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

Safety and Efficacy of Bortezomib Plus Dexamethasone for Acquired Pure Red Cell Aplasia Failure or Relapse After First-line Treatment: A Prospective Phase II Trial

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.

Study Overview

• Status: Terminated
• Conditions: Acquired Pure Red Cell Aplasia
• Intervention / Treatment: Drug: bortezomib/dexamethasone

• Study Type: Interventional
• Enrollment (Actual): 16
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Jun Shi, PhD; Phone Number: (86)2223900913; Email: shijun@ihcams.ac.cn
• Study Contact Backup: Name: Lele Zhang, MD; Phone Number: (86)15811139278; Email: zhanglele@ihcams.ac.cn

Study Locations

• China
  • Tianjin, China
    • Regenerative Medicine Center
  • Henan
    • Zhoukou, Henan, China
      • Zhoukou Central Hospital
  • Shandong
    • Tai'an, Shandong, China
      • The Second Affilated Hospital of Shandong First Medical University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: bortezomib/dexamethasone; Enrolled patients will receive the combination therapy of bortezomib and dexamethasone.	Drug: bortezomib/dexamethasone; Enrolled patients would receive the combination therapy of bortezomib and dexamethasone.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Overall response rate	within 12 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Frequency and severity of adverse events and severe adverse events	within 12 weeks
Relapse free survival	within 24 and 48 weeks

Collaborators and Investigators

• Sponsor: Institute of Hematology & Blood Diseases Hospital, China
• Investigators: Principal Investigator: Jun Shi, PhD, Institute of Hematology & Blood Diseases Hospital, China

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): September 13, 2020
• Primary Completion (Actual): January 31, 2024
• Study Completion (Actual): February 6, 2024

Study Registration Dates

• First Submitted: June 5, 2020
• First Submitted That Met QC Criteria: June 5, 2020
• First Posted (Actual): June 9, 2020

Study Record Updates

• Last Update Posted (Estimated): February 7, 2024
• Last Update Submitted That Met QC Criteria: February 5, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: Acquired Pure Red Cell Aplasia; PRCA; Failure or Relapse after First-line Treatment
• Additional Relevant MeSH Terms: Pathologic Processes; Disease Attributes; Hematologic Diseases; Anemia; Recurrence; Red-Cell Aplasia, Pure; Physiological Effects of Drugs; Autonomic Agents; Peripheral Nervous System Agents; Anti-Inflammatory Agents; Antineoplastic Agents; Antiemetics; Gastrointestinal Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Dexamethasone; Bortezomib
• Other Study ID Numbers: IHBDH-IIT2020010

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No