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

NCT04423367 | Completed Phase 2

• 1 other identifier: IIT2020010
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 3

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

Keywords

Acquired Pure Red Cell Aplasia; PRCA; Failure or Relapse after First-line Treatment

Outcome Measures

Primary Outcomes (1)

• Overall response rate

  within 12 weeks

Secondary Outcomes (2)

• Frequency and severity of adverse events and severe adverse events

  within 12 weeks

• Relapse free survival

  within 24 and 48 weeks

Study Arms (1)

bortezomib/dexamethasone

EXPERIMENTAL

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

Drug: bortezomib/dexamethasone

Interventions

bortezomib/dexamethasone DRUG

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

bortezomib/dexamethasone

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (3)

Zhoukou Central Hospital

Zhoukou, Henan, China

Location

The Second Affilated Hospital of Shandong First Medical University

Tai’an, Shandong, China

Location

Regenerative Medicine Center

Tianjin, China

Location

Related Publications (1)

• 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.

  PMID: 33957358 BACKGROUND

MeSH Terms

Interventions

Bortezomib; Dexamethasone

Intervention Hierarchy (Ancestors)

Boronic Acids; Acids, Noncarboxylic; Acids; Inorganic Chemicals; Boron Compounds; Organic Chemicals; Pyrazines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated

Study Officials

• Jun Shi, PhD

  Institute of Hematology & Blood Diseases Hospital, China

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 5, 2020
• First Posted: June 9, 2020
• Study Start: September 13, 2020
• Primary Completion: July 31, 2024
• Study Completion: September 30, 2024
• Last Updated: February 11, 2026

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

CN (3)