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Olutasidenib and Temozolomide for High-Grade Glioma

This study is testing a combination of two drugs, olutasidenib and temozolomide (TMZ), for children and young adults (ages 12-39) with newly diagnosed high-grade glioma (a type of brain tumor) that has a specific genetic change called an IDH1 mutation. Researchers want to find the best dose of olutasidenib to use with temozolomide and see if this combination can help patients live longer without their cancer getting worse. The study will also look at how safe the drugs are and how they work in the body. You would need to have an IDH1 mutation to be eligible for this study. The current status of the study is unclear, and it plans to enroll 60 participants.

Study design
This is a multicenter, international, phase II study. It will include a feasibility group to find the right dose of olutasidenib when combined with temozolomide.
What's involved
Participants will receive olutasidenib and temozolomide after radiotherapy, with treatment lasting for 13 cycles of combination therapy followed by 13 cycles of olutasidenib alone.
Compensation
Not stated in the trial record.
Follow-up
Progression-free survival will be assessed for up to 24 months from diagnosis, and olutasidenib levels will be measured for up to 24 months from the start of treatment.

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NCT06161974

Study of Olutasidenib and Temozolomide in HGG

Recruiting
PHASE2Ages 12–39InterventionalTreatment
Rigel Pharmaceuticals
~60 participants
Updated 2026-06-12 on ClinicalTrials.gov
What's tested:Olutasidenib + TMZ

At a glance

Recruiting sites
8 of 20 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Establish the RP2D of Olutasidenib and Temozolomide (Feasibility cohort)
Measured over Completion of cycle 1 (28 days) for 6-24 patients
+2 more outcomes measured

Conditions

High Grade Glioma
Astrocytoma
Astrocytoma, Grade III
Astrocytoma, Grade IV
Diffuse Intrinsic Pontine Glioma
WHO Grade III Glioma
WHO Grade IV Glioma
Metastatic Brain Tumor
Diffuse Midline Glioma, H3 K27M-Mutant
Thalamus Tumor
Spinal Tumor
IDH1 Mutation
IDH1 R132
IDH1 R132C
IDH1 R132H
IDH1 R132S
IDH1 R132G
IDH1 R132L
Oligodendroglioma

Explore related trials

AstrocytomaDiffuse Intrinsic Pontine GliomaMalignant GliomaSpinal Cord GliomaThalamic GliomaIDH1 Gene

Where it's being run

20 sites across 19 states
Ohio2
Colorado1
District of Columbia1
Florida1
Illinois1
Massachusetts1
North Carolina1
Pennsylvania1

Study leadership

  • Santosh Valvi, FRACP, MSc · STUDY_CHAIR · Perth Children's Hospital
  • Nicholas G Gottardo, MB FRACP PhD · STUDY_CHAIR · Perth Children's Hospital
  • Michael J Fisher, MD · STUDY_CHAIR · Children's Hospital of Philadelphia
  • Maryam Fouladi, MD · STUDY_CHAIR · Nationwide Children's Hospital

Who to contact

Jill DeFratis, MPH
[email protected]
650-624-1100
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Patients with a newly-diagnosed IDH1-mutant HGG including DIPG are eligible. All patients must have tumor tissue from diagnostic biopsy or resection, without exceptions. The diagnosis of HGG, including DIPG, must have been confirmed through TarGeT-SCR.
For the diagnosis of DIPG, patients must have a tumor with pontine epicenter and diffuse involvement of at least 2/3 of the pons, and histopathology consistent with diffuse WHO Grade 2-4 glioma.
All other HGG must be WHO Grade 3 or 4.
Measurable disease is not required. Patients without measurable disease are eligible.
Primary spinal tumor: Patients with a primary spinal HGG are eligible.
Patient must not have metastatic disease.
R132H, R132C, R132S, R132G or R132L.
Patients whose tumors harbor other alterations in addition to IDH1 mutation will potentially be eligible following consensus recommendation by the international multidisciplinary molecular screening committee.
Patients with IDH2 mutations are not eligible.
Patients with oligodendroglioma, IDH-mutant and 1p/19q-codeleted are not eligible.
Patients in pre-maintenance phase must enroll and start treatment no later than 21 calendar days post-completion of RT.
Patients not in pre-maintenance phase must enroll and start treatment no later than 35 calendar days post-completion of RT.
Peripheral absolute neutrophil count (ANC) ≥ 1000/mm3.
Platelet count ≥ 100,000/mm3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment).
Hemoglobin \> 8 g/dL (may be transfused). 2.5.2 Adequate Renal Function Defined as
Creatinine clearance or radioisotope GFR ≥ 70ml/min/1.73 m2 OR
Maximum serum creatinine based on age/gender as follows: 10 to \< 13 yrs=1.2 mg/dL for males and females. 13 to \< 16 yrs=1.5 mg/dL for males and 1.4 mg/dL for females.
Total bilirubin must be ≤ 1.5 × institutional ULN.
AST(SGOT)/ALT(SGPT) \< 3 × institutional ULN.
Alkaline Phosphatase \< 3 × institutional ULN. 2.5.4 Adequate Neurologic Function Defined as:
Patients with seizure disorder may be enrolled if well-controlled on anticonvulsants that are not a strong inducer or inhibitor of CYP3A4/5.
Patients must be able to swallow oral medications to be eligible for study enrollment.

Exclusion

Combined estrogen and progesterone containing hormonal contraception associated with inhibition of ovulation.
Progesterone-only hormonal contraception associated with inhibition of ovulation.
Intra Uterine Device (IUD).
Intra uterine hormone releasing system.
Bilateral tubal occlusion.
Vasectomized partner.
Sexual abstinence (avoiding heterosexual intercourse).
The following contraceptive measures are NOT considered effective:
Progesterone-only hormonal contraception (birth control pill) that that does NOT stop ovulation.
Male or female condom with or without spermicide.
Cap, diaphragm, or sponge with spermicide. 2. Using the following types of concomitant medications:
Corticosteroids: Patients receiving corticosteroids are eligible. The use of corticosteroids must be reported.
Investigational Drugs: Patients who are currently receiving another investigational drug are not eligible.
Anti-cancer Agents: Concurrent anti-cancer agents are not allowed with the exception of temozolomide given concurrently with RT and as protocol-instructed post RT maintenance therapy after enrollment on TarGeT-D.
Anticonvulsants: Patients who are receiving enzyme inducing anticonvulsants that are strong inducers of CYP3A4/5 are not eligible.
Strong CYP3A4/5 inducers: Patients who are receiving strong inducers of CYP3A4/5 are not eligible. Strong inducers of CYP3A4/5 should be avoided from 14 days prior to or 5 half-lives (whichever is longer) enrollment to the end of the study.
Patients who are receiving medications known to prolong QTc interval are not eligible
Selective serotonin reuptake inhibitors (SSRIs) such as citalopram (Celexa), escitalopram (Lexapro), fluoxetine (Prozac), fluvoxamine (Luvox), paroxetine (Paxil), sertraline (Zoloft) should be used with caution but are not contraindicated. 3. Other Criteria
Infection: Patients who have an uncontrolled infection are not eligible.
Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study are not eligible.
Patients with known clinically significant active malabsorption syndrome or other condition that could affect absorption are not eligible.
Patients with malignancy related to HIV or solid organ transplant: known history of HIV, HBV surface antigen positivity or positive HCV antibody are not eligible. Viral testing is not required unless clinically indicated in patients without a known history.
Patients with prior or ongoing clinically significant illness, medical or psychiatric condition, that, in the investigator's opinion, could affect the safety of the participant, or could impair the assessment of study results are not eligible.
Patients with any prior solid organ transplant are not eligible.
Patients with secondary/radiation-related HGG are not eligible.
Patients with metastatic/disseminated HGG who have received CSI are not eligible.

What this trial measures

  • 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 OlutasidenibFrom 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).