A Phase I/II Study of Repotrectinib in Children and Young Adults with Advanced or Metastatic Cancers Containing ALK, ROS1, or NTRK1-3 Alterations


Full Title

A Phase 1/2, Open-label, Safety, Tolerability, Pharmacokinetics, and Anti-tumor Activity Study of Repotrectinib in Pediatric and Young Adult Subjects with Advanced or Metastatic Malignancies Harboring ALK, ROS1, or NTRK1-3 Alterations (CARE)


The purpose of the phase I part of this study is to find the highest dose of the investigational drug repotrectinib that can be given in children with locally advanced or metastatic cancers containing altered versions of the ALK, ROS1, or NTRK1-3 proteins. The phase II part of the study will see how effective repotrectinib is in children and young adults with cancers containing these altered proteins.

ALK, ROS1, and NTRK1-3 are found in or on the surface of cells, and cancer cells need them to survive and grow. Repotrectinib blocks these proteins, which may cause a cancer to stop growing, grow more slowly, or shrink. Repotrectinib is a capsule that is taken orally (by mouth).

Who Can Join

To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have a locally advanced or metastatic cancer containing altered ALK, ROS1, or NTRK1-3. Examples include primary central nervous system cancer (brain or spinal cord cancer), anaplastic large cell lymphoma, and neuroblastoma.
  • Patients must have a cancer that has continued to grow despite standard therapy or for which no standard therapy exists.
  • Patients must recover from the serious side effects of previous therapies before entering the study.
  • The phase I part of the study is for patients ages 4-11; the phase II part is for patients ages 4-25.

For more information about this study and to inquire about eligibility, please contact Dr. Stephen Roberts at 212-639-4034.




Phase I/II (phases 1 and 2 combined)

Disease Status

Newly Diagnosed & Relapsed/Refractory



ClinicalTrials.gov ID