Bortezomib Plus Dexamethasone for Acquired Pure Red Cell Aplasia Failure or Relapse After First-line Treatment

Phase 2

Completed

• Conditions: Acquired Pure Red Cell Aplasia
• Interventions: Drug: bortezomib/dexamethasone

• Registration Number: NCT04423367
• Lead Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Brief Summary

This is an open-label, single-arm study to evaluate the safety and efficacy of bortezomib plus dexamethasone for acquired pure red cell aplasia failure or relapse after first-line treatment.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-06-05
• Study First Submitted QC: 2020-06-05
• Study First Posted: 2020-06-09
• Study Start: 2020-09-13
• Primary Completion: 2024-07-31
• Study Completion: 2024-09-30
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-15
• Last Update Posted: 2024-11-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

• ECOG ≤ 2.
• Age from 18 to 70.
• Diagnosed with acquired pure red cell aplasia.
• Meets the criteria of first-line treatment failure or relapse.
• Organs in good function.
• Signed informed consent.

Exclusion Criteria

• Nursing woman.
• Active bacterial, virus, fungal or parasitic infection, including HIV infection, HBsAg or HBV DNA positive, HCV DNA positive, etc.
• Secondary PRCA caused by lymphoproliferative disorders, including large granular lymphocytic leukemia, waldenström macroglobulinemia, small lymphocytic lymphoma/chronic lymphocytic leukemia, etc.
• Secondary PRCA caused by either smoldering multiple myeloma or symptomatic multiple myeloma.
• Secondary PRCA caused by either ABO major mismatched stem cell transplantation or organ transplantation.
• Secondary PRCA caused by solid tumors except for thymoma.
• Secondary PRCA caused by drugs or pregnancy.
• Secondary PRCA caused by the B19 virus.
• Have contraindications for glucocorticoids, or unable to tolerate glucocorticoids for comorbidities.
• Previously received treatment in other trials within 4 weeks before enrollment.
• Previously treated with the proteasome inhibitor.
• Experience active hemorrhage condition, including gastrointestinal bleeding, respiratory tract bleeding and central nervous system bleeding within 2 months before enrollment or during bortezomib/dexamethasone treatment.
• Have a history of malignant tumors.
• Have a history of mental illness.
• Inability to understand or to follow study procedures.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
bortezomib/dexamethasone	bortezomib/dexamethasone	Enrolled patients will receive the combination therapy of bortezomib and dexamethasone.

Primary Outcome Measures

Name	Time	Method
Overall response rate	within 12 weeks

Secondary Outcome Measures

Name	Time	Method
Frequency and severity of adverse events and severe adverse events	within 12 weeks
Relapse free survival	within 24 and 48 weeks

Trial Locations

Locations (3)

The Second Affilated Hospital of Shandong First Medical University; 🇨🇳 Tai'an, Shandong, China

Regenerative Medicine Center; 🇨🇳 Tianjin, China

Zhoukou Central Hospital; 🇨🇳 Zhoukou, Henan, China