An interventional study of biopsy or surgical resection of tumor and blood sampling in Relapsed or Refractory Pediatric Tumor, sponsored by Gustave Roussy, Cancer Campus, Grand Paris. Completed at 5 sites in 5 countries. Open to participants aged 6 Months to 18 Years. Per ClinicalTrials.gov, last updated 2026-04-01.
Sponsored by Gustave Roussy, Cancer Campus, Grand Paris · Not applicable, Interventional, and Screening
In the light of the development of high-throughput technologies enabling a biology-based reclassification of tumors and the increasing number of available specifically targeting anticancer agents the era of "precision medicine" has begun. Several clinical precision medicine trials with the aim of stratifying treatment according to molecular profiles (for example in France: 'MOlecular Screening for CAncer Treatment Optimization' MOSCATO-01, SHIVA, PROFILER, Safir01, Safir02) are ongoing in adults and have shown the feasibility of this approach. MOSCATO-01 is the first trial worldwide including pediatric patients, performing an on-purpose intervention and molecular profiling in recurrent tumors. Together with more than 500 adult patients, between December 2012 to August 2014, the tumors of 35 children and adolescents have been profiled, confirming that this approach is feasible in pediatric patients albeit with accelerated time stringencies. Importantly, the results of the first children and adolescents profiled showed that 2/3 of patients had 'actionable' alterations using hot spot mutations sequencing and CGH array (Geoerger B et al, ASCO 2014).
The project 'MAPPYACTS' will use both Whole Exome Sequencing (WES) and RNA Sequencing of tumor tissue to increase the number of targetable genomic alterations. Furthermore to improve understanding of the overall molecular profile and possible response to treatment, methylation array, miRNA expression profiles, and study of immunomodulators will be performed on tumor samples subsequently. CLIP2 (INCa-labeled early phase clinical trials centers) - SiRIC (INCa- labeled comprehensive cancer centers) molecular profiling and bioinformatics platforms will contribute with their expertise in molecular profiling projects and characterization of pediatric cancers. Data interpretation of molecular genetic alterations detected by WES and RNA Seq and treatment recommendation will be done within a multidisciplinary therapeutic molecular biology tumor board.
'MAPPYACTS' will produce one of the largest cohorts of molecularly characterized relapsed tumors reported to date, and thanks to increased access to clinical trials since the European pediatric legislation, the investigators expect that 20-30% of patients can be stratified into a targeted trial based on the detected profile.
It is the investigators' intention that this initiative paves the way to enrich ongoing clinical targeted agent trials, to increase the numbers of stratified clinical trials, to an earlier access to targeted agents, and will play a crucial role in the relevant development of these new agents in pediatric malignancies.
4,279 studies on the registry are indexed under Recurrence; 988 are open to participants now.
This study's enrollment of 791 is above the median of 50 across 3,374 interventional studies indexed under Recurrence.
Browse Recurrence studies →Gustave Roussy, Cancer Campus, Grand Paris is the lead sponsor of 242 studies on the registry; 65 are open to participants now.
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Adequate hematopoietic function for patients with solid tumor (Leukemia patients are excluded from hematological criteria) : Neutrophils >1.0 x 109/l, Platelets >100 x 109/l, Heamoglobin >80 g/l (transfusion allowed) In case of bone marrow involvment: Neutrophils >/= 0.75 x 109/l (unsupported) , Platelet count >/= 0.75 x 109/l (unsupported) Adequate hepatic function: ALAT/ASAT \<2.5 x ULN, Bilirubin ≤1.5 x ULN (in case of tumor involvement of the liver ALAT/ASAT \<5 x ULN) Adequate renal function: Serum creatinemia \<1.5 x ULN for age. In case serum creatinine >1.5 ULN according to age, creatinine clearance has to be >70mL/mL/1.73 m2 or glomerular filtration rate measurement >70% of the expected value
- Patients affiliated to a Social Security Regimen or beneficiary of the same as per local regulatory requirements
Exclusion criteria:
This is a prospective, international, multicentric clinical proof-of-concept study to stratify targeted therapies adapted to molecular profiling of relapsed or refractory pediatric tumors. The molecular screening will be done on a newly biopsied or resected tumor sample obtained at the time of relapse/progression, using high-throughput technologies, primarily WES and RNA Sequencing, and bioinformatics analysis.
Procedure: biopsy or surgical resection of tumor and blood sampling
The percentage of patients with recurrent or refractory pediatric solid tumor or leukemia that could be attributed to treatment with matched targeted agents
The percentage of patients with recurrent or refractory pediatric solid tumor or leukemia that could be attributed to treatment with matched targeted agents according to the molecular profile in their individual tumor will be estimated
Time frame: assessed four years after beginning of study
This study is completed, as verified in Mar 2026. You cannot join it, but the record below documents what was studied.
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Gustave Roussy, Cancer Campus, Grand Paris