study enrollment - FVIII inhibitor titers under and after ITI therapy - 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 | — |
Contacts
Public ContactNobuaki Suzuki
Nagoya University Hospital
Outcome results
None listed