This study led by Kezar Life Sciences is a phase two, randomized, dose-blinded, multicenter study to evaluate the safety and efficacy of KZR-616 (a selective immunoproteasome inhibitor) in the treatment of patients with autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP). KZR-616 has been shown in earlier analysis to have a positive effect on taming the inflammatory response in other autoimmune conditions in animal models, without causing immunosuppression. Study participants must be over the age of 18 years. For ITP patients specifically, eligibility is restricted to those who have a platelet count under 30,000μL on two separate blood draws over a week apart, or for ITP patients who are receiving on-going treatment and have a platelet count less than 50,000μL.
This study has multiple phases. Other active trials looking at the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of treatment with KZR-616 are now active for the following autoimmune diseases: lupus (MISSION study: https://clinicaltrials.gov/ct2/show/NCT03393013), dermatomyositis and polymyositis (PRESIDIO study: https://clinicaltrials.gov/ct2/show/NCT04033926).
USA, Italy, Poland, Russia