Non-ischaemic Heart Preservation Versus Standard Cold Storage in Human Heart Transplantation

NCT ID: NCT04066127

Last Updated: 2025-05-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

NA

Total Enrollment

66 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-07-01

Study Completion Date

2026-12-31

Brief Summary

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The overall aim of this study is to compare a new state-of-the-art ex-vivo organ preservation method with standard ischemic cold static storage of donor hearts in adult cardiac transplantation.

Standard heart preservation before transplantation consists of cold ischemic storage of the heart. Clinical studies have shown that the morbidity and mortality risk increases with the extension of the allograft ischemic time over four hours. For each additional hour the mortality risk increase with 25% the first year. This time constraint is costly and results in severe logistical problems, leading to loss of transplantable organs. The preliminary results from our safety study, where six patients transplanted with the new state-of-the-art ex-vivo organ preservation method, have shown promising results.

The study is a multicenter, prospective, open, blinded endpoint, randomized, controlled clinical trial. The primary end-point is survival free of acute cellular rejection (ACR) and retransplantation within 1-year post-transplant. ACR will be assessed blinded. The secondary end-points are ischemia/reperfusion injury, early graft dysfunction, and QoL.

Detailed Description

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SURVEY OF THE FIELD Ischemia and reperfusion (I/R) - elicited tissue injury contributes to morbidity and mortality. In organ transplantation, it is a major challenge. The imbalance in metabolic supply and demand within the ischemic organ results in tissue hypoxia and microvascular dysfunction. The following reperfusion enhances the activation of innate and adaptive immune responses resulting in a cell death program.

A damaged endothelium increases the risk of allograft rejection within 1-year after transplantation and a drug treated rejection increases the risk of chronicle rejection and five-year mortality. An improved preservation of the endothelium of the coronary arteries is therefore instrumental to achieve an improved short- and long-term outcome for the transplanted patients.

Standard heart preservation before transplantation consists of cold ischemic storage of the heart. Clinical studies have shown that the mortality risk increases sharply with extension of the allograft ischemic time over four hours. For each additional hour, the mortality risk increases with 25% the first year. Different myocardial cardioplegic preservation solutions and ex-vivo perfusion machines have been developed. Despite some promising experimental results, no consistent differences in outcome have been found. Our first-in-man pilot study, including 6 NIHP cases, show the device's feasibility and provide the first pieces of evidence that machines improves outcome of heart transplantation. However, as these results are based on a non-randomized study design with a few patients, a randomized study is needed to ensure the effect.

PURPOSE AND AIMS The purpose of this study is to compare a new state-of-the-art non ischemic heart preservation (NIHP) method, on heart allograft function, rejection episodes, and quality of life, with the standard ischemic cold static storage (SCS) method of donor hearts in adult heart transplantation.

STUDY DESIGN The study is a multicentre, prospective, randomized, open, blinded endpoint, controlled clinical trial. The study will randomly assign eligible patients to be transplanted with a donor heart preserved with either a new ex-vivo perfusion method (NIHP) or the standard cold static storage (SCS). The study will be performed at Skane University Hospital, Karolinska University Hospital, Linköping University Hospital, and Uppsala University Hospital, which cover two-thirds of the population in Sweden. Patients listed at these centres will be transplanted at Skane University Hospital and then returned to their centre for post-transplant care. The data collection, statistical analysis, and presentation of results will be done according to the CONSORT criteria. The main outcomes will be reported on intention to treat basis.

PICO Population: Adult patients with end-stage heart failure eligible for heart transplantation.

Intervention: Recipient transplanted with a donor heart preserved using NIHP. Control: Recipient transplanted with a donor heart preserved using SCS. Outcome: Survival, rejection, graft function, and quality-of-life.

STATISTICAL ANALYS PLAN Populations for analysis The full analysis set will consist of all randomized patients. Following the intent-to-treat principle, patients will be analyzed according to the preservation methods (NIHP and SCS, respectively) to which they were assigned at randomization. Efficacy variables will be analyzed based on all randomized patients as the primary population.

Patient demographics/other baseline characteristics Baseline value is defined as the last non-missing assessment prior to the transplantation. Summary statistics will be provided by treatment group for demographics and baseline characteristics, including age, sex, weight, height, body mass index (BMI), diagnosis, prior hospitalization, need for inotropic support pre-transplant and/or ECMO, CK-MB, TnI, proBNP, and vital signs. Continuous variables will be summarized using n, mean, standard deviation, median, minimum, and maximum. Categorical variables will be summarized using frequency and percentage. The difference between treatment groups will be compared using the Chi-square test for categorical variables or using Mann-Whitney U-test for continuous variables. The p-values will be provided for descriptive purposes and will not be considered to define any formal basis for determining factors to be included in statistical models.

Analysis of the primary efficacy variable and outcomes The primary objective of this study is to evaluate the survival free of allograft rejection and re-transplantation after heart transplantation comparing two preservation methods (NIHP versus SCS).

Time-to-event variables (primary efficacy variable) will be analyzed using the Cox proportional hazard model. The estimated hazards ratio and the corresponding two-sided 95% confidence interval will be provided. A supplemental log-rank test will be performed. Survival function for each treatment group will be estimated by Kaplan-Meier method and the Kaplan-Meier curves will be presented. The frequency and percentage of patients who reach the primary composite endpoint will be provided by treatment group for the population.

Analysis of secondary efficacy, exploratory, and safety variables and outcomes The secondary objects of this study are to test if the new preservation technique (NIHP) is superior to standard preservation technique (SCS) in I/R injury (cTnI and CK-MB), immediate graft function, QoL and severe adverse events. Furthermore we will analyse the postoperative renal function (minimum creatinine clearance, need for CRRT), postoperative liver function (ASAT, ALAT), postoperative pulmonary function (hours in ventilator), SAE, time to no improvement in physical activity, resource utilizations (length of stay, and number of visits to professional healthcare).

Continues variables (cTnI, CK-MB, inotrope score, minimum creatinine clearance, hours of ventilator, physical activity (calorie use), length of stay, and number of visits to professional healthcare, EQ-5D index and EQ VAS) will be analysed using Mann-Witney U-test. Additionally, the baseline value of the corresponding variable may be included as a covariate if appropriate. The estimated treatment effect and the corresponding two-sided 95% confidence interval will be provided.

Binary variables (primary graft dysfunction, LVEF\<40%, RVEF\<40%, severe adverse events) will be analysed using logistic regression. Additionally, the baseline value of the corresponding variable may be included as a covariate if appropriate. The estimated treatment effect and the corresponding two-sided 95% confidence interval will be provided. EQ-5D descriptive system will be analysed by using χ2-test and Fisher's exact test.

SAFETY The assessment of safety will be based primarily on the frequency of adverse events and laboratory abnormalities. Other safety data will be summarized as appropriate. Laboratory data will be summarized by presenting shift tables using extended normal ranges (baseline to most extreme post-baseline value), by presenting summary statistics of raw data and change from baseline values (mean, medians, standard deviations, ranges). An interim assessment of the initial 10 patients in the intervention group will be performed when all of them has passed 30-days post-transplant or expired.

Conditions

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Transplant; Failure, Heart

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

OTHER

Blinding Strategy

SINGLE

Outcome Assessors
Acute cellular rejection assessment will be performed by a blinded access

Study Groups

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Non-ischemic heart preservation (NIHP)

Non-ischemic hypothermic perfusion (NIHP): The donor heart is preserved using a portable heart-lung machine. The device perfuse the heart continuously with a a new preservation solution at a temperature of 8°C.

Group Type EXPERIMENTAL

Non-ischemic heart preservation (NIHP)

Intervention Type DEVICE

The device is a miniaturized and fully automated heart-lung machine, housed in a portable device (weight 32 kg), that enables transportation between hospitals. The reservoir is filled with 2.5 liters of the perfusion solution plus approximately 500 mL compatible washed and leucocyte-filtered red blood cells from the hospital blood bank. The NIHP system software is adjusted to maintain a mean blood pressure of 20-25 mmHg in the aortic root, giving a coronary flow between 150 mL/min and 250 mL/min.

Standard cold storage (SCS)

Standard cold static storage (SCS): The donor heart is preserved using a standard crystalloid cardioplegia. The heart is in then storage i a transport box containing ice to keep the temperature around 4-8°C. The device does not perfuse the heart.

Group Type OTHER

Standard ischemic cold static storage (SCS)

Intervention Type DEVICE

The device is a cool box that enables transportation between hospitals. The heart is stored on ice slush in the box at a temperature of approximately 4-8 °C.

Interventions

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Non-ischemic heart preservation (NIHP)

The device is a miniaturized and fully automated heart-lung machine, housed in a portable device (weight 32 kg), that enables transportation between hospitals. The reservoir is filled with 2.5 liters of the perfusion solution plus approximately 500 mL compatible washed and leucocyte-filtered red blood cells from the hospital blood bank. The NIHP system software is adjusted to maintain a mean blood pressure of 20-25 mmHg in the aortic root, giving a coronary flow between 150 mL/min and 250 mL/min.

Intervention Type DEVICE

Standard ischemic cold static storage (SCS)

The device is a cool box that enables transportation between hospitals. The heart is stored on ice slush in the box at a temperature of approximately 4-8 °C.

Intervention Type DEVICE

Eligibility Criteria

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Inclusion Criteria

* Age ≥18 years
* Signed informed consent form
* Listed for heart transplantation


* Age ≤70 years
* Accepted as heart donor by the transplant team (research consent from the donor if required in country)

Exclusion Criteria

* Previous solid organ transplantation
* Grown-up congenital heart disease (GUCH)
* Kidney failure eGFR\<40, calculated by CDK-EPI Creatinine, or ultrafiltration or dialysis or rapidly deteriorating kidney function due to a diagnosed renal disease
* Coagulopathy due to known hepatic disease or heparin induced thrombocytopenia
* Ongoing septicemia defined as positive blood culture (including with a durable VAD)
* Incompatible blood group
* Not able to understand the information provided during the informed consent procedure
* Patients under pre-transplant desensitization protocol
* Short term mechanical support pre-transplant (ECMO)
* Patient diagnosed with Systemic Lupus Erythematous, sarcoidosis or amyloidosis
* Combined organ transplantation candidates
* Patient already consented for another transplant related intervention study


* Previous sternotomy
* DCD hearts
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Lund University

OTHER

Sponsor Role collaborator

Skane University Hospital

OTHER

Sponsor Role collaborator

Region Skane

OTHER

Sponsor Role lead

Responsible Party

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Johan Nilsson, MD, PhD

Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Johan Nilsson, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Region Skane

Locations

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Skane University Hospital

Lund, Skåne County, Sweden

Site Status

Countries

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Sweden

References

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Steen S, Paskevicius A, Liao Q, Sjoberg T. Safe orthotopic transplantation of hearts harvested 24 hours after brain death and preserved for 24 hours. Scand Cardiovasc J. 2016 Jun;50(3):193-200. doi: 10.3109/14017431.2016.1154598. Epub 2016 Apr 4.

Reference Type BACKGROUND
PMID: 26882241 (View on PubMed)

Nilsson J, Jernryd V, Qin G, Nozohoor S, Goncalves DC, Ragnarsson S, Paskevicius A, Johansson M, Warheim J, Hoglund P, Sjoberg T, Steen S. Non Ischemic Heart Preservation. J Heart Lung Transpl 2018;37:S13-S.

Reference Type BACKGROUND

Jernryd V, Metzsch C, Andersson B, Nilsson J. Organ Preservation and Reperfusion Influence on Outcome after Heart Transplantation. The Journal of Heart and Lung Transplantation 2016;35:S193-S.

Reference Type BACKGROUND

Nilsson J, Ohlsson M, Stehlik J, Lund L, Andersson B. Prediction of Primary Graft Dysfunction After Heart Transplantation. J Heart Lung Transpl 2015;34:S35-S.

Reference Type BACKGROUND

Qin G, Sjoberg T, Liao Q, Sun X, Steen S. Intact endothelial and contractile function of coronary artery after 8 hours of heart preservation. Scand Cardiovasc J. 2016 Oct-Dec;50(5-6):362-366. doi: 10.1080/14017431.2016.1213876. Epub 2016 Aug 3.

Reference Type BACKGROUND
PMID: 27420646 (View on PubMed)

Steen S. Preservation of the endothelium in cardiovascular surgery-some practical suggestions--a review. Scand Cardiovasc J. 2001 Oct;35(5):297-301. doi: 10.1080/140174301317116253. No abstract available.

Reference Type BACKGROUND
PMID: 11771819 (View on PubMed)

Nilsson J, Jernryd V, Qin G, Paskevicius A, Sjoberg T, Hoglund P, Steen S. Non Ischemic Heart Preservation - Results from the Safety Study. J Heart Lung Transpl 2019;38:S26-S.

Reference Type BACKGROUND

Steen S, Sjoberg T, Pierre L, Liao Q, Eriksson L, Algotsson L. Transplantation of lungs from a non-heart-beating donor. Lancet. 2001 Mar 17;357(9259):825-9. doi: 10.1016/S0140-6736(00)04195-7.

Reference Type BACKGROUND
PMID: 11265950 (View on PubMed)

Nilsson J, Jernryd V, Qin G, Paskevicius A, Metzsch C, Sjoberg T, Steen S. A nonrandomized open-label phase 2 trial of nonischemic heart preservation for human heart transplantation. Nat Commun. 2020 Jun 12;11(1):2976. doi: 10.1038/s41467-020-16782-9.

Reference Type BACKGROUND
PMID: 32532991 (View on PubMed)

Pigot H, Steen S, Nilsson J. Comparing non-ischaemic heart preservation (NIHP) with ischaemic static cold storage of donor hearts in adult cardiac transplantation: study protocol for a randomised controlled trial. BMJ Open. 2025 Jun 25;15(6):e100553. doi: 10.1136/bmjopen-2025-100553.

Reference Type DERIVED
PMID: 40562558 (View on PubMed)

Other Identifiers

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2018/941

Identifier Type: -

Identifier Source: org_study_id

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