Adipose-derived Regenerative Cells Treatment for Congestive Heart Failure

The aim of the study is to investigate patients suffering from chronic congestive heart failure (iHF) and a left ventricular ejection fraction (LVEF) of equal or less than 40% despite best medical treatment safety and efficacy of a single retrograde intra-cardiac venous (i.cv.) injection of uncultured, autologous, adipose-derived regenerative cells (UA-ADRCs) isolated from lipoaspirate at the point of care. The cells will be isolated using the Transpose® RT / Matrase System (InGeneron, Houston, TX, USA) through an over-the-wire, small balloon catheter, advanced through the coronary sinus and located within a coronary vein at the site of interest, versus patients on best medical treatment.

This study hypothesizes that treatment of iHF with a balloon-enhanced i.cv. injection of UA-ADRCs isolated from lipoaspirate at the point of care using the Transpose RT / Matrase system (InGeneron) is safe and effective and is more effective than best medical treatment.

The primary objective of this study is to evaluate safety and efficacy of treating iHF with a single, balloon-enhanced retrograde i.cv. injection of UA-ADRCs. Efficacy will be demonstrated in a superiority study design by comparing improvement in global cardiac functional parameters demonstrated using cardiac magnetic resonance imaging (cMRI) or Cardiac CT, and by clinical endpoints after a single i.cv. injection of UA-ADRCs, to best medical treatment. Safety will be demonstrated by reporting major ad-verse cardiac events (MACE) and other adverse events.

A total of n=36 patients with iHF and a reduced LVEF (at ≤40%) will be randomly assigned to i.cv. injection of either UA-ADRCs (UA-ADRCs Group; n=24) or best medical treatment (Control Group; n=12). The enrollment will take place up to four study sites, among them the National Science Research Center of the Principal Investigator (Dr. Abay Baigenzhin) in Astana (Kazakhstan), will enroll 36 patients in the study. The study duration is the time to enroll patients in the study and will take 6 months. Individual follow-up time after the treatment is 6 months.

Follow-up schedule is as follows. Follow-up visits will take place at 1, 3, and 6 months post- treatment. Follow-up visit will include an assessment by cMRI, or cCT, physical examinations including echocardiography, patient-reported outcome questionnaires, control of medication usage, number of hospitalizations for cardiac conditions, and adverse event monitoring (including MACE). Those patients in the control group that have finished the 1, 3, and 6 month follow up visits will be offered to be treated with stem cell therapy as well. All remaining patients in both groups will be offered to participate in a subsequent registry study for 12 and 24-month follow-up vis-its, that could include physical examinations, patient-reported outcome questionnaires, medication usage, number of hospitalizations for cardiac conditions, and adverse event monitoring (including MACE) and cMRI or cCT. These long-term data would be compared to the corresponding baseline data.