Multiple MyelomaMARINANCT07559799
A Non-interventional Study of Melphalan Flufenamide (Melflufen) (Pepaxti®) and Dexamethasone in Patients With Relapsed and/or Refractory Multiple Myeloma (R/RMM)
An observational study of Multiple Myeloma, run by iOMEDICO AG at 1 site in Germany.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Multiple Myeloma
- How many people
- 50 participants
- Where
- 1 siteGermany
- Study dates
- 1 Jun 2026to 30 Aug 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: 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.
Who may be able to join
- 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.
Who cannot join
- Participation in an interventional clinical trial (except follow-up)
- Patient unable to consent
- Contraindications according to current SmPC
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Germany1 site, 1 recruiting
- Recruiting
Praxis für interdisziplinäre Onkologie & Hämatologie
Freiburg im Breisgau
Patrick Marschner, Dr. med.
About this study
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.
Read more
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.
- Conditions
- Multiple Myeloma
- Medical subject headings
- Multiple Myeloma
What is being measured
The primary outcome is the main question the study is designed to answer.
Disease control rate (DCR)
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.
Measured: max. 38 months (FPI - LPLV)
Secondary outcomes (21)
Progression-free survival (PFS)
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.
Measured: max. 38 months (FPI - LPLV)
Overall survival (OS)
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.
Measured: max. 38 months (FPI - LPLV)
Overall response rate (ORR)
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.
Measured: max. 38 months (FPI - LPLV)
Duration of treatment with melflufen
Duration of melflufen treatment will be calculated in weeks as the time from first application to the last documented application of melflufen.
Measured: max. 38 months (FPI - LPLV)
Clinical benefit rate (CBR)
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.
Measured: max. 38 months (FPI - LPLV)
Time to next treatment (TTNT)
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.
Measured: max. 38 months (FPI - LPLV)
PFS2
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.
Measured: max. 38 months (FPI - LPLV)
PFS of first subsequent treatment line
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.
Measured: max. 38 months (FPI - LPLV)
(Serious) adverse events ((S)AE)
The case- and patient-based incidence of (S)AEs will be provided.
Measured: max. 38 months (FPI - LPLV)
(Serious) adverse drug reactions ((S)ADR) related to melphalan flufenamid
The case- and patient-based incidence of (S)ADRs will be provided.
Measured: max. 38 months (FPI - LPLV)
Types of treatments prior to Melflufen
To illustrate the prior treatments, the substances administered before Melflufen are listed by treatment line.
Measured: max. 38 months (FPI - LPLV)
Melfalan flufenamid starting dose
Frequencies of patients with specific melflufen starting dose (i.e., 40mg, 30mg, other) will be provided.
Measured: max. 38 months (FPI - LPLV)
Absolute dose intensity of melphalan flufenamid
Absolute dose intensity of melphalan flufenamid will be displayed with descriptive statistics.
Measured: max. 38 months (FPI - LPLV)
Relative dose intensity of melphalan flufenamid
Relative dose intensity of melphalan flufenamid will be displayed with descriptive statistics.
Measured: max. 38 months (FPI - LPLV)
Type of dose modifications
Type of dose modifications will be displayed with descriptive statistics.
Measured: max. 38 months (FPI - LPLV)
Frequency of dose modifications
Frequency of dose modifications will be displayed with descriptive statistics.
Measured: max. 38 months (FPI - LPLV)
Reasons for dose modifications
Reasons for dose modifications will be displayed with descriptive statistics.
Measured: max. 38 months (FPI - LPLV)
Substances of subsequent antineoplastic treatment
Frequencies of substances used will be displayed in a summary table.
Measured: max. 38 months (FPI - LPLV)
Global health-related quality of life during course of treatment
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.
Measured: max. 38 months (FPI - LPLV)
Multiple myeloma related quality of life during course of treatment
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.
Measured: max. 38 months (FPI - LPLV)
Assessing parameters of physician treatment decision making
Results of therapy decision questionnaires will be displayed with frequencies in a summary table.
Measured: max. 38 months (FPI - LPLV)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Apr 2026
- Study start1 Jun 2026
- Primary completion30 Aug 2029 (estimated)
- Study completion30 Aug 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple MyelomaStudies of the same condition, open and under way.
- More trials run by iOMEDICO AGThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- iOMEDICO AG
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Oncopeptides AB
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07559799
- Sponsor's protocol ID
- IOM-120481
- Acronym
- MARINA
- Status last verified
- Jun 2026
- First submitted
- 20 Apr 2026
- Last updated
- 26 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Non-interventional Study of Melphalan Flufenamide (Melflufen) (Pepaxti®) and Dexamethasone in Patients With Relapsed and/or Refractory Multiple Myeloma (R/RMM)
- ClinicalTrials.gov ID
- NCT07559799
- Acronym / sponsor ID
- MARINA / IOM-120481
- Conditions
- Multiple Myeloma
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- iOMEDICO AGIndustry (pharma, biotech, devices)
- Collaborators
- Oncopeptides AB
- Study start
- 1 Jun 2026 (actual)
- Primary completion
- 30 Aug 2029 (estimated)
- Study completion
- 30 Aug 2029 (estimated)
- First posted
- 30 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- 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.
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (3 listed)
- Participation in an interventional clinical trial (except follow-up)
- Patient unable to consent
- Contraindications according to current SmPC
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Disease control rate (DCR)
Time frame: 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.
21 secondary outcomes
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)
Absolute dose intensity of melphalan flufenamid
Time frame: max. 38 months (FPI - LPLV)
Relative dose intensity of melphalan flufenamid
Time frame: max. 38 months (FPI - LPLV)
Type of dose modifications
Time frame: max. 38 months (FPI - LPLV)
Frequency of dose modifications
Time frame: max. 38 months (FPI - LPLV)
Reasons for dose modifications
Time frame: max. 38 months (FPI - LPLV)
Substances of subsequent antineoplastic treatment
Time frame: max. 38 months (FPI - LPLV)
Global health-related quality of life during course of treatment
Time frame: max. 38 months (FPI - LPLV)
Multiple myeloma related quality of life during course of treatment
Time frame: max. 38 months (FPI - LPLV)
Assessing parameters of physician treatment decision making
Time frame: max. 38 months (FPI - LPLV)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Multiple Myeloma, run by iOMEDICO AG at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sina Grebhardt, PhD (+49 761 / 15 242-0, info@iomedico.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Disease control rate (DCR) (time frame: max. 38 months (FPI - LPLV)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Jun 2026.