BioCardia, a developer of cellular and cell-derived therapeutics for the treatment of cardiovascular and pulmonary diseases, has announced that the US Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) has completed review of the CardiAMP cell therapy system adaptive statistical analysis design supplement for the ongoing pivotal study to treat ischemic heart failure with reduced ejection fraction (HFrEF). The adaptive statistical analysis plan is on track to be implemented at the upcoming Data Safety Monitoring Board (DSMB) meeting, scheduled for July 12, 2023.
Under the adaptive statistical analysis plan, should the CardiAMP cell therapy for heart failure study be stopped early for anticipated efficacy at this, or any future DSMB review, study sites will continue to follow all enrolled (randomized) patients until all of them have reached their one-year follow-up visit, at which time the trial results would be unblinded. Enrolment would continue after any DSMB meeting that stops randomisation for anticipated efficacy, with all subsequently enrolled patients receiving the study treatment. The open-label patients receiving the study treatment are intended to increase the amount of data available to address safety considerations for the pre-market application and market approval for the CardiAMP cell therapy system by FDA CBER.
Additional details, including the minimum number of patients treated open-label and any additional follow-up testing required, are to be confirmed with the FDA. It is our expectation that the adaptive statistical analysis plan will not extend the duration of the trial past the follow-up of the last randomised patient enrolled. The adaptive statistical analysis plan is expected to have minimal impact on statistical power of the trial. The DSMB may stop the trial early for safety or futility at any time.
“There is now potential for the CardiAMP cell therapy for heart failure trial to be stopped early for efficacy which could enable patients to have earlier access to this FDA designated breakthrough cell therapy. The most important aspect of the ongoing discussion with the FDA is our mutual focus on what is needed for a marketing application based on this trial,” said Peter Altman, president and CEO of BioCardia. “The quality of the data in this actively enrolling clinical trial is due to our top-tier clinical teams and the many dedicated patients.”