Today, the Pulmonary Fibrosis Foundation (PFF) announced enrollment of the first patient in PRECISIONS (Prospective tReatment EffiCacy in IPF uSIng genOtype for Nac Selection) clinical trial. This is the first clinical trial to apply the principles of precision medicine to the treatment of patients with idiopathic pulmonary fibrosis (IPF). The trial aims to determine if N-Acetyl-cysteine (NAC) is an effective treatment in a subset of IPF patients who carry a particular genetic variant.
The study is designed as a double-blind, multi-center, randomized, placebo-controlled trial investigating the safety and efficacy of NAC in patients with IPF who have the TOLLIP rs3750920 T/T genotype, which is present in 25% of IPF patients. The trial will enroll 200 patients from approximately 20 PFF Care Center Network (CCN) sites. Initial recruitment into the study is being facilitated by looking at phenotypic data from patients that are enrolled in the PFF Registry.
“COVID-19 presented numerous obstacles over the past several months, including acquiring the study drug from Lombardi, Italy. Thanks to the herculean efforts of the primary investigators and site coordinators in the PFF Care Center Network, and most importantly, our patient volunteers, enrollment is now underway,” said Dr. Fernando Martinez, principal investigator for PRECISIONS.
The first patient was enrolled at the University of Michigan. “IPF is a devastating disease, and we are proud to play a role in this study that we hope will lead to additional treatments for our patients who urgently need them,” said Dr. Elizabeth Belloli, principal investigator at the University of Michigan.
Dr. Kevin Flaherty, Chair, PFF Registry and CCN Steering Committee, stated “This achievement is the result of a tireless team of investigators and study coordinators working in unison under the extreme conditions of the pandemic. In addition to the clinical trial, additional aims in the PRECISIONS study seek to identify the genetic variants of IPF and other interstitial lung diseases in order to distinguish individual diseases and predict disease course.”