Pediatric Phase 1 Dose-Finding Study of Entrectinib Shows Substantial Benefit in Almost Half the Patients

by John Otrompke

A second study in pediatric oncology discussed at the ASCO presscast also was the source of surprisingly good news.

STARTRK-NG was designed as a simple phase I/IB dose-finding study, but objective responses were seen in almost half of the subjects. The study enrolled 29 patients, aged 4 to 20, with rare central nervous system tumors, neuroblastoma, or other solid tumors, of whom 28 were evaluable.

Entrectinib, a novel targeted treatment, was administered for a median of 281 days, and the tumor shrank or disappeared in 12 patients. The median time to response was 57 days, according to abstract 10009, “Phase 1/1B trial to assess the activity of entrectinib in children and adolescents with recurrent or refractory solid tumors including central nervous system (CNS) tumors” (Robinson, Gajjar, Gauvain, et al).

“The medicine was well-tolerated and there appears to be no time frame yet studied in which the medicine stops working or toxicities become limiting,” according to lead author Giles W. Robinson, MD, a pediatric neuro-oncologist at St. Jude Children's Research Hospital in Memphis, who was quoted in the ASCO press release.

No responses were observed among patients lacking the alterations targeted by entrectinib.