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

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeUp to 25
SponsorGustave Roussy, Cancer Campus, Grand Paris

About this trial

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.

Eligibility criteria

Qualifiers

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.

Disqualifiers

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 design

Treatments tested in this trial

  • Data collection

Treatment groups

1,150 Participants
are divided into 1 treatment group