There might be a clinical benefit of treating patients based on actionable molecular events. Compound screening on tumor tissue might reveal additional treatment options for children who currently have none.
ID
Bron
Verkorte titel
Aandoening
Relapsed/refractory pediatric cancer, which was established by standard diagnostic methods OR initially diagnosed pediatric cancer patients for whom no standard treatment strategy is available or who have a dismal outcome with current treatment protocols.
Ondersteuning
Onderzoeksproduct en/of interventie
Uitkomstmaten
Primaire uitkomstmaten
To determine the objective response rates (complete response (CR), very good partial response (VGPR) or partial response (PR)) in pediatric patients with very high risk, relapsed or refractory tumors harboring actionable genomic alterations, treated with pathway-targeting agents compared to a non-treated cohort.
Achtergrond van het onderzoek
Significant progress has been made in the cure of pediatric patients with cancer through optimization of conventional treatment strategies and supportive care. Despite these major advances, one in four children will ultimately die due to lack of effective protocols. One of the most promising options to improve outcomes for pediatric cancer patients is through mutation-based targeted therapeutic strategies. To be able to facilitate enrollment into clinical trials molecular profiling will be transferred to the Princess Máxima Center during this trial. However, only 50% of high risk, relapsed or refractory tumors harbor actionable events. In addition, little is known on outcomes of targeted treatment in children. The primary objective of this study is therefore, to evaluate the clinical benefit of treating patients based on actionable molecular events. Compound screening on tumor tissue might reveal additional treatment options for children who currently have none.
Doel van het onderzoek
There might be a clinical benefit of treating patients based on actionable molecular events. Compound screening on tumor tissue might reveal additional treatment options for children who currently have none.
Onderzoeksopzet
not yet known
Onderzoeksproduct en/of interventie
none
Publiek
Miriam Stumpf
+31 650006609
m.k.stumpf@prinsesmaximacentrum.nl
Wetenschappelijk
Miriam Stumpf
+31 650006609
m.k.stumpf@prinsesmaximacentrum.nl
Belangrijkste voorwaarden om deel te mogen nemen (Inclusiecriteria)
• Relapsed/refractory pediatric cancer, which was established by standard diagnostic methods OR initially diagnosed pediatric cancer patients for whom no standard treatment strategy is available or who have a dismal outcome with current treatment protocols. In the case of very high risk primary disease, the results obtained in the frame of this study may be used at the physician’s discretion to improve a standard treatment strategy and may be applied a at later time point.
• A tissue sample or tumor DNA/RNA as well as normal DNA was obtained as standard of care at diagnosis, resection or at the event of refractory or relapsed disease or is planned with the purpose to confirm the diagnosis or a suspected relapse/refractory disease.
• Life-expectancy of at least 12 weeks.
• Written informed consent according to local law and legislation
• Age <30 years.
Belangrijkste redenen om niet deel te kunnen nemen (Exclusiecriteria)
• Patients cannot participate in this study if there is no tissue or DNA/RNA available.
• If patients and parents do not want to be informed about detected germline abnormalities
Opzet
Deelname
Voornemen beschikbaar stellen Individuele Patiënten Data (IPD)
Opgevolgd door onderstaande (mogelijk meer actuele) registratie
Geen registraties gevonden.
Andere (mogelijk minder actuele) registraties in dit register
Geen registraties gevonden.
In overige registers
| Register | ID |
|---|---|
| NTR-new | NL8126 |
| Ander register | METC UMCU : Advies(niet-)WMO onderzoek 20-150 |