Access to innovative cancer treatments for young patients
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
This study is trying to gather information on new cancer treatments for kids and young adults who aren't responding to standard therapies to see how safe and effective they are.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 1150 (estimated) |
| Ages | N/A to 25 Years |
| Sex | All |
| Sponsor | Gustave Roussy, Cancer Campus, Grand Paris Academic / other |
| Drugs / interventions | chemotherapy |
| Locations | 32 sites (Villejuif, Val de Marne and 31 other locations) |
| Trial ID | NCT04477681 on ClinicalTrials.gov |
What this trial studies
This observational study aims to collect safety and efficacy data on innovative cancer treatments for children, adolescents, and young adults facing therapeutic failure or relapse. It utilizes a validated data collection tool and collaborates with a network of pediatric oncology organizations across France. The study involves over 400 pediatric oncologists and hematologists who discuss each case to determine the best therapeutic options. The goal is to ensure comprehensive data collection throughout the national territory.
Who should consider this trial
Good fit: Ideal candidates include patients aged 25 or younger with pediatric tumors or leukemia in therapeutic failure or relapse without standard treatment options.
Not a fit: Patients who are eligible for early phase clinical trials or have standard therapeutic options available may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could improve treatment options and outcomes for young patients with limited therapeutic alternatives.
How similar studies have performed: Other studies focusing on innovative treatments for pediatric cancers have shown promise, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Villejuif, Val de Marne and 31 other locations
- Gustave Roussy — Villejuif, Val de Marne, France (Recruiting)
- CHU Amiens — Amiens, France (Recruiting)
- CHU Angers — Angers, France (Recruiting)
- CHU Besançon — Besançon, France (Recruiting)
- CHU Bordeaux — Bordeaux, France (Recruiting)
- CHU Morvan — Brest, France (Recruiting)
- Chu Caen — Caen, France (Recruiting)
- CHU Clermont-Ferrand — Clermont-Ferrand, France (Recruiting)
- CHU Dijon — Dijon, France (Recruiting)
- CHU Grenoble — Grenoble, France (Recruiting)
- Centre Oscar Lambret — Lille, France (Recruiting)
- CHU Lille — Lille, France (Recruiting)
- CHU Limoges — Limoges, France (Recruiting)
- Centre Léon Bérard — Lyon, France (Recruiting)
- CHU Lyon — Lyon, France (Recruiting)
- Hôpital de La Timone — Marseille, France (Recruiting)
- CHU Montpellier — Montpellier, France (Recruiting)
- CHU Nancy — Nancy, France (Recruiting)
- CHU Nantes — Nantes, France (Recruiting)
- CHU Nice — Nice, France (Recruiting)
- Hôpital Armand Trousseau — Paris, France (Recruiting)
- Hôpital Robert-Debré — Paris, France (Recruiting)
- Institut Curie — Paris, France (Recruiting)
- CHU Poitiers — Poitiers, France (Recruiting)
- CHU Reims — Reims, France (Recruiting)
- CHU Rennes — Rennes, France (Recruiting)
- CHU Rouen — Rouen, France (Recruiting)
- CHU La Réunion — Saint-Denis, France (Recruiting)
- CHU Saint-Etienne — Saint-Etienne, France (Recruiting)
- CHU Strasbourg — Strasbourg, France (Recruiting)
- CHU Toulouse — Toulouse, France (Recruiting)
- CHU Tours — Tours, France (Recruiting)
Study contacts
- Study coordinator: Pablo BERLANGA, MD
- Email: pablo.berlanga@gustaveroussy.fr
- Phone: +33 (0)1 42 11 42 11
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.