NCT03899792

A Study of Oral LOXO-292 (Selpercatinib) in Pediatric Participants With Advanced Solid or Primary Central Nervous System (CNS) Tumors

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

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 36 (actual)
• Sponsor: Eli Lilly and Company · Industry
• Sex: All
• Age: 6 Months – 21 Years
• Healthy volunteers: Not accepted

Summary

This is an open-label, multi-center Phase 1/2 study of oral LOXO-292 in pediatric participants with an activating rearranged during transfection (RET) alteration and an advanced solid or primary CNS tumor.

Detailed description

This study includes 2 parts: phase 1 (dose escalation) and phase 2 (dose expansion). In phase 1, participants will be enrolled using a rolling 6 dose escalation scheme. The starting dose of LOXO-292 is equivalent to the adult recommended phase 2 dose of 160 milligrams (mg) twice a day (BID). Once the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) is identified, participants will be enrolled to one of four phase 2 dose expansion cohorts depending on tumor histology and tumor genotype. Cycle length will be 28 days.

Conditions

• Medullary Thyroid Cancer
• Infantile Myofibromatosis
• Infantile Fibrosarcoma
• Papillary Thyroid Cancer
• Soft Tissue Sarcoma

Interventions

Type	Name	Description
DRUG	Selpercatinib	Oral LOXO-292

Timeline

• Start date: 2019-06-13
• Primary completion: 2024-11-08
• Completion: 2029-05-01
• First posted: 2019-04-02
• Last updated: 2026-01-06
• Results posted: 2026-01-06

Locations

26 sites across 11 countries: United States, Australia, Canada, Denmark, France, Germany, Italy, Japan, South Korea, Spain, United Kingdom

Regulatory

• FDA-regulated drug study