Multicenter Trial of ECMO in Children With Severe Cardiac Failure Using the Cardiohelp System

Multicenter Trial to Evaluate the Safety and Effectiveness of the Cardiohelp System for up to 30 Days of Support in Children With Severe Cardiac Failure

The goal of this multicenter observational clinical trial is to evaluate the safety and effectiveness of the Cardiohelp System for VA-ECMO in children with cardiac failure. The main question[s] it aims to answer are:

• What is the safety and effectiveness of the Cardiohelp device for pediatric ECMO?
• What are the optimal performance specifications of the Cardiohelp device in children? Should the Cardiohelp device be FDA-cleared for children?

Children who are receiving the Cardiohelp device will be approached and consented to participate if interested. Participants will undergo a standardized data collection to estimate survival to 30 days and the prevalence of serious adverse events like stroke, bleeding, and hemolysis.

Outcomes will be compared to performance goals (PG) derived from the ECMO literature.

Funding Source -- FDA OOPD (Office of Orphan Product Development)

Study Overview

• Status: Not yet recruiting
• Conditions: Heart Failure; Cardiomyopathies; Congenital Heart Disease; Cardiogenic Shock
• Intervention / Treatment: Device: Cardiohelp device (VA-ECMO)

Detailed Description

This is a multicenter clinical trial to evaluate the safety and effectiveness of the Cardiohelp System for up to 30 days of support in children with severe cardiac failure. Despite more than 50 years of use and significant mortality, no standalone ECMO device has ever been FDA-cleared for ECMO. All ECMO use in children is off-label and this may explain why there is significant center-based variation in ECMO use that is not evidence driven. This study seeks to change that by collecting systematic safety and effectiveness data on a single standardized ECMO circuit (Cardiohelp Device). The goal is to provide the data necessary to secure FDA clearance of the first pediatric ECMO system, to determine the optimal performance range of the Cardiohelp device in infants and children, and to provide the FDA with a predicate device to support FDA clearance of other ECMO systems.

• Study Type: Observational
• Enrollment (Estimated): 50

Contacts and Locations

• Study Contact: Name: Selena Gonzales, MPH; Phone Number: 650-723-7913; Email: selena.gonzales@stanford.edu
• Study Contact Backup: Name: Christopher SD Almond, MD, MPH; Phone Number: 6507237913; Email: calmond@stanford.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

All children between 0 and 16 years of age supported with the VA-ECMO using the Cardiohelp system for primary myocardial failure where all other treatment options have failed, and clinical deterioration is expected, or risk of death is imminent.

Description

Inclusion Criteria:

1. Age 0 to 16 years of age
2. Body weight 3 to 80 kilograms
3. VA-ECMO use for primary cardiac failure using the Cardiohelp system.
4. First ECMO run during any hospitalization
5. Written informed consent from the parent or legally authorized representative.

Exclusion Criteria:Children must not meet any of the following exclusion criteria within 48 hours prior to device implant:

1. Gestational Age <37 weeks
2. Bleeding or coagulopathy that is a contraindication to anticoagulation
3. Irreversible renal, hepatic or lung failure
4. Stroke or uncertain neurological status within the past 30 days
5. Severely malnourished
6. Use of an ECMO system other than the Cardiohelp
7. VV-ECMO or ECMO for primary respiratory failure
8. Goals of patient to focus on comfort measures only.
9. Failure to separate from cardiopulmonary bypass

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Stroke-free survival to 30 days, recovery, ventricular assist device implant or transplant.	Survival in the absence of symptomatic stroke to 30 days or decannulation for recovery, transplant or VAD implant	30 days
Symptomatic stroke	symptomatic stroke	30 days
Severe hemolysis	Severe hemolysis	30 days
Circuit change due to thrombus	ECMO circuit change due to thrombus	30 days
Major device malfunction	major device malfunction of the ECMO circuit	30 days
Renal failure	renal failure	30 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of all serious adverse events	Incidence rate of all serious adverse events (per day of support)	30 days

Collaborators and Investigators

• Sponsor: Stanford University
• Collaborators: Boston Children's Hospital; Duke University
• Investigators: Principal Investigator: Lynn Sleeper, ScD, Boston Children's Hospital; Principal Investigator: Caroline Ozment, MD, Duke University

Study record dates

Study Major Dates

• Study Start (Estimated): July 1, 2024
• Primary Completion (Estimated): September 30, 2026
• Study Completion (Estimated): September 30, 2027

Study Registration Dates

• First Submitted: October 6, 2023
• First Submitted That Met QC Criteria: October 6, 2023
• First Posted (Actual): October 12, 2023

Study Record Updates

• Last Update Posted (Actual): April 1, 2024
• Last Update Submitted That Met QC Criteria: March 29, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Ischemia; Pathologic Processes; Necrosis; Myocardial Ischemia; Cardiovascular Diseases; Vascular Diseases; Congenital Abnormalities; Shock; Myocardial Infarction; Infarction; Cardiovascular Abnormalities; Heart Failure; Heart Diseases; Cardiomyopathies; Heart Defects, Congenital; Shock, Cardiogenic
• Other Study ID Numbers: 72467; 1R01FD007833-01A1 (U.S. FDA Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes