A Non-interventional Study of Melphalan Flufenamide (Melflufen) (Pepaxti®) and Dexamethasone in Patients With Relapsed and/or Refractory Multiple Myeloma (R/RMM)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Multiple Myeloma. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Germany
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
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.
Primary outcome measures
- Disease control rate (DCR) [Time frame: max. 38 months (FPI - LPLV)]
Secondary outcome measures (12)
- Progression-free survival (PFS) [Time frame: max. 38 months (FPI - LPLV)]
- Overall survival (OS) [Time frame: max. 38 months (FPI - LPLV)]
- Overall response rate (ORR) [Time frame: max. 38 months (FPI - LPLV)]
- Duration of treatment with melflufen [Time frame: max. 38 months (FPI - LPLV)]
- Clinical benefit rate (CBR) [Time frame: max. 38 months (FPI - LPLV)]
- Time to next treatment (TTNT) [Time frame: max. 38 months (FPI - LPLV)]
- PFS2 [Time frame: max. 38 months (FPI - LPLV)]
- PFS of first subsequent treatment line [Time frame: max. 38 months (FPI - LPLV)]
- (Serious) adverse events ((S)AE) [Time frame: max. 38 months (FPI - LPLV)]
- (Serious) adverse drug reactions ((S)ADR) related to melphalan flufenamid [Time frame: max. 38 months (FPI - LPLV)]
- Types of treatments prior to Melflufen [Time frame: max. 38 months (FPI - LPLV)]
- Melfalan flufenamid starting dose [Time frame: max. 38 months (FPI - LPLV)]
Eligibility criteria
Inclusion criteria
- 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.
Exclusion criteria
- Participation in an interventional clinical trial (except follow-up)
- Patient unable to consent
- Contraindications according to current SmPC
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Germany · 1 center
- Praxis für interdisziplinäre Onkologie & Hämatologie — Freiburg im Breisgau
Identifiers
NCT: NCT07559799 · IOM-120481