Full TitleA Phase 1/2 Study of the Oral TRK Inhibitor LOXO-101 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.