Oral TRK Inhibitor LOXO-101 (Larotrectinib) for Treatment of Advanced Pediatric Solid or Primary Central Nervous System Tumours

Official Title

A Phase 1/2 Study of the Oral TRK Inhibitor LOXO-101 (Larotrectinib) in Pediatric Patients With Advanced Solid or Primary Central Nervous System Tumours

Summary:

This is a multicentre, open label, Phase 1/2 study in pediatric patients with advanced solid or primary CNS tumours. LOXO‑101 (larotrectinib) will be administered orally (PO) twice daily (BID), with the dose adjusted by body surface area (BSA).

Trial Description

Primary Outcome:

  • Phase 1: To determine the safety of oral LOXO-101 (larotrectinib) in pediatric patients with advanced solid or primary central nervous system (CNS) tumours. Phase 2: To determine the overall response rate (ORR).
Secondary Outcome:
  • To characterize the pharmacokinetic (PK) properties of LOXO-101 (larotrectinib) in pediatric patients with advanced solid or primary CNS tumours
  • To identify the recommended maximum tolerated dose and/or the appropriate dose of LOXO‑101 (larotrectinib) for further clinical investigation in this patient population
  • To describe the antitumour activity of LOXO-101 (larotrectinib) in pediatric patients with advanced solid or primary CNS tumours
  • To describe pain and health related quality of life (HRQOL) in pediatric patients with advanced solid or primary CNS tumours treated with LOXO-101 (larotrectinib)
  • To assess the safety profile and tolerability of LOXO-101 (larotrectinib)
  • To evaluate the concordance of prior molecular profiling that detected an NTRK fusion within the subject's tumour with diagnostic tests being evaluated by the Sponsor
  • To characterize post-operative staging and surgical margin status in patients who have definitive surgery following treatment with LOXO-101 (larotrectinib)
  • To describe the putitive pretreatment surgical plan and capture the post treatment actual approach with an emphasis on the functional and cosmetic outcome
This is a multicentre, open label, Phase 1/2 study in pediatric patients with advanced solid or primary CNS tumours. LOXO‑101 (larotrectinib) will be administered orally (PO) twice daily (BID), with the dose adjusted by body surface area (BSA). Dose Escalation Phase has completed enrollment and it has been determined that the maximum dose for the Phase 1 Expansion and Phase 2 portions of the study will be no higher than the recommended Phase 2 dose of 100 mg BID in the adult Phase 2 trial, regardless of the patient's BSA. Phase 1 Expansion and Phase 2 portions of the study are currently enrolling.

View this trial on ClinicalTrials.gov

Interested in this trial?

Print this page and take it to your doctor to discuss your eligibilty and treatment options. Only your doctor can refer you to a clinical trial.

Resources

Canadian Cancer Society

These resources are provided in partnership with the Canadian Cancer Society