Trial of Idasanutlin and Selinexor Therapy for Children With Progressive/Relapsed AT/RT or Extra-CNS Malignant Rhabdoid Tumors

Summary

iSTAR is an open-label, multi-center, phase 1b study of oral XPO1 inhibitor selinexor and oral MDM2 inhibitor idasanutlin in children with progressive or recurrent atypical teratoid/rhabdoid tumors (AT/RT), malignant rhabdoid tumors (MRT) and synchronous/metachronous rhabdoid tumors. Primary Objectives * To determine the maximum tolerated dose (MTD) and the recommended phase 2 dose (RP2D) of combination treatment with oral idasanutlin and selinexor in children with recurrent or progressive AT/RT or MRT. * To characterize the plasma pharmacokinetics of oral idasanutlin and selinexor in children with recurrent or progressive AT/RT or MRT, to assess potential covariates to explain the inter- and intra-individual pharmacokinetic variability. Secondary Objectives * Evaluate safety of the combination treatment with oral idasanutlin and selinexor in children * Evaluate efficacy of the combination treatment of idasanutlin and selinexor as measured by objective response (partial response \[PR\] or complete response \[CR\]) rate separately in progressive/relapsed AT/RT and progressive/relapsed MRT * Estimate progression-free and overall-survival separately in progressive/relapsed AT/RT and progressive/relapsed MRT

Timeline

Start

2024-03

Primary completion

2025-09

Completion

2032-08

Drugs

Indications