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Combination ivosidenib with azacitidine and venetoclax followed by ivosidenib maintenance for IDH1‑mutated AML

A Phase II Study of IDH1 Inhibition With Ivosidenib as Maintenance Therapy After Ivosidenib, Azacitidine, and Venetoclax for Acute Myeloid Leukemia

Phase 2 Interventional Memorial Sloan Kettering Cancer Center · NCT07392242

This trial will test whether giving ivosidenib with azacitidine and venetoclax, then continuing ivosidenib alone, helps people aged 60 and older with newly diagnosed IDH1‑mutated acute myeloid leukemia.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment45 (estimated)
Ages60 Years and up
SexAll
SponsorMemorial Sloan Kettering Cancer Center Academic / other
Locations7 sites (Basking Ridge, New Jersey and 6 other locations)
Trial IDNCT07392242 on ClinicalTrials.gov

What this trial studies

This Phase 2 interventional trial gives a three‑drug induction regimen of ivosidenib, azacitidine, and venetoclax to older adults with newly diagnosed AML that carries an IDH1 mutation, followed by maintenance therapy with ivosidenib alone. Eligibility requires confirmation of an IDH1‑R132 (or related 2‑HG producing) mutation by a CLIA‑certified test and the ability to attend scheduled visits. The study will track response rates, duration of remission, safety and tolerability, and the practical time commitment for participants. Treatment is delivered at Memorial Sloan Kettering locations in New Jersey with protocol oversight by the MSK principal investigator.

Who should consider this trial

Good fit: Ideal candidates are people aged 60 or older with newly diagnosed AML confirmed to have an IDH1‑R132 (or qualifying) mutation who can comply with study visits and procedures.

Not a fit: Patients without an IDH1 mutation, those under 60 per the protocol, or people who cannot tolerate these drugs or frequent clinic visits are unlikely to benefit from this specific regimen.

Why it matters

Potential benefit: If successful, this approach could produce deeper or longer remissions for older patients with IDH1‑mutated AML and use oral maintenance to help keep the disease from returning.

How similar studies have performed: Prior studies have shown benefit from azacitidine plus venetoclax in older AML and from ivosidenib in IDH1‑mutant disease, but combining all three agents followed by ivosidenib maintenance is a newer approach still being tested.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Participant must be at least 60 years of age at the time of signing the informed consent form (ICF).
* Participant is willing and able to adhere to the study visit schedule and other protocol requirements.
* Participant has newly diagnosed AML as per World Health Organization 2022 or European leukemia Network 2022 guidelines.
* Participant has IDH1-R132 mutation present prior to initiating Ivo/Aza/Ven confirmed by CLIA approved local testing via next-generation sequencing (NGS) and/or polymerase chain reaction (PCR). Other 2-HG producing IDH1 variants may be eligible after discussion with MSK principal investigator.

  1. At MSK, this testing will utilize the MSK-REACT, a rapid multi-gene NGS panel used in all new AML diagnoses that is clinically validated by the Laboratory of Diagnostic Molecular Pathology pursuant to the requirements of CLIA'88 and approved by New York State. Other sites may use local CLIA-certified laboratories and validated clinical assays as per standard of care.
  2. The patient's chart will be utilized for screening purposes
* Participant has Eastern Cooperative Oncology Group (ECOG) performance status of 0-3
* Participant must have a WBC count \<25,000/μL at the time of initiation of study drug (leukapheresis may be performed and/or hydroxyurea may be administered to decrease the WBC count to \<25,000/μL).
* Participant has adequate organ function defined as:

  1. Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) and alanine aminotransferase (ALT/SGPT) ≤ 3 x ULN, unless considered due to leukemic organ involvement.
  2. Serum total bilirubin \< 3.0 x ULN. Higher levels are acceptable if these can be attributed to ineffective erythropoiesis, leukemia organ involvement or Gilbert's syndrome.
  3. Serum creatinine \< 2 x ULN or creatinine clearance 30 mL/min based on the Cockroft-Gault glomerular filtration rate (GFR) estimation.

Exclusion Criteria:

* Participant with acute promyelocytic leukemia
* Participants who have previously received ivosidenib or venetoclax
* Participant receiving any other investigational anti-cancer agents. Cytoreductive therapy such as hydroxyurea is permitted.
* Participants with immediate life-threatening, severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation
* Participant has active uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment).
* Participants who are planned for allogeneic stem cell transplantation based on the assessment of the treating clinician.
* Participant has significant active cardiac disease within 6 months prior to start of study treatment, including New York Heart Association (NYHA) class III or IV congestive heart failure; acute coronary syndrome (ACS); and/or stroke
* Participant has active viral infection with human immunodeficiency virus (HIV), or active infection with hepatitis B virus (HBV) or hepatitis C virus (HCV). As per exclusion criteria 5, patients receiving appropriate treatment would only be excluded if there is no improvement.
* Participant is known to have dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally.
* Participant has QTc interval (i.e., Fridericia's correction \[QTcF\]) ≥ 450 ms (mean of triplicate ECG) or other factors that increase the risk of QT prolongation or ventricular arrhythmic events (e.g. family history of long QT interval syndrome). Participants with a QTcF over 450 ms due
* Male or female participants not willing to comply with contraceptive requirements

Where this trial is running

Basking Ridge, New Jersey and 6 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Acute Myeloid LeukemiaIvosidenibAzacitidineVenetoclax25-224
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.