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

Pediatric Cancer Clinical Trial

— SACHA  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04477681
• Other study ID #: 2019-A01317-50
• Secondary ID: 2019/2848
• Status: Recruiting
• Start date: April 9, 2020
• Est. completion date: April 2026

Study information

• Verified date: June 2023
• Source: Gustave Roussy, Cancer Campus, Grand Paris
• Contact: Pablo BERLANGA, MD
• Phone: +33 (0)1 42 11 42 11
• Email: pablo.berlanga[@]gustaveroussy.fr
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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 (OIR) 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.

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 750
• Est. completion date: April 2026
• Est. primary completion date: April 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 25 Years

Eligibility

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 an ICSID as part of an ATU (nominative or cohort) 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

Related Conditions & MeSH terms

• Pediatric Cancer

Intervention

Other:
• Data collection
In particular, the patient's demographic data, medical history, previous and concomitant treatments, data on the treatment of interest (legal framework, ATU / 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

Country	Name	City	State
France	CHU Amiens	Amiens
France	CHU Angers	Angers
France	CHU Besançon	Besançon
France	CHU Bordeaux	Bordeaux
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-Étienne
France	CHU Strasbourg	Strasbourg
France	CHU Toulouse	Toulouse
France	CHU Tours	Tours
France	Gustave Roussy	Villejuif	Val De Marne

Sponsors (1)

Lead Sponsor	Collaborator
Gustave Roussy, Cancer Campus, Grand Paris

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Collect access data	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	up to 1 year