Clinical trial NCT04477681: Securing Access to Innovative Molecules in Oncology and Hematology for Children, Adolescents and Young Adults
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
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Pediatric Cancer
Lead sponsor: Gustave Roussy, Cancer Campus, Grand Paris
Sponsor class: OTHER
First posted: 2020-07-20
Enrollment: 1000
Sex: ALL
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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).

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.

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

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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

Primary outcomes:
measure: Collect access data
timeFrame: up to 1 year
Secondary outcomes:
Other outcomes:
Locations:
city: Villejuif
country: France
facility: Gustave Roussy
state: Val De Marne
city: Amiens
country: France
facility: CHU Amiens
city: Angers
country: France
facility: CHU Angers
city: Besançon
country: France
facility: CHU Besançon
city: Bordeaux
country: France
facility: CHU Bordeaux
city: Caen
country: France
facility: CHU CAEN
city: Clermont-Ferrand
country: France
facility: CHU Clermont-Ferrand
city: Dijon
country: France
facility: CHU Dijon
city: Grenoble
country: France
facility: CHU Grenoble
city: Lille
country: France
facility: Centre Oscar Lambret
city: Lille
country: France
facility: CHU Lille
city: Limoges
country: France
facility: CHU Limoges
city: Lyon
country: France
facility: Centre Léon Bérard
city: Lyon
country: France
facility: CHU Lyon
city: Marseille
country: France
facility: Hôpital de La Timone
city: Montpellier
country: France
facility: CHU Montpellier
city: Nancy
country: France
facility: CHU Nancy
city: Nantes
country: France
facility: CHU Nantes
city: Nice
country: France
facility: CHU Nice
city: Paris
country: France
facility: Hôpital Armand Trousseau
city: Paris
country: France
facility: Hôpital Robert-Debré
city: Paris
country: France
facility: Institut Curie
city: Poitiers
country: France
facility: CHU Poitiers
city: Reims
country: France
facility: CHU Reims
city: Rennes
country: France
facility: CHU Rennes
city: Rouen
country: France
facility: CHU Rouen
city: Saint-Denis
country: France
facility: CHU La Réunion
city: Saint-Étienne
country: France
facility: CHU Saint-Etienne
city: Strasbourg
country: France
facility: CHU Strasbourg
city: Toulouse
country: France
facility: CHU Toulouse
city: Tours
country: France
facility: CHU Tours
Study officials:
References:
citation: 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.