Epunamin Combined With DECP for Relapsed/Refractory Multiple Myeloma

Background:

Multiple Myeloma remains an incurable plasma cell malignancy, and patients with relapsed/refractory disease continue to face limited therapeutic options and poor clinical outcomes. Epunamin, in combination with a DECP-based regimen, may provide a novel therapeutic strategy in this setting. However, evidence from real-world clinical practice remains limited.

Methods:

This is a multicenter, single-arm, real-world observational clinical study designed to evaluate the efficacy and safety of Epunamin combined with a DECP-based regimen in patients with relapsed/refractory multiple myeloma (RRMM). A total of 48 eligible patients aged 18-75 years who meet the revised International Myeloma Working Group diagnostic criteria and have received at least one prior systemic therapy will be enrolled. Eligible participants must have an Eastern Cooperative Oncology Group performance status of 0-3 and provide written informed consent.

Major exclusion criteria include severe cardiac, pulmonary, hepatic, or renal dysfunction; unresolved toxicities from prior chemotherapy; grade ≥2 peripheral neuropathy; active severe infection; plasma cell leukemia; concurrent malignancies diagnosed within 2 years; HIV infection; pregnancy or lactation; and known hypersensitivity to study drugs.

Endpoints:

The primary endpoint is overall response rate (ORR) after completion of four treatment cycles. Secondary endpoints include very good partial response (VGPR), complete response (CR), stringent complete response (sCR), minimal residual disease (MRD), duration of response (DOR), and time to next treatment (TTNT), based on laboratory findings and revised IMWG criteria.

Statistical Analysis:

Continuous variables will be summarized using mean, median, standard deviation, minimum, and maximum values. Categorical variables will be presented as counts and percentages. Normally distributed continuous variables will be analyzed using Student's t-test or analysis of variance, while non-normally distributed variables will be compared using rank-sum tests. Categorical variables will be analyzed using the chi-square test or Fisher's exact test. Ordinal variables will be assessed using Ridit analysis or nonparametric rank-sum tests. Survival outcomes, including progression-free survival (PFS) and overall survival (OS), will be estimated using the Kaplan-Meier method, with comparisons performed using the log-rank test. All statistical analyses will be two-sided, with P < 0.05 considered statistically significant.

Study Period:

Patient enrollment and follow-up are planned from October 2025 to September 2027.

Inclusion criteria

Adults aged 18 to 75 years with relapsed/refractory Multiple Myeloma diagnosed according to revised IMWG criteria.

Received at least one prior systemic treatment regimen. Eastern Cooperative Oncology Group Performance Status score of 0-3. Able to understand treatment-related changes and management options during therapy and follow-up, with good treatment adherence and follow-up compliance.

Provided written informed consent. -

Exclusion criteria

Severe dysfunction of major organs, including cardiac, pulmonary, hepatic, or renal impairment, defined as left ventricular ejection fraction <50%, diffusion capacity for carbon monoxide <50% of predicted value due to chronic respiratory disease, serum bilirubin >2 mg/dL, alanine aminotransferase or aspartate aminotransferase >2.5 × upper limit of normal, or estimated glomerular filtration rate <30 mL/min.

Toxicities from prior chemotherapy not recovered to baseline or grade ≤1. Peripheral neuropathy grade ≥2, or grade 1 with pain. Major surgery, radiotherapy, infection requiring systemic antibiotic treatment, or other severe infection within 14 days before enrollment.

High-risk plasma cell leukemia with peripheral blood plasma cells ≥20%. Psychiatric disorders, cognitive impairment, or other conditions affecting self-control or study compliance.

Pregnant or breastfeeding women, or fertile patients unwilling to use adequate contraception during the study. Male patients unwilling to use effective contraception or refrain from sperm donation during treatment and for 3 months after the last dose.

Diagnosis or treatment of another malignancy within 2 years before enrollment. Known hypersensitivity to Epunamin, Dexamethasone, Cisplatin, Etoposide, or Cyclophosphamide.

Human Immunodeficiency Virus Infection positive. Participation in another clinical trial within 30 days before enrollment or during the study period.

Any condition considered unsuitable for participation by the investigator.