Keywords: Gene Therapy , Heart Failure , Regenerative Medicine , Cardiology
IRB Number: 00067119
Specialty: Cardiothoracic Surgery, Cardiothoracic Surgery, Cardiothoracic Surgery
Sub Specialties: Heart Stem Cell Therapy, Heart Failure
The primary objective of this Phase I/II study is to assess the safety and clinical efficacy of delivering single doses of JVS-100 by a retrograde delivery infusion into the coronary sinus in subjects with chronic ischemic heart failure. The first 2 cohorts are open label. Subjects in the first cohort will receive 30 mgs of JVS-100 while patients in the second cohort will receive 45 mg of JVS-100. Following a safety DSMC review after 7 days following the completion of enrollment in each cohort, additional subjects will be randomized to receive either a single dose of JVS-100 (30 or 45 mg) or placebo in a parallel arm design. These results will be used to design a Phase IIb or Phase III clinical trial.
To assess safety and tolerability of single doses of JVS-100 delivered via retrograde coronary sinus administration through the Oscor Venos Occlusion Balloon catheter.
To investigate the impact of single doses of JVS-100 (either 30 or 45 mg) delivered retrograde via the coronary sinus through the Oscor Venos Occlusion Balloon catheter on 6 minute walk distance and/or quality of life measured by the Minnesota Living with Heart Failure Questionnaire compared to placebo at 4 months post dosing.
To assess the impact of a single dose of JVS-100 on heart failure symptoms (e.g. quality of life and NYHA class), functional (e.g. 6 minute walk, treadmill exercise test), LV function (e.g. LVESV, LVEF), biomarker (e.g. NTproBNP) and morbidity/mortality (e.g. time to first heart failure decompensation, cardiovascular, and all-cause mortality) parameters compared to placebo at 4 and/or 12 months post dosing.