Ivosidenib and Venetoclax for IDH1-Mutated Blood Cancers

This study is looking at how safe and effective a combination of medicines is for people with certain blood cancers (hematologic malignancies) that have a specific genetic change called an IDH1 mutation. The medicines being tested are ivosidenib and venetoclax, given with or without another medicine called azacitidine. Ivosidenib and venetoclax may work by blocking enzymes that cancer cells need to grow. Azacitidine is a chemotherapy drug that helps stop cancer cell growth. The goal is to see if adding azacitidine makes the treatment work better. To join, you must be over 18, have an IDH1 mutation, and have certain types of relapsed/refractory acute myeloid leukemia (AML) or newly diagnosed AML if you can't have standard chemotherapy. The study will measure how many people respond to the treatment and any side effects over up to three years. The current status of this study is unclear.

Study design
This is an interventional study, meaning participants will receive specific treatments. It aims to find the best dose and measure how many people respond to the treatment.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The study will track overall response and side effects for up to three years, and dose-limiting toxicity for up to 56 days.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT03471260

Ivosidenib and Venetoclax With or Without Azacitidine in Treating Patients With IDH1 Mutated Hematologic Malignancies

Recruiting
PHASE1Ages 18+InterventionalTreatment
M.D. Anderson Cancer Center
~96 participants
Updated 2026-09-14 on ClinicalTrials.gov
What's tested:AzacitidineIvosidenibVenetoclax

At a glance

Recruiting sites
4 of 4 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Overall response rate (ORR)
Measured over Up to 3 years
+2 more outcomes measured
Acute Myeloid Leukemia
Hematopoietic and Lymphoid System Neoplasm
Myelodysplastic Syndrome
Myeloproliferative Neoplasm
Recurrent Acute Myeloid Leukemia
Refractory Acute Myeloid Leukemia

NCT03471260

Where you'd take part

This study runs at 4 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Cleveland Clinic Taussig Cancer Institute, Case Comprehensive Cancer Center

    Cleveland, Ohiostudy coordinator listed

    Recruiting

  • Dana-Farber Cancer Institute

    Boston, Massachusettsstudy coordinator listed

    Recruiting

  • M D Anderson Cancer Center

    Houston, Texasstudy coordinator listed

    Recruiting

  • Oregon Health and Science University

    Portland, Oregonstudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

  • Courtney DiNardo, MD · PRINCIPAL_INVESTIGATOR · M.D. Anderson Cancer Center

Opens a ready-to-send draft in your own email app — review before sending.

  • Overall response rate (ORR)Up to 3 years

    Defined as complete response (CR), CR with incomplete blood count recovery (CRi), morphologic leukemia-free state (MLFS), and partial response (PR) based on revised International Working Group (IWG) response criteria. Analysis will be performed for enrolled subjects.

  • Incidence of adverse eventsUp to 3 years

    Will be collected using leukemia adverse event guidelines.

  • Dose-limiting toxicityUp to 56 days

    Defined as any grade 3 or 4, clinically significant non-hematologic adverse event or abnormal laboratory value.