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Venetoclax 400

Phase 3

Acute Myeloid Leukemia | Small molecule | Oncology |United Therapeutics Corporation|Last Updated: Aug 19, 2025

Target and mechanism

Molecular targetBCL2
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment227

FDA Designations

No designations recorded

Clinical trial landscape

Venetoclax 400 · 1 trial · 1 indication

Phase 3 1
NCT07075016Ivosidenib and Azacitidine With or Without Venetoclax in Adult Patients With Newly Diagnosed IDH1-Mutated AML or MDS/AML Considered Ineligible for Intensive ChemotherapyAcute Myeloid Leukemia
RECRUITING227 Analytics
PHASE3RECRUITING
Ivosidenib and Azacitidine With or Without Venetoclax in Adult Patients With Newly Diagnosed IDH1-Mutated AML or MDS/AML Considered Ineligible for Intensive Chemotherapy
Acute Myeloid LeukemiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Event-free survival (EFS) in patients with newly diagnosed IDH1-mutated AML ineligible for intensive chemotherapy
12 months after inclusion of last AML patient

Event-free survival (EFS) in patients with newly diagnosed IDH1-mutated AML ineligible for intensive chemotherapy, measured from the date of randomization to the date of treatment failure, hematologic relapse from CR/CRh or death from any cause, whichever occurs first. Treatment failure is defined as lack of obtaining either CR or CRh by week 24. Patients evaluable for response but not achieving CR or CRh by week 24 will be considered a treatment failure at day 1 post randomization to avoid ambiguities of variable or prolonged periods without response. Patients who die before week 24 without response assessments will also be considered treatment failures at day 1 post randomization. Patients who are alive but not evaluable for response will be censored at day 1 post randomization. Patients who achieved CR or CRh by week 24 and are not known to have morphologic relapse or died will be censored at the date of the last clinical assessment.

Secondary Endpoints

Overall survival (OS) in patients with newly diagnosed IDH1-mutated AML
12 months after inclusion of last AML patient
Rate of CR/CRh in patients with newly diagnosed IDH1-mutated AML
12 months after inclusion of last AML patient
Rate of CR in patients with newly diagnosed IDH1-mutated AML
12 months after inclusion of last AML patient
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Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Placebo comparator: Venetoclax-placeboPLACEBO_COMPARATORday 1-28 Placebo Treatment will be on a continuous 28-day cycle schedule continued until disease relapse, disease progression, development of unacceptable toxicity, death, withdrawal of subject or other protocol defined criteria for discontinuation (which ever comes first)
Experimental: VenetoclaxEXPERIMENTALday 1-28 Venetoclax Treatment will be on a continuous 28-day cycle schedule continued until disease relapse, disease progression, development of unacceptable toxicity, death, withdrawal of subject or other protocol defined criteria for discontinuation (which ever comes first)

Interventions

NameTypeDescription
Venetoclax 400DRUGday 1-28 per cycle
PlaceboDRUGday 1-28 per cycle
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites119

Inclusion Criteria: 1. Patient with newly diagnosed IDH1-mutated AML, or IDH1-mutated MDS/AML according to the 2022 International Consensus Classification (Appendix A). Patients with AML with both IDH1 and IDH2 mutation are eligible as well. Of note: in case both NPM1 and IDH1 are mutated and both ...

Countries:AustriaBelgiumDenmarkEstoniaFinlandFranceGermanyIrelandItalyLithuaniaNetherlandsNorwaySpainSwedenSwitzerlandUnited Kingdom
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Frequently asked questions about Venetoclax 400

What is Venetoclax 400 used for?

Venetoclax 400 is an investigational small molecule being studied for the treatment of Acute Myeloid Leukemia (AML). It is currently in Phase 3 clinical development as part of a combination therapy for adult patients with newly diagnosed IDH1-mutated AML or MDS/AML who are considered ineligible for intensive chemotherapy.

Who makes Venetoclax 400?

Venetoclax 400 is being developed by United Therapeutics Corporation, a biopharmaceutical company traded on the NASDAQ under the ticker symbol UTHR. The company is conducting a Phase 3 clinical trial to evaluate the drug in combination with other agents for the treatment of Acute Myeloid Leukemia.

What phase is Venetoclax 400 in?

Venetoclax 400 is currently in Phase 3 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The ongoing Phase 3 trial is recruiting participants to evaluate its safety and efficacy in patients with Acute Myeloid Leukemia.

What clinical trials is Venetoclax 400 in?

Venetoclax 400 is being studied in a Phase 3 clinical trial registered as NCT07075016. This randomized, double-blind, placebo-controlled trial is recruiting 227 participants across multiple European countries, including Austria, Belgium, Denmark, Finland, France, Germany, Ireland, Italy, and others.

Is Venetoclax 400 the same as venetoclax?

Venetoclax 400 is a specific formulation or dosage of the drug venetoclax, which is being studied in combination with ivosidenib and azacitidine for Acute Myeloid Leukemia. The trial name refers to the 400 mg dose of venetoclax used in the study regimen.