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Olutasidenib+ TMZ

Phase 2

High Grade Glioma | Small molecule | Oncology |Rigel Pharmaceuticals, Inc.|Last Updated: Sep 1, 2026

Target and mechanism

Molecular targetIDH1
Target classInhibitor
ModalitySmall molecule

Also known as Olutasidenib

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment60

FDA Designations

No designations recorded

Clinical trial landscape

Olutasidenib+ TMZ · 1 trial · 19 indications

Phase 2 1
NCT06161974Study of Olutasidenib and Temozolomide in HGGHigh Grade Glioma
RECRUITING60 Analytics
PHASE2RECRUITING
Study of Olutasidenib and Temozolomide in HGG
High Grade GliomaUnlock trial analytics

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 to 39 Years
SexALL
Healthy VolunteersNo
Study Sites20

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 StatesAustraliaCanadaGermanyNetherlandsNew ZealandUnited Kingdom
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Recent Changes (Last 90 Days)

LOWSep 1, 2026NCT06161974lastUpdatePostDate: changed
LOWSep 1, 2026NCT06161974lastUpdatePostDate: changed
LOWJun 12, 2026NCT06161974startDate: changed
LOWJun 12, 2026NCT06161974startDate: changed

Frequently asked questions about Olutasidenib+ TMZ

What is Olutasidenib used for?

Olutasidenib is an investigational small molecule being studied for Acute Myeloid Leukemia with IDH1 mutation and High Grade Glioma. It is in Phase 2 clinical development for these oncology indications.

What does Olutasidenib target?

Olutasidenib is a kinase inhibitor, belonging to the -nib class of drugs. It is being studied in biomarker-selected patients with IDH1 mutations, including subtypes such as IDH1 R132C, R132H, R132S, R132G, and R132L.

Who makes Olutasidenib?

Olutasidenib is being developed by Rigel Pharmaceuticals, Inc., which trades under the ticker RIGL. The company is conducting multiple Phase 2 trials for the drug in IDH1-mutated cancers.

What phase is Olutasidenib in?

Olutasidenib is in Phase 2 clinical development. It is not FDA approved and remains investigational. There are three active or planned Phase 2 trials studying the drug in Acute Myeloid Leukemia and High Grade Glioma.

What clinical trials is Olutasidenib in?

Olutasidenib is being studied in three Phase 2 trials. NCT06161974 evaluates olutasidenib with temozolomide in High Grade Glioma. NCT06782542 studies olutasidenib with venetoclax and azacitidine in newly diagnosed AML. NCT07471841 examines olutasidenib in relapsed IDH1-mutated AML after venetoclax.

Is Olutasidenib the same as Olutasidenib plus TMZ?

Olutasidenib is the same drug as Olutasidenib plus TMZ, where TMZ refers to temozolomide. The combination name is used in the context of the High Grade Glioma trial NCT06161974, which tests olutasidenib together with temozolomide.