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Acute Myeloid LeukemiaPhase 2OLUVENAZANCT06782542

Olutasidenib, Venetoclax, and Azacitidine in IDH1 Mutated Newly Diagnosed Acute Myeloid Leukemia Patients Eligible for Intensive Induction Chemotherapy

A Phase 2 study testing Olutasidenib, Venetoclax and Azacitidine in people with Acute Myeloid Leukemia and IDH1 Mutation, run by Justin Watts, MD at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

The purpose of this study is as follows: 1. Determine whether people receiving the combination treatment of olutasidenib, venetoclax, and azacitidine have the same, more, or fewer side effects compared to the usual chemotherapy treatment that people with this condition receive. 2. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Acute Myeloid Leukemia, IDH1 Mutation
What is being tested?
Olutasidenib, Venetoclax and Azacitidine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
1 siteUnited States
Study dates
31 Mar 2026to 31 Mar 2029
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Participant is an adult male or female participant aged 18-75 years considered eligible to undergo intensive induction chemotherapy at the time of signing the informed consent form (ICF).
  • Eastern Cooperative Oncology Group (ECOG) performance status ≤2
  • Confirmed diagnosis of:
  • Newly diagnosed AML Isocitrate dehydrogenase 1 (IDH1) R132 mutated disease as assessed locally. Note: historical results from within 30 days of informed consent will be accepted if the participant did not receive systemic treatment after collection.
  • Secondary AML, including prior hypomethylating agents (HMA) exposure for myelodysplastic syndrome (MDS), myeloproliferative neoplasms (MPN), or MDS/MPN is allowed.
  • Participant must have adequate organ function, defined by the following:
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  • Aspartate transaminase (AST) and alanine aminotransferase (ALT) values ≤3 × upper limit of normal (ULN) or ≤5 × ULN for participants with leukemic involvement.
  • Bilirubin ≤2 ULN (≤3 × ULN in participants with Gilbert Syndrome) or ≤3 × ULN for participants with leukemic involvement.
  • Creatinine clearance ≥30 mL/min (using Cockcroft-Gault), or serum creatinine ≤1.5 × ULN.
  • The interval from prior treatment for an antecedent hematologic disorder to the first dose of study treatment (C1D1) will be at least 7 days for cytotoxic or non-cytotoxic (immunotherapy) agents. In addition, the following will be allowed:
  • Intrathecal chemotherapy for prophylactic use or for controlled central nervous system (CNS) leukemia.
  • Use of hydroxyurea for participants with rapidly proliferative disease is allowed before the start of study therapy and for the first 4 weeks on study treatment.
  • Recovery from 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) version 5.0 classification (excluding infertility, alopecia, or Grade 1 neuropathy).
  • Baseline QT interval corrected using the Fridericia equation (QTcF) ≤ 480 msec. Note: This criterion does not apply to participants with a bundle branch block (BBB); for participants with BBB, a cardiology consult is recommended to ensure that QTcF is not prolonged.
  • Female participants who are women of childbearing potential (WOCBP) must have a negative serum or urine (beta-human chorionic gonadotropin (βhCG)) pregnancy test at screening and negative serum or urine test documented within the 24-hour period prior to the first dose of study drug. WOCBP are defined as sexually mature women without prior hysterectomy or who have had any evidence of menses in the past 12 months. However, women who have been amenorrheic for 12 or more months are still considered to be of childbearing potential if the amenorrhea is possibly due to prior chemotherapy, anti-estrogens, or ovarian suppression (Section 4.8.1).
  • Sexually active female participants who are WOCBP and male participants who are sexually active partners of WOCBP must agree to use a highly effective method of contraception during the course of the study from the date of informed consent and for at least 3 months after their last dose of study drug. Effective birth control methods include the following:
  • Intrauterine device (IUD) plus one barrier method.
  • Stable doses of hormonal contraception for at least 3 months (eg, oral, injectable, implant, transdermal).
  • 2 barrier methods; effective barrier methods are male or female condoms, diaphragms, and spermicides (creams or gels that contain a chemical to kill sperm).
  • A vasectomized partner (where vasectomy was done at least 4 months prior to first dose of study treatment).
  • True abstinence (ie, abstinence that is in line with the preferred and usual lifestyle of the participant).
  • Female and male participants must agree to refrain from egg/ova retrieval either for their own use or donation or from sperm donation, respectively, from the date of informed consent until 3 months after the last dose of study treatment.
  • Participant is willing and able to participate and comply with all study requirements and to provide signed and dated written informed consent prior to initiation of any study procedures.

Who cannot join

  • Relapsed/Refractory AML.
  • ELN (2022) favorable risk AML, except for nucleophosmin 1 (NPM1) mutated AML, which is allowed.
  • Acute promyelocytic leukemia (APL).
  • Positive Fms related receptor tyrosine kinase 3-Internal tandem duplication (FLT3-ITD) mutation.
  • Active CNS involvement by leukemia (other extramedullary disease is allowed).
  • Participants <18 years or >75 years of age.
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  • Female participant who is pregnant or breastfeeding.
  • Participant plans to become pregnant or father a child (including ova or sperm donation) while enrolled in this study or within 3 months after last dose of study treatment (Section 4.8).
  • Participant has a known allergy or history of hypersensitivity to study drugs or their excipients.
  • Previous therapy with olutasidenib (or ivosidenib or other IDH1 inhibitor) or venetoclax (or another B cell lymphoma 2 (BCL-2) inhibitor).
  • Participant has active evidence of clinically significant unstable medical condition such as uncontrolled infection, severe metabolic abnormality, poorly controlled psychiatric illness, or symptomatic coronary artery disease (other than stable angina), which could place the participant at unacceptable risk of study treatment, per the Investigator's judgement.
  • Participants receiving treatment with strong Cytochrome P450, family 3, subfamily A (CYP3A) inhibitors within 7 to 14 days or 5 half-lives (whichever is longer) prior to the first dose of study medication. Azoles are allowed with appropriate venetoclax dose reductions. Please note that participants receiving these medications would qualify for this study after undergoing a washout period of 7 to 14 days or 5 half-lives, whatever is longer for the inhibitor/inducer.
  • Participants receiving treatment with strong CYP3A inducers within 7 to 14 days or 5 half-lives (whichever is longer) prior to the first dose of study medication. Please note that participants receiving these medications would qualify for this study after undergoing a washout period of 7 to 14 days or 5 half-lives, whatever is longer for the inhibitor/inducer.
  • History of allogeneic hematopoietic stem cell transplant (HSCT) for a diagnosis other than AML if there is clinically significant active graft-versus-host disease (GVHD) or ongoing immunosuppressive therapy is required beyond prednisone 10 mg daily or equivalent. Otherwise, prior allogeneic HSCT is allowed.
  • Participants with a concurrent active malignancy under treatment.
  • Known history of active hepatitis B (HBV) or hepatitis C (HCV) infection or human immunodeficiency virus (HIV) infection (clinically detectable viral load).
  • Major surgery within 28 days prior to the first dose. Participants must have recovered from surgery and be without current complications.
  • Participants with impaired decision-making capacity.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Justin Watts, MD contact

(305) 2438986

jxw401@miami.edu

United States1 site, 1 recruiting

  • University of Miami

    Miami, Florida, 33136

    Justin Watts, MD, 305-243-8986, jxw401@miami.edu

    Justin Watts, MD

    Recruiting

Other trials in United States

About this study

Official title: Single-Arm Phase 2 Study of Olutasidenib, Venetoclax, and Azacitidine in IDH1 Mutated Newly Diagnosed Acute Myeloid Leukemia Patients Who Are Eligible for Intensive Induction Chemotherapy

The purpose of this study is as follows:

  1. Determine whether people receiving the combination treatment of olutasidenib, venetoclax, and azacitidine have the same, more, or fewer side effects compared to the usual chemotherapy treatment that people with this condition receive.
  2. Determine how well the combination treatment of olutasidenib, venetoclax, and azacitidine works compared to the usual chemotherapy treatment that people with this condition receive.
Being tested
Olutasidenib, Venetoclax, Azacitidine
Medical subject headings
Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • OlutasidenibDrug

    Olutasidenib will be supplied as 150 mg capsules to be administered orally, twice per day (BID) on an empty stomach (fasting at least 1 hour before or 2 hours after a meal), starting on Cycle 1 Day 1, and will be given continuously.

  • VenetoclaxDrug

    Participants 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

  • AzacitidineDrug

    Participants will receive Azacitidine 75 mg/m2 per day via subcutaneous (SC) injection or intravenous (IV) infusion on Days 1-7 of each cycle.

Study groups in NCT06782542
Study groupRoleWhat they receive
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
Receives: Drug: Olutasidenib; Drug: Venetoclax; Drug: Azacitidine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants Experiencing Excessive Toxicity

    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.

    Measured: Up to 12 months

  2. Composite Complete Remission (CRc)

    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.

    Measured: Up to 3 months

Secondary outcomes (20)

  1. Number of Participants Experiencing Unacceptable Toxicity

    The number of participants experiencing unacceptable toxicity in entire cohort will be reported over the duration of study treatment. All toxicity will be assessed using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.

    Measured: Up to 13 months

  2. Number of Participants Experiencing Treatment-Emergent Adverse Events

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

    Measured: Up to 13 months

  3. Number of Participants Experiencing Serious Adverse Events

    The number of participants experiencing serious adverse events (SAEs) will be reported over the duration of study treatment. All toxicity will be assessed using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.

    Measured: Up to 13 months

  4. Number of Participants Experiencing Adverse events of special interest (AESIs)

    The number of participants experiencing adverse events of special interest (AESIs) will be reported over the duration of study treatment. All toxicity will be assessed using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.

    Measured: Up to 13 months

  5. Number of participants who died within 30-days

    The number of participants that die within 30-days after starting study therapy will be reported.

    Measured: Up to 30 days

  6. Number of participants who died within 60-days

    The number of participants that die within 60-days after starting study therapy will be reported.

    Measured: Up to 60 days

  7. Duration of Composite Complete Remission (CRc)

    Duration of CR over the duration of study treatment will be reported. Duration of CR is defined as time in months from CRc is first achieved until relapse, death, or off-study for other acute myeloid leukemia (AML) therapy.

    Measured: Up to 12 months

  8. Time to Composite Complete Remission (CRc)

    Time to composite complete remission (CRc) among participants will be reported in months. Time to CRc is defined is the period from Cycle 1 Day 1 (C1D1) to the date CRc is first achieved.

    Measured: Up to 12 months

  9. Percentage of Participants Receiving Allogeneic HCST

    The percentage of participants receiving allogenic stem cell transplant (HCST), over the duration of study treatment will be reported.

    Measured: Up to 12 months

  10. Percentage of Participants Achieving Complete Response (CR)

    The percentage of study participants achieving complete response (CR) to study treatment will be reported. Response will be assessed using on modified 2022 European LeukemiaNet (ELN) criteria for AML.

    Measured: Up to 12 months

  11. Overall Response Rate

    Overall response rate (ORR) (reported as a percentage), which includes complete remission (CR), complete remission with partial hematologic recovery (CRh), complete remission with incomplete hematologic recovery (CRi), morphologic leukemia free state (MLFS), and partial response (PR) over the duration of study treatment, as defined by modified 2022 European LeukemiaNet (ELN) criteria.

    Measured: Up to 12 months

  12. Duration of response (DOR)

    Duration of response (DOR) over the duration of study treatment will be reported. The DOR is defined as the number of days from the date of initial response to the date of first documented disease progression/relapse or death, whichever occurs first.

    Measured: Up to 12 months

  13. Time to Response (TTR)

    Time to response (TTR) over the duration of study treatment will be reported in months. TTR is defined as the elapsed time from Cycle 1 Day 1 until complete remission (CR), complete remission with partial hematologic recovery (CRh), complete remission with incomplete hematologic recovery (CRi), morphologic leukemia free state (MLFS), or partial response (PR) is first achieved.

    Measured: Up to 12 months

  14. Event-free survival (EFS)

    Event-free survival (EFS) among study participants over the duration of study treatment will be reported. EFS is defined as time in months from the date of treatment initiation (i.e., C1D1) to the date of documented treatment failure, relapse, or death from any cause, whichever occurs first, and will be calculated for all participants.

    Measured: Up to 12 months

  15. Overall survival (OS)

    Overall survival (OS) is defined as the elapsed time in months from the start of treatment until death from any cause. Alive participants will be censored at the last date known to be alive.

    Measured: Up to 36 months

  16. Minimal residual disease (MRD) negative rates

    Minimal residual disease (MRD) negative rates as assessed by multi-parameter flow cytometry (MPFC) and/or next generation sequencing (NGS)/polymerase chain reaction (PCR) based technology performed locally, will be reported as a percentage over the duration of study treatment.

    Measured: Up to 12 months

  17. Pharmacokinetics (PK): Cmax

    Maximum plasma concentration (Cmax) among study participants will be reported via the collection and analysis of blood samples. The peak concentration(Cmax) is the highest level of plasma concentration that occurs after drug administration.

    Measured: Up to 9 months

  18. Pharmacokinetics: Tmax

    Time to maximum plasma concentration (Tmax) among study participants will be reported via the collection and analysis of blood samples

    Measured: Up to 9 months

  19. Pharmacokinetics: Cmin

    Minimum plasma concentration (Cmin) among study participants will be reported via the collection and analysis of blood samples.

    Measured: Up to 9 months

  20. Pharmacokinetics: AUC

    Area under the plasma concentration-time curve (AUC)(0-24)

    Measured: Up to 9 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Jan 2025
  2. Study start31 Mar 2026
  3. Primary completion31 Mar 2029 (estimated)
  4. Study completion31 Mar 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
16 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06782542
Sponsor's protocol ID
20240763
Acronym
OLUVENAZA
Status last verified
Apr 2026
First submitted
15 Jan 2025
Last updated
16 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Olutasidenib, Venetoclax and Azacitidine in people with Acute Myeloid Leukemia and IDH1 Mutation, run by Justin Watts, MD at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 23 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 16 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Justin Watts, MD ((305) 2438986, jxw401@miami.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants Experiencing Excessive Toxicity (time frame: Up to 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06782542.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Apr 2026.

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