RecruitingNCT04477681

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

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


Sponsor

Gustave Roussy, Cancer Campus, Grand Paris

Enrollment

1,150 participants

Start Date

Apr 9, 2020

Study Type

OBSERVATIONAL

Conditions

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.


Eligibility

Max Age: 25 Years

Inclusion Criteria6

  • 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 Criteria3

  • 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

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Interventions

OTHERData 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 necessary.


Locations(32)

Gustave Roussy

Villejuif, Val de Marne, France

CHU Amiens

Amiens, France

CHU Angers

Angers, France

CHU Besançon

Besançon, France

CHU Bordeaux

Bordeaux, France

CHU Morvan

Brest, France

CHU CAEN

Caen, France

CHU Clermont-Ferrand

Clermont-Ferrand, France

CHU Dijon

Dijon, France

CHU Grenoble

Grenoble, France

Centre Oscar Lambret

Lille, France

CHU Lille

Lille, France

CHU Limoges

Limoges, France

Centre Léon Bérard

Lyon, France

CHU Lyon

Lyon, France

Hôpital de La Timone

Marseille, France

CHU Montpellier

Montpellier, France

CHU Nancy

Nancy, France

CHU Nantes

Nantes, France

CHU Nice

Nice, France

Hôpital Armand Trousseau

Paris, France

Hôpital Robert-Debré

Paris, France

Institut Curie

Paris, France

CHU Poitiers

Poitiers, France

CHU Reims

Reims, France

CHU Rennes

Rennes, France

CHU Rouen

Rouen, France

CHU La Réunion

Saint-Denis, France

CHU Saint-Etienne

Saint-Etienne, France

CHU Strasbourg

Strasbourg, France

CHU Toulouse

Toulouse, France

CHU Tours

Tours, France

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NCT04477681


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