At a glance
ClinicalIndex Comparison Record- ✓Diagnosed with hematologic or solid tumor malignancy that has progressed despite standard therapy or for which no effective standard therapy exists
- ✓Age < 18 years at inclusion; patients 18+ may be included after sponsor discussion if they have pediatric recurrent/refractory malignancy
- ✓Advanced molecular profiling (WES/WGS +/- RNAseq) of recurrent or refractory tumor at disease progression/relapse
- ✓Evaluable or measurable disease by standard imaging criteria for tumor type
- ✕Symptomatic CNS metastases that are neurologically unstable or require increasing corticosteroid doses or local CNS-directed therapy
- ✕Impairment of gastrointestinal function or disease significantly altering drug absorption (ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption)
- ✕Clinically significant, uncontrolled heart disease including cardiac arrhythmias, unstable ischemia, congestive heart failure within 12 months
- ✕Active viral hepatitis, HIV infection, or any other uncontrolled infection
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
European Proof-of-Concept Therapeutic Stratification Trial of Molecular Anomalies in Relapsed or Refractory Tumors
In Brief
A Phase 2 clinical trial evaluating Ribociclib, Topotecan, and 22 other interventions for Pediatric Cancer. Currently recruiting, targeting 472 participants across 21 sites in 6 countries.
Signals
Detailed 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.