Larotrectinib as TRK-inhibitor in children with solid tumors

"Phase 1/2 Study of the Oral TRK Inhibitor LOXO-101 in Pediatric Patients with Advanced Sold or Primary Central Nervous System Tumors"

In this study, children and adolescents aged 1 to 21 years with an advanced or metastatic solid tumor - a primary tumor of the central nervous system or IFS (infantile fibrosarcoma) - are treated with LOXO-101 (Larotrectinib). Larotrectinib specifically inhibits tropomyosin receptor kinases (TRK), a group of signaling proteins that play an important role in cellular communication and tumor growth. The study therefore treats tumors whose genetic information has changed in this area (NTRK fusion) and where the tumor has recurred or progressed after standard therapy. Larotrectinib is taken orally over a 28-day cycle.

The inclusion of patients in Heidelberg has been possible since 22.06.2017.

Inclusion criteria for phase 2:

  • Children and adolescents between 0 and 21 years with a locally advanced or metastatic IF with a positive ETS gene variation 6 (ETV6) detected by FISH or by molecular analysis
  • Children and adolescents between 0 and 21 years with a locally advanced solid tumors or primary central nervous system tumors with NTRK gene fusion
  • At least one measurable lesion according to RECIST or RANO
  • Karnofsky oder Lansky > 50

For more information, see the Study Registry: The study is registered with the U.S. National Institutes of Health (ClinicalTrials.gov).
See NCT02637687

EudraCT-number: 2016-003498-16 (see EU clinical trial register)