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Olutasidenib

Phase 2

IDH1 Mutation | Small molecule | Oncology |Rigel Pharmaceuticals, Inc.|Last Updated: Apr 16, 2026

Success Probability
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Market & Valuation
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment25
FDA Designations
No designations recorded
Clinical trial landscape

Olutasidenib · 2 trials · 3 indications

Phase 2 2
NCT07471841Olutasidenib in Relapsed IDH1 Mutated AML Patients Who Have Previously Received VenetoclaxIDH1 Mutation
NOT YET_RECRUITING25 Analytics
NCT06782542Olutasidenib, Venetoclax, and Azacitidine in IDH1 Mutated Newly Diagnosed Acute Myeloid Leukemia Patients Eligible for Intensive Induction ChemotherapyAcute Myeloid Leukemia
RECRUITING16 Analytics
PHASE2NOT YET_RECRUITING
Olutasidenib in Relapsed IDH1 Mutated AML Patients Who Have Previously Received Venetoclax
IDH1 MutationUnlock trial analytics
PHASE2RECRUITING
Olutasidenib, Venetoclax, and Azacitidine in IDH1 Mutated Newly Diagnosed Acute Myeloid Leukemia Patients Eligible for Intensive Induction Chemotherapy
Acute Myeloid LeukemiaUnlock trial analytics
Study Endpoints
Primary Endpoints
Composite complete remission rate
2 years

Composite complete remission (CRc) rate is defined as patients that meet the criteria for CR + CRh + CRi per the modified European LeukemiaNet (mELN) 2022. * Complete Remission (CR) is defined as absence of leukemia cells in the bone marrow (\< 5% blasts), normal blood counts (absolute neutrophil count ≥ 1000/μL and platelet count ≥ 100,000/μL), and the absence of circulating blasts, and extramedullary disease * Complete Remission with Hematologic recovery (CRh) is defined as meeting all criteria for CR as Bone marrow myeloblasts \< 5%; absence of circulating blasts; absence of extramedullary disease; both ANC ≥ 0.5x109/L (500/µL) and platelet count ≥ 50×109/L (50 000/µL) * Complete Remission with Incomplete hematologic recovery (CRi) is defined as meeting all criteria for Complete Remission (CR) except for either a low neutrophil count (neutropenia) or a low platelet count (thrombocytopenia)

Number of Participants Experiencing Excessive Toxicity
Up to 12 months

The number of participants experiencing excessive toxicity in six (6) patient safety lead-in over the duration of study treatment will be reported. All treatment-emergent adverse events (TEAEs) will be graded using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.

Composite Complete Remission (CRc)
Up to 3 months

Composite complete remission (CRc) among participants will be reported as a percentage. CRc is includes number of participants experiencing complete remission \[CR\], complete remission with partial hematologic recovery \[CRh\] or complete remission with incomplete hematologic recovery \[CRi\] after up to 3 cycles as defined by modified 2022 European LeukemiaNet (ELN) criteria.

Secondary Endpoints
Overall response rate (ORR)
2 years
Overall survival
2 years
Duration of response (DoR)
2 years
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Olutasidenib and AzacitidineEXPERIMENTALEach cycle will last for 28 days. Patients will receive olutasidenib 150 mg orally twice daily Day 1 through Day 28. After 3 cycles of olutasidenib, azacitidine 75 mg/m2 given on Day 1 through Day 7 may be added at the discretion of the treating investigator if the patient has not achieved a complete remission or for whom loss of response is suspected. Subjects with at least a PR after 6 cycles of treatment will continue treatment.. Subjects without at least a PR after 6 cycles of treatment will move to long term follow up.
OLUVENAZA Treatment GroupEXPERIMENTALParticipants in this group will receive combination treatment of Olutasidenib, Venetoclax and Azacitidine orally for up to 12 cycles, each cycle lasting 28 days. Total participation duration is about 14 months
Interventions
NameTypeDescription
OlutasidenibDRUGOlutasidenib 150 mg orally twice a day
Azacitidine (AZA)DRUGAzacitidine 75 mg/m2 subcutaneously or IV (over 10-40 minutes)
VenetoclaxDRUGParticipants will receive Venetoclax as a 100mg tablet to be self-administered orally with a meal and water once daily, two hours after starting Olutasidenib administration, starting on Cycle 1 Day 1. The dosing regimen of Venetoclax is as follows: * Cycle 1: Days 1 - 21 over a 28-day cycle * Cycle 1 Week 1: Ramp-up dosing schedule up to 400mg (4 x 100mg/tablet) * For participants with blast clearance: Cycle 2 and beyond: Days 1 - 14 over a 28-day cycle * For participants with persistent clearance: Cycle 2 through 4: Days 1 - 21
AzacitidineDRUGParticipants will receive Azacitidine 75 mg/m2 per day via subcutaneous (SC) injection or intravenous (IV) infusion on Days 1-7 of each cycle.
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Eligibility Criteria
Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. Written informed consent and HIPAA authorization for release of personal health information prior to registration. NOTE: HIPAA authorization may be included in the informed consent or obtained separately. 2. Age ≥ 18 years at the time of consent. 3. ECOG Performance Status of...

Countries:United States
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Competitive Landscape -Genetic Mutations 6 trials (matched to "IDH1 Mutation")
Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT06782542studyFirstPostDate: changed
LOWMay 24, 2026NCT07471841studyFirstPostDate: changed