Study of Organ Perfusion in Heart Transplantation in Children

A Prospective Randomized Single Blind Multicenter Phase II Study of Organ Perfusion With Custodiol-N Compared With Custodiol in Heart Transplantation in Children

The goal of this prospective randomized single blind multicenter phase II study is to compare organ perfusion with Custodiol-N and Custodiol in heart transplantation in children of all ages (birth to <18 years) being listed on the waiting list for heart transplantation. The main question it aims to answer is:

to compare the safety of Custodiol-N in heart transplantation in children in comparison to its precursor product Custodiol.

Participants will receive either a heart to be transplanted, either perfused with Custodiol-N or Custodiol to Researchers will compare the two solutions to see if the new solution Custodiol-N is safe in heart transplantation in children.

Study Overview

• Status: Active, not recruiting
• Conditions: Heart Transplantation
• Intervention / Treatment: Drug: Custodiol-N; Drug: Custodiol

Detailed Description

Orthotopic Heart transplantation (or cardiac transplantation) is a procedure performed in patients with end stage heart failure when other treatments have failed.

The surgical procedures for orthotopic heart transplantation are essentially the same in adult and paediatric patients . Due to anatomical variants in congenital heart disease the procurement procedure may be modified with regard to the extent of the Vena cava superior and the great vessels. In implantation most frequently the bicaval technique is used but the biatrial technique which is leaving the back wall of both atria in situ may also be employed.

A main difference between adult and paediatric heart transplantation (HTx) are the indications for transplantation: In adults the majority of patients undergo HTx as treatment of end stage heart failure due to cardiomyopathy or severe coronary artery disease when other treatments have failed. Infants younger than one year of age congenital heart disease (CHD) is the most common diagnosis representing more than every second case followed by dilated cardiomyopathy (DCM) in more than one third of cases. In older patients DCM is the most frequent underlying diagnosis. CHD may include patients who have undergone corrective surgery and in whom myocardial dysfunction develops later .

• Study Type: Interventional
• Enrollment (Estimated): 15
• Phase: Phase 2

Contacts and Locations

Study Locations

• Germany
  • Berlin, Germany, 13353
    • Deutsches Herzzentrum der Charite
  • Gießen, Germany, 35385
    • Universitätsklinikum Gießen (UKGM), Kinderherzzentrum, Klinikum für Kinderherzchirurgie
  • München, Germany, 81377
    • Klinikum der Universität München (LMU), Klinik und Poliklinik für Herzchirurgie, Chirurgische Klinik

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Age from birth to less than18 years
• Recipients awaiting their first transplant
• Ability of the patients and/ or their legal guardians to understand character and individual consequences of the clinical trial
• written informed consent of the patients and/ or their legal guardians (must be available before enrolment in the study)
• Patient listed on the waiting list for heart transplantation

Exclusion Criteria:

• Patients who have participated within 30 days or are still participating in any other interventional studies
• history of severe organic disease other than concerning the heart
• history/demonstration of HIV antibodies or AIDS
• multiorgan transplantation
• machine-perfused organ
• the explantation team is affiliated to another clinic than transplantation team
• Failing Fontan patients
• Patients who have been incarcerated or involuntarily institutionalized by court order or by the authorities
• Pregnancy and lactation

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Custodiol-N; organ will be perfused with Custodiol-N solution	Drug: Custodiol-N; heart will preserved in and will be treated with Custodiol-N solution; Other Names: Solution for organ preservation in transplantation
Active Comparator: Custodiol; organ will be perfused with Custodiol solution	Drug: Custodiol; heart will preserved in and will be treated with Custodiol solution; Other Names: Solution for organ preservation in transplantation

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
adverse event reporting	Safety assessment	up to 3 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
heart rate	vital parameters	up to day 7 after release of the aortic cross clamp
blood pressure (systolic (SBP) and diastolic (DBP))	vital parameters	up to day 7 after release of the aortic cross clamp
body temperature	vital parameters	up to day 7 after release of the aortic cross clamp
concentration of cardiac troponin	laboratory tests	up to day 7 after release of the aortic cross clamp
concentration of creatinine kinase (CK-MB)	laboratory tests	up to day 7 after release of the aortic cross clamp
(SBP (systolic blood pressure), DBP (diastolic blood pressure))	Haemodynamics	from termination of cardiopulmonary bypass until transfer to intensive care unit
Heart Rhythm (HR)	Haemodynamics	from termination of cardiopulmonary bypass until transfer to intensive care unit
Pulmonary artery pressure (PAP (if available))	Haemodynamics	from termination of cardiopulmonary bypass until transfer to intensive care unit
Cerebrovascular resistance (CVR (if available))	Haemodynamics	from termination of cardiopulmonary bypass until transfer to intensive care unit
Death	survival	up to 12 months
function of transplanted organ (heart)	Graft survival	up to 3 months
Readmission to intensive care unit (ICU)	return to intensive care unit	up to 3 months
Length of ICU stays	duration of stay in intensive care unit	up to 3 months
Catecholamine requirement	(yes/no)	up to day 7
Antihypertensives intake	(yes/no)	up to 12 months
Milrinone support	(yes/no)	up to day 7
Need for pacemaker therapy	(yes/no)	up to day 7
Left and right ventricular assist device (LVAD, RVAD)	Device therapy (each yes/no)	up to 3 months
Extracorporeal membrane oxygenation (ECMO)	Device therapy (each yes/no)	up to 3 months
Biventricular assist Device (BIVAD) or percutaneous LVAD	Device therapy (each yes/no)	up to 3 months
Ejection fraction	Echocardiographic markers of function and rejection	up to day 7
enddiastolic and endsystolic ventricle	Echocardiographic markers of function and rejection	up to day 7
Cardiac arrhythmias	occurrence, severity, type	up to day 7

Collaborators and Investigators

• Sponsor: Dr. F. Köhler Chemie GmbH
• Investigators: Principal Investigator: Christoph Knosalla, Prof. Dr., German Heart Institute

Study record dates

Study Major Dates

• Study Start (Actual): August 15, 2023
• Primary Completion (Estimated): April 1, 2025
• Study Completion (Estimated): December 1, 2025

Study Registration Dates

• First Submitted: August 21, 2023
• First Submitted That Met QC Criteria: September 8, 2023
• First Posted (Actual): September 11, 2023

Study Record Updates

• Last Update Posted (Actual): September 11, 2023
• Last Update Submitted That Met QC Criteria: September 8, 2023
• Last Verified: April 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pharmaceutical Solutions
• Other Study ID Numbers: CL-N-HTX-Paed-II/10/20

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: No