Securing Access to Innovative Molecules in Oncology and Hematology for Children, Adolescents and Young Adults

Recruiting

• Conditions: Pediatric Cancer
• Interventions: Other: Data collection

• Registration Number: NCT04477681
• Lead Sponsor: Gustave Roussy, Cancer Campus, Grand Paris

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

Study Timeline

• Study Start: 2020-04-09
• Study First Submitted: 2020-07-15
• Study First Submitted QC: 2020-07-15
• Study First Posted: 2020-07-20
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-10
• Last Update Posted: 2025-01-13
• Primary Completion: 2026-04
• Study Completion: 2027-04

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 1000

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients in therapeutic failure or relapse	Data collection	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 an ATU or outside AMM, in one of the centers of the SFCE (Société Française Cancer Enfant)

Primary Outcome Measures

Name	Time	Method
Collect access data	up to 1 year	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

Trial Locations

Locations (31)

CHU Lyon; 🇫🇷 Lyon, France

Institut Curie; 🇫🇷 Paris, France

CHU Strasbourg; 🇫🇷 Strasbourg, France

CHU Nancy; 🇫🇷 Nancy, France

CHU Nantes; 🇫🇷 Nantes, France

CHU Poitiers; 🇫🇷 Poitiers, France

CHU Amiens; 🇫🇷 Amiens, France

CHU Angers; 🇫🇷 Angers, France

CHU Limoges; 🇫🇷 Limoges, France

Hôpital Armand Trousseau; 🇫🇷 Paris, France

CHU Reims; 🇫🇷 Reims, France

Hôpital Robert-Debré; 🇫🇷 Paris, France

Gustave Roussy; 🇫🇷 Villejuif, Val De Marne, France

CHU Besançon; 🇫🇷 Besançon, France

CHU Clermont-Ferrand; 🇫🇷 Clermont-Ferrand, France

CHU Dijon; 🇫🇷 Dijon, France

Centre Oscar Lambret; 🇫🇷 Lille, France

CHU Lille; 🇫🇷 Lille, France

Centre Léon Bérard; 🇫🇷 Lyon, France

CHU Montpellier; 🇫🇷 Montpellier, France

Hôpital de La Timone; 🇫🇷 Marseille, France

CHU Nice; 🇫🇷 Nice, France

CHU Rennes; 🇫🇷 Rennes, France

CHU Rouen; 🇫🇷 Rouen, France

CHU La Réunion; 🇫🇷 Saint-Denis, France

CHU Saint-Etienne; 🇫🇷 Saint-Étienne, France

CHU Bordeaux; 🇫🇷 Bordeaux, France

CHU CAEN; 🇫🇷 Caen, France

CHU Grenoble; 🇫🇷 Grenoble, France

CHU Tours; 🇫🇷 Tours, France

CHU Toulouse; 🇫🇷 Toulouse, France