NCT02813135

European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 472 (estimated)
• Sponsor: Gustave Roussy, Cancer Campus, Grand Paris · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This proof-of-concept platform trial is designed to cover the targeting of several survival pathways in oncogenesis that are currently not adequately employed for pediatric patients in Europe (Geoerger 2017; Geoerger 2019). The aims of the trial are: 1. To determine the recommended phase II dose (RP2D) of a specific anticancer agent and/or a relevant combination in a pediatric population, to document its tolerability and 2. To explore first signals of activity in a molecularly enriched study population.

Detailed description

The first molecular profiling protocols have been launched in Europe (MOSCATO-01 (Geoerger 2014), MAPPYACTS, INFORM, iTHER, SM-PAEDS, etc.) determining multiple actionable alterations in pediatric recurrent cancers. Increasingly, stratified approaches are being implemented to enrich clinical trials of molecularly targeted agents and possibly improve outcomes in specific populations i.e. a molecularly enriched/predictive biomarker-driven approach. The diversity and heterogeneity of the detected molecular alterations and the low number of pediatric patients mandate an adapted, innovative trial design for the attributed treatment options in order to satisfy the current unmet medical need. This basket trial is designed to cover the targeting of several survival pathways in oncogenesis that are currently not adequately employed for pediatric patients in Europe.

Conditions

• Pediatric Cancer

Interventions

Type	Name	Description
DRUG	Ribociclib
DRUG	Topotecan
DRUG	Temozolomide
DRUG	Everolimus
DRUG	Adavosertib
DRUG	Carboplatin
DRUG	Olaparib
DRUG	Irinotecan
DRUG	Vistusertib
DRUG	Nivolumab
DRUG	Cyclophosphamide
DRUG	Selumetinib
DRUG	Enasidenib
DRUG	Lirilumab
DRUG	Fadraciclib
DRUG	Cytarabine
DRUG	Dexamethasone
DRUG	Ceralasertib
DRUG	Futibatinib
DRUG	Capmatinib
DRUG	Avelumab
DRUG	Peposertib
DRUG	Capivasertib
DRUG	Vinorelbine

Timeline

• Start date: 2016-08-03
• Primary completion: 2031-02-01
• Completion: 2031-02-01
• First posted: 2016-06-24
• Last updated: 2026-01-16

Locations

21 sites across 6 countries: Denmark, France, Italy, Netherlands, Spain, United Kingdom