Selpercatinib, a RET-Inhibitor for children with solid tumours and brain tumours

 “A Phase 1/2 Study of the Oral RET Inhibitor LOXO-292 in Pediatric Patients with Advanced RET-Altered Solid or Primary Central Nervous System Tumors Safety and Tolerability, Efficacy and Pharmacokinetics in Paediatric Patients with Solid Tumours (LOXO-RET-18036)”

In this clinical trial children, adolescents and young adults between 12 and 21 years will be treated with Selpercatinib (Loxo-292).

Selpercatinib specifically inhibits the Receptor-Tyrosine Kinase (RET), meaning it blocks a type of enzyme (kinase) and helps prevent the cancer cells from growing. Before beginning treatment, the identification of a RET gene alteration must be determined using laboratory testing.

Therefore, in this clinical trial adnvanced tumours with RET-alterations are treated after standard therapy. Selpercatinib i staken orally over a 28-day cycle as gelatine capsules or as a liquid suspension.

Inclusion of patients in Heidelberg has been possible since 16 July 2020.

  • Pediatric patients ≥ 12 years of age and ≤ 21 years of age at Cycle 1 Day 1 (C1D1) with a locally advanced or metastatic solid or primary CNS tumor that has relapsed, progressed or was nonresponsive to available therapies and/or for which no standard or available systemic curative therapy exists
  • Evidence of an activating RET gene alteration in tumor and/or blood (e.g., gene rearrangement and/or mutation, excluding synonymous, frameshift, or nonsense mutations)
  • Karnofsky oder Lansky > 50

For more information, see the Study Registry: The study is registered with the U.S. National Institutes of Health (
See NCT03899792

EudraCT-Number: 2019-000212-28