Full TitleA Phase 1/2 Study of the Oral TRK Inhibitor Larotrectinib in Pediatric Patients with Advanced Solid or Primary Central Nervous System Tumors
The purpose of this study is to evaluate the safety and effectiveness of the investigational drug LOXO-101 to treat children with infantile fibrosarcoma, primary brain tumors, or other advanced solid tumors. LOXO-101 works by inhibiting a group of genes called NTRK 1/2/3. These genes are thought to play a role in the development of several different childhood cancers. LOXO-101 is taken orally (by mouth).
To be eligible for this study, patients must meet several criteria, including but not limited to the following:
- For the Phase I portion of this study, patients must be between the ages of 1 and 21 years of age and have a brain tumor or other advanced solid tumor that is not responding to standard therapy; be age 1 month or older with a persistent cancer containing an NTRK gene fusion; or be age 1 month or older and have infantile fibrosarcoma that would otherwise require disfiguring surgery or limb amputation.
- For the Phase II portion of this study, patients must be age 1 month or older and have infantile fibrosarcoma that would otherwise require disfiguring surgery or limb amputation; be age 1 month to age 21 years and have a persistent cancer containing an NTRK gene fusion; or be older than age 21 years and have a pediatric cancer with an NTRK gene fusion.
- Patients must recover from the serious side effects of prior therapies before entering the study.