Pediatric circulatory support contractors' meeting: Report of the clinical trials working group

Based on the broad discussions of the Clinical Working Group, the following conclusions and recommendations were developed. (1) Pediatric patients requiring mechanical circulatory support represent a highly hetero-geneous population and this argues in favor of evaluation of multiple devices. (2) Randomized controlled clinical trials are unlikely to be achieved for investigational devices. (3) Comparator groups should be defined, utilized, and shared among studies whenever feasible. (4) Adult devices modified for pediatric use require studies in pediatric patients prior to approval through any regulatory pathway. (5) All studies should use uniform pediatric appropriate definitions. (6) IDE studies should use as homogeneous populations as possible and these should reflect the common populations to be supported during commercialization. (7) More challenging (and heterogeneous) populations can be studied in parallel with the primary cohort of IDE studies with comparable data collection, using a prospective registry design. (8) A consortium approach is suggested for preand post-approval clinical studies. This would comprise a network of centers that would perform studies within the framework of a collaborative agreement coordinated through the funding agency (the most obvious candidate being NHLBI). (9) INTERMACS represents the most advanced tool for data collection, and has already been modified for pediatric usage. It is recommended that funding be sought to modify INTERMACS to serve as the main repository for all pediatric VAD studies, including IDE studies, data analysis of emergency and "compassionate" usage (for patients outside of IDE primary cohort) and postapproval studies. Both short and long-term device usage should be studied within this mechanism. (10) Post-approval studies are essential for VADs designed for pediatric use given the small number of patients that will be enrolled in pre-market IDE clinical studies. The objective of these studies would be to evaluate device performance and potential device-related problems in a broader population using pre-defined AEs over an extended period of time, after pre-market establishment of reasonable safety and effectiveness or probable benefit. Note: It is recognized that there are other forms of circulatory support than VADs, and that oxygenation is a frequent requirement during circulatory failure in children. However, most of the attendees have focused their efforts on the development of pediatric circulatory support with VADs. Thus, most discussion focused on this form of support. This does not negate the importance of study of other forms of circulatory support in children, nor does it imply that these devices could not be included in the above-mentioned network design.