POEMS SyndromePhase 2NCT07652879
KCD (Carfilzomib/Cyclophosphamide/Dexamethasone) Regimen for the Treatment of Newly Diagnosed POEMS Syndrome
A Phase 2 study testing CARFILZOMIB, CYCLOPHOSPHAMIDE, DEXAMETHASONE in people with POEMS Syndrome, run by Shanghai Changzheng Hospital at 1 site in China.
Trial summary
This study is a single-center, prospective, open-label clinical study to evaluate the efficacy and safety of KCD(Carfilzomib/Cyclophosphamide/Dexamethasone) regimen in subjects with newly diagnosed POEMS Syndrome. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- POEMS Syndrome
- What is being tested?
- CARFILZOMIB, CYCLOPHOSPHAMIDE, DEXAMETHASONE
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 15 Jun 2026to Jan 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Newly diagnosed POEMS syndrome meeting the Dispenzieri diagnostic criteria (2023 version);
- Age 18-75 years;
- ECOG performance status 0-3, with an estimated life expectancy >3 months;
- No active infective diseases;
- No prior anti-POEMS therapy except for corticosteroids;
- No severe organic impairment of major organs, meeting the following laboratory requirements: creatinine clearance ≥40 mL/min, total bilirubin ≤1.5 × upper limit of normal (ULN); AST and ALT ≤2.5 × ULN; cardiac enzymes <2 × ULN; left ventricular ejection fraction within normal range on echocardiography, and no clinically significant electrocardiogram abnormalities;
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- Absolute neutrophil count ≥1.5 × 10^9/L without prior growth factor support; platelet count ≥50 × 10^9/L without platelet transfusion within 7 days prior to screening; hemoglobin ≥60 g/L;
- Ability to swallow and take medication orally;
- Completion of all screening and assessments as outlined in the study protocol;
- Signed informed consent for chemotherapy.
Who cannot join
- POEMS syndrome complicated by multiple myeloma, light chain amyloidosis, or Waldenström macroglobulinemia;
- HIV positivity, or active hepatitis A, hepatitis B, or hepatitis C infection; or hepatitis B virus DNA >10^2 copies/mL;
- Concurrent severe unstable medical conditions, including heart failure, renal failure, liver failure, bleeding disorders, arterial/venous thrombotic events within 6 months, uncontrolled diabetes mellitus, or a history of active hemorrhagic cystitis;
- History of autoimmune diseases (e.g., Crohn's disease, rheumatoid arthritis, systemic lupus erythematosus) within the past 2 years that caused end-organ damage or required systemic immunosuppressive or disease-modifying therapy;
- Severe active infections (e.g., untreated tuberculosis, pulmonary aspergillosis);
- Presence of other malignancies (except non-melanoma skin cancer, in situ cervical, bladder, or breast cancer with disease-free survival >5 years);
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- Epilepsy requiring medication, dementia, or other mental status abnormalities that interfere with understanding or complying with the study protocol;
- Drug use, medical, psychological, or social conditions that may interfere with study participation or outcome assessment;
- Pregnancy or breastfeeding;
- Any condition deemed by the investigator to make the patient unsuitable for enrollment.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Xuerou Yu contact
About this study
Official title: A Single-center, Prospective, Open-label Investigation of the KCD (Carfilzomib/Cyclophosphamide/Dexamethasone) Regimen for the Treatment of Newly Diagnosed POEMS Syndrome
This study is a single-center, prospective, open-label clinical study to evaluate the efficacy and safety of KCD(Carfilzomib/Cyclophosphamide/Dexamethasone) regimen in subjects with newly diagnosed POEMS Syndrome.
Read the detailed description
POEMS syndrome is a rare plasma cell disorder, with an incidence of approximately 0.3 per 100,000 individuals. Its typical clinical manifestations encompass peripheral neuropathy, organomegaly, endocrine abnormalities, M -proteinemia, and cutaneous alterations, frequently accompanied by papilledema, fluid retention, thrombocytosis, osteosclerosis, and elevated levels of vascular endothelial growth factor (VEGF). Plasma cell clonal proliferation and VEGF overexpression are regarded as the key pathogenic mechanisms of POEMS syndrome. Currently, there is no standardized treatment protocol for POEMS syndrome, and anti-plasma cell therapy remains the primary therapeutic approach. Clinically recommended treatments involve alkylating agents, immunomodulators, proteasome inhibitors, and autologous hematopoietic stem cell transplantation (ASCT). Carfilzomib, a second-eneration proteasome inhibitor, has been extensively utilized in multiple myeloma. The evidence supporting the use of carfilzomib in POEMS syndrome is restricted to case reports and small-scale retrospective studies, and prospective clinical trials evaluating carfilzomib-based regimens as first-line therapy are still lacking. Therefore, this study designed a centralized, prospective, open-label clinical trial to evaluate the KCD regimen(carfilzomib + cyclophosphamide + dexamethasone) in patients with newly-diagnosed POEMS syndrome. This study plans to enroll 20 subjects, all subjects will receive KCD regimen for 6-8 cycles. The primary endpoints include overall hematological response rate, neurological response rate, and vascular endothelial growth factor (VEGF) response rate. Secondary endpoints include hematological and non-hematological toxicities, progression-free survival (PFS), and overall survival (OS).
- Conditions
- POEMS Syndrome
- Being tested
- CARFILZOMIB, CYCLOPHOSPHAMIDE, DEXAMETHASONE
- Keywords
- POEMS syndrome, Carfilzomib
- Medical subject headings
- POEMS Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CARFILZOMIB, CYCLOPHOSPHAMIDE, DEXAMETHASONEDrug
Also called KCD
carfilzomib at a dose of 27 mg/m2 (20 mg/m2 only in the first infusion) intravenously (iv) on days 1, 8, and 15,cyclophosphamide at a dose of 200 mg/m2 iv on days 1, 8 and 15 and dexamethasone at a dose of 20 mg (10 mg for patients >75 years) days 1, 2, 8, 9, 15 and 16
| Study group | Role | What they receive |
|---|---|---|
| carfilzomib, cyclophosphamide and dexamethasone | Experimental | carfilzomib at a dose of 27 mg/m2 (20 mg/m2 only in the first infusion) intravenously (iv) on days 1, 8, and 15, cyclophosphamide at a dose of 200 mg/m2 iv on days 1, 8 and 15, and dexamethasone at a dose of 20 mg (10 mg for patients >75 years) days 1, 2, 8, 9, 15 and 16 in 28 days cycles Receives: Drug: CARFILZOMIB, CYCLOPHOSPHAMIDE, DEXAMETHASONE |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall hematological response rate
hematological response rate:
- Complete Remission (CR_H): Normal bone marrow; negative serum and urine immunofixation electrophoresis; disappearance of M-protein.
- Very Good Partial Remission (VGPR_H): Reduction of M-protein by >90% (baseline M-protein ≥5 g/L).
- Partial Remission (PR_H): Reduction of M-protein by ≥50% (baseline M-protein ≥10 g/L).
- No Response (NR_H): Failure to meet criteria for PR_H.
- Progressive Disease (PD): Reappearance of M-protein in serum and/or urine, or an increase of >25% from the lowest level (with absolute M-protein increase ≥5 g/L).
Measured: 2 years
Overall VEGF response rate
VEGF response rate:
- Complete Remission (CR_V): Normalization of serum VEGF (elevation typically defined as serum VEGF >2 times the upper limit of normal).
- Partial Remission (PR_V): Reduction of VEGF by ≥50%.
- No Response (NR_V): Failure to meet criteria for PR_V.
- Progressive Disease (PD): Persistent (≥2 consecutive measurements) elevation of VEGF , or persistent elevation of VEGF by 50% from the post-treatment nadir.
Measured: 2 years
Overall neurological response rate
Neurological response rate:
Neurologic improvement assessed by neurophysiologic examination, modified Rankin Scale, or Overall Neuropathy Limitations Scale (ONLS).
- Complete response: 0 point;
- Improvement: Improved by 1 point;
- Progression: Worsened by 1 point
Measured: 2 years
Secondary outcomes (3)
the incidence of adverse events and severe adverse events
Major Adverse Events
- Hematologic toxicity: neutropenia, thrombocytopenia, etc.
- Infections: bacterial pneumonia, sepsis, etc.
- Worsening of neuropathy
- Capillary leak syndrome
- Thrombotic events Grading and Definition of Serious Adverse Event All adverse events are graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0. Gr
Measured: 2 years
the 2-year overall survival rate
Two-year overall survival (2-year OS) is defined as the proportion of patients who remain alive at two years following the start of treatment (or from the date of diagnosis).
Measured: 2 years
Two-year progression-free survival
Two-year progression-free survival (2-year PFS) is defined as the proportion of patients who remain alive and have not experienced disease progression at two years following the start of treatment (or from the time of diagnosis)
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jun 2026
- Study start15 Jun 2026 (estimated)
- Primary completionJan 2028 (estimated)
- Study completionJan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for POEMS SyndromeStudies of the same condition, open and under way.
- More trials run by Shanghai Changzheng HospitalThe 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
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Changzheng Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Haiyan He, Shanghai Changzheng Hospital
- Investigators
- Haiyan He, Dr., Principal investigator, Shanghai Changzheng Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07652879
- Sponsor's protocol ID
- CZ-POEMS-01
- Status last verified
- Jun 2026
- First submitted
- 24 May 2026
- Last updated
- 17 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: POEMS syndrome is a rare disease, and its clinical data are highly valuable
Study at a glance
From the registry record, last updated by the sponsor on 17 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-center, Prospective, Open-label Investigation of the KCD (Carfilzomib/Cyclophosphamide/Dexamethasone) Regimen for the Treatment of Newly Diagnosed POEMS Syndrome
- ClinicalTrials.gov ID
- NCT07652879
- Acronym / sponsor ID
- CZ-POEMS-01
- Conditions
- POEMS Syndrome
- Keywords
- POEMS syndrome, Carfilzomib
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Shanghai Changzheng HospitalUniversity, hospital or other
- Study start
- 15 Jun 2026 (estimated)
- Primary completion
- Jan 2028 (estimated)
- Study completion
- Jan 2028 (estimated)
- First posted
- 17 Jun 2026 (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 study is a single-center, prospective, open-label clinical study to evaluate the efficacy and safety of KCD(Carfilzomib/Cyclophosphamide/Dexamethasone) regimen in subjects with newly diagnosed POEMS Syndrome.
Detailed description
POEMS syndrome is a rare plasma cell disorder, with an incidence of approximately 0.3 per 100,000 individuals. Its typical clinical manifestations encompass peripheral neuropathy, organomegaly, endocrine abnormalities, M -proteinemia, and cutaneous alterations, frequently accompanied by papilledema, fluid retention, thrombocytosis, osteosclerosis, and elevated levels of vascular endothelial growth factor (VEGF). Plasma cell clonal proliferation and VEGF overexpression are regarded as the key pathogenic mechanisms of POEMS syndrome. Currently, there is no standardized treatment protocol for POEMS syndrome, and anti-plasma cell therapy remains the primary therapeutic approach. Clinically recommended treatments involve alkylating agents, immunomodulators, proteasome inhibitors, and autologous hematopoietic stem cell transplantation (ASCT). Carfilzomib, a second-eneration proteasome inhibitor, has been extensively utilized in multiple myeloma. The evidence supporting the use of carfilzomib in POEMS syndrome is restricted to case reports and small-scale retrospective studies, and prospective clinical trials evaluating carfilzomib-based regimens as first-line therapy are still lacking. Therefore, this study designed a centralized, prospective, open-label clinical trial to evaluate the KCD regimen(carfilzomib + cyclophosphamide + dexamethasone) in patients with newly-diagnosed POEMS syndrome. This study plans to enroll 20 subjects, all subjects will receive KCD regimen for 6-8 cycles. The primary endpoints include overall hematological response rate, neurological response rate, and vascular endothelial growth factor (VEGF) response rate. Secondary endpoints include hematological and non-hematological toxicities, progression-free survival (PFS), and overall survival (OS).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| carfilzomib, cyclophosphamide and dexamethasone | Experimental | Drug: CARFILZOMIB, CYCLOPHOSPHAMIDE, DEXAMETHASONEcarfilzomib at a dose of 27 mg/m2 (20 mg/m2 only in the first infusion) intravenously (iv) on days 1, 8, and 15, cyclophosphamide at a dose of 200 mg/m2 iv on days 1, 8 and 15, and dexamethasone at a dose of 20 mg (10 mg for patients >75 years) days 1, 2, 8, 9, 15 and 16 in 28 days cycles |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Newly diagnosed POEMS syndrome meeting the Dispenzieri diagnostic criteria (2023 version);
- Age 18-75 years;
- ECOG performance status 0-3, with an estimated life expectancy >3 months;
- No active infective diseases;
- No prior anti-POEMS therapy except for corticosteroids;
Key exclusion criteria (10 listed)
- POEMS syndrome complicated by multiple myeloma, light chain amyloidosis, or Waldenström macroglobulinemia;
- HIV positivity, or active hepatitis A, hepatitis B, or hepatitis C infection; or hepatitis B virus DNA >10^2 copies/mL;
- Concurrent severe unstable medical conditions, including heart failure, renal failure, liver failure, bleeding disorders, arterial/venous thrombotic events within 6 months, uncontrolled diabetes mellitus, or a history of active hemorrhagic cystitis;
- History of autoimmune diseases (e.g., Crohn's disease, rheumatoid arthritis, systemic lupus erythematosus) within the past 2 years that caused end-organ damage or required systemic immunosuppressive or disease-modifying therapy;
- Severe active infections (e.g., untreated tuberculosis, pulmonary aspergillosis);
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 |
|---|---|---|
| CARFILZOMIB, CYCLOPHOSPHAMIDE, DEXAMETHASONEAlso: KCDcarfilzomib at a dose of 27 mg/m2 (20 mg/m2 only in the first infusion) intravenously (iv) on days 1, 8, and 15,cyclophosphamide at a dose of 200 mg/m2 iv on days 1, 8 and 15 and dexamethasone at a dose of 20 mg (10 mg for patients >75 years) days 1, 2, 8, 9, 15 and 16 | Drug | carfilzomib, cyclophosphamide and dexamethasone |
Primary outcome
Overall hematological response rate
Time frame: 2 years
hematological response rate: * Complete Remission (CR_H): Normal bone marrow; negative serum and urine immunofixation electrophoresis; disappearance of M-protein. * Very Good Partial Remission (VGPR_H): Reduction of M-protein by >90% (baseline M-protein ≥5 g/L). * Partial Remission (PR_H): Reduction of M-protein by ≥50% (baseline M-protein ≥10 g/L). * No Response (NR_H): Failure to meet criteria for PR_H. * Progressive Disease (PD): Reappearance of M-protein in serum and/or urine, or an increase of >25% from the lowest level (with absolute M-protein increase ≥5 g/L).
Overall VEGF response rate
Time frame: 2 years
VEGF response rate: * Complete Remission (CR_V): Normalization of serum VEGF (elevation typically defined as serum VEGF >2 times the upper limit of normal). * Partial Remission (PR_V): Reduction of VEGF by ≥50%. * No Response (NR_V): Failure to meet criteria for PR_V. * Progressive Disease (PD): Persistent (≥2 consecutive measurements) elevation of VEGF , or persistent elevation of VEGF by 50% from the post-treatment nadir.
Overall neurological response rate
Time frame: 2 years
Neurological response rate: Neurologic improvement assessed by neurophysiologic examination, modified Rankin Scale, or Overall Neuropathy Limitations Scale (ONLS). 1. Complete response: 0 point; 2. Improvement: Improved by 1 point; 3. Progression: Worsened by 1 point
3 secondary outcomes
the incidence of adverse events and severe adverse events
Time frame: 2 years
the 2-year overall survival rate
Time frame: 2 years
Two-year progression-free survival
Time frame: 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jun 2026.
- Individual participant dataSharing planned: No. POEMS syndrome is a rare disease, and its clinical data are highly valuable
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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.
Xuerou Yu contact
Haiyan He, Dr. principal investigator
Shanghai Changzheng Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CARFILZOMIB, CYCLOPHOSPHAMIDE, DEXAMETHASONE in people with POEMS Syndrome, run by Shanghai Changzheng Hospital at 1 site 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 10 inclusion and 10 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: looking for participants now, as of the sponsor's last update on 17 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Haiyan He, Dr. (+86 13661513012, doctorhehaiyan@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall hematological response rate (time frame: 2 years). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 Jun 2026.