IDH1 mutation

Clinical trials

Active trials (332)

Trials with an active ClinicalTrials.gov status, an intervention mapped to a derived drug and a condition mapped to an associated cancer. 184 recruiting · 50 active phase 3 · 857 in any status. Most recently updated first, 50 per page.

Computedrule biomarker-links-v1

clinicaltrialsPublishedShowing 1–50 of 332 active studies · status and phase as posted by the registrant; each row links to the study record.

NCT	Title	Status	Phase	Enrollment (n)	Sponsor	Countries	Last update	Source
NCT02465060	Targeted Therapy Directed by Genetic Testing in Treating Patients With Advanced Refractory Solid Tumors, Lymphomas, or Multiple Myeloma (The MATCH Screening Trial)	active not recruiting	Phase 2

Literature

Linked publications (25)

Publications linked to the scope gene(s) through PubMed entity edges; 25 per page, newest first.

pubmedPublishedShowing 1–25 of 25 publications · bibliographic data as recorded by PubMed; abstracts are not reproduced.

Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study.
· Lancet Oncol · 2020 · PMID 32416072 · validated civic_curation pubmed
Vorasidenib in IDH1- or IDH2-Mutant Low-Grade Glioma.
· N Engl J Med · 2023 · PMID 37272516 · validated civic_curation pubmed
Durable Remissions with Ivosidenib in IDH1-Mutated Relapsed or Refractory AML.
· N Engl J Med · 2018 · PMID 29860938 · validated civic_curation pubmed
IDH1 and IDH2 mutations in gliomas.
· N Engl J Med · 2009 · PMID 19228619 · validated civic_curation

IDH1 Mutation4
Ivosidenib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	A	Supports Sensitivity Response	3	accepted	EID7278 I phase 1 trial for patients with IDH1-mutated AML, patients who received ivosidenib (AG-120). The rate of complete remission was 21.6%, the over all response rate was 30.4%. PMID 29860938 · DiNardo et al., 2018 · Open in CIViC	civic
Venetoclax	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	C	Supports Sensitivity Response	3	accepted	EID8856 In this case series, 7 elderly patients with secondary AML (sAML) not eligible for intensive chemotherapy and refractory to HMA (including 1 patient with antecedent myeloproliferative neoplasm) were t… (full text at CIViC) PMID 30725494 · Huemer et al., 2019 · Open in CIViC	civic
Ivosidenib	Cholangiocarcinoma	Predictive	B	Supports Sensitivity Response	3	accepted	EID7447 In a phase1 trial, 73 patients with cholangiocarcinoma harboring IDH1 mutations were treated with mutant IDH1 inhibitor ivosidenib. The response rate was 5%, median progression free survival was 3.8 m… (full text at CIViC) PMID 31300360 · Lowery et al., 2019 · Open in CIViC	civic
Olaparib	Brain GliomaUNRESOLVED	Predictive	D	Supports Sensitivity Response	3	accepted	EID2933 Mutations in isocitrate dehydrogenase 1 and 2, which result in overproduction of 2-hydroxyglutarate (2HG). 2HG is considered to be an oncometabolite, or a metabolite that promotes carcinogenesis, and … (full text at CIViC) PMID 28148839 · Sulkowski et al., 2017 · Open in CIViC	civic

IDH1 Mutation4

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Sensitivity Response

accepted

EID7278

I phase 1 trial for patients with IDH1-mutated AML, patients who received ivosidenib (AG-120). The rate of complete remission was 21.6%, the over all response rate was 30.4%.

PMID 29860938 · DiNardo et al., 2018 · Open in CIViC

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Sensitivity Response

accepted

EID8856

In this case series, 7 elderly patients with secondary AML (sAML) not eligible for intensive chemotherapy and refractory to HMA (including 1 patient with antecedent myeloproliferative neoplasm) were t… (full text at CIViC)

PMID 30725494 · Huemer et al., 2019 · Open in CIViC

Cholangiocarcinoma

Predictive

Supports Sensitivity Response

accepted

EID7447

In a phase1 trial, 73 patients with cholangiocarcinoma harboring IDH1 mutations were treated with mutant IDH1 inhibitor ivosidenib. The response rate was 5%, median progression free survival was 3.8 m… (full text at CIViC)

PMID 31300360 · Lowery et al., 2019 · Open in CIViC

Olaparib

Brain GliomaUNRESOLVED

Predictive

Supports Sensitivity Response

accepted

EID2933

Mutations in isocitrate dehydrogenase 1 and 2, which result in overproduction of 2-hydroxyglutarate (2HG). 2HG is considered to be an oncometabolite, or a metabolite that promotes carcinogenesis, and … (full text at CIViC)

PMID 28148839 · Sulkowski et al., 2017 · Open in CIViC

IDH1 R132C + IDH1 R132H1
Ivosidenib	Acute Myeloid LeukemiaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	4	accepted	EID12903 Popovici-Muller et al. reported preliminary efficacy data from an ongoing phase 1 study of ivosidenib (AG-120) monotherapy in patients with IDH1-mutated malignancies. In patients with relapsed or refr… (full text at CIViC) PMID 29670690 · Popovici-Muller et al., 2018 · Open in CIViC	civic

IDH1 R132C + IDH1 R132H1

Acute Myeloid LeukemiaCURATED_BROADER

Predictive

Supports Sensitivity Response

accepted

EID12903

Popovici-Muller et al. reported preliminary efficacy data from an ongoing phase 1 study of ivosidenib (AG-120) monotherapy in patients with IDH1-mutated malignancies. In patients with relapsed or refr… (full text at CIViC)

PMID 29670690 · Popovici-Muller et al., 2018 · Open in CIViC

IDH1 R1323
Ivosidenib	Cholangiocarcinoma	Predictive	A	Supports Sensitivity Response	5	accepted	EID10886 The multicenter randomized double-blind phase 3 study included patients with IDH1-mutant cholangiocarcinoma who had progressed on previous therapy. Patients were randomly assigned to Ivosidenib or pla… (full text at CIViC) PMID 32416072 · Abou-Alfa et al., 2020 · Open in CIViC	civic
Bevacizumab + SunitinibSubstitutes	High-Grade Glioma, NOS	Predictive	B	Supports Sensitivity Response	2	accepted	EID2020 In a clinical study of 63 recurrent glioma patients, the combined cohort of patients treated with either bevacizumab or sunitinib showed that those harboring IDH1 codon 132 mutation were associated wi… (full text at CIViC) PMID 22199315 · Lv et al., 2011 · Open in CIViC	civic
Dasatinib	Intrahepatic Cholangiocarcinoma	Predictive	D	Supports Sensitivity Response	4	accepted	EID6439 Through a high-throughput drug screen of a large panel of cancer cell lines, including 17 biliary tract cancers, this work found that IDH mutant (IDHm) ICC cells demonstrate a striking response to the… (full text at CIViC) PMID 27231123 · Saha et al., 2016 · Open in CIViC	civic

IDH1 R1323

Cholangiocarcinoma

Predictive

Supports Sensitivity Response

accepted

EID10886

The multicenter randomized double-blind phase 3 study included patients with IDH1-mutant cholangiocarcinoma who had progressed on previous therapy. Patients were randomly assigned to Ivosidenib or pla… (full text at CIViC)

PMID 32416072 · Abou-Alfa et al., 2020 · Open in CIViC

Bevacizumab + SunitinibSubstitutes

High-Grade Glioma, NOS

Predictive

Supports Sensitivity Response

accepted

EID2020

In a clinical study of 63 recurrent glioma patients, the combined cohort of patients treated with either bevacizumab or sunitinib showed that those harboring IDH1 codon 132 mutation were associated wi… (full text at CIViC)

PMID 22199315 · Lv et al., 2011 · Open in CIViC

Intrahepatic Cholangiocarcinoma

Dasatinib

Predictive

Supports Sensitivity Response

accepted

EID6439

Through a high-throughput drug screen of a large panel of cancer cell lines, including 17 biliary tract cancers, this work found that IDH mutant (IDHm) ICC cells demonstrate a striking response to the… (full text at CIViC)

PMID 27231123 · Saha et al., 2016 · Open in CIViC

IDH1 Mutation + IDH2 MutationIDH1 IDH21
Vorasidenib	Glioma	Predictive	A	Supports Sensitivity Response	4	accepted	EID11708 In this study, 331 patients with IDH-mutant grade 2 gliomas were either given vorasidenib (n = 168) or a placebo (n = 163). A median PFS of 27.7 months was reported in the vorasidenib group, compared … (full text at CIViC) PMID 37272516 · Mellinghoff et al., 2023 · Open in CIViC	civic

IDH1 Mutation + IDH2 MutationIDH1 IDH21

Glioma

Predictive

Supports Sensitivity Response

accepted

EID11708

In this study, 331 patients with IDH-mutant grade 2 gliomas were either given vorasidenib (n = 168) or a placebo (n = 163). A median PFS of 27.7 months was reported in the vorasidenib group, compared … (full text at CIViC)

PMID 37272516 · Mellinghoff et al., 2023 · Open in CIViC

IDH1 R132C + IDH1 R132G + IDH1 R132H + IDH1 R132L + IDH1 R132S1
Vorasidenib	Low Grade Glioma	Predictive	A	Supports Sensitivity Response	5	accepted	EID11508 This phase 3 clinical trial was conducted to assess the progression free survival in patients with IDH-mutant gliomas treated with Vorasidenib, an oral brain-penetrant inhibitor, which showed prelimin… (full text at CIViC) PMID 37272516 · Mellinghoff et al., 2023 · Open in CIViC	civic

IDH1 R132C + IDH1 R132G + IDH1 R132H + IDH1 R132L + IDH1 R132S1

Low Grade Glioma

Predictive

Supports Sensitivity Response

accepted

EID11508

This phase 3 clinical trial was conducted to assess the progression free survival in patients with IDH-mutant gliomas treated with Vorasidenib, an oral brain-penetrant inhibitor, which showed prelimin… (full text at CIViC)

PMID 37272516 · Mellinghoff et al., 2023 · Open in CIViC

IDH1 R132H2
AGI-5198	OligodendrogliomaCURATED_BROADER	Predictive	D	Supports Sensitivity Response	4	accepted	EID979 In pre-clinical studies, an IDH1 inhibitor (AG-5198) impaired growth of glioma / anaplastic oligodendroglioma cell lines with IDH1 R132H mutations. A phase I clinical trial of the AG-120 IDH1 inhibit… (full text at CIViC) PMID 23558169 · Rohle et al., 2013 · Open in CIViC	civic
Ivosidenib	Pancreatic Ductal Adenocarcinoma	Predictive	C	Does Not Support Sensitivity Response	2	accepted	EID5987 A 48-year-old female was diagnosed with metastatic pancreatic ductal adenocarcinoma and subsequently started on standard of care chemotherapy, during which her hepatic lesions progressed. Detailed mol… (full text at CIViC) PMID 27466707 · Brody et al., 2018 · Open in CIViC	civic

IDH1 R132H2

AGI-5198

OligodendrogliomaCURATED_BROADER

Predictive

Supports Sensitivity Response

accepted

EID979

In pre-clinical studies, an IDH1 inhibitor (AG-5198) impaired growth of glioma / anaplastic oligodendroglioma cell lines with IDH1 R132H mutations. A phase I clinical trial of the AG-120 IDH1 inhibit… (full text at CIViC)

PMID 23558169 · Rohle et al., 2013 · Open in CIViC

Pancreatic Ductal Adenocarcinoma

Predictive

Does Not Support Sensitivity Response

accepted

EID5987

A 48-year-old female was diagnosed with metastatic pancreatic ductal adenocarcinoma and subsequently started on standard of care chemotherapy, during which her hepatic lesions progressed. Detailed mol… (full text at CIViC)

PMID 27466707 · Brody et al., 2018 · Open in CIViC

Drug	Jurisdiction · authority	Cancer	Indication	Status	Approval date	Source
Venetoclax	US FDA	—	Efficacy supplement 2018-09-07 (see label)	approved	Sep 7, 2018	openfda
Venetoclax	US FDA	—	Efficacy supplement 2018-06-08 (see label)	approved	Jun 8, 2018	openfda
Venetoclax	US FDA	—	Efficacy supplement 2018-06-08 (see label)	approved	Jun 8, 2018	openfda
Venetoclax	US FDA	—	Efficacy supplement 2017-12-20 (see label)	approved	Dec 20, 2017	openfda
Venetoclax	US FDA	Acute Myeloid Leukemia	In combination with azacitidine, or decitabine, or low-dose cytarabine for the treatment of newly diagnosed acute myeloid leukemia (AML) in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.	approved	Apr 11, 2016	openfda
Venetoclax	US FDA	—	For the treatment of adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).	approved	Apr 11, 2016	openfda
Vorasidenib	CA Health Canada	—	Marketed in Canada as VORANIGO (DIN 02551233) under ATC L01XM04 VORASIDENIB. Indications are not published in the Drug Product Database — see the Health Canada Product Monograph.	approvedsource: Marketed	Oct 21, 2024	health-canada-dpd
Vorasidenib	CA Health Canada	—	Marketed in Canada as VORANIGO (DIN 02551225) under ATC L01XM04 VORASIDENIB. Indications are not published in the Drug Product Database — see the Health Canada Product Monograph.	approvedsource: Marketed	Oct 21, 2024	health-canada-dpd
Vorasidenib	EU EMA	Grade 2 Follicular Lymphoma	Voranigo as monotherapy is indicated for the treatment of predominantly non‑enhancing Grade 2 astrocytoma or oligodendroglioma with an IDH1 R132 or IDH2 R172 mutation in adult and adolescent patients aged 12 years and older and weighing at least 40 kg who only had surgical intervention and are not in immediate need of radiotherapy or chemotherapy (see section 5.1).	approved	Sep 17, 2025	ema
Vorasidenib	US FDA	Grade 2 Follicular Lymphoma	VORANIGO is indicated for the treatment of adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 (IDH1) or isocitrate dehydrogenase-2 (IDH2) mutation, as detected by an FDA-approved test, following surgery including biopsy, sub-total resection, or gross total resection [see Dosage and Administration (2.1) , Clinical Pharmacology (12.1) and Clinical Studies (14) ]. VORANIGO is an isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2) inhibitor indicated for the treatment of adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, as detected by an FDA-approved test, following surgery including biopsy, sub-total resection, or gross total resection. ( 1 )	approved	Aug 6, 2024	openfda

Drug

Jurisdiction · authority

Cancer

Indication

Status

Approval date

Source

US FDA

—

Efficacy supplement 2018-09-07 (see label)

approved

Sep 7, 2018

US FDA

—

Efficacy supplement 2018-06-08 (see label)

approved

Jun 8, 2018

US FDA

—

Efficacy supplement 2018-06-08 (see label)

approved

Jun 8, 2018

US FDA

—

Efficacy supplement 2017-12-20 (see label)

approved

Dec 20, 2017

US FDA

Acute Myeloid Leukemia

In combination with azacitidine, or decitabine, or low-dose cytarabine for the treatment of newly diagnosed acute myeloid leukemia (AML) in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy.

approved

Apr 11, 2016

US FDA

—

For the treatment of adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

approved

Apr 11, 2016

CA Health Canada

—

Marketed in Canada as VORANIGO (DIN 02551233) under ATC L01XM04 VORASIDENIB. Indications are not published in the Drug Product Database — see the Health Canada Product Monograph.

approvedsource: Marketed

Oct 21, 2024

health-canada-dpd

CA Health Canada

—

Marketed in Canada as VORANIGO (DIN 02551225) under ATC L01XM04 VORASIDENIB. Indications are not published in the Drug Product Database — see the Health Canada Product Monograph.

approvedsource: Marketed

Oct 21, 2024

health-canada-dpd

Grade 2 Follicular Lymphoma

EU EMA

Voranigo as monotherapy is indicated for the treatment of predominantly non‑enhancing Grade 2 astrocytoma or oligodendroglioma with an IDH1 R132 or IDH2 R172 mutation in adult and adolescent patients aged 12 years and older and weighing at least 40 kg who only had surgical intervention and are not in immediate need of radiotherapy or chemotherapy (see section 5.1).

approved

Sep 17, 2025

ema

Grade 2 Follicular Lymphoma

US FDA

VORANIGO is indicated for the treatment of adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 (IDH1) or isocitrate dehydrogenase-2 (IDH2) mutation, as detected by an FDA-approved test, following surgery including biopsy, sub-total resection, or gross total resection [see Dosage and Administration (2.1) , Clinical Pharmacology (12.1) and Clinical Studies (14) ]. VORANIGO is an isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2) inhibitor indicated for the treatment of adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation, as detected by an FDA-approved test, following surgery including biopsy, sub-total resection, or gross total resection. ( 1 )

approved

Aug 6, 2024