What is GIST?

Gastrointestinal stromal tumor (GIST) is a disease in which abnormal cells form in the tissues of the gastrointestinal tract: stomach, small intestine, and large intestine. GIST is the most common sarcoma of the gastrointestinal (GI) tract.

In approximately 5% of patients with GIST, defects in a gene called PDGFRα, known as D842V, lead to primary resistance to existing approved medications. Currently, there are no approved targeted therapy options for PDGFRα D842V mutant GIST.

Prior results have demonstrated the clinical safety and efficacy of crenolanib in patients with PDGFRα D842V mutant GIST

For more information about GIST and for patient support, please visit the following resources:

PDGFR Summary

Aberrations in PDGFR have an incidence of about 30% in cancer, including mutations, deletions, and amplifications. Tumor types for which PDGFR is altered in at least 10% of cases includes GIST, glioblastoma, lung, melanoma, bladder, prostate, colorectal and ovarian cancers.

Crenogist (ARO-012): an ongoing study for D842V Mutant GIST

  • This is a Phase III clinical trial of crenolanib in patients with advanced or metastatic gastrointestinal stromal tumors (GIST) with a specific mutation (D842V) in the PDGFRA gene.
  • This study is currently recruiting at multiple centers in the United States and Europe.
  • This study aims to find out how safe and effective crenolanib is, compared with a placebo, in prolonging the amount of time patients avoid disease progression.

For more information about the study, please contact a study site or visit clinicaltrials.gov/ct2/show/NCT02847429


  1. Crenolanib has shown benefit in PDGFRA-mutant GIST.
  2. Patients who progress after treatment with prior TKIs may still remain sensitive to crenolanib.
  3. Crenolanib has favorable pharmacokinetics and does not accumulate with repeated dosing.

Recent Peer-reviewed Publications

  1. Blay J-Y, Heinrich MC, Hohenberger P, et al. A randomized, double-blind, placebo-controlled, phase III study of crenolanib in advanced or metastatic GIST patients bearing a D842V mutation in PDGFRA: The CrenoGIST study. J Clin Oncol 2017; 35 (suppl; abstr TPS11080).
  2. von Mehren M, Tetzlaff ED, Macaraeg M, et al. Dose escalating study of crenolanib besylate in advanced GIST patients with PDGFRA D842V activating mutations. J Clin Oncol 2016; 34 (suppl; abstr 11010).
  3. Matro JM, Yu JQ, Heinrich MC, Ramachandran A, Ku N, Mehren Mv. Correlation of PET/CT and CT RECIST response in GIST patients with PDGFRA D842V gene mutations treated with crenolanib. J Clin Oncol 2014; 32:5s (suppl; abstr 10546).
  4. Heinrich MC, Griffith D, McKinley A, et al. Crenolanib inhibits the drug-resistant PDGFRA D842V mutation associated with imatinib-resistant gastrointestinal stromal tumorsClin Cancer Res 2012; 18(16): 4375-84.
  5. Michael M, Vlahovic G, Khamly K, Pierce KJ, Guo F, Olszanski AJ. Phase Ib study of CP-868,596, a PDGFR inhibitor, combined with docetaxel with or without axitinib, a VEGFR inhibitor. Br J Cancer 2010; 103(10): 1554-61.
  6. Lewis NL, Lewis LD, Eder JP, et al. Phase I study of the safety, tolerability, and pharmacokinetics of oral CP-868,596, a highly specific platelet-derived growth factor receptor tyrosine kinase inhibitor in patients with advanced cancers. J Clin Oncol 2009; 27(31): 5262-9.