- ICH GCP
- US Clinical Trials Registry
- Klinisk utprøving NCT07652879
KCD (Carfilzomib/Cyclophosphamide/Dexamethasone) Regimen for the Treatment of Newly Diagnosed POEMS Syndrome
16. juni 2026 oppdatert av: Haiyan He, Shanghai Changzheng Hospital
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.
Studieoversikt
Status
Rekruttering
Forhold
Intervensjon / Behandling
Detaljert beskrivelse
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).
Studietype
Intervensjonell
Registrering (Antatt)
20
Fase
- Fase 2
Kontakter og plasseringer
Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.
Studiekontakt
- Navn: Haiyan He, Dr.
- Telefonnummer: +86 13661513012
- E-post: doctorhehaiyan@126.com
Studer Kontakt Backup
- Navn: Xuerou Yu
Studiesteder
-
-
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Shanghai, Kina
- Rekruttering
- Shanghai Changzheng Hospital
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Deltakelseskriterier
Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Voksen
- Eldre voksen
Tar imot friske frivillige
Nei
Beskrivelse
Inclusion Criteria:
- 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;
- 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.
Exclusion Criteria:
- 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);
- 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.
Studieplan
Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Eksperimentell: carfilzomib, cyclophosphamide and dexamethasone
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
|
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
Andre navn:
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Overall hematological response rate
Tidsramme: 2 years
|
hematological response rate:
|
2 years
|
|
Overall VEGF response rate
Tidsramme: 2 years
|
VEGF response rate:
|
2 years
|
|
Overall neurological response rate
Tidsramme: 2 years
|
Neurological response rate: Neurologic improvement assessed by neurophysiologic examination, modified Rankin Scale, or Overall Neuropathy Limitations Scale (ONLS).
|
2 years
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
the incidence of adverse events and severe adverse events
Tidsramme: 2 years
|
Major Adverse Events
|
2 years
|
|
the 2-year overall survival rate
Tidsramme: 2 years
|
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).
|
2 years
|
|
Two-year progression-free survival
Tidsramme: 2 years
|
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)
|
2 years
|
Samarbeidspartnere og etterforskere
Det er her du vil finne personer og organisasjoner som er involvert i denne studien.
Sponsor
Etterforskere
- Hovedetterforsker: Haiyan He, Dr., Shanghai Changzheng Hospital
Studierekorddatoer
Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.
Studer hoveddatoer
Studiestart (Antatt)
15. juni 2026
Primær fullføring (Antatt)
1. januar 2028
Studiet fullført (Antatt)
1. januar 2028
Datoer for studieregistrering
Først innsendt
24. mai 2026
Først innsendt som oppfylte QC-kriteriene
16. juni 2026
Først lagt ut (Faktiske)
17. juni 2026
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
17. juni 2026
Siste oppdatering sendt inn som oppfylte QC-kriteriene
16. juni 2026
Sist bekreftet
1. juni 2026
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
- Sykdommer i nervesystemet
- Nevromuskulære sykdommer
- Sykdommer i immunsystemet
- Sykdommer i det perifere nervesystemet
- Hematologiske sykdommer
- Immunproliferative lidelser
- Medfødte abnormiteter
- Abnormiteter, flere
- Paraproteinemier
- Blodproteinforstyrrelser
- Polynevropatier
- Medfødte, arvelige og neonatale sykdommer og abnormiteter
- Hemic og lymfatiske sykdommer
- DIKT syndrom
- Organiske kjemikalier
- Hydrokarboner
- Polysykliske forbindelser
- Gravadienes
- Gravaner
- Steroider
- Smeltede ringforbindelser
- Steroider, fluorert
- Fosforamid -sennep
- Nitrogen sennepsforbindelser
- Sennepsforbindelser
- Hydrokarboner, halogenert
- Fosforamider
- Organofosforforbindelser
- Gravadienetrioler
- Deksametason
- Cyklofosfamid
- carfilzomib
Andre studie-ID-numre
- CZ-POEMS-01
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
NEI
IPD-planbeskrivelse
POEMS syndrome is a rare disease, and its clinical data are highly valuable
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Nei
Studerer et amerikansk FDA-regulert enhetsprodukt
Nei
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