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

Prospective Evaluation of Ivosidenib Maintenance Following Allogeneic Stem Cell Transplantation in Patients With Acute Myeloid Leukemia or High-risk Myelodysplastic Neoplasia With IDH1 (Isocitrate Dehydrogenase 1) Mutation

Phase 2

Not yet recruiting

Conditions: AML
MDS

Interventions: Drug: Ivosidenib 250 MG (milligram)

First Posted Date: 2024-12-05

Last Posted Date: 2024-12-05

Lead Sponsor: Technische Universität Dresden

Target Recruit Count: 76

Registration Number: NCT06717958

Ivosidenib As Post-HSCT Maintenance for AML

Phase 2

Not yet recruiting

Conditions: IDH1 Mutation
Acute Myeloid Leukemia (AML)
Hematopoietic Stem Cell Transplant (HSCT)

Interventions: Drug: Ivosidenib
Drug: Placebo

First Posted Date: 2024-11-27

Last Posted Date: 2024-11-27

Lead Sponsor: Massachusetts General Hospital

Target Recruit Count: 75

Registration Number: NCT06707493

Locations: 🇺🇸
Massachusetts General Hospital, Boston, Massachusetts, United States
🇺🇸
Dana Farber Cancer Institute, Boston, Massachusetts, United States

Lvosidenib (AK112) Combined With CapeOX and Radiotherapy in Patients With Unresectable Metastatic MSS-type Colorectal Cancer

Phase 2

Not yet recruiting

Conditions: CRC
MSS
Metastatic Cancer

Interventions: Drug: Ivosidenib (AK112)

First Posted Date: 2024-09-12

Last Posted Date: 2024-09-12

Lead Sponsor: Fudan University

Target Recruit Count: 36

Registration Number: NCT06593548

Locations: 🇨🇳
Xinxiang Li, Shanghai, Shanghai, China

Ivosidenib Plus Durvalumab and Gemcitabine/Cisplatin as First-Line Therapy in Participants With Locally Advanced or Metastatic Cholangiocarcinoma With an IDH1 Mutation

Phase 1

Recruiting

Conditions: Locally Advanced, Unresectable or Metastatic Cholangiocarcinoma With an IDH1 Mutation

Interventions: Drug: Ivosidenib
Drug: Durvalumab (for the first 8, 21-day, cycles)
Drug: Gemcitabine (for the first 8, 21-day, cycles)
Drug: Cisplatin (for the first 8, 21-day, cycles)
Drug: Durvalumab (starting from cycle 9)
Drug: Ivosidenib Recommended Combination Dose (RCD)

First Posted Date: 2024-07-15

Last Posted Date: 2025-04-08

Lead Sponsor: Institut de Recherches Internationales Servier

Target Recruit Count: 52

Registration Number: NCT06501625

Locations: 🇺🇸
Northwestern Medicine, Chicago, Illinois, United States
🇺🇸
Memorial Sloan Kettering Cancer Center, New York, New York, United States
🇺🇸
Gibbs Cancer Center, Spartanburg, South Carolina, United States

and more 24 locations

Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation

Phase 3

Recruiting

Conditions: Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS)
Myelodysplastic Syndromes (MDS)

Interventions: Drug: Azacitidine
Drug: Ivosidenib

First Posted Date: 2024-06-20

Last Posted Date: 2025-02-17

Lead Sponsor: Institut de Recherches Internationales Servier

Target Recruit Count: 48

Registration Number: NCT06465953

Locations: 🇺🇸
University of Chicago, Duchossois Center for Advanced Medicine (DCAM), Chicago, Illinois, United States
🇺🇸
Massachusetts General Hospital, Boston, Massachusetts, United States
🇺🇸
MSKCC, New York, New York, United States

and more 18 locations

Ivosidenib and Ruxolitinib in Patients With Advanced Myeloproliferative Neoplasms (MPNs) That Have an IDH1 Gene Mutation

Phase 1

Recruiting

Conditions: Myeloproliferative Neoplasms

Interventions: Drug: Ivosidenib
Drug: Ruxolitinib

First Posted Date: 2024-03-04

Last Posted Date: 2024-10-16

Lead Sponsor: University of Chicago

Target Recruit Count: 18

Registration Number: NCT06291987

Locations: 🇺🇸
University of Chicago Medicine Comprehensive Cancer Center, Chicago, Illinois, United States

Ivosidenib (TIBSOVO®) Combined With Azacitidine According to Current SmPC

Recruiting

Conditions: Acute Myeloid Leukemia (AML)

First Posted Date: 2023-12-26

Last Posted Date: 2024-06-24

Lead Sponsor: iOMEDICO AG

Target Recruit Count: 100

Registration Number: NCT06181734

Locations: 🇩🇪
Praxis für interdisziplinäre Onkologie & Hämatologie, Freiburg, Germany

Ivosidenib in Participants With Locally Advanced or Metastatic Conventional Chondrosarcoma Untreated or Previously Treated With 1 Systemic Treatment Regimen

Phase 3

Recruiting

Conditions: Locally Advanced or Metastatic Conventional Chondrosarcoma With an IDH1 Mutation, Untreated or Previously Treated With 1 Systemic Treatment Regimen

Interventions: Drug: Ivosidenib 500mg
Drug: Placebo

First Posted Date: 2023-11-13

Last Posted Date: 2025-05-07

Lead Sponsor: Servier Bio-Innovation LLC

Target Recruit Count: 136

Registration Number: NCT06127407

Locations: 🇧🇷
Instituto Nacional Do Câncer - Inca, Rio De Janeiro, Brazil
🇧🇷
Icesp - Instituto Do Câncer Do Estado de São Paulo, São Paulo, Brazil
🇧🇷
Fundação Amaral Carvalho - Jaú/ Sp, Jaú, Brazil

and more 55 locations

A Phase 2 Study of Ivosidenib in Previously Treated Japanese Subjects With Nonresectable or Metastatic Cholangiocarcinoma With an IDH1 Mutation

Phase 2

Active, not recruiting

Conditions: Cholangiocarcinoma Non-resectable
Cholangiocarcinoma Metastatic

Interventions: Drug: Ivosidenib

First Posted Date: 2023-10-13

Last Posted Date: 2025-01-10

Lead Sponsor: Servier

Target Recruit Count: 12

Registration Number: NCT06081829

Locations: 🇯🇵
National Cancer Center Hospital East (JPN-002), Kashiwa, Japan
🇯🇵
Kumamoto University Hospital (JPN-004), Kumamoto, Japan
🇯🇵
National Hospital Organization Shikoku Cancer Center (JPN-007), Matsuyama, Japan

and more 4 locations

Ivosidenib, Nivolumab, and Ipilimumab Combination in Previously Treated Subjects With Nonresectable or Metastatic IDH1 Mutant Cholangiocarcinoma

Phase 1

Completed

Conditions: IDH1-mutant Cholangiocarcinoma

Interventions: Drug: Recommended Combination Dose (RCD) of ivosidenib
Drug: Ivosidenib
Drug: Nivolumab
Drug: Ipilimumab

First Posted Date: 2023-06-27

Last Posted Date: 2024-12-17

Lead Sponsor: Servier Bio-Innovation LLC

Target Recruit Count: 7

Registration Number: NCT05921760

Locations: 🇺🇸
UCSF - Medical Center at Mission Bay, San Francisco, California, United States
🇺🇸
Ucsf Helen Diller Family Comprehensive Cancer Center, San Francisco, California, United States
🇺🇸
Sidney Kimmel Comprehensive Cancer Center At Johns Hopkins, Baltimore, Maryland, United States

and more 2 locations

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