Trial of new driver for pediatric VAD could transform research for children with HF
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The first prospective FDA-regulated device trial using a pediatric ventricular assist device registry was launched in November. It could transform how research is conducted in children with advanced heart failure.
Because of small patient populations and lack of significant industry support, prospective data on treatments for children with advanced HF are few and far between. To address that, the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) was formed in 2017. ACTION connects stakeholders such as patients, families, clinicians, researchers, payors and industry and has developed a quality improvement and research-based network, including a registry. The network now includes 60 sites with more than 1,000 members.
ACTION is now playing a major role in a prospective trial, the Berlin Heart ACTIVE Driver Trial, by serving as the clinical research organization and having its registry serve as the repository for the data. The trial is of a next-generation driver (Active) for a pediatric VAD (Excor, Berlin Heart) that is smaller than the previous-generation driver (Ikus) and enables children to be more mobile and aims to allow children to leave the hospital while they await a heart transplant.
On Aug. 15, 2023, ACTION announced that the enrollment goal of 40 children was met 10 months after launch.
Healio spoke with Angela Lorts, MD, MBA, co-founder and co-executive director of ACTION, professor of pediatrics at the University of Cincinnati and director of the ventricular assist device program at Cincinnati Children’s Hospital, about the challenges of conducting trials in children with HF, the role of ACTION in the trial, what the researchers hope to learn from the trial and how the trial might influence the care of and research for children with advanced heart disease.
Healio: What have traditionally been the barriers to conducting prospective trials in children with advanced HF?
Lorts: There are many barriers to doing device and medication trials in children. The number of patients at each site is much smaller than the number of adult heart failure patients, making it difficult to do prospective device and drug trials. There is also a lack of economic incentive for device and pharmaceutical companies due to the relatively small number of patients that may benefit from the therapies when compared to the adult population. Finally, there is a lack of awareness that heart failure is a problem in children due to the diverse etiologies of the disease in this population.
Healio: How does the novel driving unit work and how might it impact the performance of the Excor VAD compared with the current unit?
Lorts: The Berlin Heart Excor is the only durable ventricular assist device approved for small children. The current driving unit, the Ikus, is very large and has an extremely short battery life, hindering rehabilitation and therefore quality of life of these children. The children on the Berlin Heart Excor must remain inpatient while they are waiting for a transplant, and they will often be in the hospital for 6 months or longer.
The Berlin Heart Active is the new driving unit that is being studied through the ACTION learning network, and once approved it will replace the Ikus and allow for more mobility and rehabilitation of the children. The first step is to study the Active driving unit for inpatient care, but the goal will be to allow the patients to go home on their ventricular assist device while they await a suitable organ.
Healio: What is the role of ACTION and the ACTION Registry in this trial?
Lorts: ACTION is a learning network that was founded in 2017 and includes all stakeholders: patients and families, providers, regulatory agencies and industry. One initiative of ACTION includes a registry that has been designed to collect data for prospective trials. The adverse events collected in the registry are adjudicated, and the data are audited. Using this data and structure for device and drug trials will improve efficiency of the trials. More sites can be included in the trial since they are already participating in ACTION and many of the regulatory and legal aspects have been addressed. The data collection is efficient and prevents patients’ data from being entered into multiple databases. In addition, the providers and industry will be working together to design the trials and ensure they are feasible and that the results of the trial will improve the outcomes of children with heart disease.
Healio: What are you hoping to learn from this trial?
Lorts: The ACTIVE Driver Trial is allowing us to bring a new device component to children in the United States. We are also learning how to design and execute a trial using a learning network. We hope to learn that the driver is safe and effective and to understand the usability. After the trial is complete, we aim to design the next phase, which will allow these children to go home on a VAD. Once we have an established process for prospective trials, we will be able to study medications and devices for children quickly and at a much lower cost.
Healio: When do you expect the trial to be completed?
Lorts: The ACTIVE Driver Trial is halfway through enrollment. We have enrolled 20 of the 40 expected patients. We expect the enrollment to be complete by the end of the summer.
Healio: If the trial is successful, how might that impact the care of children with advanced HF?
Lorts: If the trial is successful, we expect that the driver will replace the old driver and children at all centers will be able to be more mobile and have a better quality of life while waiting for transplant. The success of the trial will also alter how we do trials for children with heart failure in the future. If we can be more efficient and effective, by using learning networks such as ACTION, we may see more device and pharmaceutical companies willing to embark on advancing therapies for children with heart failure.
Healio: What kind of research initiatives for children with heart disease might we see in the future?
Lorts: We will see new devices for children with heart failure in the future. There are many ventricular assist devices that appear promising as therapies for children, but they have not made it to the bedside due to the cost of clinical device trials. As a community, we will need to be creative to make the next-generation devices small enough to save the lives of all children that need cardiac support.
Healio: Is there anything else you would like to mention?
Lorts: Currently, there are 60 sites, mostly in North America, participating in ACTION. We are launching a global initiative that is aiming to enroll 20 more sites from at least 10 countries. We will continue to advance if we learn from each other and include perspectives across the world. In addition, our data will be richer, and we can help get devices and medications to children all over the world.
To learn more or to get involved, please reach out to info@actionlearningnetwork.org or go to actionlearningnetwork.org. To read more about ACTION’s outcomes and research, please visit ACTION’s publications page.
Editor’s Note: This article was updated on Aug. 16, 2023 when the trial’s enrollment goal was met.
Reference:
ACTION. https://www.actionlearningnetwork.org/news/first-pediatric-cardiac-device-ide-trial. Published Aug. 15, 2023. Accessed Aug. 16, 2023.
For more information:
Angela Lorts, MD, MBA, can be reached at info@actionlearningnetwork.org.
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