Идёт набор NCT07559799

A Non-interventional Study of Melphalan Flufenamide (Melflufen) (Pepaxti®) and Dexamethasone in Patients With Relapsed and/or Refractory Multiple Myeloma (R/RMM)

— Наблюдательное Multiple Myeloma

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: Это наблюдательное исследование: исследуемое лечение участникам по протоколу не назначают.
Кому может быть актуально: Состояния в реестре: Multiple Myeloma. Базовые параметры: от 18 лет · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: Германия
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

Не всё понятно в терминах? Прочитайте наш гид для пациентов →

Обзор

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.

Первичные конечные точки

Disease control rate (DCR) [Срок оценки: max. 38 months (FPI - LPLV)]

Вторичные конечные точки (12)

Progression-free survival (PFS) [Срок оценки: max. 38 months (FPI - LPLV)]
Overall survival (OS) [Срок оценки: max. 38 months (FPI - LPLV)]
Overall response rate (ORR) [Срок оценки: max. 38 months (FPI - LPLV)]
Duration of treatment with melflufen [Срок оценки: max. 38 months (FPI - LPLV)]
Clinical benefit rate (CBR) [Срок оценки: max. 38 months (FPI - LPLV)]
Time to next treatment (TTNT) [Срок оценки: max. 38 months (FPI - LPLV)]
PFS2 [Срок оценки: max. 38 months (FPI - LPLV)]
PFS of first subsequent treatment line [Срок оценки: max. 38 months (FPI - LPLV)]
(Serious) adverse events ((S)AE) [Срок оценки: max. 38 months (FPI - LPLV)]
(Serious) adverse drug reactions ((S)ADR) related to melphalan flufenamid [Срок оценки: max. 38 months (FPI - LPLV)]
Types of treatments prior to Melflufen [Срок оценки: max. 38 months (FPI - LPLV)]
Melfalan flufenamid starting dose [Срок оценки: max. 38 months (FPI - LPLV)]

Критерии участия

Критерии включения

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.