Clinical trial NCT04423367: Bortezomib Plus Dexamethasone for Acquired Pure Red Cell Aplasia Failure or Relapse After First-line Treatment
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Acquired Pure Red Cell Aplasia
Keywords: Acquired Pure Red Cell Aplasia
PRCA
Failure or Relapse after First-line Treatment
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
First posted: 2020-06-09
Enrollment: 18
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Overall response rate
timeFrame: within 12 weeks
Secondary outcomes:
measure: Frequency and severity of adverse events and severe adverse events
timeFrame: within 12 weeks
measure: Relapse free survival
timeFrame: within 24 and 48 weeks
Other outcomes:
Locations:
city: Zhoukou
country: China
facility: Zhoukou Central Hospital
state: Henan
city: Tai'an
country: China
facility: The Second Affilated Hospital of Shandong First Medical University
state: Shandong
city: Tianjin
country: China
facility: Regenerative Medicine Center
Study officials:
affiliation: Institute of Hematology & Blood Diseases Hospital, China
name: Jun Shi, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.