Acquired Pure Red Cell AplasiaPhase 2BID-PERALNCT04423367
Bortezomib Plus Dexamethasone for Acquired Pure Red Cell Aplasia Failure or Relapse After First-line Treatment
A Phase 2 study testing bortezomib/dexamethasone in people with Acquired Pure Red Cell Aplasia, run by Institute of Hematology & Blood Diseases Hospital, China at 3 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Acquired Pure Red Cell Aplasia
- What is being tested?
- bortezomib/dexamethasone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 3 sitesChina
- Study dates
- 13 Sep 2020to 30 Sep 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
3 sites in 1 country.
China3 sites
The Second Affilated Hospital of Shandong First Medical University
Tai’an, Shandong
Regenerative Medicine Center
Tianjin
Zhoukou Central Hospital
Zhoukou, Henan
About this study
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
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
- Being tested
- bortezomib/dexamethasone
- Keywords
- Acquired Pure Red Cell Aplasia, PRCA, Failure or Relapse after First-line Treatment
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
bortezomib/dexamethasoneDrug
Enrolled patients would receive the combination therapy of bortezomib and dexamethasone.
| Study group | Role | What they receive |
|---|---|---|
| bortezomib/dexamethasone | Experimental | Enrolled patients will receive the combination therapy of bortezomib and dexamethasone. Receives: Drug: bortezomib/dexamethasone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate
Measured: within 12 weeks
Secondary outcomes (2)
Frequency and severity of adverse events and severe adverse events
Measured: within 12 weeks
Relapse free survival
Measured: within 24 and 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2020
- Study start13 Sep 2020
- Primary completion31 Jul 2024
- Study completion30 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Acquired Pure Red Cell AplasiaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jun Shi, PhD, Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04423367
- Sponsor's protocol ID
- IIT2020010
- Acronym
- BID-PERAL
- Status last verified
- Nov 2024
- First submitted
- 5 Jun 2020
- Last updated
- 11 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
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.
PubMed 33957358Zhang L, Shen Y, Li R, Pan H, Gao Z, Li W, Zhao J, Liang Q, Fang L, Shi J. Treatment-free survival to bortezomib combined with dexamethasone in patients with refractory/relapsed pure red cell aplasia: a prospective clinical study. Ann Med. 2025 Dec;57(1):2540016. doi: 10.1080/07853890.2025.2540016. Epub 2025 Aug 7.
PubMed 40772317
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04423367
- Acronym / sponsor ID
- BID-PERAL / IIT2020010
- Conditions
- Acquired Pure Red Cell Aplasia
- Keywords
- Acquired Pure Red Cell Aplasia, PRCA, Failure or Relapse after First-line Treatment
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 13 Sep 2020 (actual)
- Primary completion
- 31 Jul 2024 (actual)
- Study completion
- 30 Sep 2024 (actual)
- First posted
- 9 Jun 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| bortezomib/dexamethasone | Experimental | Drug: bortezomib/dexamethasoneEnrolled patients will receive the combination therapy of bortezomib and dexamethasone. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (15 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| bortezomib/dexamethasoneEnrolled patients would receive the combination therapy of bortezomib and dexamethasone. | Drug | bortezomib/dexamethasone |
Primary outcome
Overall response rate
Time frame: within 12 weeks
2 secondary outcomes
Frequency and severity of adverse events and severe adverse events
Time frame: within 12 weeks
Relapse free survival
Time frame: within 24 and 48 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Feb 2026.
- Background: PubMed 33957358Zhang 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.
- Derived from this study: PubMed 40772317Zhang L, Shen Y, Li R, Pan H, Gao Z, Li W, Zhao J, Liang Q, Fang L, Shi J. Treatment-free survival to bortezomib combined with dexamethasone in patients with refractory/relapsed pure red cell aplasia: a prospective clinical study. Ann Med. 2025 Dec;57(1):2540016. doi: 10.1080/07853890.2025.2540016. Epub 2025 Aug 7.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jun Shi, PhD principal investigator
Institute of Hematology & Blood Diseases Hospital, China
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing bortezomib/dexamethasone in people with Acquired Pure Red Cell Aplasia, run by Institute of Hematology & Blood Diseases Hospital, China at 3 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (time frame: within 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04423367, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Feb 2026.