Pediatric CancerSACHANCT04477681
Securing Access to Innovative Molecules in Oncology and Hematology for Children, Adolescents and Young Adults
An observational study of Pediatric Cancer, run by Gustave Roussy, Cancer Campus, Grand Paris at 32 sites in France.
Trial summary
It involves collecting safety and efficacy data, under the actual conditions of use of medicines in children and adolescents, using a validated tool (Ennov EDC) and relying on the network of Interregional pediatric oncology appeal organizations (RIOs) identified by INCa since 2010 and responsible for the organization… Read more about this study
- Who is it for?
- Ages Up to 25 years
- Conditions the study is for
- Pediatric Cancer
- What is being tested?
- Data collection
- How many people
- 1,150 participants
- Where
- 32 sitesFrance
- Study dates
- 9 Apr 2020to Apr 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Up to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Any child, adolescent or young adult, treated for a pediatric tumor or leukemia, in therapeutic failure or relapse without standard treatment option, not eligible / refusal of inclusion in a clinical study open on the territory and treated with an innovative drug within the framework of compassionate use or outside marketing authorization, in one of the centers of the SFCE (Société Française des Cancers de l' Enfant).
Who may be able to join
- Age ≤ 25 years old at the time of inclusion in the study
- Patient with a pediatric tumor or leukemia in therapeutic failure or relapse without standard therapeutic options. Exceptionally, patients on the first line of treatment without standard therapeutic options may also be included (eg g: inoperable plexiform neurofibroma and MEK inhibitors, childhood fibrosarcoma and NTRK inhibitors).
- Patient not eligible for an early phase clinical trial open to inclusion on French territory or refusal of inclusion
- Patient treated with a new drug discussed at a RCPPI as part of a compassionate use issued by the ANSM or an off-label prescription of a drug of interest already authorized in adults.
- Patients treated in one of the SFCE centers authorized to prescribe chemotherapy
- Patient who did not object to participate after being informed of the study. The patient must be able and willing to cooperate in the study.
Who cannot join
- Patient included in an early phase clinical trial open to inclusions on French territory.
- Oral refusal to participate by the patient or their legal representatives, in reading the information note specific to the study
- Patient under guardianship or curatorship, deprived of liberty or in the impossibility of expressing opposition to participating in the study
Trial locations and contacts
32 sites in 1 country. Contact the study team or a site near you to ask about taking part.
32 sites in 1 country
France32 sites, 32 recruiting
- Recruiting
CHU Amiens
Amiens
Leslie ANDRY, MD, Andry.Leslie@chu-amiens.fr
- Recruiting
CHU Angers
Angers
Emilie DE CARLI, MD, EmDeCarli@chu-angers.fr
- Recruiting
CHU Besançon
Besançon
Sébastien KLEIN, MD, s1klein@chu-besancon.fr
- Recruiting
CHU Bordeaux
Bordeaux
Stéphane DUCASSOU, MD, stephane.ducassou@chu-bordeaux.fr
- Recruiting
CHU Morvan
Brest, 29200
Liana Carausu, liana.carausu@chu-brest.fr
- Recruiting
CHU CAEN
Caen
Damien BODET, MD, bodet-d@chu-caen.fr
- Recruiting
CHU Clermont-Ferrand
Clermont-Ferrand
Justyna KANOLD, jkanold@chu-clermontferrand.fr
- Recruiting
CHU Dijon
Dijon
Claire BRIANDET, MD, claire.briandet@chu-dijon.fr
- Recruiting
CHU Grenoble
Grenoble
Dominique PLANTAZ, MD, Dplantaz@chu-grenoble.fr
- Recruiting
Centre Oscar Lambret
Lille
Sandra RAIMBAULT, MD, s-raimbault@o-lambret.fr
- Recruiting
CHU Lille
Lille
Brigitte NELKEN, MD, brigitte.nelken@chru-lille.fr
- Recruiting
CHU Limoges
Limoges
Christophe PIGUET, MD, Christophe.Piguet@chu-limoges.fr
- Recruiting
Centre Léon Bérard
Lyon
Nadège CORRADINI, MD, Nadege.CORRADINI@ihope.fr
- Recruiting
CHU Lyon
Lyon
Carine HALFON-DOMENECH, MD, carine.halfondomenech@ihope.fr
- Recruiting
Hôpital de La Timone
Marseille
Nicolas ANDRE, MD, Nicolas.ANDRE@ap-hm.fr
- Recruiting
CHU Montpellier
Montpellier
Laure SAUMET, MD, l-saumet@chu-montpellier.fr
- Recruiting
CHU Nancy
Nancy
Pascal CHASTAGNER, MD, p.chastagner@chru-nancy.fr
- Recruiting
CHU Nantes
Nantes
Morgane CLEIREC, MD, morgane.cleirec@chu-nantes.fr
- Recruiting
CHU Nice
Nice
Joy BENADIBA, MD, benadiba.j@chu-nice.fr
- Recruiting
Hôpital Armand Trousseau
Paris
Arnaud PETIT, MD, arnaud.petit@aphp.fr
- Recruiting
Hôpital Robert-Debré
Paris
Marion STRULLU, MD, marion.strullu@aphp.fr
- Recruiting
Institut Curie
Paris
Isabelle AERTS, MD, isabelle.aerts@curie.fr
- Recruiting
CHU Poitiers
Poitiers
Frederic MILLOT, MD, f.millot@chu-poitiers.fr
- Recruiting
CHU Reims
Reims
Claire PUCHART, MD, cpluchart@chu-reims.fr
- Recruiting
CHU Rennes
Rennes
Chloé PUISIEUX, MD, Chloe.PUISEUX@chu-rennes.fr
- Recruiting
CHU Rouen
Rouen
Pascal SCHNEIDER, Pascale.Schneider@chu-rouen.fr
- Recruiting
CHU La Réunion
Saint-Denis
Yves REGUERRE, MD, yves.reguerre@chu-reunion.fr
- Recruiting
CHU Saint-Etienne
Saint-Etienne
Sandrine THOUVENIN, MD, sandrine.thouvenin@chu-st-etienne.fr
- Recruiting
CHU Strasbourg
Strasbourg
Natacha ENTZ-WERLE, Natacha.Entz-Werle@chru-strasbourg.fr
- Recruiting
CHU Toulouse
Toulouse
Marion Gambart, MD, gambart.m@chu-toulouse.fr
- Recruiting
CHU Tours
Tours
Jill SERRE, MD, J.SERRE@chu-tours.fr
- Recruiting
Gustave Roussy
Villejuif, Val de Marne, 94805
Pablo BERLANGA, MD, +33 (0)1 42 11 42 11, pablo.berlanga@gustaveroussy.fr
About this study
Official title: Securing Access to Innovative Molecules in Oncology and Hematology for Children, Adolescents and Young Adults in Therapeutic Failure or Relapse and Not Eligible for a Clinical Trial: a Project of the SFCE
It involves collecting safety and efficacy data, under the actual conditions of use of medicines in children and adolescents, using a validated tool (Ennov EDC) and relying on the network of Interregional pediatric oncology appeal organizations (RIOs) identified by INCa since 2010 and responsible for the organization of Pluridisciplinary Pediatric Interregional (RCPPI) and National Consultative Meetings which discuss each case of relapse in order to define the best therapeutic options.
Read the detailed description
The objective is to aim for completeness throughout the national territory. This is why this project will be carried out in close collaboration with the RIOs and RCPPI and that of the 31 centers of the SFCE which bring together more than 400 doctors pediatric oncologists and hematologists on the French territory
- Conditions
- Pediatric Cancer
- Being tested
- Data collection
- Medical subject headings
- Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Data collectionOther
In particular, the patient's demographic data, medical history, previous and concomitant treatments, data on the treatment of interest (legal framework, compassionate use/ off-label), dosage, start date, etc.), clinical data will be collected. biological (including molecular profiling of the tumor if available) and radiological follow-up, information on adverse effects. The data to be filled in is that of the CERFA pharmacovigilance form of the ANSM, but additional fields can be created if necessary.
| Study group | Role | What they receive |
|---|---|---|
| Patients in therapeutic failure or relapse | Any child, adolescent or young adult, treated for a pediatric tumor or leukemia, in therapeutic failure or relapse without standard treatment option, not eligible / refusal of inclusion in a clinical study open on the territory and treated with an innovative drug within the framework of a compassionate use or outside marketing authorization, in one of the centers of the SFCE (Société Française des Cancers de l' Enfant) Receives: Other: Data collection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Collect access data
Collect access data of children, adolescents and young adults in therapeutic failures and not eligible for a clinical trial with innovative molecules, whether they are targeted therapies, immunotherapies or chemotherapies
Measured: From enrollment to the end of treatment maximum 7 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2020
- Study start9 Apr 2020
- Primary completionApr 2027 (estimated)
- Study completionApr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Pediatric CancerStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Gustave Roussy, Cancer Campus, Grand Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04477681
- Sponsor's protocol ID
- 2019-A01317-50
- Other IDs
- 2019/2848
- Acronym
- SACHA
- Status last verified
- Mar 2026
- First submitted
- 15 Jul 2020
- Last updated
- 31 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Berlanga P, Ndounga-Diakou LA, Aerts I, Corradini N, Ducassou S, Strullu M, de Carli E, Andre N, Entz-Werle N, Raimbault S, Roumy M, Renouard M, Gueguen G, Plantaz D, Reguerre Y, Cleirec M, Petit A, Puiseux C, Andry L, Klein S, Bodet D, Kanold J, Briandet C, Halfon-Domenech C, Nelken B, Piguet C, Saumet L, Chastagner P, Benadiba J, Millot F, Pluchart C, Schneider P, Thouvenin S, Gambart M, Serre J, Abbou S, Leruste A, Cayzac H, Gandemer V, Laghouati S, Vassal G. Measuring Safety and Outcomes for the Use of Compassionate and Off-Label Therapies for Children, Adolescents, and Young Adults With Cancer in the SACHA-France Study. JAMA Netw Open. 2023 Jul 3;6(7):e2321568. doi: 10.1001/jamanetworkopen.2023.21568.
PubMed 37399010Pasqualini C, Proust S, Schleiermacher G, Gambart M, Jannier S, Petit A, Dupraz C, Thebaud E, Reguerre Y, Ndounga-Diakou LA, Laghouat S, Defachelles AS, Berlanga P. Chemo-Immunotherapy Rescue for High-Risk Neuroblastoma Patients With Progressive Disease Before High-Dose Chemotherapy: Real-World Data From the SACHA-France Study. Pediatr Blood Cancer. 2025 Dec;72(12):e32080. doi: 10.1002/pbc.32080. Epub 2025 Oct 3.
PubMed 41044876Berlanga P, Aerts I, Corradini N, Ndounga-Diakou LA, Entz-Werle N, Ducassou S, Andre N, Sevrin F, Chastagner P, Puiseux C, Cleirec M, Plantaz D, De Carli E, Gambart M, Khanfar C, Thouvenin S, Petit A, Klein S, Briandet C, Millot F, Pluchart C, Reguerre Y, Schneider P, Serre J, Halfon-Domenech C, Carausu L, Piguet C, Saumet L, Benadiba J, Abbou S, Laghouati S, Geoerger B, Vassal G. Centralized Investigator Review of Radiological and Functional Imaging Reports in Real-World Oncology Studies: The SACHA-France Experience. Pediatr Blood Cancer. 2025 Feb;72(2):e31449. doi: 10.1002/pbc.31449. Epub 2024 Nov 19.
PubMed 39558828
Study at a glance
From the registry record, last updated by the sponsor on 31 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Securing Access to Innovative Molecules in Oncology and Hematology for Children, Adolescents and Young Adults in Therapeutic Failure or Relapse and Not Eligible for a Clinical Trial: a Project of the SFCE
- ClinicalTrials.gov ID
- NCT04477681
- Acronym / sponsor ID
- SACHA / 2019-A01317-50
- Conditions
- Pediatric Cancer
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 1,150 participants (estimated)
- Lead sponsor
- Gustave Roussy, Cancer Campus, Grand ParisUniversity, hospital or other
- Study start
- 9 Apr 2020 (actual)
- Primary completion
- Apr 2027 (estimated)
- Study completion
- Apr 2027 (estimated)
- First posted
- 20 Jul 2020 (actual)
- 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.
It involves collecting safety and efficacy data, under the actual conditions of use of medicines in children and adolescents, using a validated tool (Ennov EDC) and relying on the network of Interregional pediatric oncology appeal organizations (RIOs) identified by INCa since 2010 and responsible for the organization of Pluridisciplinary Pediatric Interregional (RCPPI) and National Consultative Meetings which discuss each case of relapse in order to define the best therapeutic options.
Detailed description
The objective is to aim for completeness throughout the national territory. This is why this project will be carried out in close collaboration with the RIOs and RCPPI and that of the 31 centers of the SFCE which bring together more than 400 doctors pediatric oncologists and hematologists on the French territory
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients in therapeutic failure or relapse | – | Other: Data collectionAny child, adolescent or young adult, treated for a pediatric tumor or leukemia, in therapeutic failure or relapse without standard treatment option, not eligible / refusal of inclusion in a clinical study open on the territory and treated with an innovative drug within the framework of a compassionate use or outside marketing authorization, in one of the centers of the SFCE (Société Française des Cancers de l' Enfant) |
Who may qualify
- Ages
- Up to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Any child, adolescent or young adult, treated for a pediatric tumor or leukemia, in therapeutic failure or relapse without standard treatment option, not eligible / refusal of inclusion in a clinical study open on the territory and treated with an innovative drug within the framework of compassionate use or outside marketing authorization, in one of the centers of the SFCE (Société Française des Cancers de l' Enfant).
Key inclusion criteria (6 listed)
- Age ≤ 25 years old at the time of inclusion in the study
- Patient with a pediatric tumor or leukemia in therapeutic failure or relapse without standard therapeutic options. Exceptionally, patients on the first line of treatment without standard therapeutic options may also be included (eg g: inoperable plexiform neurofibroma and MEK inhibitors, childhood fibrosarcoma and NTRK inhibitors).
- Patient not eligible for an early phase clinical trial open to inclusion on French territory or refusal of inclusion
- Patient treated with a new drug discussed at a RCPPI as part of a compassionate use issued by the ANSM or an off-label prescription of a drug of interest already authorized in adults.
- Patients treated in one of the SFCE centers authorized to prescribe chemotherapy
Key exclusion criteria (3 listed)
- Patient included in an early phase clinical trial open to inclusions on French territory.
- Oral refusal to participate by the patient or their legal representatives, in reading the information note specific to the study
- Patient under guardianship or curatorship, deprived of liberty or in the impossibility of expressing opposition to participating in the study
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 |
|---|---|---|
| Data collectionIn particular, the patient's demographic data, medical history, previous and concomitant treatments, data on the treatment of interest (legal framework, compassionate use/ off-label), dosage, start date, etc.), clinical data will be collected. biological (including molecular profiling of the tumor if available) and radiological follow-up, information on adverse effects. The data to be filled in is that of the CERFA pharmacovigilance form of the ANSM, but additional fields can be created if necessary. | Other | Patients in therapeutic failure or relapse |
Primary outcome
Collect access data
Time frame: From enrollment to the end of treatment maximum 7 years
Collect access data of children, adolescents and young adults in therapeutic failures and not eligible for a clinical trial with innovative molecules, whether they are targeted therapies, immunotherapies or chemotherapies
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Mar 2026.
- Background: PubMed 37399010Berlanga P, Ndounga-Diakou LA, Aerts I, Corradini N, Ducassou S, Strullu M, de Carli E, Andre N, Entz-Werle N, Raimbault S, Roumy M, Renouard M, Gueguen G, Plantaz D, Reguerre Y, Cleirec M, Petit A, Puiseux C, Andry L, Klein S, Bodet D, Kanold J, Briandet C, Halfon-Domenech C, Nelken B, Piguet C, Saumet L, Chastagner P, Benadiba J, Millot F, Pluchart C, Schneider P, Thouvenin S, Gambart M, Serre J, Abbou S, Leruste A, Cayzac H, Gandemer V, Laghouati S, Vassal G. Measuring Safety and Outcomes for the Use of Compassionate and Off-Label Therapies for Children, Adolescents, and Young Adults With Cancer in the SACHA-France Study. JAMA Netw Open. 2023 Jul 3;6(7):e2321568. doi: 10.1001/jamanetworkopen.2023.21568.
- Derived from this study: PubMed 41044876Pasqualini C, Proust S, Schleiermacher G, Gambart M, Jannier S, Petit A, Dupraz C, Thebaud E, Reguerre Y, Ndounga-Diakou LA, Laghouat S, Defachelles AS, Berlanga P. Chemo-Immunotherapy Rescue for High-Risk Neuroblastoma Patients With Progressive Disease Before High-Dose Chemotherapy: Real-World Data From the SACHA-France Study. Pediatr Blood Cancer. 2025 Dec;72(12):e32080. doi: 10.1002/pbc.32080. Epub 2025 Oct 3.
- Derived from this study: PubMed 39558828Berlanga P, Aerts I, Corradini N, Ndounga-Diakou LA, Entz-Werle N, Ducassou S, Andre N, Sevrin F, Chastagner P, Puiseux C, Cleirec M, Plantaz D, De Carli E, Gambart M, Khanfar C, Thouvenin S, Petit A, Klein S, Briandet C, Millot F, Pluchart C, Reguerre Y, Schneider P, Serre J, Halfon-Domenech C, Carausu L, Piguet C, Saumet L, Benadiba J, Abbou S, Laghouati S, Geoerger B, Vassal G. Centralized Investigator Review of Radiological and Functional Imaging Reports in Real-World Oncology Studies: The SACHA-France Experience. Pediatr Blood Cancer. 2025 Feb;72(2):e31449. doi: 10.1002/pbc.31449. Epub 2024 Nov 19.
- Institutional website (Gustave Roussy)Link provided by the sponsor.
- Individual participant dataSharing planned: Undecided
Related trials for Pediatric Cancer
Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
RecruitingObservational study
Psychological Benefits of a Normalized Camping Experience for Children With Cancer
Pediatric Cancer
Active, not recruitingPhase 3
An Extension Study Evaluating 6-week Treatment Cycles of Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
RecruitingObservational study
The Decision To Enroll In Therapeutic Clinical Trials In A Pediatric Cancer Clinical Network
Knowledge, Attitudes, Practice
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.
32 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pediatric Cancer, run by Gustave Roussy, Cancer Campus, Grand Paris at 32 sites in France.
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 6 inclusion and 3 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: looking for participants now, as of the sponsor's last update on 31 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in France. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Pablo BERLANGA, MD (+33 (0)1 42 11 42 11, pablo.berlanga@gustaveroussy.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Collect access data (time frame: From enrollment to the end of treatment maximum 7 years). 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 NCT04477681, which also lists 3 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 31 Mar 2026.