BioCardia, Inc. [Nasdaq: BCDA] is thrilled to announce that the Data Safety Monitoring Board (DSMB) has completed their review of the Phase III CardiAMP® Cell Therapy Heart Failure Trial (NCT02438306) and recommended a pause while awaiting the one-year follow-up results. As a reminder, this trial was granted FDA Breakthrough Designation and is showing tremendous promise as a potential life-saving treatment for cardiovascular and pulmonary diseases.
The Data and Safety Monitoring Board (DSMB) has recommended a pause on enrolling new patients and conducting crossover treatments in light of an outcomes assessment of patients already participating in the one-year follow-up and those due to undergo their scheduled treatment. This assessment, which follows an analysis of the trial data and the primary FS composite endpoint assessment, as well as a supplemental analysis conducted on July 19th, was not initiated due to any emergent safety events.
The DSMB has advised that patients currently enrolled in the trial and completing their treatment will have their participation paused once their scheduled treatment has been completed, in order to review the intermediate study results. Thank you for your contributions to the trial, and stay tuned for further updates!
At this stage, the Data Safety Monitoring Board suggests that the blind should remain in place in order to guarantee the trustworthiness of the results not yet collected, and to ensure that the research can be picked up and continued without interruption once the one-year evaluation has been completed.
The Finkelstein Schoenfeld (FS) composite endpoint was used by the Data and Safety Monitoring Board (DSMB) for an interim review of 132 procedures involving 111 randomized patients. The FS composite endpoint considered factors such as survival, major adverse cardiac and cerebrovascular events, and six-minute walk distance at 12 months, with no imputation of data for patients yet to receive the one-year follow-up.
Other important endpoints such as New York Heart Association classification, quality of life assessments, and heart function assessment were not included in the pre-specified interim analysis.
Data from a blinded report to the Company, which groups together treated and control patients, reveals promising survival rates at follow up compared to similar large pivotal trials in patients with heart failure with reduced ejection fraction.
Furthermore, the data shows positive health outcomes for patients who had follow up appointments through all key dates up to twelve months, measured by the six-minute walk test, New York Heart Association system, Minnesota Living with Heart Failure questionnaire, and assessment of left ventricular ejection fraction. However, the data does not show the difference in outcomes between the treated and control group of patients.
The Dat Safety Monitoring Board (DSMB) is a panel of experts consisting of two cardiologists with experience in heart failure and interventional cardiology and a biostatistician. With patient safety and data integrity as top priorities, their mission is to ensure that any therapy included in a clinical trial is worth the potential risk and will reach the primary endpoint of the trial. Through thorough risk-benefit analyses, they evaluate the potential benefit versus risk of a therapy to ensure patient safety and the integrity of the data.
The DSMB initially reviewed an analysis of unblinded data and concluded that the trial was unlikely to meet its primary FS composite endpoint, though the criteria for termination had not been met; the slow rate of enrollment was also cited.
Subsequently, the DSMB was provided with statistical analysis conducted in accordance with the prespecified data review plan, health outcome analysis beyond the six-minute walk, and information on the increased enrollment rates across many clinical centers.
Consequently, the DSMB recommended that the Company pause enrollment of new patients and continue treating those already enrolled while preserving the data blind, so that, post-12-month outcome analysis for all enrolled patients, the trial may be continued.
BioCardia is delighted to be able to provide the requested analysis at the twelve-month follow-up for the Data Safety Monitoring Board. With no reported treatment-related safety issues and positive patient health outcomes in the trial, we are more motivated than ever to accelerate our CardiAMP Chronic Myocardial Ischemia trial and CardiALLO Heart Failure trial with our impressive clinical partners.
About the CardiAMP Cell Therapy Program
CardiAMP Cell Therapy has developed a revolutionary stem cell therapy for the treatment of heart failure that holds tremendous promise. Through a minimally-invasive, catheter-based procedure, the therapy utilizes a patient’s bone marrow cells to potentially stimulate the body’s natural healing response.
What sets this advanced therapy apart from other similar treatments is its three proprietary elements: pre-procedural cell analysis for patient selection, a high target dosage of cells, and a proprietary delivery system that has been shown to be safer and more successful in cell retention than other intramyocardial delivery systems.
The CardiAMP Cell Therapy Heart Failure Trial is currently underway and may potentially revolutionize treatments for heart failure. However, please note that it is still in its investigational phase and is limited by United States law for investigational use only.
About BioCardia®
BioCardia, a Sunnyvale, California based company, is developing revolutionary cell-based treatments for heart and lung illnesses. Their groundbreaking CardiAMP™ autologous and NK1R+ allogeneic cell therapies are giving hope to countless patients, with four product candidates currently in development.
In 2019, the FDA awarded their CardiAMP Cell Therapy Heart Failure Trial the Breakthrough Designation, and the program was additionally supported by the Maryland Stem Cell Research Fund and now has the added status of having CMS reimbursement.
Moreover, BioCardia is partnering with other biotherapeutic companies to lend their delivery systems and further the development of treatments for heart failure, chronic myocardial ischemia, and acute myocardial infarction.