Study to Evaluate Rituximab and Bortezomib in Patients With Newly Diagnosed Acquired Hemophilia A

Phase 2

• Conditions: Acquired Hemophilia A
• Interventions: Drug: Bortezomib; Drug: Rituximab

• Registration Number: NCT03700229
• Lead Sponsor: Peking Union Medical College Hospital

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.

Study Timeline

• Study First Submitted: 2018-10-04
• Study First Submitted QC: 2018-10-06
• Study First Posted: 2018-10-09
• Study Start: 2018-10-10
• Status Verified: 2018-11
• Last Update Submitted: 2018-11-20
• Last Update Posted: 2018-11-23
• Primary Completion: 2019-10-15
• Study Completion: 2020-04-15

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 22

Inclusion Criteria

1. Must be ≥ 18 years;
2. Understand and voluntarily sign an ICD prior to any study related assessments/procedures are conducted;
3. Diagnosis of acquired hemophilia A；
4. acute bleeding episodes(≥once).

Exclusion Criteria

1. Uncontrolled systemic infection;
2. Allergy to rituximab;
3. Positive for Lupus anticoagulant;
4. Life expectancy < 3 months;
5. Pregnant and breastfeeding women;
6. Neuropathy>Grade 1;
7. Positive for Hepatitis B surface antigen or hepatitis C antibody or human immunodeficiency virus（HIV）antibody;
8. Patients with poor compliance；
9. Patient who is considered by the investigator not suitable for clinical study.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Bortezomib +Rituximab	Bortezomib	Bortezomib +Rituximab
Bortezomib +Rituximab	Rituximab	Bortezomib +Rituximab

Primary Outcome Measures

Name	Time	Method
The time to attain first complete remission (CR)	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 Outcome Measures

Name	Time	Method
Adverse events	During 24 month	Include major bleeding, infection, nerve toxicity, and so on.
The time to durable treatment response	During 24 month	The time to durable treatment response was defined as the time from date of achieving CR until the date of relapse or date of death from any cause（whichever came first）
Overall survival	During 24 month	Overall survival was defined as the time from date of first diagnosis until the date of death from any cause，and death from any cause will be recorded.

Trial Locations

Locations (1)

Peking Union Medical College Hospital; 🇨🇳 Beijing, Beijing, China