(20290) A Phase 1/2 Study of the Oral TRK Inhibitor Larotrectinib in Pediatric Patients with Advanced Solid or Primary Central Nervous System Tumors

Solid tumors harboring NTRK fusion

Phase 1: Number of subjects in an assigned dose cohort with treatment emergent adverse events (TEAEs) by grade assessed by National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) v 4.03 who experience a Dose-limiting toxicity (DLT)., Phase 1: Number of participants with TEAEs, Phase 1: Severity of TEAEs, Phase 2: Overall response rate (ORR) by IRRC

Phase 1: Maximum concentration of larotrectinib in plasma (Cmax), Phase 1: Area under the concentration versus time curve from time 0 to t (AUC0–t) of larotrectinib in plasma, Phase 1: Oral clearance (CL/F), Phase 1: Cerebral spinal fluid/plasma ratio of larotrectinib, Phase 1: Maximum tolerated dose (MTD), Phase 1: Recommended dose for Phase 2, Phase 1: Overall Response Rate (ORR), Phase 1: Mean change from baseline in Pain scores as assessed by the Wong-Baker Faces scale, Phase 1: Mean change in Health-related quality of life scores by Pediatrics Quality of Life - Core Module (PedsQL-Core), Phase 2: Best overall response (BOR), Phase 2: Duration of response (DOR), Phase 2: Proportion of patients with any tumor regression (i.e., any decrease from baseline of the longest diameters of target lesions) as a best response, Phase 2: Progression-free survival (PFS), Phase 2: Overall survival (OS), Phase 2: Number of participants with Treatment emergent adverse events (TEAEs), Severity of adverse events as assessed by NCI-CTCAE grading V 4.03, Phase 2: Clinical Benefit Rate (CBR), Phase 2: Concordance coefficient, Phase 2: Post-operative tumor staging, Phase 2: Post-operative surgical margin assessment, Phase 2: Pre-treatment surgical plan to preserve function and cosmetic outcome, Phase 2: Post-treatment plans to conserve function and cosmetic outcome

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