Assess Safety and Probable Benefit of the EXCOR® Pediatric Ventricular Assist Device

Not Applicable

Completed

Brief Summary: The purpose of the study is to determine whether use of the Berlin Heart EXCOR® Pediatric Ventricular Assist Device for bridge-to-transplant is associated with a reasonable assurance of safety and probable benefit such that the EXCOR® Pediatric merits approval by the Food and Drug Administration under a Humanitarian Device Exception (HDE).

Detailed Description: Berlin Heart Inc. is sponsoring a prospective, multi-center, single arm study to access the safety and probable benefit of the Berlin Heart EXCOR® Pediatric Ventricular Assist Device \[EXCOR® Pediatric\]. The study will compare the EXCOR® Pediatric with a historical control population supported with extra-corporeal membrane oxygenation (ECMO) as a bridge to cardiac transplantation in children.

Recruitment & Eligibility

Inclusion Criteria

Severe New York Heart Association (NYHA) Functional Class IV (or Ross Functional Class IV for patients <= 6 years) heart failure refractory to optimal medical therapy, and has met at least one of the following criteria:

Interagency Registry for Mechanically Assisted Circulatory Support (INTERMACS) profile status 1 or 1a, i.e. critical cardiogenic shock (low BP unresponsive to support), compromised end organ perfusion, < 24 hour survival without mechanical support; may be due to Ventricular Tachycardia (VT)/Ventricular Fibrillation (VF) (1A) OR
INTERMACS profile status or 2A (i.e progressive decline): not in imminent danger, but worsening despite optimal inotropic therapy; may be due to VT/VF (2A) AND at least one of the following criteria: Decline in renal functions, Decline in nutritional status, Decline in mobility/ambulation

Support with extra-corporeal membrane oxygenation (ECMO) or other mechanical circulatory support device OR
Unable to separate from cardiopulmonary bypass
- Listed (UNOS status 1A or equivalent) for cardiac transplantation
- Two-ventricle circulation, including cardiomypathy, repaired structural heart disease or acquired heart disease
- Age 0 to 16 years
- Weight >= 3 kg and <= 60 kg
- Legal guardian (and patient if age-appropriate) understands the nature of the procedure, is willing to comply with associated follow-up evaluations, and provide written informed consent and assent prior to the procedure

Exclusion Criteria

Support on ECMO for >= 10 days
Cardiopulmonary resuscitation (CPR) duration >= 30 minutes within 48 hours of implantation
Body weight < 3.0 kg or Body Surface Area > 1.5 m2
Presence of mechanical aortic valve
Unfavorable or technically-challenging cardiac anatomy including single ventricle lesions, complex heterotaxy, and restrictive cardiomyopathy
Evidence of intrinsic hepatic disease
Evidence of intrinsic renal disease
Evidence of intrinsic pulmonary disease
Hemodialysis or peritoneal dialysis (not including dialysis or continuous veno-venous hemofiltration (CVVH) for fluid removal)
Moderate or severe aortic and/or pulmonic valve insufficiency
Apical Ventricular Septal Defects (VSD) or other compromise that is technically challenging to repair at implant
Documented heparin induced thrombocytopenia (HIT)
Documented coagulopathy
Hematologic disorder
Active Infection within 48 hours of implant (positive blood culture or White Blood Cell Count >15,000 and fever > 38 degrees C)
Documented Human immunodeficiency virus (HIV) infection or acquired immunodeficiency syndrome (AIDS)
Evidence of recent life-limiting malignant disease
Stroke within 30 days prior to enrollment
Psychiatric or behavioral disease
Currently participating in another Investigational Device Exemption (IDE) or Investigational New Drug Application (IND) trial
Patient is pregnant or nursing

Arm && Interventions

Group	Intervention	Description
EXCOR Pediatric	EXCOR Pediatric	Implantation of the EXCOR Pediatric Ventricular Assist Device

Primary Outcome Measures

Name	Time	Method
The Safety of EXCOR® Pediatric Was Evaluated by Summarizing the Serious Adverse Event Rate Experienced While the Subject Was Supported on the Device.	Participants were followed while on device support, an average of 58 days	The serious adverse event rate was calculated by totaling the number of serious adverse events all subjects experienced during device support (from implant to explant, an average of 58 days) divided by the total support time (in days) for all subjects. The serious adverse event rates were calculated separately for each primary study cohort.
Efficacy of the EXCOR® Pediatric Was Estimated by Showing Survival of All Participants Who Were Supported by the Device.	Participants were followed while on device support, an average of 58 days	Efficacy of the EXCOR® Pediatric was estimated by showing survival of all participants who were supported by the device.

Secondary Outcome Measures

Name	Time	Method

Locations (17): Arkansas Children's Hospital
🇺🇸
Little Rock, Arkansas, United States
Children's Healthcare of Atlanta
🇺🇸
Atlanta, Georgia, United States
St. Louis Children's Hospital
🇺🇸
St. Louis, Missouri, United States
Lucille Packard Children's Hospital / Stanford University
🇺🇸
Palo Alto, California, United States
University of Alabama at Birmingham
🇺🇸
Birmingham, Alabama, United States
The Children's Hospital Denver
🇺🇸
Denver, Colorado, United States
C.S. Mott Children's Hospital
🇺🇸
Ann Arbor, Michigan, United States
The Mt. Sinai Hospital
🇺🇸
New York, New York, United States
Texas Children's Hospital
🇺🇸
Houston, Texas, United States
Stollery Children's Hospital
🇨🇦
Edmonton, Alberta, Canada
Children's Hospital of Pittsburgh
🇺🇸
Pittsburgh, Pennsylvania, United States
Riley Hospital for Children
🇺🇸
Indianapolis, Indiana, United States
University of Minnesota - Fairview
🇺🇸
Minneapolis, Minnesota, United States
The Hospital for Sick Children
🇨🇦
Toronto, Ontario, Canada
Children's Hospital of Wisconsin
🇺🇸
Milwaukee, Wisconsin, United States
Children's Hospital of Boston
🇺🇸
Boston, Massachusetts, United States
Seattle Children's Hospital
🇺🇸
Seattle, Washington, United States