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

Официальное название

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

Обзор

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.

Подробное описание

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

Вмешательства

• Другое Data collection
  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 neces

Первичные конечные точки

• Collect access data [Срок оценки: From enrollment to the end of treatment maximum 7 years]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Франция · 32 центра

• Gustave Roussy — Villejuif
• CHU Amiens — Amiens
• CHU Angers — Angers
• CHU Besançon — Besançon
• CHU Bordeaux — Bordeaux
• CHU Morvan — Brest
• CHU CAEN — Caen
• CHU Clermont-Ferrand — Clermont-Ferrand
• … и ещё 24 центра

Публикации

• 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 PMID 37399010
• Pasqualini 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. PMID 41044876

Идентификаторы

NCT: NCT04477681 · 2019-A01317-50 · 2019/2848

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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