A Non-interventional Study of Melphalan Flufenamide (Melflufen) (Pepaxti®) and Dexamethasone in Patients With Relapsed and/or Refractory Multiple Myeloma (R/RMM)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- iOMEDICO AG
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Disease control rate (DCR)
研究概览
简要总结
Multiple myeloma is the second most common hematologic malignancy in adults and despite the new therapies that have been developed in the last decades it remains incurable. Over the course of the disease, patients eventually become refractory to the various treatments. Therefore, new therapeutic options which utilize new mechanisms of action are essential.
Melphalan flufenamide (melflufen) represents such an additional therapeutic approach. Melflufen is a peptide-drug conjugate (PDC) which is highly lipophilic and rapidly incorporated into the tumor cells. Once inside the tumor cell, melflufen is hydrolyzed by peptidases, including aminopeptidases and esterases, to release its alkylator payload. The alkylating agent then induces DNA damage resulting in cell death.
Melphalan flufenamid in combination with Dexamethason was approved by the European Medicines Agency (EMA) in August 2022 for the treatment of patients with triple class refractory relapsed/refractory Multiple Myeloma who have received at least 3 prior lines of therapy. For patients with prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation.
The non-interventional study MARINA aims to address open scientific questions regarding the effectiveness, as well as therapy and safety management of melflufen in a real-world setting. By collecting comprehensive real-world data - including the Disease Control Rate (DCR) as a key endpoint, which is of most value for patients in this late disease stage - MARINA will investigate the therapeutic benefit of melflufen in routine clinical practice.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with R/RMM who have previously been treated with at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who relapsed on or after the last therapy
- •Indication and decision for fourth- or later-line treatment with melflufen (Pepaxti®) and dexamethasone, according to current SmPC as assessed by the treating physician
- •Signed and dated written informed consent*.
- •Treatment decision before inclusion into this non-interventional study
- •Age ≥18 years
- •Patients are allowed to be enrolled up to 28 days (+ 14 days) after their first dose of melflufen+dexamethasone,, but before any response assessment and second dose of melflufen+dexamethasone. These patients will not participate in the PRO assessments.
排除标准
- •Participation in an interventional clinical trial (except follow-up)
- •Patient unable to consent
- •Contraindications according to current SmPC
结局指标
主要结局
Disease control rate (DCR)
时间窗: max. 38 months (FPI - LPLV)
DCR is defined as the proportion of patients achieving a remission (i.e., sCR, CR, VGPR or PR or MR) or stable disease as best response according to local medical standards during treatment with melflufen. Patients without response measurement are considered non-responders.
次要结局
- Progression-free survival (PFS)(max. 38 months (FPI - LPLV))
- Overall survival (OS)(max. 38 months (FPI - LPLV))
- Overall response rate (ORR)(max. 38 months (FPI - LPLV))
- Duration of treatment with melflufen(max. 38 months (FPI - LPLV))
- Clinical benefit rate (CBR)(max. 38 months (FPI - LPLV))
- Time to next treatment (TTNT)(max. 38 months (FPI - LPLV))
- PFS2(max. 38 months (FPI - LPLV))
- PFS of first subsequent treatment line(max. 38 months (FPI - LPLV))
- (Serious) adverse events ((S)AE)(max. 38 months (FPI - LPLV))
- (Serious) adverse drug reactions ((S)ADR) related to melphalan flufenamid(max. 38 months (FPI - LPLV))
- Types of treatments prior to Melflufen(max. 38 months (FPI - LPLV))
- Melfalan flufenamid starting dose(max. 38 months (FPI - LPLV))
- Absolute dose intensity of melphalan flufenamid(max. 38 months (FPI - LPLV))
- Relative dose intensity of melphalan flufenamid(max. 38 months (FPI - LPLV))
- Type of dose modifications(max. 38 months (FPI - LPLV))
- Frequency of dose modifications(max. 38 months (FPI - LPLV))
- Reasons for dose modifications(max. 38 months (FPI - LPLV))
- Substances of subsequent antineoplastic treatment(max. 38 months (FPI - LPLV))
- Global health-related quality of life during course of treatment(max. 38 months (FPI - LPLV))
- Multiple myeloma related quality of life during course of treatment(max. 38 months (FPI - LPLV))
- Assessing parameters of physician treatment decision making(max. 38 months (FPI - LPLV))