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

June 2, 2023 updated by: Gustave Roussy, Cancer Campus, Grand Paris

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed 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

Study Type

Observational

Enrollment (Estimated)

750

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 25 years (Child, Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Any child, adolescent or young adult, treated for a pediatric tumor or leukemia, in therapeutic failure or relapse without standard treatment option, not eligible / refusal of inclusion in a clinical study open on the territory and treated with an innovative drug within the framework of an ATU or outside AMM, in one of the centers of the SFCE (Société Française Cancer Enfant).

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Patients in therapeutic failure or relapse
Any child, adolescent or young adult, treated for a pediatric tumor or leukemia, in therapeutic failure or relapse without standard treatment option, not eligible / refusal of inclusion in a clinical study open on the territory and treated with an innovative drug within the framework of an ATU or outside AMM, in one of the centers of the SFCE (Société Française Cancer Enfant)
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Collect access data
Time Frame: up to 1 year
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Gustave Roussy, Cancer Campus, Grand Paris

Collaborators

Société Française des Cancers de l'Enfant (SFCE)

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 9, 2020

Primary Completion (Estimated)

April 1, 2025

Study Completion (Estimated)

April 1, 2026

Study Registration Dates

First Submitted

July 15, 2020

First Submitted That Met QC Criteria

July 15, 2020

First Posted (Actual)

July 20, 2020

Study Record Updates

Last Update Posted (Actual)

June 6, 2023

Last Update Submitted That Met QC Criteria

June 2, 2023

Last Verified

June 1, 2023

More Information

Terms related to this study

Other Study ID Numbers

  • 2019-A01317-50
  • 2019/2848 (Other Identifier: CSET number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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