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

NCT ID: NCT04423367

Last Updated: 2024-11-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 18 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-09-13
• Study Completion Date: 2024-09-30

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.

Conditions

Acquired Pure Red Cell Aplasia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

bortezomib/dexamethasone

Enrolled patients will receive the combination therapy of bortezomib and dexamethasone.

Group Type: EXPERIMENTAL

bortezomib/dexamethasone

Intervention Type: DRUG

Enrolled patients would receive the combination therapy of bortezomib and dexamethasone.

Interventions

bortezomib/dexamethasone

Enrolled patients would receive the combination therapy of bortezomib and dexamethasone.

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Institute of Hematology & Blood Diseases Hospital, China

OTHER

Sponsor Role: lead

Responsible Party

Jun Shi

Director, Regenerative Medicine Clinic Center

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Jun Shi, PhD

Role: PRINCIPAL_INVESTIGATOR

Institute of Hematology & Blood Diseases Hospital, China

Locations

Zhoukou Central Hospital

Zhoukou, Henan, China

The Second Affilated Hospital of Shandong First Medical University

Tai’an, Shandong, China

Regenerative Medicine Center

Tianjin, , China

Countries

China

References

Zhang L, Chen N, Xu Z, Liang Q, Pan H, Zhao J, Fang L, Shi J. Good treatment-free survival of monoclonal gammopathy of undetermined significance associated pure red cell aplasia after bortezomib plus dexamethasone. Blood Cells Mol Dis. 2021 Jul;89:102573. doi: 10.1016/j.bcmd.2021.102573. Epub 2021 Apr 27.

Reference Type: BACKGROUND

PMID: 33957358 (View on PubMed)

Other Identifiers

IIT2020010

Identifier Type: -

Identifier Source: org_study_id