Calcium upregulation by percutaneous administration of gene therapy in cardiac disease (CUPID Trial), a first-in-human phase 1/2 clinical trial

BE Jaski, ML Jessup, DM Mancini, TP Cappola… - Journal of cardiac …, 2009 - Elsevier
BACKGROUND: SERCA2a deficiency is commonly seen in advanced heart failure (HF).
This study is designed to investigate safety and biological effects of enzyme replacement
using gene transfer in patients with advanced HF. METHODS AND RESULTS: A total of 9
patients with advanced HF (New York Heart Association [NYHA] Class III/IV, ejection fraction
[EF]≤ 30%, maximal oxygen uptake [VO2 max]< 16 mL· kg· min, with maximal
pharmacological and device therapy) received a single intracoronary infusion of …