Relapsed or Refractory Pediatric TumorMAPPYACTSNCT02613962
Proof -of -Concept Study To Stratify Targeted Therapies Adapted To Molecular Profiling
A study testing biopsy or surgical resection of tumor and blood sampling in people with Relapsed or Refractory Pediatric Tumor, run by Gustave Roussy, Cancer Campus, Grand Paris at 5 sites in 5 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 6 months to 18 years
- Conditions the study is for
- Relapsed or Refractory Pediatric Tumor
- What is being tested?
- biopsy or surgical resection of tumor and blood sampling
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 791 participants
- Where
- 5 sitesin 5 countries
- Study dates
- 13 Jan 2016to 16 Nov 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Written informed consent signed by the patient, or parents or legal representative and assent of the minor child to perform biopsy/surgical resection/aspiral/sample and molecular analysis of the tumor and blood sample
- Patient with confirmed solid tumor or leukemia which is recurrent or refractory to standard treatment and who is eligible for an early phase clinical trial
- In case of solid tumor, lesion must be accessible for biopsy or surgical resection or cytological puncture
- Age: Patients aged ≥ 6 months at time of inclusion and aged ≤ 18 years at the time of initial diagnosis
- Performance status and life expectancy that allows treatment in an experimental trial: Karnofsky performance status ≥ 70% for patients > 12 years of age, Lansky play scale ≥ 70% for patients ≤ 12 years of age
- Adequate organ function:
- 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
Who cannot join
- Life expectancy ≤ 3 months
- Symptomatic CNS metastases
- In case of leukemia, are excluded patients with isolated meningitis relapses.
- Coagulation disorder that prevents the accomplishment of a biopsy or surgery
- Uncontrolled infections not responsive to antibiotics, antiviral medicines, or antifungal medicines
- Presence of ≥ CTCAE grade 2 toxicity (except alopecia, ototoxicity, lymphopenia which are not excluded if grade 3 or less) due to prior cancer therapy
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- Malignant disease other than that being treated in this study. Exceptions to this exclusion include the following: malignancies that were treated curatively and have not recurred within 3 years prior to study entry
- Any concurrent illness or laboratory abnormality that in opinion of investigator may interfere with the interpretation of study results, may suppose a risk for the realization of biopsy/surgery, and in the judgment of the investigator would make the patient inappropriate for the study
- Evidence of active viral Hepatitis B or C or known diagnosis of human immunodeficiency virus infection
Trial locations and contacts
5 sites in 5 countries.
5 sites in 5 countries
About this study
Official title: A Prospective, Multicentric Clinical Proof-of-concept Study to Stratify Targeted Therapies Adapted to Molecular Profiling of Relapsed or Refractory Pediatric Tumors
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.
Read more
'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.
- Conditions
- Relapsed or Refractory Pediatric Tumor
- Being tested
- biopsy or surgical resection of tumor and blood sampling
- Medical subject headings
- Recurrence, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
biopsy or surgical resection of tumor and blood samplingProcedure or surgery
| Study group | Role | What they receive |
|---|---|---|
| relapsed or refractory pediatric tumor | Experimental | 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. Receives: Procedure: biopsy or surgical resection of tumor and blood sampling |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: assessed four years after beginning of study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2015 (estimated)
- Study start13 Jan 2016
- Primary completion16 Nov 2022
- Study completion16 Nov 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Relapsed or Refractory Pediatric TumorStudies of the same condition, open and under way.
- More trials run by Gustave Roussy, Cancer Campus, Grand ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 791 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gustave Roussy, Cancer Campus, Grand Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Innovative Therapies For Children with Cancer Consortium
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02613962
- Sponsor's protocol ID
- 2015-A00464-45
- Other IDs
- 2015/2244
- Acronym
- MAPPYACTS
- Status last verified
- Mar 2026
- First submitted
- 23 Nov 2015
- Last updated
- 1 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
Publications
Chaix J, Schleiermacher G, Corradini N, Andre N, Thebaud E, Gambart M, Defachelles AS, Entz-Werle N, Chastagner P, De Carli E, Ducassou S, Landman-Parker J, Adam-de-Beaumais T, Larive A, Michiels S, Vassal G, Valteau-Couanet D, Geoerger B, Berlanga P. Clinical trial inclusion in patients with relapsed/refractory neuroblastoma following the European Precision Cancer Medicine trial MAPPYACTS. Eur J Cancer. 2024 Apr;201:113923. doi: 10.1016/j.ejca.2024.113923. Epub 2024 Feb 15.
PubMed 38377775Marques Da Costa ME, Zaidi S, Scoazec JY, Droit R, Lim WC, Marchais A, Salmon J, Cherkaoui S, Morscher RJ, Laurent A, Malinge S, Mercher T, Tabone-Eglinger S, Goddard I, Pflumio F, Calvo J, Redini F, Entz-Werle N, Soriano A, Villanueva A, Cairo S, Chastagner P, Moro M, Owens C, Casanova M, Hladun-Alvaro R, Berlanga P, Daudigeos-Dubus E, Dessen P, Zitvogel L, Lacroix L, Pierron G, Delattre O, Schleiermacher G, Surdez D, Geoerger B. A biobank of pediatric patient-derived-xenograft models in cancer precision medicine trial MAPPYACTS for relapsed and refractory tumors. Commun Biol. 2023 Sep 18;6(1):949. doi: 10.1038/s42003-023-05320-0.
PubMed 37723198Berlanga P, Pierron G, Lacroix L, Chicard M, Adam de Beaumais T, Marchais A, Harttrampf AC, Iddir Y, Larive A, Soriano Fernandez A, Hezam I, Chevassus C, Bernard V, Cotteret S, Scoazec JY, Gauthier A, Abbou S, Corradini N, Andre N, Aerts I, Thebaud E, Casanova M, Owens C, Hladun-Alvaro R, Michiels S, Delattre O, Vassal G, Schleiermacher G, Geoerger B. The European MAPPYACTS Trial: Precision Medicine Program in Pediatric and Adolescent Patients with Recurrent Malignancies. Cancer Discov. 2022 May 2;12(5):1266-1281. doi: 10.1158/2159-8290.CD-21-1136.
PubMed 35292802Morscher RJ, Brard C, Berlanga P, Marshall LV, Andre N, Rubino J, Aerts I, De Carli E, Corradini N, Nebchi S, Paoletti X, Mortimer P, Lacroix L, Pierron G, Schleiermacher G, Vassal G, Geoerger B. First-in-child phase I/II study of the dual mTORC1/2 inhibitor vistusertib (AZD2014) as monotherapy and in combination with topotecan-temozolomide in children with advanced malignancies: arms E and F of the AcSe-ESMART trial. Eur J Cancer. 2021 Nov;157:268-277. doi: 10.1016/j.ejca.2021.08.010. Epub 2021 Sep 17.
PubMed 34543871Peneder P, Stutz AM, Surdez D, Krumbholz M, Semper S, Chicard M, Sheffield NC, Pierron G, Lapouble E, Totzl M, Erguner B, Barreca D, Rendeiro AF, Agaimy A, Boztug H, Engstler G, Dworzak M, Bernkopf M, Taschner-Mandl S, Ambros IM, Myklebost O, Marec-Berard P, Burchill SA, Brennan B, Strauss SJ, Whelan J, Schleiermacher G, Schaefer C, Dirksen U, Hutter C, Boye K, Ambros PF, Delattre O, Metzler M, Bock C, Tomazou EM. Multimodal analysis of cell-free DNA whole-genome sequencing for pediatric cancers with low mutational burden. Nat Commun. 2021 May 28;12(1):3230. doi: 10.1038/s41467-021-23445-w.
PubMed 34050156
Study at a glance
From the registry record, last updated by the sponsor on 1 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Multicentric Clinical Proof-of-concept Study to Stratify Targeted Therapies Adapted to Molecular Profiling of Relapsed or Refractory Pediatric Tumors
- ClinicalTrials.gov ID
- NCT02613962
- Acronym / sponsor ID
- MAPPYACTS / 2015-A00464-45
- Conditions
- Relapsed or Refractory Pediatric Tumor
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 791 participants (actual)
- Lead sponsor
- Gustave Roussy, Cancer Campus, Grand ParisUniversity, hospital or other
- Collaborators
- Innovative Therapies For Children with Cancer Consortium
- Study start
- 13 Jan 2016 (actual)
- Primary completion
- 16 Nov 2022 (actual)
- Study completion
- 16 Nov 2022 (actual)
- First posted
- 25 Nov 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Screening
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| relapsed or refractory pediatric tumor | Experimental | Procedure: biopsy or surgical resection of tumor and blood samplingThis 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. |
Who may qualify
- Ages
- 6 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Written informed consent signed by the patient, or parents or legal representative and assent of the minor child to perform biopsy/surgical resection/aspiral/sample and molecular analysis of the tumor and blood sample
- Patient with confirmed solid tumor or leukemia which is recurrent or refractory to standard treatment and who is eligible for an early phase clinical trial
- In case of solid tumor, lesion must be accessible for biopsy or surgical resection or cytological puncture
- Age: Patients aged ≥ 6 months at time of inclusion and aged ≤ 18 years at the time of initial diagnosis
- Performance status and life expectancy that allows treatment in an experimental trial: Karnofsky performance status ≥ 70% for patients > 12 years of age, Lansky play scale ≥ 70% for patients ≤ 12 years of age
Key exclusion criteria (9 listed)
- Life expectancy ≤ 3 months
- Symptomatic CNS metastases
- In case of leukemia, are excluded patients with isolated meningitis relapses.
- Coagulation disorder that prevents the accomplishment of a biopsy or surgery
- Uncontrolled infections not responsive to antibiotics, antiviral medicines, or antifungal medicines
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| biopsy or surgical resection of tumor and blood sampling | Procedure or surgery | relapsed or refractory pediatric tumor |
Primary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Apr 2026.
- Derived from this study: PubMed 38377775Chaix J, Schleiermacher G, Corradini N, Andre N, Thebaud E, Gambart M, Defachelles AS, Entz-Werle N, Chastagner P, De Carli E, Ducassou S, Landman-Parker J, Adam-de-Beaumais T, Larive A, Michiels S, Vassal G, Valteau-Couanet D, Geoerger B, Berlanga P. Clinical trial inclusion in patients with relapsed/refractory neuroblastoma following the European Precision Cancer Medicine trial MAPPYACTS. Eur J Cancer. 2024 Apr;201:113923. doi: 10.1016/j.ejca.2024.113923. Epub 2024 Feb 15.
- Derived from this study: PubMed 37723198Marques Da Costa ME, Zaidi S, Scoazec JY, Droit R, Lim WC, Marchais A, Salmon J, Cherkaoui S, Morscher RJ, Laurent A, Malinge S, Mercher T, Tabone-Eglinger S, Goddard I, Pflumio F, Calvo J, Redini F, Entz-Werle N, Soriano A, Villanueva A, Cairo S, Chastagner P, Moro M, Owens C, Casanova M, Hladun-Alvaro R, Berlanga P, Daudigeos-Dubus E, Dessen P, Zitvogel L, Lacroix L, Pierron G, Delattre O, Schleiermacher G, Surdez D, Geoerger B. A biobank of pediatric patient-derived-xenograft models in cancer precision medicine trial MAPPYACTS for relapsed and refractory tumors. Commun Biol. 2023 Sep 18;6(1):949. doi: 10.1038/s42003-023-05320-0.
- Derived from this study: PubMed 35292802Berlanga P, Pierron G, Lacroix L, Chicard M, Adam de Beaumais T, Marchais A, Harttrampf AC, Iddir Y, Larive A, Soriano Fernandez A, Hezam I, Chevassus C, Bernard V, Cotteret S, Scoazec JY, Gauthier A, Abbou S, Corradini N, Andre N, Aerts I, Thebaud E, Casanova M, Owens C, Hladun-Alvaro R, Michiels S, Delattre O, Vassal G, Schleiermacher G, Geoerger B. The European MAPPYACTS Trial: Precision Medicine Program in Pediatric and Adolescent Patients with Recurrent Malignancies. Cancer Discov. 2022 May 2;12(5):1266-1281. doi: 10.1158/2159-8290.CD-21-1136.
- Derived from this study: PubMed 34543871Morscher RJ, Brard C, Berlanga P, Marshall LV, Andre N, Rubino J, Aerts I, De Carli E, Corradini N, Nebchi S, Paoletti X, Mortimer P, Lacroix L, Pierron G, Schleiermacher G, Vassal G, Geoerger B. First-in-child phase I/II study of the dual mTORC1/2 inhibitor vistusertib (AZD2014) as monotherapy and in combination with topotecan-temozolomide in children with advanced malignancies: arms E and F of the AcSe-ESMART trial. Eur J Cancer. 2021 Nov;157:268-277. doi: 10.1016/j.ejca.2021.08.010. Epub 2021 Sep 17.
- Derived from this study: PubMed 34050156Peneder P, Stutz AM, Surdez D, Krumbholz M, Semper S, Chicard M, Sheffield NC, Pierron G, Lapouble E, Totzl M, Erguner B, Barreca D, Rendeiro AF, Agaimy A, Boztug H, Engstler G, Dworzak M, Bernkopf M, Taschner-Mandl S, Ambros IM, Myklebost O, Marec-Berard P, Burchill SA, Brennan B, Strauss SJ, Whelan J, Schleiermacher G, Schaefer C, Dirksen U, Hutter C, Boye K, Ambros PF, Delattre O, Metzler M, Bock C, Tomazou EM. Multimodal analysis of cell-free DNA whole-genome sequencing for pediatric cancers with low mutational burden. Nat Commun. 2021 May 28;12(1):3230. doi: 10.1038/s41467-021-23445-w.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing biopsy or surgical resection of tumor and blood sampling in people with Relapsed or Refractory Pediatric Tumor, run by Gustave Roussy, Cancer Campus, Grand Paris at 5 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in France, Ireland, Israel, Italy, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02613962, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Apr 2026.