TriangleHEALTH PROFESSIONAL · SOURCE READING
IDH1 inhibitors
Source: Bile Duct Cancer (Cholangiocarcinoma) Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: March 28, 2025 · Captured 2026-09-09.
Selected source text with whitespace normalised. This Triangle page is not an NCI PDQ summary.
Up to 15% of bile duct cancers have IDH1 variants.
Evidence (IDH1 inhibitors):
The phase III ClarIDHy trial (NCT02989857) included 187 patients with cholangiocarcinoma and IDH1 variants. Patients had disease that had progressed during previous systemic therapy. Patients were randomly assigned to receive either the IDH1 inhibitor ivosidenib or placebo. The primary end point was PFS.[16,17][Level of evidence B1]
Source links and citations
The phase III ClarIDHy trial (NCT02989857) included 187 patients with cholangiocarcinoma and IDH1 variants. Patients had disease that had progressed during previous systemic therapy. Patients were randomly assigned to receive either the IDH1 inhibitor ivosidenib or placebo. The primary end point was PFS.[16,17][Level of evidence B1]
The median PFS was improved among patients treated with ivosidenib (2.7 months) compared with placebo (1.4 months) (HR, 0.37; 95% CI, 0.25−0.54; P < .001). PFS rates at 6 months and 12 months were 32% and 21.9%, respectively, in the ivosidenib arm. No patients in the placebo group were progression free at 6 months.
The phase III ClarIDHy trial (NCT02989857) included 187 patients with cholangiocarcinoma and IDH1 variants. Patients had disease that had progressed during previous systemic therapy. Patients were randomly assigned to receive either the IDH1 inhibitor ivosidenib or placebo. The primary end point was PFS.[16,17][Level of evidence B1]
In the intention-to-treat analysis, median OS was 10.3 months in the ivosidenib group compared with 7.5 months for the placebo group (HR, 0.79; one-sided P = .09), despite crossover of 57% of placebo patients to ivosidenib. When adjusted for crossover, median OS for the placebo group was 5.1 months.
The phase III ClarIDHy trial (NCT02989857) included 187 patients with cholangiocarcinoma and IDH1 variants. Patients had disease that had progressed during previous systemic therapy. Patients were randomly assigned to receive either the IDH1 inhibitor ivosidenib or placebo. The primary end point was PFS.[16,17][Level of evidence B1]
Grades 3 and 4 toxicities occurred in 46% of patients in the ivosidenib group and 36% of patients in the placebo group.
Studies cited in this source section
Source citation is not a determination that a study applies to you.
Publication references
Read the original reference and check its publication notices.
Preserved source evidence · Not medical advice