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

Study Overview
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Brief Summary
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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
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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.

Official Title
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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]

Conditions
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Heart Failure, Cardiomyopathies

Intervention / Treatment
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* Device: EXCOR Pediatric


Participation Criteria
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Eligibility Criteria
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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 OR 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

Ages Eligible for Study
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Minimum Age: 1 Day
Maximum Age: 16 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: EXCOR Pediatric<br>Implantation of the EXCOR Pediatric Ventricular Assist Device | Device: EXCOR Pediatric<br>* Extracorporeal Ventricular Assist Device<br>* Other names: EXCOR® Pediatric VAD;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The Safety of EXCOR® Pediatric Was Evaluated by Summarizing the Serious Adverse Event Rate Experienced While the Subject Was Supported on the Device. | 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. | 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. | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Ventricular Assist Device, Pediatric Ventricular Assist Device