Securing Access to Innovative Molecules in Oncology and Hematology for Children, Adolescents and Young Adults
SACHA
2 other identifiers
observational
1,150
1 country
32
Brief 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 of Pluridisciplinary Pediatric Interregional (RCPPI) and National Consultative Meetings which discuss each case of relapse in order to define the best therapeutic options.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Apr 2020
Longer than P75 for all trials
32 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 9, 2020
CompletedFirst Submitted
Initial submission to the registry
July 15, 2020
CompletedFirst Posted
Study publicly available on registry
July 20, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
April 1, 2027
March 31, 2026
March 1, 2026
7 years
July 15, 2020
March 27, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
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
From enrollment to the end of treatment maximum 7 years
Study Arms (1)
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)
Interventions
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.
Eligibility Criteria
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).
You may qualify if:
- 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 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.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (32)
Gustave Roussy
Villejuif, Val de Marne, 94805, France
CHU Amiens
Amiens, France
CHU Angers
Angers, France
CHU Besançon
Besançon, France
CHU Bordeaux
Bordeaux, France
CHU Morvan
Brest, 29200, France
CHU CAEN
Caen, France
CHU Clermont-Ferrand
Clermont-Ferrand, France
CHU Dijon
Dijon, France
CHU Grenoble
Grenoble, France
Centre Oscar Lambret
Lille, France
CHU Lille
Lille, France
CHU Limoges
Limoges, France
Centre Léon Bérard
Lyon, France
CHU Lyon
Lyon, France
Hôpital de La Timone
Marseille, France
CHU Montpellier
Montpellier, France
CHU Nancy
Nancy, France
CHU Nantes
Nantes, France
CHU Nice
Nice, France
Hôpital Armand Trousseau
Paris, France
Hôpital Robert-Debré
Paris, France
Institut Curie
Paris, France
CHU Poitiers
Poitiers, France
CHU Reims
Reims, France
CHU Rennes
Rennes, France
CHU Rouen
Rouen, France
CHU La Réunion
Saint-Denis, France
CHU Saint-Etienne
Saint-Etienne, France
CHU Strasbourg
Strasbourg, France
CHU Toulouse
Toulouse, France
CHU Tours
Tours, France
Related Publications (2)
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.
PMID: 37399010BACKGROUNDPasqualini 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.
PMID: 41044876DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Intervention Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 15, 2020
First Posted
July 20, 2020
Study Start
April 9, 2020
Primary Completion (Estimated)
April 1, 2027
Study Completion (Estimated)
April 1, 2027
Last Updated
March 31, 2026
Record last verified: 2026-03