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"A Privacy-protecting Environment for Child Transplants Health Related and Genomic Data Integration in the European Reference Network"

Not yet recruiting
Conditions
Transplant Complication
Kidney Transplant
Liver Transplant
Registration Number
NCT07194057
Lead Sponsor
Instituto de Investigación Hospital Universitario La Paz
Brief Summary

Protect\_Child\_101 is an observational study to be performed in children that have undergone a liver or renal transplant.

The aim of this study is to analyse small variations in the genetic material (DNA) of transplanted children. The investigators will also study a type of chemical 'marks' called methylations, which do not change the DNA itself, but can affect how it functions. These marks can influence how certain diseases develop or how the body responds to transplantation.

Specifically, investigators seek to discover:

* Whether there are genetic or epigenetic (methylation) alterations that may explain why some children develop serious diseases that require transplantation.

* If these alterations can help us predict possible complications after transplantation, such as organ rejection, infections, organ failure, cancer development.

Within this study, data from the child's medical history will be collected. The data to be collected are demographic data (gender, age, ethnicity), clinical data, personal and family history possibly related to his/her disease, course and evolution of the disease, and complementary and laboratory examinations collected from his/her clinical history.

The only non-routine tests to be performed will be the genomic and methylomic tests. Nevertheless, these determinations will be performed on samples obtained during the child's routine care. No extra intervention is planned as part of this study.

Samples and clinical data will be collected at different time points after transplantation. Schematically, collection is planned for months 0, 1, 3, 6, 12 and 24 post-transplant. In addition to these pre-established points, comprehensive data collection will be attempted when the child suffers a relevant clinical event, e.g. infection, treatment toxicity, organ rejection (post-transplant complication).

Detailed Description

Not available

Recruitment & Eligibility

Status
NOT_YET_RECRUITING
Sex
All
Target Recruitment
200
Inclusion Criteria
  • ● Paediatric patients (6 months to 18 years old) with liver or kidney transplant.

Both patients with de novo transplantation or in follow-up can be included in the study.

  • For the retrospective cohort, only patients within the first 5 years after transplantation will be included.
  • Patients and/or parents agreeing to participate in the study and provide consent for the obtention of clinical data and samples for genomic and methylomic analysis and the use of the information according to the protocol.
Exclusion Criteria
  • Patients that are not being followed up in the clinical site.
  • Subjects alternating between different clinical sites. Subjects/Tutors that don't understand the informed consent form.
  • Subject or their legally authorized representative does not sign the informed consent document.
  • Re-transplantation or AB0-incompatible transplantation.

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Epstein Barr InfectionFrom transplant until end of post-transplant follow-up period (up to 7years)

Number of Espstein Barr infections defined as \>3500 copies in PCR in peripheral blood

Cytomegalovirus infectionFrom transplant until end of post-transplant follow-up period (up to 7 years)

A) Primary CMV infection after transplant with or without CMV disease (\>1000 copies/ml in peripheral blood in patients with previous negative CMV serology) B) Secondary CMV infection after transplant (any PCR with CMV disease or CMV \>1000 copies/ml in asymptomatic patients)

BK virus infectionFrom transplant until end of post-transplant follow-up period (up to 7 years)

Positive BK viremia (define cut-off level) and/or histological evidence of BK nephropathy

CholangitisFrom transplant until end of post-transplant follow-up period

Worsening of liver function tests accompanied by an elevation in inflammatory markers, with or without a positive blood or bile culture.

Urinary Tract InfectionFrom transplant until end of post-transplant follow-up period (up to 7 years)

Positive urine cultures AND increased inflammation marker (e.g. CRP) or fever

SepsisFrom transplant until end of post-transplant follow-up period (up to 7 years)

SIRS in relation to infectious cause +/- positive blood cultures

Renal Calcineurin Inhibitors toxicityFrom transplant until end of post-transplant follow-up period (up to 7 years)

Histological evidence of kidney CNI-related kidney damage

Mycophenolate mofetil toxicityFrom transplant until end of post-transplant follow-up period (up to 7 years)

Evidence of myelosuppression during therapy without any other proven cause and/or Clinical/histological evidence of MMF-related enteropathy

mTOR inhibitor toxicityFrom transplant until end of post-transplant follow-up period (up to 7 years)

mTOR induced-proteinuria (occurrence of proteinuria after mTOR exposure with resolution after treatment suspension)

Thrombotic microangiopathyFrom transplant until end of post-transplant follow-up period (up to 7 years)

Ocurrence of no non immune-mediated hemolytic anemia and/or thrombocytopenia and/or hypertension and/or proteinuria with histological evidence of kidney TMA

Kidney rejection episodeFrom transplant until end of post-transplant follow-up period (up to 7 years)

Histological evidence based on Banff criteria

Liver rejection episodeFrom transplant until end of post-transplant follow-up period (up to 7 years)

Histological evidence based on Banff criteria

Chronic liver rejectionFrom transplant until end of post-transplant follow-up period (up to 7 years)

Histological evidence based on Banff criteria

Chronic kidney rejectionFrom transplant until end of post-transplant follow-up period (up to 7 years)

Histological evidence based on Banff criteria

Chronic renal failure after pLTxFrom transplant until end of post-transplant follow up period (up to 7 years)

Elevation of serum-creatinine for 3\>months

Chronic liver failure (graft chirrosis and fibrosis)From transplant until end of post-transplant follow up period (up to 7 years)

Ocurrence of portal hypertension diagnosis both clinical (ascites, splenomegaly, varices) and analytical (thrombocytopenia) presentation.

Secondary Outcome Measures
NameTimeMethod
Liver Primary non-functionFrom transplant to post-trasnplant follow-up period (up to 7 years)

Ocurrence of at least 2 of the following within 7 days post-transplant:

* Alanine aminotransferase (ALT) greater than or equal to 2,000 U/L

* INR greater than or equal to 10 mg/dl

* Acidosis (Defined as one of the following: a) Arterial PH less or equal to 7,30. b) Venous pH less than or equal to 7,25. c) Lactate greater than or equal to 4 mmol/L)

Kidney primary non-functionFrom transplant until end of post-transplant follow-up period (up to 7 years)

Persistence of dialysis status or eGFR \<15 ml/min/1.7 m2

Liver early allograft dysfunctionFrom transplant until end of post-transplant follow-up period (up to 7 years)

One of the following laboratory criteria within the 7 first days post transplant:

* Serum bilirubin ≥10 mg/dL (171 μmol/L) on day 7

* INR ≥1.6 on day 7

* AST or ALT \>2000 IU/L within the first 7 days.

Or, for 5 consecutive days after day 7:

* Bilirubin \> 10 mg/ dL

* INR \> 1.6

* Serum Urea \> 100 mg/dL

Delayed kidney Graft FunctionFrom transplant until end of post-transplant follow-up period (up to 7 years)

Need for dialysis within the first 7 days after kidney transplantation

Vascular ComplicationsFrom transplant until end of post-transplant follow-up period (Up to 7 years)

Hepatic artery thrombosis (HAT) or portal vein stenosis (may lead to ischemic injury and chronic dysfunction)

Biliary ComplicationsFrom transplant until end of post-transplant follow-up period (up to 7 years)

Biliary structures, leaks, or ischemic cholangiopathy due to vascular insufficiency can cause chronic dysfunction.

Urological complicationsFrom transplant until end of post-transplant follow-up period (up to 7 years)

Need of re-intervention due to post-surgical events

Post-transplant lymphoproliferative diseaseFrom transplant until end of post-transplant follow-up period (up to 7 years)

Radiological diagnosis of PTLD: heterogeneous extranodal masses, allograft involvement, and CNS or visceral lesions.

DiabetesFrom transplant until end of post-transplant follow-up period (up to 7 years)

The diagnosis is confirmed when one of the following criteria is met on two separate occasions:

Fasting plasma glucose (FPG): ≥126 mg/dL (7.0 mmol/L) after at least 8 hours fasting.

Oral glucose tolerance test (OGTT): 2-hour plasma glucose ≥200 mg/dL (11.1 mmol/L) after a 75 g oral glucose load.

Hemoglobin A1c (HbA1c): ≥6.5%, using a standardized assay.

Random plasma glucose: ≥200 mg/dL (11.1 mmol/L) in the presence of classic symptoms of hyperglycemia (polyuria, polydipsia, weight loss).

Posterior reversible encelopathy (PRES)From transplant until end of post-transplant follow-up period (up to 7 years)

Ocurrence of acute neurologic symptoms (headache, seizures, altered consciousness or visual disturbances) with typical neuroimaging (CT, MRI) findings (bilateral areas of white matter edema in the posterior cerebral hemispheres).

MortalityFrom transplant until end of post-transplant follow-up period (up to 7 years)

Death by any cause

Relapse of primary immune mediated diseaseFrom transplant until end of post-transplant follow-up period (up to 7 years)

histological findings of Focal segmental glomerulosclerosis (FSGS) in kidney biopsy in a patient with primary FSGS

Graft survivalFrom transplant until end of post-transplant follow-up period (up 7 years)

Time from transplant to the need for dialysis or entry onto the re- transplant list.

Trial Locations

Locations (4)

University Medical Center Hamburg-Eppendorf (UKE),

🇩🇪

Hamburg, Germany

Mediterranean Institute for Transplantation (ISMETT)

🇮🇹

Palermo, Sicily, Italy

University Hospital Padova

🇮🇹

Padua, Italy

Hospital Universitario La Paz

🇪🇸

Madrid, Madrid, Spain

University Medical Center Hamburg-Eppendorf (UKE),
🇩🇪Hamburg, Germany
Jan Beime, MD
Contact
+49 40 74100
j.beime@uke.de

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