Basic Study of Combination Therapy Based on APCC-induced FVllI Activation in Hemophilia A Patients With Inhibitors

• Conditions: Hemophilia A With Inhibitor

• Registration Number: NCT02554526
• Lead Sponsor: Nara Medical University

Brief Summary

Using whole blood samples and plasma samples obtained from some hemophilia A patients with inhibitors, the investigators will perform the coagulation assessment in the co-presence of aPCC and factor VIII by comprehensive coagulation assays and flow chamber analysis under blood flow conditions.

Detailed Description

Comprehensive coagulation assays using recently popular rotational thromboelastometry (ROTEM), factor Xa/thrombin/plasmin generation test, and coagulation wave analysis and recently developed flow chamber system under blood flow conditions (T-TAS®) will be used. A bypassing agent, aPCC (Feiba®) and recombinant factor VIII (Advate®) will be added ex vivo to whole blood and plasma samples from patients with hemophilia A with inhibitors to compare with the coagulation effects of aPCC in the presence of factor VIII and those of aPCC in its absence under the conditions close to the physiological condition. Furthermore, the difference in the coagulation effects by the difference in inhibitor epitopes will be examined.

Study Timeline

• Status Verified: 2015-09
• Study Start: 2015-09
• Study First Submitted: 2015-09-15
• Study First Submitted QC: 2015-09-17
• Last Update Submitted: 2015-09-17
• Study First Posted: 2015-09-18
• Last Update Posted: 2015-09-18
• Primary Completion: 2017-03
• Study Completion: 2017-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: Male
• Target Recruitment: 10

Inclusion Criteria

• Patients with hemophilia A with inhibitors (Inhibitor titer; >0.5 Bethesda U/ml)
• Patients with agreement consents with the patients signatures

Exclusion Criteria

• Patients that do not fill the conditions described above

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Evaluation of coagulation effects using patients' whole bloods by ROTEM and T-TAS	up to 15 months
Evaluation of coagulation effects using patients' plasmas by FXa/thrombin/plasmin generation tests and clot waveform analysis	up to 10 months

Trial Locations

Locations (1)

Department of Pediatrics; 🇯🇵 Kashihara, Nara, Japan