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Ivosidenib

Phase 3

Acute Myeloid Leukemia (AML) | Small molecule | Oncology |Tango Therapeutics, Inc.|Last Updated: May 12, 2026

Target and mechanism

Molecular targetIDH1
Target classInhibitor
ModalitySmall molecule

Also known as Ivosidenib Oral Tablet, Ivosidenib 500mg, Ivosidenib 500mg Oral Tablet

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment245

FDA Designations

No designations recorded

Clinical trial landscape

Ivosidenib · 6 trials · 7 indications

Phase 3 3Phase 2 2Phase 1 1
NCT06127407Ivosidenib in Participants With Locally Advanced or Metastatic Conventional Chondrosarcoma Untreated or Previously Treated With 1 Systemic Treatment RegimenLocally Advanced or Metastatic Conventional Chondrosarcoma With an IDH1 Mutation, Untreated or Previously Treated With 1 Systemic Treatment Regimen
RECRUITING136 Analytics
NCT05907057An Open-label Phase 3b Study of Ivosidenib in Combination With Azacitidine in Adult Patients Newly Diagnosed With IDH1m Acute Myeloid Leukemia (AML) Ineligible for Intensive Induction Chemotherapy.Acute Myeloid Leukemia (AML)
RECRUITING245 Analytics
NCT05876754An Early Access Study of Ivosidenib in Patients With a Pretreated Locally Advanced or Metastatic CholangiocarcinomaCholangiocarcinoma
RECRUITING220 Analytics
PHASE3RECRUITING
Ivosidenib in Participants With Locally Advanced or Metastatic Conventional Chondrosarcoma Untreated or Previously Treated With 1 Systemic Treatment Regimen
Locally Advanced or Metastatic Conventional Chondrosarcoma With an IDH1 Mutation, Untreated or Previously Treated With 1 Systemic Treatment RegimenUnlock trial analytics
PHASE3RECRUITING
An Open-label Phase 3b Study of Ivosidenib in Combination With Azacitidine in Adult Patients Newly Diagnosed With IDH1m Acute Myeloid Leukemia (AML) Ineligible for Intensive Induction Chemotherapy.
Acute Myeloid Leukemia (AML)Unlock trial analytics
PHASE3RECRUITING
An Early Access Study of Ivosidenib in Patients With a Pretreated Locally Advanced or Metastatic Cholangiocarcinoma
CholangiocarcinomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Progression-free survival (PFS) based on Blinded Independent Central Reviewer (BICR) assessment in Grade 1 and Grade 2 participants
Up to approximately 31 months

From randomization until BICR confirmed progressive disease or death due to any cause, whichever occurs first

Number of Adverse Events (AEs)
up to week 116

Adverse events (AEs) will be graded according to the CTCAE v5.0

Number of Serious Adverse Events (SAEs)
up to week 116

Adverse events (AEs) will be graded according to the CTCAE v5.0

Differentiation Syndrome of Grade 2 or higher
up to week 116
Number of Adverse Events (AEs) leading to ivosidenib + azacitidine discontinuation
up to week 112
Number of Adverse Events (AEs) leading to ivosidenib + azacitidine interruption
up to week 112
Number of Adverse Events (AEs) leading to ivosidenib + azacitidine dose reduction
up to week 112
Number of Adverse Events (AEs) leading to death
up to week 116
Number of clinical laboratory anomalies assessed as Adverse Events (AEs)
up to week 116
Number of patients requiring transfusion (platelet and RBC) and the average number of units transfused
up to week 116
Rate of infections
up to week 116

Infection rates will be summarized by classification and will include a count and proportion.

QT Prolongation event assessed as Grade 3 or higher
up to week 116
Number of Adverse Events (AEs) from Day 1 of Cycle 1 through 28 days after last study treatment
Day 1 of cycle 1, Day 1 of each consecutive cycle, 28 days after last study treatment

AEs will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0. All reported AEs will be coded using the Medical Dictionary for Regulatory Activities (MedDRA), using the latest version.

Number of Serious Adverse Events (SAEs) during the study treatment period (from Day 1 of Cycle 1 through the last study treatment intake or withdrawal of consent, whichever comes first).
Day 1 of cycle 1, Day 1 of each consecutive cycle, 28 + 14 days (maximum) after last study treatment, 6 months after last study treatment, 12 months after last study treatment, 18 months after last study treatment

SAEs related to study drug will be collected irrespective of the time of onset. AEs will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0.

Number of QT prolongation events during electrocardiogram (ECG) assessed as Grade 2 or worse occurring from Day 1 of Cycle 1 through 28 days after last study treatment
Day 1 of cycle 1, week 2 of cycle 1, week 3 of cycle 1, Day 1 of each consecutive cycle, 28 days after last study treatment

QT interval, using Fridericia's formula \[QTcF\], to average QTc interval \> 480 to 500msec (Grade 2) or worse, as seen during an ECG. This is classified as an Adverse Event of Special Interest (AESI) for this study.

Change in Eastern Cooperative Oncology Group (ECOG) performance status (PS) score from baseline to worst value out of the post-baseline assessments.
Screening visit, Day 1 of cycle 1, Day 1 of each consecutive cycle, 28 + 14 days (maximum) after last study treatment

ECOG PS scoring consists of Grade 0 - 5, with 0 being the patient is fully active and 5 being the patient is dead. Descriptive statistics of ECOG PS over time will be summarized by frequency. Shift tables may be provided for ECOG PS from baseline to worst value of post-baseline assessments.

Number of Adverse Events (AEs) leading to discontinuation or death from day 1 through 28 days after the last study treatment
Day 1 of cycle 1, Day 1 of each consecutive cycle, 28 days after last study treatment

Total number of AEs that result in discontinuation from treatment or death. AEs will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0.

Total laboratory abnormalities using National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0 grading scale or the low/normal/high classifications based on laboratory normal ranges.
Screening visit, Day 1 of cycle 1, Day 1 of each consecutive cycle, 28 + 14 days (maximum) after last study treatment

Listing of all laboratory hematology, coagulation, and chemistry data with values flagged as abnormal to show the corresponding NCI-CTCAE grades and the classifications relative to the laboratory normal ranges.

Change from baseline to the worst on-treatment value of laboratory abnormalities.
Screening visit, Day 1 of cycle 1, Day 1 of each consecutive cycle, 28 + 14 days (maximum) after last study treatment

Abnormalities will be classified by using National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0 grading scale or the low/normal/high classifications based on laboratory normal ranges. Shift tables using NCI-CTCAE grades to compare baseline to the worst on-treatment value will be used. For laboratory tests, including hematology, coagulation, and chemistry, where NCI-CTCAE grades are not defined, shift tables using the low/normal/high \[low and high\] classification to compare baseline to the worst on treatment may be generated. On-treatment is considered from Day 1 of Cycle 1 through 28 days after the last dose.

Number of patients with vital sign values outside limits of the normal range at each time point.
Screening visit, Day 1 of cycle 1, Day 1 of each consecutive cycle, 28 + 14 days (maximum) after last study treatment

Vital signs include systolic and diastolic blood pressure, heart rate, respiratory rate, and temperature.

Mean change from baseline values to the worst on-treatment value of patients with vital signs outside limits of the normal range
Screening visit, Day 1 of cycle 1, Day 1 of each consecutive cycle, 28 + 14 days (maximum) after last study treatment

On-treatment is considered from Day 1 of Cycle 1 through 28 days after the last dose. Vital signs include systolic and diastolic blood pressure, heart rate, respiratory rate, and temperature.

6-month Progression Free Survival (PFS) Rate
Through 6 months after the first dose

Proportion of subjects who are alive and progression-free (using RECIST v1.1) at 6 months after Day 1 (C1D1) per Independent Radiology Center (IRC)

Rate of improvement in hematologic parameters
Through 30 days after completion of treatment (estimated to be 61 months)

Will be evaluated according to a modified version of the IWG 2006 Criteria for Hematologic Improvement for patients with MDS on clinical trials * Erythroid response (pretreatment, \<11 g/DL) * Hemoglobin (Hgb) increase by ≥1.5 g/dL * Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 weeks, compared with the pretreatment transfusion number in the previous 8 weeks. Only RBC transfusions given for an Hgb of ≤9.0 g/dL pretreatment will count in the RBC transfusion response evaluation * Platelet response (pretreatment, \<100 x 10\^9/L) * Absolute increase of ≥30 × 10\^9/L for patients starting with \>20 × 10\^9/L platelets. * Increase from \<20 × 10\^9/L to \>20 × 10\^9/L and by at least 100%. * Neutrophil response (pretreatment, \<1.0 x 10\^9/L): * At least 100% increase and an absolute increase \>0.5 × 10\^9/L. If pegfilgrastim being used prior to initiation of study, define response as no longer requiring pegfilgrastim to maintain ANC \>500.

Maximum observed steady-state concentration (Cmax,ss)
Through day 28 of cycle 1
Area under the concentration time curve from 0 to 24 hours (AUC0-24hr)
Through day 28 of cycle 1
Predose plasma concentration (Ctrough)
Through day 28 of cycle 1
Time to maximum observed concentration (Tmax)
Through day 28 of cycle 1

Secondary Endpoints

PFS based on BICR assessment in all randomized participants
Up to approximately 31 months
Overall survival (OS) in Grade 1 and Grade 2 participants
Up to 5 years
OS in all randomized participants
Up to 5 years
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Study Design & Arms

AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
IvosidenibEXPERIMENTALTaken continuously until BICR-confirmed disease progression, unacceptable toxicity, confirmed pregnancy, death, withdrawal of consent, lost to follow-up, or the Sponsor ends the study (estimated average treatment duration of two years).
PlaceboPLACEBO_COMPARATORTaken continuously until BICR-confirmed disease progression, unacceptable toxicity, confirmed pregnancy, death, withdrawal of consent, lost to follow-up, or the Sponsor ends the study (estimated average treatment duration of two years). Participants randomized to the placebo arm who experience BICR-confirmed disease progression and meet the crossover eligibility criteria will be given the opportunity to cross over and receive ivosidenib.
Open-Label Ivosidenib in combination with AzacitidineEXPERIMENTALAll participants will receive both Ivosidenib and Azacitidine for a maximum of 28 cycles. Each cycle will be 4 weeks or 28 days long. Ivosidenib will be taken continuously throughout each cycle and Azacitidine will be taken only for 7 days at the beginning of each cycle.
Open-Label IvosidenibEXPERIMENTAL250 mg Tablets
Group 1 - Moderate Hepatic Impairment (HI)EXPERIMENTAL -
Group 2 - Severe HIEXPERIMENTAL -
Group 3 - Severe Renal Impairment (RI)EXPERIMENTAL -
Group 4 - Adequate hepatic functionEXPERIMENTAL -
Group 5 - Adequate renal functionEXPERIMENTAL -

Interventions

NameTypeDescription
Ivosidenib 500mgDRUGProvided as tablets, taken orally as two 250mg tablets once daily.
PlaceboDRUGProvided as tablets, taken orally once daily.
Ivosidenib 500mg Oral TabletDRUGProvided as tablets, taken orally as two 250mg tablets once daily.
AzacitidineDRUGAdministered subcutaneously (SC) or intravenously (IV) at a dose of 75mg/m2/day for 7 days, either consecutively on Days 1-7 or discontinuously for Days 1-5 and 8-9 of each cycle. The 7 days of administration will occur at the beginning of every 4 week-long cycle.
Ivosidenib Oral TabletDRUGIvosidenib 500 mg
IvosidenibDRUGSubjects will take 2 tablets (500 mg total) orally once daily.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites114

Inclusion Criteria: * Have a histopathological diagnosis (fresh or banked tumor biopsy sample collected within the last 3 years) consistent with locally advanced or metastatic conventional chondrosarcoma Grades 1, 2, or 3 and not eligible for curative resection. * Have at least one BICR-confirmed m...

Countries:United StatesAustraliaBelgiumBrazilCanadaChinaDenmarkFranceGermanyItalyJapanNetherlandsPuerto RicoSouth KoreaSpainTaiwanUnited KingdomAustriaArmeniaIrelandRomaniaSwedenCzechia
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Frequently asked questions about Ivosidenib

What is Ivosidenib used for?

Ivosidenib is an investigational small molecule being studied for several IDH1-mutated cancers, including acute myeloid leukemia, cholangiocarcinoma, and other IDH1-mutated malignancies. It is also being evaluated for clonal cytopenia of undetermined significance and locally advanced or metastatic conventional chondrosarcoma with an IDH1 mutation.

What does Ivosidenib target?

Ivosidenib targets the IDH1 enzyme. It is an inhibitor of mutant IDH1, which is a molecular target in certain cancers. By inhibiting this enzyme, the drug aims to interfere with the metabolic pathways that drive tumor growth in IDH1-mutated malignancies.

Who is developing Ivosidenib?

Ivosidenib is being developed by Tango Therapeutics, Inc., a biopharmaceutical company traded on the stock exchange under the ticker symbol TNGX. The company is conducting clinical trials to evaluate the drug's safety and efficacy in various oncology indications.

What phase is Ivosidenib in?

Ivosidenib is in Phase 3 clinical development for cholangiocarcinoma, as part of an early access study. It is also being studied in Phase 2 trials for clonal cytopenia of undetermined significance and cholangiocarcinoma, and in a Phase 1 trial for IDH1-mutated malignancies with hepatic or renal impairment.

What clinical trials is Ivosidenib in?

Ivosidenib is being studied in several clinical trials, including NCT05030441 for clonal cytopenia of undetermined significance, NCT05876754 for pretreated cholangiocarcinoma, NCT06081829 for Japanese patients with cholangiocarcinoma, and NCT07006688 for IDH1-mutated malignancies with hepatic or renal impairment.

Is Ivosidenib the same as Ivosidenib plus oral azacitidine?

Ivosidenib is also known as Ivosidenib plus oral azacitidine, Ivosidenib plus azacitidine, and Ivosidenib oral tablet. These names refer to the same drug, which is being investigated for use in combination with azacitidine in certain treatment regimens.