Proof -of -Concept Study To Stratify Targeted Therapies Adapted To Molecular Profiling (MAPPYACTS)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02613962|
Recruitment Status : Recruiting
First Posted : November 25, 2015
Last Update Posted : February 17, 2020
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.
|Condition or disease||Intervention/treatment||Phase|
|Relapsed or Refractory Pediatric Tumor||Procedure: biopsy or surgical resection of tumor and blood sampling||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||700 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Prospective, Multicentric Clinical Proof-of-concept Study to Stratify Targeted Therapies Adapted to Molecular Profiling of Relapsed or Refractory Pediatric Tumors|
|Actual Study Start Date :||January 13, 2016|
|Estimated Primary Completion Date :||January 2022|
|Estimated Study Completion Date :||January 2022|
Experimental: relapsed or refractory pediatric tumor
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 [ Time Frame: assessed four years after beginning of study ]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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02613962
|Contact: Birgit Geoerger, MD||0142114661 ext +email@example.com|
|Contact: Tiphaine Adam de Beaumais||0142113136 ext +33||TIPHAINE.ADAM-DE-BEAUMAIS@gustaveroussy.fr|
|Villejuif, Val De Marne, France, 94805|
|Contact: Birgit Geoerger, MD 0142114661 ext +33 firstname.lastname@example.org|
|Our Lady's Children's Hospital||Recruiting|
|Dublin, Crumlin, Ireland, 12|
|Contact: Cormac Owens, MD 314096659 ext +35 email@example.com|
|Schneider Children's Medical Center||Not yet recruiting|
|Petach Tikva, Israel, 49202|
|Contact: Isaac Yaniv, MD 39253669 ext 972 firstname.lastname@example.org|
|Fondazione IRCCS Istituto||Recruiting|
|Milano, Italy, 20133|
|Contact: Michela Casanova, MD 0223902594 ext +39 email@example.com|
|Hospital Universitari Vall D'Hebron||Not yet recruiting|
|Barcelona, Cataluna, Spain, 08035|
|Contact: Raquel Hladun-Alvaro, MD 934893090 ext +34 firstname.lastname@example.org|