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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

1000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-04-09

Study Completion Date

2027-04-30

Brief Summary

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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.

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Detailed Description

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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

Conditions

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Pediatric Cancer

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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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 a compassionate use or outside marketing authorization, in one of the centers of the SFCE (Société Française des Cancers de l' Enfant)

Data collection

Intervention Type OTHER

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.

Interventions

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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 necessary.

Intervention Type OTHER

Eligibility Criteria

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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 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 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

Maximum Eligible Age

25 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

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France

Central Contacts

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Pablo BERLANGA, MD

Role: CONTACT

[email protected]

+33 (0)1 42 11 42 11

Lee Aymar NDOUNGA DIAKOU, PhD

Role: CONTACT

[email protected]

+33 (0)1 42 11 42 11

Facility Contacts

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Pablo BERLANGA, MD

Role: primary

[email protected]

References

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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, Abbou S, Leruste A, Cayzac H, Gandemer V, Laghouati S, Vassal G. Measuring Safety and Outcomes for the Use of Compassionate and Off-Label Therapies for Children, Adolescents, and Young Adults With Cancer in the SACHA-France Study. JAMA Netw Open. 2023 Jul 3;6(7):e2321568. doi: 10.1001/jamanetworkopen.2023.21568.

Reference Type BACKGROUND

PMID: 37399010 (View on PubMed)

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 Oct 3:e32080. doi: 10.1002/pbc.32080. Online ahead of print.

Reference Type DERIVED

PMID: 41044876 (View on PubMed)

Other Identifiers

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2019/2848

Identifier Type: OTHER

Identifier Source: secondary_id

2019-A01317-50

Identifier Type: -

Identifier Source: org_study_id

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Study Results

Basic Information

Brief Summary

Related Clinical Trials

Proof -of -Concept Study To Stratify Targeted Therapies Adapted To Molecular Profiling

Open Dose Escalating Trial to Determine the Maximum Tolerated Dose in Paediatric Patients With Advanced Cancers for Whom no Therapy is Known

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Detailed Description

Conditions

Study Design

Study Groups

Patients in therapeutic failure or relapse

Data collection

Interventions

Data collection

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

Gustave Roussy, Cancer Campus, Grand Paris

Responsible Party

Locations

Gustave Roussy

CHU Amiens

CHU Angers

CHU Besançon

CHU Bordeaux

CHU CAEN

CHU Clermont-Ferrand

CHU Dijon

CHU Grenoble

Centre Oscar Lambret

CHU Lille

CHU Limoges

Centre Léon Bérard

CHU Lyon

Hôpital de La Timone

CHU Montpellier

CHU Nancy

CHU Nantes

CHU Nice

Hôpital Armand Trousseau

Hôpital Robert-Debré

Institut Curie

CHU Poitiers

CHU Reims

CHU Rennes

CHU Rouen

CHU La Réunion

CHU Saint-Etienne

CHU Strasbourg

CHU Toulouse

CHU Tours

Countries

Central Contacts

Pablo BERLANGA, MD

Lee Aymar NDOUNGA DIAKOU, PhD

Facility Contacts

Pablo BERLANGA, MD

Leslie ANDRY, MD

Emilie DE CARLI, MD

Sébastien KLEIN, MD

Stéphane DUCASSOU, MD

Damien BODET, MD

Justyna KANOLD

Claire BRIANDET, MD

Dominique PLANTAZ, MD

Sandra RAIMBAULT, MD

Brigitte NELKEN, MD

Christophe PIGUET, MD

Nadège CORRADINI, MD

Carine HALFON-DOMENECH, MD

Nicolas ANDRE, MD

Laure SAUMET, MD

Pascal CHASTAGNER, MD

Morgane CLEIREC, MD

Joy BENADIBA, MD