A Non-interventional Study of Melphalan Flufenamide (Melflufen) (Pepaxti®) and Dexamethasone in Patients With Relapsed and/or Refractory Multiple Myeloma (R/RMM) (MARINA)
27 aprile 2026 aggiornato da: iOMEDICO AG
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.
Panoramica dello studio
Stato
Non ancora reclutamento
Condizioni
Tipo di studio
Osservativo
Iscrizione (Stimato)
50
Contatti e Sedi
Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.
Contatto studio
- Nome: Sina Grebhardt, PhD
- Numero di telefono: +49 761 / 15 242-0
- Email: info@iomedico.com
Criteri di partecipazione
I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.
Criteri di ammissibilità
Età idonea allo studio
- Adulto
- Adulto più anziano
Accetta volontari sani
No
Metodo di campionamento
Campione non probabilistico
Popolazione di studio
Adult patients (≥18 years old) with relapsed and/or refractory multiple myeloma (R/RMM) pretreated with a proteasome inhibitor, an immunomodulatory drug and a CD38 antibody with decision for treatment with melflufen (Pepaxti®) and dexamethasone in fourth or later line according to SmPC.
Descrizione
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
Piano di studio
Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.
Come è strutturato lo studio?
Dettagli di progettazione
Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Disease control rate (DCR)
Lasso di tempo: 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.
|
max. 38 months (FPI - LPLV)
|
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Progression-free survival (PFS)
Lasso di tempo: max. 38 months (FPI - LPLV)
|
PFS is defined as the time from start of melflufen treatment until first progression or death from any cause, whichever comes first.
Patients without disease progression or death at the time of analysis will be censored at their date of last contact.
PFS will be calculated using the Kaplan-Meier method.
|
max. 38 months (FPI - LPLV)
|
|
Overall survival (OS)
Lasso di tempo: max. 38 months (FPI - LPLV)
|
OS is defined as the time from start of melflufen treatment until death from any cause.
Patients without documented death will be censored with their date of last contact.
OS will be calculated using the Kaplan-Meier method.
|
max. 38 months (FPI - LPLV)
|
|
Overall response rate (ORR)
Lasso di tempo: max. 38 months (FPI - LPLV)
|
ORR is defined as the proportion of patients achieving a remission (sCR, CR, VGPR or PR or MR) as best response according to local medical standards.
Patients without response measurement are considered non-responders.
|
max. 38 months (FPI - LPLV)
|
|
Duration of treatment with melflufen
Lasso di tempo: max. 38 months (FPI - LPLV)
|
Duration of melflufen treatment will be calculated in weeks as the time from first application to the last documented application of melflufen.
|
max. 38 months (FPI - LPLV)
|
|
Clinical benefit rate (CBR)
Lasso di tempo: max. 38 months (FPI - LPLV)
|
CBR is defined as the proportion of patients achieving a sCR, CR, VGPR, PR or MR as best response according to local medical standards during treatment with melflufen.
Patients without response measurement are considered non-responders.
|
max. 38 months (FPI - LPLV)
|
|
Time to next treatment (TTNT)
Lasso di tempo: max. 38 months (FPI - LPLV)
|
TTNT is defined as the time from start of melflufen treatment until start of the following therapy line or death, whichever comes first.
Patients alive and without subsequent treatment at the time of analysis will be censored at their date of last contact.
TTNT will be calculated using the Kaplan-Meier method.
|
max. 38 months (FPI - LPLV)
|
|
PFS2
Lasso di tempo: max. 38 months (FPI - LPLV)
|
PFS2 is defined as the time from start of melflufen treatment until progression or death during first subsequent treatment line, whichever comes first.
If subsequent line is not reached, previous-line death will serve as event.
Patients without progression after the start of the subsequent line or death at the time of analysis will be censored at their date of last contact.
PFS2 will be calculated using the Kaplan-Meier method.
|
max. 38 months (FPI - LPLV)
|
|
PFS of first subsequent treatment line
Lasso di tempo: max. 38 months (FPI - LPLV)
|
PFS of first subsequent treatment line is defined as time from start of first subsequent treatment line until first progression thereafter, or death from any cause, whatever comes first.
Patients without progression or death at the time of analysis will be censored with their date of last contact.
PFS of first subsequent treatment line will be calculated using the Kaplan-Meier method.
|
max. 38 months (FPI - LPLV)
|
|
(Serious) adverse events ((S)AE)
Lasso di tempo: max. 38 months (FPI - LPLV)
|
The case- and patient-based incidence of (S)AEs will be provided.
|
max. 38 months (FPI - LPLV)
|
|
(Serious) adverse drug reactions ((S)ADR) related to melphalan flufenamid
Lasso di tempo: max. 38 months (FPI - LPLV)
|
The case- and patient-based incidence of (S)ADRs will be provided.
|
max. 38 months (FPI - LPLV)
|
|
Types of treatments prior to Melflufen
Lasso di tempo: max. 38 months (FPI - LPLV)
|
To illustrate the prior treatments, the substances administered before Melflufen are listed by treatment line.
|
max. 38 months (FPI - LPLV)
|
|
Melfalan flufenamid starting dose
Lasso di tempo: max. 38 months (FPI - LPLV)
|
Frequencies of patients with specific melflufen starting dose (i.e., 40mg, 30mg, other) will be provided.
|
max. 38 months (FPI - LPLV)
|
|
Absolute dose intensity of melphalan flufenamid
Lasso di tempo: max. 38 months (FPI - LPLV)
|
Absolute dose intensity of melphalan flufenamid will be displayed with descriptive statistics.
|
max. 38 months (FPI - LPLV)
|
|
Relative dose intensity of melphalan flufenamid
Lasso di tempo: max. 38 months (FPI - LPLV)
|
Relative dose intensity of melphalan flufenamid will be displayed with descriptive statistics.
|
max. 38 months (FPI - LPLV)
|
|
Type of dose modifications
Lasso di tempo: max. 38 months (FPI - LPLV)
|
Type of dose modifications will be displayed with descriptive statistics.
|
max. 38 months (FPI - LPLV)
|
|
Frequency of dose modifications
Lasso di tempo: max. 38 months (FPI - LPLV)
|
Frequency of dose modifications will be displayed with descriptive statistics.
|
max. 38 months (FPI - LPLV)
|
|
Reasons for dose modifications
Lasso di tempo: max. 38 months (FPI - LPLV)
|
Reasons for dose modifications will be displayed with descriptive statistics.
|
max. 38 months (FPI - LPLV)
|
|
Substances of subsequent antineoplastic treatment
Lasso di tempo: max. 38 months (FPI - LPLV)
|
Frequencies of substances used will be displayed in a summary table.
|
max. 38 months (FPI - LPLV)
|
|
Global health-related quality of life during course of treatment
Lasso di tempo: max. 38 months (FPI - LPLV)
|
The change from baseline (i.e., difference) in the scales of the EORTC QLQ-C30 (European Organisation for Research and Treatment of Cancer quality of life questionnaire C30) will be displayed for each point in time, using boxplots.
|
max. 38 months (FPI - LPLV)
|
|
Multiple myeloma related quality of life during course of treatment
Lasso di tempo: max. 38 months (FPI - LPLV)
|
The change from baseline (i.e., difference) in the scales of the EORTC QLQ-MY20 (European Organisation for Research and Treatment of Cancer quality of life questionnaire MY20) will be displayed for each point in time, using boxplots.
|
max. 38 months (FPI - LPLV)
|
|
Assessing parameters of physician treatment decision making
Lasso di tempo: max. 38 months (FPI - LPLV)
|
Results of therapy decision questionnaires will be displayed with frequencies in a summary table.
|
max. 38 months (FPI - LPLV)
|
Collaboratori e investigatori
Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.
Sponsor
Collaboratori
Studiare le date dei record
Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.
Studia le date principali
Inizio studio (Stimato)
15 maggio 2026
Completamento primario (Stimato)
30 giugno 2029
Completamento dello studio (Stimato)
30 giugno 2029
Date di iscrizione allo studio
Primo inviato
20 aprile 2026
Primo inviato che soddisfa i criteri di controllo qualità
27 aprile 2026
Primo Inserito (Effettivo)
30 aprile 2026
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
30 aprile 2026
Ultimo aggiornamento inviato che soddisfa i criteri QC
27 aprile 2026
Ultimo verificato
1 aprile 2026
Maggiori informazioni
Termini relativi a questo studio
Termini MeSH pertinenti aggiuntivi
- Malattie vascolari
- Malattia cardiovascolare
- Neoplasie
- Malattie del sistema immunitario
- Neoplasie per tipo istologico
- Malattie ematologiche
- Malattie linfoproliferative
- Disturbi immunoproliferativi
- Neoplasie, plasmacellule
- Disturbi emostatici
- Paraproteinemie
- Disturbi delle proteine del sangue
- Disturbi emorragici
- Malattie emiche e linfatiche
- Mieloma multiplo
Altri numeri di identificazione dello studio
- IOM-120481
Informazioni su farmaci e dispositivi, documenti di studio
Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti
No
Studia un dispositivo regolamentato dalla FDA degli Stati Uniti
No
Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .