TY - JOUR
T1 - Berlin Heart EXCOR Pediatric ventricular assist device Investigational Device Exemption study
T2 - Study design and rationale
AU - Almond, Christopher S.
AU - Buchholz, Holger
AU - Massicotte, Patricia
AU - Ichord, Rebecca
AU - Rosenthal, David N.
AU - Uzark, Karen
AU - Jaquiss, Robert D B
AU - Kroslowitz, Robert
AU - Kepler, Mary Beth
AU - Lobbestael, Aaron
AU - Bellinger, David
AU - Blume, Elizabeth D.
AU - Fraser, Charles D.
AU - Bartlett, Robert H.
AU - Thiagarajan, Ravi
AU - Jenkins, Kathy
N1 - Funding Information:
Support: This study is supported, in part, by a Food and Drug Administration grant from the Office of Orphan Product Development ( R01 FD003557 ). Dr Almond is supported by the same grant.
Copyright:
Copyright 2018 Elsevier B.V., All rights reserved.
PY - 2011/9
Y1 - 2011/9
N2 - Background: Currently, there are no Food and Drug Administration-approved devices available that can provide long-term mechanical circulatory support to smaller children with severe heart failure as a bridge to heart transplant (HT). In recent years, the Berlin Heart EXCOR Pediatric ventricular assist device (VAD) has emerged as a potential treatment option. Systematic data on the safety and efficacy of the EXCOR are limited. Methods: The Investigational Device Exemption (IDE) clinical study is designed to evaluate the safety and probable benefit of the EXCOR to support regulatory review of the device under the Humanitarian Device Exemption regulation. The study design and rationale are reviewed in light of the well-described challenges inherent in small population studies. Results: The Berlin Heart EXCOR IDE clinical study is a prospective, multicenter, single-arm, clinical cohort study. Children aged 0 to 16 years with severe heart failure (Interagency Registry for Mechanically Assisted Circulatory Support profile 1 or 2) due to 2-ventricle heart disease and actively listed for HT comprise the primary study cohort. The control population is a propensity-matched retrospective cohort of children supported with extracorporeal membrane oxygenation, the only bridge device available to smaller children before the EXCOR. The primary efficacy end point is survival to heart transplantation or recovery. The primary safety end point is the incidence of serious adverse events as defined by pediatric Interagency Registry for Mechanically Assisted Circulatory Support criteria. The study will enroll a total of 48 subjects in 2 cohorts based on body surface area (cohort 1 <0.7 m2, cohort 2 0.7-1.5 m2) and is powered to show safety superiority to a prespecified performance goal of 0.25 serious adverse events per day of support. Children ineligible for the primary cohort will still have access to the device in a third compassionate-use cohort where adverse event data will be collected for additional safety characterization of the device. Conclusion: The Berlin Heart IDE clinical study will be the first bridge-to-HT VAD study designed exclusively for children. It is anticipated that the study will provide important information on the safety and efficacy of the Berlin Heart EXCOR Pediatric in children while providing valuable lessons into the design and conduct of future VAD studies in children.
AB - Background: Currently, there are no Food and Drug Administration-approved devices available that can provide long-term mechanical circulatory support to smaller children with severe heart failure as a bridge to heart transplant (HT). In recent years, the Berlin Heart EXCOR Pediatric ventricular assist device (VAD) has emerged as a potential treatment option. Systematic data on the safety and efficacy of the EXCOR are limited. Methods: The Investigational Device Exemption (IDE) clinical study is designed to evaluate the safety and probable benefit of the EXCOR to support regulatory review of the device under the Humanitarian Device Exemption regulation. The study design and rationale are reviewed in light of the well-described challenges inherent in small population studies. Results: The Berlin Heart EXCOR IDE clinical study is a prospective, multicenter, single-arm, clinical cohort study. Children aged 0 to 16 years with severe heart failure (Interagency Registry for Mechanically Assisted Circulatory Support profile 1 or 2) due to 2-ventricle heart disease and actively listed for HT comprise the primary study cohort. The control population is a propensity-matched retrospective cohort of children supported with extracorporeal membrane oxygenation, the only bridge device available to smaller children before the EXCOR. The primary efficacy end point is survival to heart transplantation or recovery. The primary safety end point is the incidence of serious adverse events as defined by pediatric Interagency Registry for Mechanically Assisted Circulatory Support criteria. The study will enroll a total of 48 subjects in 2 cohorts based on body surface area (cohort 1 <0.7 m2, cohort 2 0.7-1.5 m2) and is powered to show safety superiority to a prespecified performance goal of 0.25 serious adverse events per day of support. Children ineligible for the primary cohort will still have access to the device in a third compassionate-use cohort where adverse event data will be collected for additional safety characterization of the device. Conclusion: The Berlin Heart IDE clinical study will be the first bridge-to-HT VAD study designed exclusively for children. It is anticipated that the study will provide important information on the safety and efficacy of the Berlin Heart EXCOR Pediatric in children while providing valuable lessons into the design and conduct of future VAD studies in children.
UR - http://www.scopus.com/inward/record.url?scp=80052283184&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=80052283184&partnerID=8YFLogxK
U2 - 10.1016/j.ahj.2011.05.026
DO - 10.1016/j.ahj.2011.05.026
M3 - Article
C2 - 21884857
AN - SCOPUS:80052283184
SN - 0002-8703
VL - 162
SP - 425-435.e6
JO - American heart journal
JF - American heart journal
IS - 3
ER -