A Phase 1/1b, Open-Label, Dose-Escalation and Expansion Study of Entrectinib (RXDX-101) in Children and Adolescents with Recurrent or Refractory Solid Tumors and Primary CNS Tumors, with or without TRK, ROS1, or ALK Fusions

Protocol: 
AAAR2419
Phase: 
I

A Phase 1/1b, Open-Label, Dose-Escalation and Expansion Study of Entrectinib (RXDX-101) in Children and Adolescents with Recurrent or Refractory Solid Tumors and Primary CNS Tumors, with or without TRK, ROS1, or ALK Fusions

The purpose of this study is to find out the effect and safety of an investigational (experimental) drug called entrectinib in children, adolescents and young adults with treatment-resistant solid tumor in brain or body. This study consists in two phase, Phase 1 part dose escalation, will determine which is the right dose of entrectinib to be used in children/adolescents/adults with relapsed (has returned) or refractory (did not respond to standard treatment) solid tumor.
Phase 1b part dose expansion portion (one dose will be tested) will further evaluate the effect and safety of entrectinib in pediatric patients whose cancer was previously shown via molecular testing to have specific changes in cancer cells.

Are you Eligible? (Inclusion Criteria)

- Male or female age ≥ 2 years and < 22 years unless enrolled in Part E
- Ability to swallow capsules unless enrolled in Part E
-Tumor type:
Part A: Relapsed or refractory extracranial solid tumors
Part B: Relapsed or refractory primary brain tumors with NTRK1/2/3, ROS1, or ALK
molecular alterations.
Part C: Relapsed or refractory neuroblastoma
Part D: Relapsed or refractory non-neuroblastoma, extracranial solid tumors with
NTRK1/2/3, ROS1, or ALK gene fusions
Part E: Any subject eligible for Parts A (expansion), B, C, or D age ≥ 2 years and < 22
years, who is unable to swallow capsules, and all subjects < 2 years.
-Other Inclusion Criteria may apply.

Trial Location

Herbert Irving Comprehensive Cancer Center
161 Fort Washington Ave.
Herbert Irving Pavilion
New York, NY 10032
United States