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| Diagnosis | Solid or brain tumour with a change in the RET gene | Study Status | Open |
| Phase | I/II |
| Age | Child, Adult - (6 Months to 21 Years) | Randomisation | NO |
| Line of treatment | Disease relapse or progression |
| Routes of Treatment Administration | Selpercatinib is taken by mouth (capsules or oral suspension)
Participants <18 years of age may be offered to try a tablet formulation of selpercatinib. |
| Last Posted Update | 2024-01-03 |
| ClinicalTrials.gov # | NCT03899792 |
International Sponsor
Loxo Oncology, Inc.Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Daniel MorgensternCentres
Study Description
Brief 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.
With a recent amendment, stable patients who have been on study for over 2 years may discuss the option of a drug treatment holiday with their clinical team.
Inclusion Criteria
- Advanced or metastatic solid or primary CNS tumor which has failed standard of care therapies
- Evidence of an activating RET gene alteration in the tumor and/or blood
- Measurable or non-measurable disease
- Karnofsky (participants 16 years and older) or Lansky (participants younger than 16) performance score of at least 50
- Participant with primary CNS tumors or cerebral metastases must be neurologically stable for 7 days prior and must not have required increasing doses of steroids within the last 7 days
- Adequate hematologic, hepatic and renal function.
- Ability to receive study drug therapy orally or via gastric access
- Willingness of men and women of reproductive potential to observe conventional and effective birth control
Exclusion Criteria
- Major surgery within two weeks prior to planned start of LOXO-292
- Clinically significant, uncontrolled cardiac, cardiovascular disease or history of myocardial infarction within 6 months prior to planned start of LOXO-292
- Active uncontrolled systemic bacterial, viral, fungal or parasitic infection
- Clinically significant active malabsorption syndrome
- Pregnancy or lactation
- Uncontrolled symptomatic hyperthyroidism or hypothyroidism (i.e. the participant required a modification to current thyroid medication in the 7 days before start of LOXO-292)
- Uncontrolled symptomatic hypercalcemia or hypocalcemia
- Known hypersensitivity to any of the components of the investigational agent, LOXO-292 or Ora-Sweet® SF and OraPlus®, for participants who will receive LOXO-292 suspension
- Prior treatment with a selective RET inhibitor(s) (including investigational selective RET inhibitor[s])
