An Open-label Phase II Study to Determine the Efficacy and Safety of Rituximab and Bortezomib in Patients With Newly Diagnosed Acquired Hemophilia A
Overview
- Phase
- Phase 2
- Intervention
- Rituximab
- Conditions
- Acquired Hemophilia A
- Sponsor
- Peking Union Medical College Hospital
- Enrollment
- 22
- Locations
- 1
- Primary Endpoint
- The time to attain first complete remission (CR)
- Last Updated
- 7 years ago
Overview
Brief Summary
The purpose of this prospective study is to determine the efficacy and safety of Rituximab plus Bortezomib in patients with newly diagnosed acquired hemophilia A.
Detailed Description
This is a prospective, single-arm, open-label clinical trial to evaluate the efficacy and safety the regimen of Bortezomib with rituximab as first-line treatment to eradicate anti-factor VIII antibodies in newly diagnosed acquired hemophilia A. All of enrolled patients in this study will be injected Bortezomib plus rituximab. This study will be performed for about 2 years and approximately 22 patients will be enrolled in our insititution. After obtaining the written informed consent from the patients, the information of demographic and medical history will be collected and laboratory tests will be performed. Patients who meet the inclusion/exclusion criteria will be received the regimens: Bortezomib (1.3mg/m2 d1,4,8,11) with rituximab (375mg/m2 for one dose). The information of adverse events will be collected. In case that the evaluation of treatment response might be performed in regular clinical practice, those data will also be collected as well.
Investigators
Tienan Zhu
Associate chief physician
Peking Union Medical College Hospital
Eligibility Criteria
Inclusion Criteria
- •Must be ≥ 18 years;
- •Understand and voluntarily sign an ICD prior to any study related assessments/procedures are conducted;
- •Diagnosis of acquired hemophilia A；
- •acute bleeding episodes(≥once).
Exclusion Criteria
- •Uncontrolled systemic infection;
- •Allergy to rituximab;
- •Positive for Lupus anticoagulant;
- •Life expectancy \< 3 months;
- •Pregnant and breastfeeding women;
- •Neuropathy\>Grade 1;
- •Positive for Hepatitis B surface antigen or hepatitis C antibody or human immunodeficiency virus（HIV）antibody;
- •Patients with poor compliance；
- •Patient who is considered by the investigator not suitable for clinical study.
Arms & Interventions
Bortezomib +Rituximab
Bortezomib +Rituximab
Intervention: Rituximab
Bortezomib +Rituximab
Bortezomib +Rituximab
Intervention: Bortezomib
Outcomes
Primary Outcomes
The time to attain first complete remission (CR)
Time Frame: Last day of the treatment regimen (up to 3 months)
Complete remission defined as titer FVIII inhibitor lower than 0.6 Bethesda unit, factor VIII level\> 50% and no bleeding events without bypass treatments for 24 hours
Secondary Outcomes
- Adverse events(During 24 month)
- The time to durable treatment response(During 24 month)
- Overall survival(During 24 month)