Clonal cytopenia of undetermined significance (CCUS) is a condition characterized by clonal hematopoiesis (CH) and cytopenia, and it significantly increases the risk of developing acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). About 15-20% of AML patients and 5% of patients with MDS carry IDH1/2 mutations, and those with CH and these mutations have an even higher risk of developing myeloid neoplasms.

Targeted therapies, such as IDH1/2 inhibitors, have shown promising results in patients with relapsed/refractory (R/R) AML and more recently in MDS. Notably, these inhibitors have been particularly effective in cases with low mutational burden, suggesting they might also be beneficial in patients with IDH1-mutant CCUS, who typically have a lower mutational burden.

The Preventing IDH Mutant Myeloid Neoplasm (PIMMs; NCT05030441) clinical trial is the first of its kind and will investigate the safety and efficacy of a drug called ivosidenib in patients with CCUS. This trial aims to determine whether ivosidenib is effective in preventing the progression of CCUS and later development of myeloid neoplasms. The study will enroll 15 patients and is being conducted in a decentralized manner, allowing any patient residing in the US to participate.

For those interested in more information about the trial, for enrolling patients, or becoming a participating site, please contact (314) 747-3543 or email PIMM@wustl.edu or bolton@wustl.edu.

Written by Elitsa Kamberska
Edited by Thomas Southgate

Publishing date: 25/07/2023