A Study of Oral LOXO-292 (Selpercatinib) in Pediatric Participants With Advanced Solid or Primary Central Nervous System (CNS) Tumors
Sponsor: Loxo Oncology, Inc.
ClinicalTrials ID:NCT03899792
This open-label, multi-center Phase 1/2 study evaluates oral LOXO-292 in pediatric patients with advanced solid or primary CNS tumors with RET alterations. Phase 1 involves dose escalation to determine MTD/RP2D, followed by Phase 2 dose expansion. Eligible participants must have failed standard therapies, show RET alterations, and meet specific health criteria. LOXO-292 is administered orally.
Patient Parameters
| Parameter | Options |
|---|
Program Overview
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.
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.
Eligibility Criteria
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])
Locations
| FACILITY | ZIP | CITY | STATE |
|---|---|---|---|
| Childrens Hospital of Los Angeles | 90027 | Los Angeles | California |
| The Children's Hospital for Cancer and Blood Disorders | 80045 | Aurora | Colorado |
| Nemours Children's Health | 32827 | Orlando | Florida |
| Dana-Farber Cancer Institute | 02215 | Boston | Massachusetts |
| University Of Minnesota Hospital | 55454 | Minneapolis | Minnesota |
| Memorial Sloan Kettering Cancer Center | 10065 | New York | New York |
| Cincinnati Children's Hospital Medical Center | 45229-3039 | Cincinnati | Ohio |
| Children's Hospital of Philadelphia | 19104 | Philadelphia | Pennsylvania |
| St. Jude Children's Research Hospital | 38105 | Memphis | Tennessee |
| University of Texas Southwestern Medical Center at Dallas | 75390-9063 | Dallas | Texas |
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