Clinical trial NCT00583661: Assess Safety and Probable Benefit of the EXCOR® Pediatric Ventricular Assist Device
Official title: 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]
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Heart Failure
Cardiomyopathies
Keywords: Ventricular Assist Device
Pediatric Ventricular Assist Device
Lead sponsor: Berlin Heart, Inc
Sponsor class: INDUSTRY
First posted: 2007-12-31
Enrollment: 48
Sex: ALL
Minimum age (years): 1
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: The Safety of EXCOR® Pediatric Was Evaluated by Summarizing the Serious Adverse Event Rate Experienced While the Subject Was Supported on the Device.
timeFrame: Participants were followed while on device support, an average of 58 days
measure: Efficacy of the EXCOR® Pediatric Was Estimated by Showing Survival of All Participants Who Were Supported by the Device.
timeFrame: Participants were followed while on device support, an average of 58 days
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Palo Alto
country: United States
facility: Lucille Packard Children's Hospital / Stanford University
state: California
city: Denver
country: United States
facility: The Children's Hospital Denver
state: Colorado
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Boston
country: United States
facility: Children's Hospital of Boston
state: Massachusetts
city: Ann Arbor
country: United States
facility: C.S. Mott Children's Hospital
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota - Fairview
state: Minnesota
city: St. Louis
country: United States
facility: St. Louis Children's Hospital
state: Missouri
city: New York
country: United States
facility: The Mt. Sinai Hospital
state: New York
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh
state: Pennsylvania
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin
state: Wisconsin
city: Edmonton
country: Canada
facility: Stollery Children's Hospital
state: Alberta
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
Study officials:
affiliation: Texas Children's Hospital / Baylor College of Medicine
name: Charles D Fraser, MD, FACS
role: PRINCIPAL_INVESTIGATOR
References:
citation: Jordan LC, Ichord RN, Reinhartz O, Humpl T, Pruthi S, Tjossem C, Rosenthal DN. Neurological complications and outcomes in the Berlin Heart EXCOR(R) pediatric investigational device exemption trial. J Am Heart Assoc. 2015 Jan 22;4(1):e001429. doi: 10.1161/JAHA.114.001429.
citation: Fraser CD Jr, Jaquiss RD, Rosenthal DN, Humpl T, Canter CE, Blackstone EH, Naftel DC, Ichord RN, Bomgaars L, Tweddell JS, Massicotte MP, Turrentine MW, Cohen GA, Devaney EJ, Pearce FB, Carberry KE, Kroslowitz R, Almond CS; Berlin Heart Study Investigators. Prospective trial of a pediatric ventricular assist device. N Engl J Med. 2012 Aug 9;367(6):532-41. doi: 10.1056/NEJMoa1014164.
citation: Almond CS, Buchholz H, Massicotte P, Ichord R, Rosenthal DN, Uzark K, Jaquiss RD, Kroslowitz R, Kepler MB, Lobbestael A, Bellinger D, Blume ED, Fraser CD Jr, Bartlett RH, Thiagarajan R, Jenkins K. Berlin Heart EXCOR Pediatric ventricular assist device Investigational Device Exemption study: study design and rationale. Am Heart J. 2011 Sep;162(3):425-35.e6. doi: 10.1016/j.ahj.2011.05.026. Epub 2011 Jul 30.