Ivosidenib

Generic Name: Ivosidenib

Brand Names: Tibsovo

Drug Type: Small Molecule

Chemical Formula: C_₂₈H_₂₂ClF_₃N_₆O_₃

CAS Number: 1448347-49-6

Unique Ingredient Identifier: Q2PCN8MAM6

Background: Ivosidenib is a first-in-class isocitrate dehydrogenase-1 (IDH1) inhibitor. IDH1 is an enzyme that is often mutated and overexpressed in some cancers, leading to aberrant cell growth and proliferation. Ivosidenib inhibits mutated IDH1, blocking the enzymatic activity and further differentiation of cancer cells.

Ivosidenib was granted accelerated approval by the FDA in July 2018 for the treatment of relapsed of refractory acute myeloid leukemia in adults. It is currently approved to also treat newly diagnosed acute myeloid leukemia in older adults in combination azacitidine or as monotherapy, as well as locally advanced or metastatic cholangiocarcinoma in adults. The drug is only effective in patients with a susceptible IDH1 mutation.

In February 2023, the EMA's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion of ivosidenib and recommended it be granted marketing authorization for the treatment of acute myeloid leukemia and cholangiocarcinoma. It was fully approved by the EMA in May 2023.

Indication: Ivosidenib is an isocitrate dehydrogenase-1 (IDH1) inhibitor approved for use in the US and Europe. It is indicated for the treatment of patients with a susceptible IDH1 mutation with:

Associated Conditions: Acute Myeloid Leukemia, Locally Advanced Cholangiocarcinoma, Metastatic Cholangiocarcinoma, Refractory Acute Myeloid Leukemia (AML), Relapsed Acute Myelogenous Leukemia (AML)

Clinical Trials

Glasdegib-Based Treatment Combinations for the Treatment of Patients With Relapsed Acute Myeloid Leukemia Who Have Undergone Hematopoietic Cell Transplantation

Phase 1

Withdrawn

Conditions: Recurrent Acute Myeloid Leukemia

Interventions: Drug: Gilteritinib Fumarate
Drug: Enasidenib Mesylate
Drug: Glasdegib Maleate
Drug: Bosutinib Monohydrate
Drug: Decitabine
Drug: Ivosidenib
Drug: Venetoclax

First Posted Date: 2020-12-07

Last Posted Date: 2022-06-09

Lead Sponsor: City of Hope Medical Center

Registration Number: NCT04655391

Locations: 🇺🇸
City of Hope Meidcal Center, Duarte, California, United States

CPX-351 and Ivosidenib for the Treatment of IDH1 Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Phase 2

Recruiting

Conditions: Myelodysplastic Syndrome
Acute Myeloid Leukemia With Gene Mutations
Recurrent Acute Myeloid Leukemia
Myeloproliferative Neoplasm
Refractory Acute Myeloid Leukemia

Interventions: Drug: Ivosidenib
Drug: Liposome-encapsulated Daunorubicin-Cytarabine

First Posted Date: 2020-07-30

Last Posted Date: 2025-04-23

Lead Sponsor: M.D. Anderson Cancer Center

Target Recruit Count: 30

Registration Number: NCT04493164

Locations: 🇺🇸
M D Anderson Cancer Center, Houston, Texas, United States

AG-120 in People With IDH1 Mutant Chondrosarcoma

Phase 2

Active, not recruiting

Conditions: Chondrosarcoma, Grade 3
IDH1 Gene Mutation
Chondrosarcoma
Chondrosarcoma, Grade 2

Interventions: Drug: AG-120

First Posted Date: 2020-02-20

Last Posted Date: 2025-05-16

Lead Sponsor: Memorial Sloan Kettering Cancer Center

Target Recruit Count: 6

Registration Number: NCT04278781

Locations: 🇺🇸
Johns Hopkins Hospital (Data Collection Only), Baltimore, Maryland, United States
🇺🇸
Columbia University (Specimen Analysis Only), New York, New York, United States
🇺🇸
Memorial Sloan Kettering Cancer Center, New York, New York, United States

and more 2 locations

Ivosidenib and Combination Chemotherapy for the Treatment of IDH1 Mutant Relapsed or Refractory Acute Myeloid Leukemia

Phase 1

Active, not recruiting

Conditions: Recurrent Myelodysplastic Syndrome
Recurrent Acute Myeloid Leukemia
Refractory Acute Myeloid Leukemia
Refractory Myelodysplastic Syndrome
Recurrent Myeloproliferative Neoplasm

Interventions: Drug: Cytarabine
Biological: Filgrastim
Drug: Fludarabine
Drug: Fludarabine Phosphate
Drug: Ivosidenib

First Posted Date: 2020-01-31

Last Posted Date: 2025-01-13

Lead Sponsor: Northwestern University

Target Recruit Count: 2

Registration Number: NCT04250051

Locations: 🇺🇸
Northwestern University, Chicago, Illinois, United States

Ivosidenib in Treating Patients With Advanced Solid Tumors, Lymphoma, or Histiocytic Disorders With IDH1 Mutations (A Pediatric MATCH Treatment Trial)

Phase 2

Active, not recruiting

Conditions: Recurrent Ependymoma
Recurrent Rhabdoid Tumor
Recurrent Hepatoblastoma
Recurrent Malignant Germ Cell Tumor
Recurrent Malignant Solid Neoplasm
Recurrent Ewing Sarcoma
Recurrent Langerhans Cell Histiocytosis
Recurrent Medulloblastoma
Recurrent Neuroblastoma
Recurrent Osteosarcoma

Interventions: Drug: Ivosidenib

First Posted Date: 2019-12-12

Last Posted Date: 2025-05-13

Lead Sponsor: National Cancer Institute (NCI)

Target Recruit Count: 3

Registration Number: NCT04195555

Locations: 🇺🇸
Banner Children's at Desert, Mesa, Arizona, United States
🇺🇸
Phoenix Childrens Hospital, Phoenix, Arizona, United States
🇺🇸
Banner University Medical Center - Tucson, Tucson, Arizona, United States

and more 171 locations

A China Bridging Study of Ivosidenib in r/r AML Subjects With an IDH1 Mutation

Phase 1

Completed

Conditions: Relapsed or Refractory Acute Myeloid Leukemia

Interventions: Drug: ivosidenib

First Posted Date: 2019-11-25

Last Posted Date: 2023-02-13

Lead Sponsor: CStone Pharmaceuticals

Target Recruit Count: 30

Registration Number: NCT04176393

Locations: 🇨🇳
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences, Tianjin, Tianjin, China

Gemcitabine and Cisplatin With Ivosidenib or Pemigatinib for the Treatment of Unresectable or Metastatic Cholangiocarcinoma

Phase 1

Terminated

Conditions: Unresectable Cholangiocarcinoma
Advanced Cholangiocarcinoma
Metastatic Cholangiocarcinoma

Interventions: Drug: Cisplatin
Drug: Gemcitabine
Drug: Ivosidenib
Drug: Pemigatinib

First Posted Date: 2019-09-12

Last Posted Date: 2024-05-06

Lead Sponsor: Academic and Community Cancer Research United

Target Recruit Count: 8

Registration Number: NCT04088188

Locations: 🇺🇸
M D Anderson Cancer Center, Houston, Texas, United States
🇺🇸
Mayo Clinic in Rochester, Rochester, Minnesota, United States
🇺🇸
Emory University Hospital/Winship Cancer Institute, Atlanta, Georgia, United States

and more 4 locations