NCT02073994

Study of Orally Administered AG-120 in Subjects With Advanced Solid Tumors, Including Glioma, With an IDH1 Mutation

Completed Phase 1 Last updated 17 February 2026

What this trial tests

Phase 1 trial testing AG-120 in Cholangiocarcinoma in 174 participants. Completed in 4 January 2024.

Timeline

1 March 2014

Primary endpoint
4 January 2024

4 January 2024

Quick facts

Lead sponsor	Institut de Recherches Internationales Servier
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	174
Start date	1 March 2014
Primary completion	4 January 2024
Estimated completion	4 January 2024
Sites	12 locations across France, United States

Drugs / interventions tested

• AG-120 — full drug profile →

Conditions studied

• Cholangiocarcinoma — all drugs for Cholangiocarcinoma →
• Chondrosarcoma — all drugs for Chondrosarcoma →
• Glioma — all drugs for Glioma →
• Other Advanced Solid Tumors — all drugs for Other Advanced Solid Tumors →

Sponsor

Institut de Recherches Internationales Servier — full company profile →

Who can join

18 and older, any sex, with Cholangiocarcinoma or Chondrosarcoma. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-120 in advanced solid tumors, including glioma, that harbor an IDH1 mutation. The first portion of the study is a dose escalation phase where cohorts of patients will receive ascending oral doses of AG-120 to determine maximum tolerated dose (MTD) and/or the recommended Phase II dose. The second portion of the study is a dose expansion phase where four arms of patients will receive AG-120 to further evaluate the safety, tolerability, and clinical activity of the recommended Phase II dose. Anticipated time on study treatment is until disease progression, unacceptable toxicity occurs or at Investigator discretion.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Cholangiocarcinoma - evolving concepts and therapeutic strategies.
   Ilyas SI, Khan SA, Hallemeier CL, Kelley RK, et al · · 2018 · cited 1192× · PMID 28994423 · DOI 10.1038/nrclinonc.2017.157
2. Liver Cancer Cell of Origin, Molecular Class, and Effects on Patient Prognosis.
   Sia D, Villanueva A, Friedman SL, Llovet JM. · · 2017 · cited 864× · PMID 28043904 · DOI 10.1053/j.gastro.2016.11.048
3. Whole-Genome and Epigenomic Landscapes of Etiologically Distinct Subtypes of Cholangiocarcinoma.
   Jusakul A, Cutcutache I, Yong CH, Lim JQ, et al · · 2017 · cited 735× · PMID 28667006 · DOI 10.1158/2159-8290.cd-17-0368
4. Metabolic dysregulation and emerging therapeutical targets for hepatocellular carcinoma.
   Du D, Liu C, Qin M, Zhang X, et al · · 2022 · cited 520× · PMID 35256934 · DOI 10.1016/j.apsb.2021.09.019
5. New Horizons for Precision Medicine in Biliary Tract Cancers.
   Valle JW, Lamarca A, Goyal L, Barriuso J, et al · · 2017 · cited 475× · PMID 28818953 · DOI 10.1158/2159-8290.cd-17-0245
6. Final Overall Survival Efficacy Results of Ivosidenib for Patients With Advanced Cholangiocarcinoma With IDH1 Mutation: The Phase 3 Randomized Clinical ClarIDHy Trial.
   Zhu AX, Macarulla T, Javle MM, Kelley RK, et al · · 2021 · cited 354× · PMID 34554208 · DOI 10.1001/jamaoncol.2021.3836
7. Glioblastoma targeted therapy: updated approaches from recent biological insights.
   Touat M, Idbaih A, Sanson M, Ligon KL. · · 2017 · cited 308× · PMID 28863449 · DOI 10.1093/annonc/mdx106
8. Altered metabolism in cancer: insights into energy pathways and therapeutic targets.
   Tufail M, Jiang CH, Li N. · · 2024 · cited 265× · PMID 39294640 · DOI 10.1186/s12943-024-02119-3