SACHA: Securing Access to Innovative Molecules in Oncology and Hematology for Children, Adolescents and Young Adults
Study Details
Study Description
Brief Summary
It involves collecting safety and efficacy data, under the actual conditions of use of medicines in children and adolescents, using a validated tool (VIGINOM) 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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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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 French territory
Study Design
Arms and Interventions
| Arm | 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.
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Outcome Measures
Primary Outcome Measures
- Collect access data [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
Eligibility Criteria
Criteria
Inclusion Criteria:
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Age ≤ 25 years old at the time of inclusion in the study
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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).
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Patient not eligible for an early phase clinical trial open to inclusion on French territory or refusal of inclusion
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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.
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Patients treated in one of the SFCE centers authorized to prescribe chemotherapy
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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:
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Patient included in an early phase clinical trial open to inclusions on French territory.
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Oral refusal to participate by the patient or their legal representatives, in reading the information note specific to the study
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Patient under guardianship or curatorship, deprived of liberty or in the impossibility of expressing opposition to participating in the study
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Gustave Roussy | Villejuif | Val De Marne | France | 94805 |
Sponsors and Collaborators
- Gustave Roussy, Cancer Campus, Grand Paris
- Société Française des Cancers de l'Enfant (SFCE)
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 2019-A01317-50
- 2019/2848