CONNECT TarGeT D: Phase 2 Study of Olutasidenib with Temozolomide as Maintenance Therapy in Pediatric and Young Adult Patients Newly Diagnosed with High-Grade Glioma (HGG), including Diffuse Intrinsic Pontine Glioma (DIPG), which Harbor IDH1 Mutations
CONNECT TarGeT D
What is the goal of the study?
This study consists of a safety and feasibility study (Part 1, Figure 1) in which patients 12- 39 years of age with newly-diagnosed IDH1-mutant HGG and DIPG will be treated with the post-RT combination of olutasidenib and temozolomide to identify the dose of olutasidenib that is feasible when given in combination with temozolomide as maintenance therapy after completion of focal radiotherapy. Once the recommended phase 2 dose (RP2D) for the combination of olutasidenib and temozolomide has been established, the phase 2 study will open to enrollment. In the Phase 2 study (Part 2, Figure 2) patients 12-39 years of age with newly-diagnosed IDH1-mutant HGG and DIPG will be treated with the post-RT combination of olutasidenib and temozolomide. Efficacy of this molecularly-targeted therapy will be assessed by progression-free survival (PFS) (HGG [Strata A and B]) and OS (DIPG [Stratum C]) with key longitudinal biomarker correlatives as above. Stratum A will include 33 patients with IDH1-mutant Grade 3 astrocytoma per 2021 World Health Organization (WHO) classification of CNS Tumors. Outcomes among patients with IDH1-mutant WHO Grade 4 astrocytoma, DIPG, primary thalamic and spinal cord, HGG (Strata B and C) will be descriptively analyzed. Strata B and C will enroll up to 15 patients each. In order to characterize the pharmacokinetic (PK) and pharmacodynamic (PD) properties of olutasidenib administered to pediatric patients (i.e., 12 to < 18 years of age), these patients will receive olutasidenib monotherapy for 2 weeks (Pre-maintenance cycle) before proceeding with Cycle 1 of Maintenance therapy with olutasidenib and temozolomide. A minimum of 6 patients aged 12 to < 18 years will be enrolled in the Pre-maintenance cycle.
Who can participate in the study?
Please contact the study team listed below to learn more.