MoleculAr Profiling for Pediatric and Young Adult Cancer Treatment Stratification 2

Обзор

FMG2025 continues the previous efforts to propose treatment for patients based on the molecular characteristics of their tumor at treatment failure in cancer precision medicine trials within standard of care in France. However, whereas FMG2025 is a descriptive effort providing the basis for clinical decisions, MAPPYACTS 2 will translate these findings to clinical actions. The symbiosis is critical to advance patient care. Since 2012, the molecular profiling trials "MOlecular Screening for CAncer Treatment Optimization" (MOSCATO-01) and "MoleculAr Profiling for Pediatric and Young Adult Cancer Treatment Stratification" (MAPPYACTS) have included pediatric and adolescent patients with recurrent or refractory malignancy that underwent on-purpose biopsy or surgical intervention. Whole Exome Sequencing of tumor and normal tissue and RNA Sequencing of tumor tissue have been applied to detect genomic alterations that could lead to an adapted targeted treatment. Furthermore, ancillary studies were associated exploring circulating tumor DNA, the immune contexture of tumors and developing Patient-Derived Xenografts (PDX). The FMG2025 project transfers the molecular profiling of advanced pediatric cancers into a global approach that is now considered standard of care in France. Subsequent clinical recommendations and decisions will be made based on discussions with biologists, scientist and physicians in the molecular and clinical molecular tumor boards. Associated ancillary research studies and links to clinical interventional studies remain essential elements of the program to provide clinical, translational and basic research in order to improve scientific knowledge. The program is articulated in two main parts that are closely interacting: FMG2025 - Cancers et leucémies pédiatriques en échec de traitement or equivalent international projects that cover the sequencing of tumor and blood samples and provide molecular reports. The clinical study MAPPYACTS 2 that provides clinical and therapeutic discussions of the sequencing results and therapy recommendations via the clinical molecular tumor board reports. It collects molecular and comprehensive clinical data of the patients registered in FMG2025 or equivalent international projects and thereby constitutes the critical link to clinical interventional studies and its sponsors ensuring facilitated access to these trials. It also covers and coordinates ancillary research studies.

Подробное описание

MAPPYACTS 2 is a prospective international multicentric clinical study to provide clinical therapeutic recommendations, to set up the molecular and comprehensive clinical database of patients with relapsed or refractory pediatric malignancies in FMG2025 - Cancers et leucémies pédiatriques en échec de traitement or equivalent international projects, to collect the follow-up data on treatment and patients' outcome, in order to determine the outcome of the program in regard to benefit to the patient, all the patients and to health care. It will also serve as a central link to interventional study platforms and international precision medicine programs, and to cover and coordinate ancillary research studies that lead to improved treatment and outcome for children with advanced malignancies.

Subsequent clinical recommendations and decisions will be made based on discussions with biologists, scientists and physicians in the molecular and clinical molecular tumor boards run by the MAPPYACTS 2 study teams.

Associated ancillary research studies and links to clinical interventional studies are essential components of the program to perform clinical, translational and basic research in order to generate scientific knowledge and develop new treatment strategies that improve outcome of these patients. Research projects will be discussed and agreed on in the MAPPYACTS 2 study committee.

Вмешательства

• Биопрепарат Solid tumor and CT DNA
  Biopsy and blood sample

Первичные конечные точки

• Overall survival (OS) [Срок оценки: 3 years]
• The number of additional technologies and new treatment strategies that are introduced in the care for advanced pediatric cancers through this research program. [Срок оценки: 3 years]
• The types of additional technologies and new treatment strategies that are introduced in the care for advanced pediatric cancers through this research program. [Срок оценки: 3 years]

Вторичные конечные точки (3)

• Objective response rate on treatment attributed by the molecular tumor board in the clinical trial, overall and according to the treatment decision [Срок оценки: 3 years]
• Progression-free survival (PFS) [Срок оценки: 3 years]
• 5-year overall survival [Срок оценки: 5 years]

Критерии участия

Критерии включения

• Patient referred for sequencing of the tumor within the FMG2025 or equivalent program and written informed consent for FMG2025 "Cancers et leucémies pédiatriques en échec de traitement" or equivalent, according to local regulations
• Written informed consent of MAPPYACTS 2 to collect molecular and comprehensive clinical data on cancer diagnosis, therapies, therapy outcomes, to provide clinical therapeutic recommendations, to collect follow-up data on treatment and patients' outcome; optional written consents to perform to ancillary research studies, according to local regulations. The written consent will include access to reimbursement data from the French national health insurance through linkage with the Système National des Données de Santé (SNDS) or equivalent.
• Patient with histologically/cytologically confirmed solid tumor or leukemia which is relapsed or refractory to standard treatment and who is potentially eligible for an experimental treatment or an early phase clinical trial
• Planned tumor biopsy, surgical resection, bone marrow or blood sample or recently (preferably within the last 3 months) archived frozen tumor material available of the current recurrent or refractory disease
• Patients aged ≤ 25 years at the time of initial diagnosis
• Performance status and life expectancy > 3 months expected to allow enrolment into an clinical trial
• Patients affiliated with a Social Security Regimen or beneficiary of the same, as per local regulatory requirements

Критерии исключения

• Any concurrent illness or laboratory abnormality that, in the opinion of the investigator, is likely to interfere with the interpretation of study results
• Pregnant women

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Да

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Диагностика

Центры проведения

Франция · 29 центров

• Gustave Roussy — Paris
• CHU Amiens-Picardie — Amiens
• CHU Angers — Angers
• CHU Besançon — Besançon
• CHU Pellegrin — Bordeaux
• CHRU Morvan — Brest
• CHU de Caen — Caen
• CHU Estaing — Clermont-Ferrand
• … и ещё 21 центр

Идентификаторы

NCT: NCT05691608 · 2021-A02990-41 · 2021-3321

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗