Trial reportClinical cancer research : an official journal of the American Association for Cancer Research2022
FDA Approval Summary: Ivosidenib for the Treatment of Patients with Advanced Unresectable or Metastatic, Chemotherapy Refractory Cholangiocarcinoma with an IDH1 Mutation.
Trial report in Clinical cancer research : an official journal of the American Association for Cancer Research, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 42 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
42 citing papers in PubMed, 61 citations in OpenAlex.
- Metabolites and cancer metastasis.Oncogene · 2026Review
- Molecularly Guided Therapy and the Tumor Microenvironment in Advanced Cholangiocarcinoma: Current Evidence and Emerging Strategies Targeting FGFR2, HER2, and IDH1 with Pemigatinib, Zanidatamab, and Ivosidenib.Journal of clinical medicine · 2026Review
- APOBEC3 activity and DNA polymerase-ε deficiency are associated with distinct IDH1 R132 hotspot mutations.Molecular oncology · 2026Article
- An Ultra-Fast UPLC-MS/MS Method for Quantifying Ivosidenib: Application to Assessment of Metabolic Stability in Human Liver Microsomes.Archiv der Pharmazie · 2026Article
- Integrating locoregional therapy with systemic and emerging cellular therapies in advanced intrahepatic cholangiocarcinoma.Discover oncology · 2026Review
- Global patterns of mutational profiles in biliary tract cancer.Journal of hepatology · 2026Article
- Paired tumor-normal metabolomic profiling reveals alterations in choline phospholipid precursor metabolism in biliary tract cancer.Journal of gastroenterology · 2026Article
- Targeted therapies in adolescent and young adult patients with central nervous system tumors.Neuro-oncology advances · 2026Review
- A note on rank-preserving structural failure time models to account for crossover.Clinical trials (London, England) · 2026Article
- New and Emerging Therapies for Patients with Low-Grade Glioma.CNS drugs · 2026Review
- Advancing systemic therapy for biliary tract cancer: current strategies and emerging paradigms.Frontiers in oncology · 2026Review
- Targeted therapies and immunotherapies for unresectable cholangiocarcinoma.Chinese medical journal · 2025Review
- Advances in the Molecular Biology of Chondrosarcoma for Drug Discovery and Precision Medicine.Cancers · 2025Review
- Review
- Machine learning-based QSAR and structure-based virtual screening guided discovery of novel mIDH1 inhibitors from natural products.Journal of computer-aided molecular design · 2025Article
- Tumour-agnostic kinase inhibitors.Nature reviews. Drug discovery · 2025Review
- ERK1/2 Signaling in Intrahepatic Cholangiocarcinoma: From Preclinical Advances to Therapeutic Strategies.Biology · 2025Review
- Advancing Systemic Therapy in Chondrosarcoma: New Horizons.Oncology and therapy · 2025Article
- Chondrosarcoma: New Molecular Insights, Challenges in Near-Patient Preclinical Modeling, and Therapeutic Approaches.International journal of molecular sciences · 2025Review
- How do I prescribe and manage mIDH inhibitors in patients with IDH-mutant glioma?Neuro-oncology practice · 2025Review
Corrections and comments
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Authors and funding
17 authors at 2 institutions in 1 country.
Funding
Abstract
On August 25, 2021, the FDA approved ivosidenib for the treatment of adult patients with unresectable locally advanced or metastatic hepatocellular isocitrate dehydrogenase 1 (IDH1) mutated cholangiocarcinoma (CCA) as detected by an FDA-approved test with disease progression after 1 to 2 prior lines of systemic therapy for advanced disease. The approval was based on data from Study AG120-C-005 (ClarIDHy), a double-blind placebo-controlled trial that randomly allocated (2:1) patients to receive either ivosidenib or placebo. Independently assessed progression-free survival (PFS) was the primary endpoint. With a median follow-up of 6.9 months, the HR for PFS was 0.37 [95% confidence interval (CI), 0.25-0.54; P < 0.0001). Overall survival (OS) was the key secondary endpoint. At the final analysis of OS, with 70.5% of patients in the placebo arm receiving ivosidenib post disease progression, a non-statistically significant improvement in the ivosidenib arm with an HR = 0.79 (95% CI, 0.56-1.12) and median OS of 10.3 months (95% CI, 7.8-12.4) and 7.5 months (95% CI, 4.8-11.1) in the ivosidenib and placebo arms, respectively, were reported. Adverse reactions occurring in >20% of patients receiving ivosidenib were fatigue/asthenia, nausea, diarrhea, abdominal pain, ascites, vomiting, cough, and decreased appetite. Adverse reactions occurring in >20% of patients receiving placebo were fatigue/asthenia, nausea, abdominal pain, and vomiting. This is the first approval for the subset of patients with CCA harboring an IDH1 mutation.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.