Access to innovative cancer treatments for young patients

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study aims to collect safety and efficacy data on innovative cancer treatments for children, adolescents, and young adults facing therapeutic failure or relapse. It utilizes a validated data collection tool and collaborates with a network of pediatric oncology organizations across France. The study involves over 400 pediatric oncologists and hematologists who discuss each case to determine the best therapeutic options. The goal is to ensure comprehensive data collection throughout the national territory.

Why it matters

Who can join: the full rules

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

Where it's running

• Gustave Roussy
  Villejuif, Val de Marne, France Recruiting
• CHU Amiens
  Amiens, France Recruiting
• CHU Angers
  Angers, France Recruiting
• CHU Besançon
  Besançon, France Recruiting
• CHU Bordeaux
  Bordeaux, France Recruiting
• CHU Morvan
  Brest, France Recruiting

Study contacts

• Study coordinator: Pablo BERLANGA, MD
• Email: pablo.berlanga@gustaveroussy.fr
• Phone: +33 (0)1 42 11 42 11

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.