MoleculAr Profiling for Pediatric and Young Adult Cancer Treatment Stratification 2 (MAPPYACTS2)

• ClinicalTrials.gov Identifier: NCT05691608
• Recruitment Status: Recruiting
• First Posted: January 20, 2023
• Last Update Posted: January 20, 2023
• Sponsor: Gustave Roussy, Cancer Campus, Grand Paris
• Information provided by (Responsible Party): Gustave Roussy, Cancer Campus, Grand Paris

Tracking Information

• First Submitted Date: December 12, 2022
• First Posted Date: January 20, 2023
• Last Update Posted Date: January 20, 2023
• Actual Study Start Date: September 9, 2022
• Estimated Primary Completion Date: September 9, 2030 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: January 19, 2023)

• Overall survival (OS) [ Time Frame: 3 years ]
  Defined as the time from study entry to death whatever the cause of death, after 3 years, for the whole cohort and according to cancer type.
• The number of additional technologies and new treatment strategies that are introduced in the care for advanced pediatric cancers through this research program. [ Time Frame: 3 years ]
  The number of patients receiving matched targeted treatments The number of new treatment strategies that are introduced in the care for advanced pediatric cancers through this research program The number of additional technologies that are introduced in the care for advanced pediatric cancers through this research program
• The types of additional technologies and new treatment strategies that are introduced in the care for advanced pediatric cancers through this research program. [ Time Frame: 3 years ]
  The type of patients receiving matched targeted treatments The type of new treatment strategies that are introduced in the care for advanced pediatric cancers through this research program The type of additional technologies that are introduced in the care for advanced pediatric cancers through this research program

• Original Primary Outcome Measures: Same as current
• Change History: No Changes Posted

Current Secondary Outcome Measures (submitted: January 19, 2023)

• Objective response rate on treatment attributed by the molecular tumor board in the clinical trial, overall and according to the treatment decision [ Time Frame: 3 years ]
  Objective tumor response measured according to the standard for each tumor entity (i.e. RECIST 1.1, INRC, RANO, etc.). Assessed for the whole cohort, for each disease entity as well as for each suggested treatment and until last follow-up. The number and proportion of patients that are suggested to receive a matched targeted agent Direct medical costs including the planned or recently performed intervention
• Progression-free survival (PFS) [ Time Frame: 3 years ]
  Defined as the time interval from start of attributed treatment (targeted or not) to progression or death. They will be assessed for the whole cohort, for each disease entity as well as for each suggested treatment and until last follow-up.
• 5-year overall survival [ Time Frame: 5 years ]
  Defined as the time from study entry to death whatever the cause of death, after 5 years. They will be assessed for the whole cohort, for each disease entity as well as for each suggested treatment and until last follow-up.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: MoleculAr Profiling for Pediatric and Young Adult Cancer Treatment Stratification 2
• Official Title: MoleculAr Profiling for Pediatric and Young Adult Cancer Treatment Stratification 2

Brief Summary

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.

Detailed Description

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.

• Study Type: Interventional
• Study Phase: Not Applicable
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Diagnostic

Condition

• Solid Tumor
• Leukemia

Intervention

Biological: Solid tumor and CT DNA

Biopsy and blood sample

Study Arms

Experimental: Patient with solid tumor or leukemia (no treatment)

Patient with solid tumor or leukemia (no treatment)

Intervention: Biological: Solid tumor and CT DNA

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: January 19, 2023): 1800
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: September 9, 2030
• Estimated Primary Completion Date: September 9, 2030 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• 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

Exclusion Criteria:

• 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 6 Years to 25 Years (Child, Adult)
• Accepts Healthy Volunteers: Yes

Contacts

Contact: Birgit GEOERGER, MD; +33 (0)1 42 11 46 61; Birgit.GEOERGER@gustaveroussy.fr

Contact: Jean-Luc JOANNIC, PhD; +33 (0)1 42 11 42 11 ext 47 94; jeanluc.joannic@gustaveroussy.fr

• Listed Location Countries: France

Administrative Information

• NCT Number: NCT05691608
• Other Study ID Numbers: 2021-A02990-41; 2021-3321 ( Other Identifier: CSET NUMBER )
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Yes
• Plan Description: Study protocol Informed Consent Form
• Supporting Materials: Study Protocol
• Supporting Materials: Informed Consent Form (ICF)
• Time Frame: Data will become available in January 2023 The duration of the availability is up to 2030.

• Current Responsible Party: Gustave Roussy, Cancer Campus, Grand Paris
• Original Responsible Party: Same as current
• Current Study Sponsor: Gustave Roussy, Cancer Campus, Grand Paris
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Birgit GEOERGER, MD; Gustave Roussy, Cancer Campus, Grand Paris

• PRS Account: Gustave Roussy, Cancer Campus, Grand Paris
• Verification Date: January 2023