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Olutasidenib + TMZ

Phase 2

High Grade Glioma | Small molecule | Oncology |Rigel Pharmaceuticals, Inc.|Last Updated: Feb 21, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment60
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06161974Study of Olutasidenib and Temozolomide in HGGPHASE2 RECRUITING 60 — — Mar 1, 2025Jun 1, 2035Feb 21, 202518 United States, Australia +4
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Study Endpoints
Primary Endpoints
Establish the RP2D of Olutasidenib and Temozolomide (Feasibility cohort)
Completion of cycle 1 (28 days) for 6-24 patients

To identify the dose of olutasidenib that is feasible when given post-RT in combination with temozolomide as maintenance therapy in pediatric and young adult patients newly diagnosed with IDH1-mutant high-grade glioma

Assess Progression-Free Survival (PFS) in Grade 3 IDH1-mutant Astrocytoma (Stratum A)
From date of diagnosis until date of Progressive Disease or death due to any cause or date of last follow-up, assessed up 24 months

To assess the post-RT efficacy of olutasidenib in newly diagnosed patients with WHO Grade 3 IDH1-mutant Astrocytoma treated with maintenance olutasidenib and temozolomide for 13 cycles followed by 13 cycles of single agent olutasidenib compared to molecularly-stratified and matched historical controls

Maximum plasma concentration [Cmax] of Olutasidenib
From Day 1 of treatment until date of first documented progression or date of death from any cause, whichever comes first, assessed up to 24 months

To characterize the plasma pharmacokinetic (PK) properties of olutasidenib in pediatric patients (e.g., 12 to \< 18 years of age), administered in combination with temozolomide (first year) and as single agent (second year) as maintenance chemotherapy by measuring the Maximum Concentration \[Cmax\] and Area Under the Curve (AUC) of olutasidenib in plasma (All strata).

Secondary Endpoints
Evaluate objective response rate (ORR) in HGG (All Strata)
From day 1 of protocol treatment through 30 days following end of protocol treatment
Evaluate Health-Related Quality of Life Outcomes (All Strata)
From pre-maintenance (2 weeks before the first cycle), and at the start of even numbered cycles (each cycle is 28 days) and at the End of Treatment visit (can have up to 26 cycles)
Evaluate Overall Survival in IDH1-mutant Grade 3 Astrocytoma (Stratum A)
From date of diagnosis until date of death due to any cause or date of last follow-up, assessed up to 60 months
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Stratum AEXPERIMENTALPatients with localized, intracranial, non-pontine, and non-thalamic IDH 1 mutant Astrocytoma, CNS WHO Grade 3.
Stratum BEXPERIMENTALPatients with localized, intracranial, non-pontine, and non-thalamic IDH 1 mutant Astrocytoma, CNS WHO Grade 4.
Stratum CEXPERIMENTALPatients with IDH-1 mutant DIPG, primary thalamic and spinal cord IDH-1 mutant HGG.
Interventions
NameTypeDescription
Olutasidenib + TMZDRUGOlutasidenib 150 mg PO BID + Temozolomide 200 mg/m2 PO QD
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Eligibility Criteria
Age Range12 Years — 39 Years
SexALL
Healthy VolunteersNo
Study Sites18

Criteria TarGeT-D study strata definitions * Stratum A: Patients with localized, intracranial, non-pontine, and non-thalamic IDH 1 mutant Astrocytoma, CNS WHO Grade 3. * Stratum B: Patients with localized, intracranial, non-pontine, and non-thalamic IDH 1 mutant Astrocytoma, CNS WHO Grade 4. * Stra...

Countries:United StatesAustraliaCanadaGermanyNetherlandsUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06161974primaryCompletionDate: changed
LOWMay 24, 2026NCT06161974studyFirstPostDate: changed