Study Status: RECRUITING
Recruit Status: RECRUITING
Study Type: INTERVENTIONAL
Official Title: Safety and Efficacy of Venetoclax in Idiopathic Pulmonary Fibrosis
Brief Summary: Based on preclinical data, investigators hypothesize that apoptosis resistance in monocyte-derived macrophages (MDMs) have a decisive role in the development of idiopathic pulmonary fibrosis (IPF).
Specifically, macrophages from subjects with IPF have increased expression of Bcl-2 in mitochondria.
In preclinical models of IPF, a conditional deletion of Bcl-2 in MDMs reverses established fibrosis by inducing apoptosis.
Additional evidence to suggest that Bcl-2 expression in MDM mitochondria is a therapeutic target for IPF as administration of the Bcl-2 inhibitor, ABT-199 (Venetoclax), showed marked efficacy in preclinical models of IPF by inducing apoptosis of MDMs and reversing established fibrosis.
ABT-199 is an orally available mimetic of the BH3 domain of Bcl-2, which is the domain the anchors Bcl-2 in the mitochondria to inhibit apoptosis.
ABT-199 has shown therapeutic efficacy and good safety and tolerability in patients with chronic lymphocytic leukemia.
Investigators anticipate that treatment with ABT-199 could result in significant benefit for IPF patients that have a life expectancy of 3-5 years.
As there is no curative therapy for IPF, this clinical trial has the potential to substantially alter treatment approaches in patients with IPF.