Table 3.
List of completed and ongoing clinical trials that evaluate(d) IDH inhibitors for the treatment of CCA. The IDH mutation types and their frequency are also reported.
| Cholangiocarcinoma | |||||
|---|---|---|---|---|---|
| IDH Mutation Types | IDH1 R132X, IDH2 R172X. | ||||
| Frequency of IDH Mutations | 40% (iCCA) | ||||
| Trial Name | Phase | Year | Drug Tested | Target Population | Outcome Measure |
| Clinical Trials–Completed | |||||
| NCT02989857 (ClarIDHy trial) [121] | III | 2017–2021 | Ivosidenib vs. placebo | Pre-treated advanced IDH1mut iCCA | mPFS 2.7 vs. 1.4 mo, mOS 10.8 vs. 9.7 mo |
| NCT03684811 | Ib/II | 2018–2022 | Olutasidenib | Relapsed/refractory IDH1mut advanced solid tumors | ORR 12.5% |
| NCT04088188 | I | 2021–2023 | Ivosidenib (combined with cisplatin/gemcitabine) | IDH1mut unresectable or metastatic CCA (Arm A) | mOS 22.9 mo, mPFS 15.4 mo |
| Clinical Trials—Ongoing | |||||
| NCT04521686 [122] | I | 2020 | LY3410738 | IDH1mut or IDH2mut advanced solid tumors | |
| NCT04762602 [72] | I | 2021 | HMPL-306 | IDHmut solid tumors | |
| NCT05814536 | I | 2023 | Safusidenib | IDH1mut advanced CCA and other solid tumors | |
iCCA: intrahepatic cholangiocarcinoma; mPFS: median progression-free survival; mo: months; mOS: median overall survival; ORR: objective response rate; CCA: cholangiocarcinoma.