The risk-benefit assessment determined that there are no significant safety concerns.
Following a prespecified data review of the ongoing phase 3 clinical trial (NCT02438306) of BioCardia’s CardiAMP cell therapy, an investigational autologous cell therapy for the treatment of heart failure, an independent Data Safety Monitoring Board (DSMB) has given a positive review and recommendation to continue.1
The CardiAMP cell therapy uses patients’ bone marrow cells in a catheter-based procedure with the intent of stimulating a natural healing process. Based on data from 101 patients who were at least 12 months post-treatment, the DSMB’s risk-benefit assessment determined that there are no significant safety concerns. Blinded patient data from both patients treated with CardiAMP and patients in the control group(3 treatment : 2 sham control, randomly assigned) were available for 93% of all patients in the trial, and demonstrated that patients in this trial had a greater survival rate than patients with heart failure with reduced ejection fraction who were involved in several other similar large pivotal trials (NCT03057977 and NCT01035255). Furthermore, patients involved in the trial showed favorable trends for meaningful clinical improvement on 6-minute walk distance test; patient status, assessed by New York Heart Association class; quality of life measured using the Minnesota Living with Heart Failure questionnaire (MLHFQ); and heart function assessments. However, due to the blinding, it is not known at this time whether any significant difference exists in these outcome measures between the control group and the group that received treatment with CardiAMP.
“It is outstanding news that available blinded results show the trial integrity remains intact with few patients lost to follow-up visits,” Peter Altman, PhD, chief executive officer, BioCardia, said in a statement.1 “The phenomenal follow-up during these difficult times is to the credit of our dedicated clinical partners and the patients participating in the study. The patients also appear to be doing relatively well. It has long been noted that patients participating in clinical trials do better. The meaningful improvements on average across both treated and control patients being observed here are great for those participating in the study. We would not expect the control patients in this study to improve where control placebo patients in our phase 2 trial did not.”
The multi-center clinical trial is expected to enroll 260 patients between 21 years and 90 years of age who have been diagnosed with chronic ischemic left ventricular dysfunction secondary to myocardial infarction. Participants are required to have been treated with medical and device therapy for heart failure or post-infarction left ventricular dysfunction for at least 3 months before being randomly assigned to a group. Participants must also have a left ventricular ejection fraction between 20% and 40%, and a pre-procedure screening of bone-marrow aspiration.
Patients in the experimental group will receive placement of an introducer guidewire and have a left ventriculogram performed. Treatment will be administered with the CardiAMPcell therapy system, which includes the CardiAMP Cell Separator, a cardiac delivery catheter, and a flexible tip guide catheter. Patients in the sham comparator control group will only receive placement of the introducer guidewire and the left ventriculogram. The primary end point is an outcomes composite score based on a 3-tiered Finkelstein-Schoenfeld (FS) hierarchical analysis, which will be conducted at 12 months post-treatment. Secondary end points include non-inferiority and superiority survival rates, occurrence of major adverse cardiac events (MACE), time to first MACE after baseline measurements, and mean change in score on the MLHFQ.
The trial will take place across 40 locations in the United States and Canada, and the estimated primary completion date is December 2023. In addition to the positive review, the DSMB also recommended that BioCardia implement an adaptive statistical analysis plan capable of early readout for treatment efficacy into their clinical trial plans.
“The DSMB’s support for implementing an adaptive design based on the data in hand is encouraging and we will drive toward implementing an adaptive statistical analysis plan in the coming months with the involvement of the study’s executive steering committee and the FDA,” Altman added to the statement.1 “An adaptive statistical analysis plan will enable the study to be powered based on the ongoing trial data and may allow earlier study readout.”