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 Phase II clinical results suggest promising efficacy of crenolanib in patients with PDGFRα D842V mutant GIST.

PDGFR Summary

Platelet derived growth factor receptors (PDGFRs) are tyrosine kinase receptors, that are involved in many normal cellular processes, including organogenesis, cell proliferation, angiogenesis, etc. These receptors come in two isoforms called PDGFRA and PDGFRB. PDGFR signaling can be altered by gene amplification or activating mutations. These aberrations in PDGFR have an incidence of about 30% in cancer. 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 aims to find out how safe and effective crenolanib is, compared with a placebo, in prolonging the amount of time patients avoid disease progression.

This study is currently recruiting at multiple centers in the United States and Europe – find a location below.

State Center City
California Sarcoma Oncology Center Santa Monica
Florida University of Miami Miami
North Carolina Duke Cancer Institute Durham
Oregon Oregon Health and Science University Portland
Pennsylvania Fox Chase Cancer Center Philadelphia
Texas MD Anderson Cancer Center Houston
Country Center City
France Institut Bergonie Bordeaux
Centre Oscar Lambret Lille
Centre Leon Berard Lyon
La Timone University Hospital Marseille
Centre Hospitalier Universitaire (CHU) de Reims Reims
Germany HELIOS Klinikum Berlin-Buch Berlin
Mannheim University Medical Centre, University of Heidelberg Mannheim
Universitätsklinikum München Munich
Italy Policlinico S. Orsola-Malpighi Bologna
Istituto Nazionale Tumori Milan
Institut Regina Elena / IFO Rome
Candiolo Cancer Institute – FPO, IRCCS Turin
Norway University Hospital The Norwegian Radium Hospital Oslo
Poland M Sklodowska-Curie Memorial Cancer Centre and Institute of Oncology Warsaw
Spain Vall d’Hebron University Hospital Barcelona
Hospital Universitario Puerta de Hierro Madrid
Hospital Virgen del Rocio Sevilla
Fundación Instituto Valenciano de Oncología Valencia

For more information about the study, please contact a study site by visiting HERE