Olutasidenib and Azacitidine for IDH1-mutated AML
This study is looking at how well a combination of two drugs, olutasidenib and azacitidine, works for people with acute myeloid leukemia (AML) that has a specific change called an IDH1 mutation. You would have already received treatment with venetoclax and a hypomethylating agent (HMA-Ven). The study will first give you both olutasidenib and azacitidine, and then you would continue with olutasidenib alone. Researchers want to see if this treatment can help prevent the cancer from coming back or delay its return. They will measure how long it takes for the treatment to fail, meaning the cancer comes back, gets worse, or treatment has to stop. The study plans to enroll 28 participants, but its current status is unclear.
- Study design
- This is an interventional study, meaning participants will receive a specific treatment. It is not specified if it's a single-arm or randomized study, nor is the phase mentioned. It plans to enroll 28 participants.
- What's involved
- You would take olutasidenib by mouth twice daily and receive azacitidine by IV or under the skin for seven days each cycle. Cycles repeat every 28 days for up to 4 cycles, followed by ongoing olutasidenib. You will also have bone marrow and blood tests.
- Compensation
- Not stated in the trial record.
- Follow-up
- After treatment, you will be followed up within 30 days and then every 4 months until you withdraw consent, are lost to follow-up, the study ends, or you pass away.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT07304011
Olutasidenib With Azacitidine Followed by Olutasidenib Maintenance for the Treatment of IDH1-mutated Acute Myeloid Leukemia in Patients With Prior Treatment With Venetoclax Plus a Hypomethylating Agent
Recruiting
PHASE2Ages 18+InterventionalTreatmentUniversity of California, DavisInvestigator-initiated
~28 participants
Updated 2026-07-02 on ClinicalTrials.gov
What's tested:AzacitidineOlutasidenib
At a glance
Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Treatment failure
Measured over At 12 months from complete response (CR)/complete remission with incomplete count recovery (CRi)
+1 more outcome measured
Conditions
Where it's being run
1 sites across 1 statesCalifornia1
Study leadership
- Brian Jonas, MD · PRINCIPAL_INVESTIGATOR · University of California, Davis
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Pathologically documented AML (except acute promyelocytic leukemia with the t(15;17) translocation) as defined by the World Health Organization (WHO) or International Consensus Classification criteria
Achieved complete response (CR)/complete remission with incomplete count recovery (CRi) response to first line HMA-Ven according to the European Leukemia Net (ELN) recommendations for diagnosis as determined by investigator review
Documented IDH1-R132 mutations (≥ 0.01%) detected in the bone marrow or blood. Mutation must be present at the time of AML diagnosis or after initiating HMA-Ven
Receiving first-line HMA-Ven with less than or equal to 4 cycles of HMA-Ven at the time of enrollment. Participants must discontinue venetoclax (Ven) at least 1 week (or 5 half-lives, whichever is shorter) from initiating study treatment
Candidate for standard of care azacitidine
Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 (Karnofsky ≥ 50%)
No prior solid organ allograft
Recovery from the non-hematologic toxic effects of prior treatment to grade ≤ 1, or baseline value according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) classification (excluding infertility or alopecia)
Aged ≥ 18 at the time of consent
Creatinine clearance ≥ 40 mL/min (calculated by the Cockcroft-Gault formula or measured by 24-hour urine collection)
Serum alanine aminotransferase (ALT) ≤ 3 × upper limit of normal (ULN)
Serum aspartate aminotransferase (AST) ≤ 3 × ULN
Bilirubin ≤ 2 x ULN unless due to Gilbert's syndrome or controlled autoimmune hemolytic anemia (not requiring immunosuppressive other than ≤ 20 mg of prednisolone daily)
Note: Patients with Gilbert's syndrome may be included if total bilirubin is ≤ 3 × ULN and direct bilirubin is ≤ 2 × ULN
Prothrombin time (PT) or international normalized ratio (INR)/activated partial thromboplastin time (aPTT) ≤ 1.5 × ULN
Women of child-bearing potential, men, and their respective partners, must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for 90 days after the last dose of olutasidenib
Must sign and date the informed consent form prior to undergoing any study procedures
Exclusion
Prior IDH1 inhibitor (IDH1i) targeted therapy
Prior AML therapy except for HMA-Ven
History of a different malignancy unless they have been disease-free for at least 12 months and are deemed by the investigator to be at low risk for recurrence of that malignancy. Individuals with a history of other malignancies within 12 months and without any evidence of disease progression or requiring therapy may be considered, but only after consideration and approval by the Overall Principal Investigator (PI). Individuals with the following cancers are eligible if diagnosed and/or treated within the past 12 months: cervical cancer in situ, breast ductal carcinoma in situ (DCIS), and basal cell or squamous cell carcinoma of the skin
Patients with symptomatic central nervous system (CNS) metastases or other tumor location (such as spinal cord compression, other compressive mass, uncontrolled painful lesion, bone fracture, etc.) necessitating an urgent therapeutic intervention, palliative care, surgery or radiation therapy
Patients with previous allogeneic hematopoietic stem cell transplantation (HSCT) for non-AML indications, if they meet any of the following criteria: \< 100 days from time of HSCT; active acute or chronic graft versus (vs.) host disease (GvHD); or receiving immunosuppressive therapy as treatment or prophylaxis against GvHD
Note: Doses \< 20 mg methylprednisolone (or its equivalent) daily are not an exclusion criterion
Treatment with radiation therapy or major surgery (requiring general anesthesia) within 2 weeks prior to study drug dosing
Patients unable to swallow oral medications, or patients with gastrointestinal conditions (e.g., malabsorption, resection, etc.) deemed by the Investigator to jeopardize intestinal absorption
Congestive heart failure (New York Heart Association Class III or IV) or unstable angina pectoris; previous history of myocardial infarction within one year prior to study entry, uncontrolled hypertension, or uncontrolled arrhythmias
Patients with a baseline corrected QT interval by Fridericia's formula (QTcF) of \> 480 msec
Note: This criterion does not apply to patients with a bundle branch block (BBB); for participants with BBB, a cardiology consult is recommended to ensure that QTcF is not prolonged
Concomitant medication(s) known to cause Torsades de Pointes (TdP) initiated less than the duration required to reach steady-state plasma concentration (approximately five half-lives) before first dose of study drug. Medications used as needed (PRN), e.g. Zofran, and common AML supportive care drugs (e.g. levofloxacin, azoles, etc.) are exempt
Concurrent treatment with chronic corticosteroids except if chronic treatment with \< 20 mg of methylprednisolone daily or equivalent (pulse steroids for treatment or prophylaxis are allowed \[e.g., for transfusion or medication reactions\])
Known history of seropositivity for HIV infection
Active, uncontrolled bacterial, viral, or fungal infections, requiring systemic therapy (prophylactic systemic antimicrobials permitted)
Uncontrolled disease-related metabolic disorder (e.g., hypercalcemia)
Pregnant, breastfeeding, or planning to become pregnant while enrolled in this trial or within 90 days after the last dose of olutasidenib. Pregnant or breastfeeding
NOTE: Breast milk cannot be stored for future use while the mother is being treated on study)
Plans to donate sperm or conceive a child through intercourse while enrolled in this trial or within 90 days after the last dose of olutasidenib
Unwillingness or inability to comply with procedures either required in this protocol or considered standard of care
Medical, uncontrolled disease-related metabolic disorder, psychiatric, cognitive, or other conditions that may, in the opinion of the investigator, compromise the patient's ability to understand the patient information, give informed consent, comply with the study protocol, or complete the study
What this trial measures
- Treatment failureAt 12 months from complete response (CR)/complete remission with incomplete count recovery (CRi)
Defined as the percent of patients who reached death due to acute myeloid leukemia (AML), relapse, or discontinuation of treatment due to an adverse event, at 12 months from the time of CR/CRi in patients with AML who begin first line venetoclax plus a hypomethylating agent regimen and subsequently transition to olutasidenib maintenance. The Kaplan-Meier method will be employed to summarize the duration from the initiation of study treatment to treatment failure and to report the probability of being event-free at one year. The 1-year treatment failure rate will be reported along with its 95% confidence interval.
- Median time to treatment failureFrom CR/CRi through death due to AML, relapse, or treatment discontinuation due to adverse event, assessed up to 4 years
Will be reported along with its 95% confidence interval.