40 Pediatric Patients Enrolled Over 10 Months at 15 ACTION Sites.
CINCINNATI — A leading pediatric heart failure organization ACTION, the Advanced Cardiac Therapies Improving Outcomes Network (Cincinnati, OH)), in conjunction with Berlin Heart, Inc. (The Woodlands, TX), launched the first prospective FDA regulated device trial using a pediatric ventricular assist device (VAD) registry in November 2022. Enrollment in the trial has been completed as of August 11, 2023, just 10 months after launch.
The Berlin Heart EXCOR® Active Driver Trial is sponsored by Berlin Heart, Inc. ACTION is serving as the clinical research organization (CRO) with the ACTION registry serving as the repository of the clinical trial data.
The goal of the trial is to gain FDA approval for the new EXCOR® Active driving unit, which will replace the approved IKUS driving unit that powers the commercially available EXCOR® Pediatric Ventricular Assist Device system. Of the 60+ children’s hospitals who are member sites in ACTION, 15 were chosen to participate in the trial.
Historically, there has been a paucity of pediatric device and drug trials due to cost, patient volume and variability in heart failure etiology. ACTION is collaborating with industry, patients and families and regulatory agencies to develop novel ways to bring innovation to the bedside and improve the treatment and outcomes for the pediatric population with heart disease.
The trial will allow young heart failure patients implanted with a Berlin Heart VAD to rehabilitate and become stronger while awaiting heart transplantation. The currently approved IKUS driver is large, heavy and cumbersome. The new EXCOR® Active driver is smaller and allows patients much greater mobility and quality of life.
“We are thrilled to partner with Berlin Heart on this important study,” said Angela Lorts, MD, MBA and Co-Executive Director of ACTION. “We are using the ACTION registry data as ‘real world data’ for both prospective clinical trials and to apply for expanded FDA indications for pediatrics. This study and others will continue our important work in making a difference to improve outcomes for patients with heart failure, especially children.”
Now that enrollment has been completed, ACTION will finish adjudicating adverse events and perform data audits once all patients have met the appropriate trial endpoint. Data will be compiled and submitted to the FDA, in conjunction with the sponsor, Berlin Heart, Inc. While the FDA reviews the data, study sites will have an ongoing continued access protocol (CAP) to use the EXCOR® Active.
ACTION (the Advanced Cardiac Therapies Improving Outcomes Network) is an organization that unites and connects a global healthcare community to improve outcomes for patients with heart failure, especially children. Since 2017, ACTION has continued to grow and engage an international community by uniting key stakeholders: patients, families, clinicians, researchers, payors and industry.
Based in Cincinnati, OH, ACTION uses a quality improvement and research-based approach. ACTION works in collaboration with network sites and network members, sharing data, finding better solutions and discovering new innovations. Currently, there are 60+ network sites and 1,185 network members. Learn more at actionlearningnetwork.org.