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Biomarkers in Pediatric Congenital Heart Disease and PAH

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ClinicalTrials.gov Identifier: NCT04130243
Recruitment Status : Recruiting
First Posted : October 17, 2019
Last Update Posted : October 17, 2019
Sponsor:
Information provided by (Responsible Party):
Prof. dr. R.M.F. Berger, University Medical Center Groningen

Brief Summary:

Nowadays, biomarkers are broadly used in clinical practice. Blood-derived biomarkers fulfil an important role in the field of cardiology. However, most biomarkers have been investigated for adult left ventricular disease. In congenital heart diseases (CHD) and pulmonary arterial hypertension (PAH), which involves children and mostly the right ventricle, less is known about the clinical and predictive value of blood-derived biomarkers. Since the group of survivors of CHD and PAH is growing because of the improved techniques nowadays, development of better tools to maintain the quality of life for the longer term in these patients is urgently needed. Blood-derived biomarkers are minimally invasive biomarkers, are quantitative and have shown to be able to reveal pathological processes in an early stage. Hence, blood-derived biomarkers may be a good addition to current diagnostic means in CHD and PAH.

Objective: The primary objective of this study is to investigate cross-sectionally the association between various emerging blood-derived biomarkers and right ventricular (RV) function:defined as tricuspid annular plane systolic excursion (TAPSE) measured with echocardiography, in children with (a history of ) an abnormally loaded, volume and/or pressure loaded, right ventricle associated with CHD and/or PAH.


Condition or disease Intervention/treatment
Congenital Heart Disease Pulmonary Arterial Hypertension Other: Blood test

  Show Detailed Description

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 380 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 4 Years
Official Title: Biomarkers in Congenital Heart Diseases and Pulmonary Arterial Hypertension
Actual Study Start Date : December 5, 2017
Estimated Primary Completion Date : December 1, 2021
Estimated Study Completion Date : December 1, 2021


Group/Cohort Intervention/treatment
Congenital Heart disease
In patients with (a history of) pressure- and/or volume loaded right ventricle due to congenital heart disease extra blood will be withdrawn for a blood test; serumbiomarker
Other: Blood test
Withdrawal of (extra) blood to measure serum biomarkers with a blood test

Pulmonary Arterial Hypertension
In patients with (a history of) pressure- and/or volume loaded right ventricle due to pulmonary arterial hypertension extra blood will be withdrawn for a blood test; serumbiomarker
Other: Blood test
Withdrawal of (extra) blood to measure serum biomarkers with a blood test




Primary Outcome Measures :
  1. TAPSE (mm) TAPSE (tricuspid annular plane systolic excursion) [ Time Frame: At baseline (t=0) ]
    Right ventricular function defined as TAPSE assessed by echocardiography


Secondary Outcome Measures :
  1. WHO functional class [ Time Frame: At baseline (t=0) ]
    Disease severity defined as WHO-functional class

  2. WHO functional class [ Time Frame: At 1 year ]
    Disease severity defined as WHO-functional class

  3. Six-minute walk distance (6MWD) [ Time Frame: At baseline (t=0) ]
    Disease severity defined as distance walked at 6MWD

  4. Six-minute walk distance (6MWD) [ Time Frame: At 1 year ]
    Disease severity defined as distance walked at 6MWD

  5. RV fractional area change (RV-FAC) [ Time Frame: At 1 year ]
    Disease severity defined as RV-FAC

  6. RV fractional shortening (RV-FS) [ Time Frame: At baseline (t=0) ]
    Disease severity defined as RV-FS

  7. RV fractional shortening (RV-FS) [ Time Frame: At 1 year ]
    Disease severity defined as RV-FS

  8. RV-function assessed with eyeballing (good, moderate, bad) [ Time Frame: At baseline (t=0) ]
    Disease severity defined RV-function assessed with eyeballing

  9. RV-function assessed with eyeballing (good, moderate, bad) [ Time Frame: At 1 year ]
    Disease severity defined RV-function assessed with eyeballing

  10. NYHA classification [ Time Frame: At baseline (t=0) ]
    Disease severity defined NYHA classification

  11. NYHA classification [ Time Frame: At 1 year ]
    Disease severity defined NYHA classification


Other Outcome Measures:
  1. Heart/Lung transplantation per participant [ Time Frame: During follow-up of the study; from enrollment till end of the study (5 years later) ]
    Disease severity defined as undergoing a heart/lung transplantation

  2. Number of Hospitalizations per participant [ Time Frame: During follow-up of the study; from enrollment till end of the study (5 years later) ]
    Hospitalization due to any cause during follow up study

  3. Number of participants that do not survive during follow-up study [ Time Frame: During follow-up of the study; from enrollment till end of the study (5 years later) ]
    Mortality defined as dead due to any cause during follow up study: from enrollment till end of study (5 years later)



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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The study population concerns all patients aged 0-18 years with (a history of) an abnormally loaded right ventricle (due to right-sided congenital heart disease or pulmonary arterial hypertension) who visit the outpatient clinic or will be electively admitted at the Center of Congenital Heart Disease - University Medical Center Groningen (CHH-UMCG).
Criteria

Inclusion Criteria:

  • Age 0-18 years
  • Patients with Pulmonary Arterial hypertension: diagnosis confirmed according to the standards of the National Expertise Center for PH in childhood.
  • Patients with CHD with an abnormally loaded right ventricle including tetralogy of Fallot (TOF), pulmonary (valve) stenosis (PS), pulmonary insufficiency (PI), atrial septal defect (ASD), ventricular septal defect (VSD) and total anomalous pulmonary venous return (TAPVR): diagnosis confirmed by echocardiography or cardiac MRI in UMCG-CCH.

Exclusion Criteria:

  • Age >18 years
  • Not familiar with Dutch language
  • Pregnant
  • Concomitant musculoskeletal disease
  • No echocardiography available within three months before or after blood collection
  • Being under examination for non-diagnosed disease at time of investigation

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04130243


Contacts
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Contact: Meindina G Haarman, MD +31(0)503613363 m.g.haarman@umcg.nl
Contact: Anne-Marie C Koop, BSc a.c.koop@umcg.nl

Locations
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Netherlands
University Medical Center Groningen Recruiting
Groningen, Netherlands, 9713GZ
Contact: Meindina G Haarman, MD    +31(0)503613363    m.g.haarman@umcg.nl   
Sub-Investigator: Anne-Marie C Koop, MD         
Sub-Investigator: Meindina G Haarman, MD         
Principal Investigator: Rudolphus MF Berger, MD PhD         
Sponsors and Collaborators
University Medical Center Groningen
Investigators
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Study Chair: A AE Verhagen, MD PhD University Medical Center Groningen

Publications:
Knofczynski GT, Mundfrom D. Sample sizes when using multiple linear regression for prediction. Educational and Psychological Measurement. 2008 June; 68(3):431-442.
Thygesen K, Alpert JS, White HD; Joint ESC/ACCF/AHA/WHF Task Force for the Redefinition of Myocardial Infarction, Jaffe AS, Apple FS, Galvani M, Katus HA, Newby LK, Ravkilde J, Chaitman B, Clemmensen PM, Dellborg M, Hod H, Porela P, Underwood R, Bax JJ, Beller GA, Bonow R, Van der Wall EE, Bassand JP, Wijns W, Ferguson TB, Steg PG, Uretsky BF, Williams DO, Armstrong PW, Antman EM, Fox KA, Hamm CW, Ohman EM, Simoons ML, Poole-Wilson PA, Gurfinkel EP, Lopez-Sendon JL, Pais P, Mendis S, Zhu JR, Wallentin LC, Fernández-Avilés F, Fox KM, Parkhomenko AN, Priori SG, Tendera M, Voipio-Pulkki LM, Vahanian A, Camm AJ, De Caterina R, Dean V, Dickstein K, Filippatos G, Funck-Brentano C, Hellemans I, Kristensen SD, McGregor K, Sechtem U, Silber S, Tendera M, Widimsky P, Zamorano JL, Morais J, Brener S, Harrington R, Morrow D, Lim M, Martinez-Rios MA, Steinhubl S, Levine GN, Gibler WB, Goff D, Tubaro M, Dudek D, Al-Attar N. Universal definition of myocardial infarction. Circulation. 2007 Nov 27;116(22):2634-53. Epub 2007 Oct 19.

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Responsible Party: Prof. dr. R.M.F. Berger, Head of Pediatric Cardiology, University Medical Center Groningen
ClinicalTrials.gov Identifier: NCT04130243     History of Changes
Other Study ID Numbers: 201700243
First Posted: October 17, 2019    Key Record Dates
Last Update Posted: October 17, 2019
Last Verified: October 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Prof. dr. R.M.F. Berger, University Medical Center Groningen:
Biomarker
Congenital Heart Disease
Pulmonary Arterial Hypertension
Additional relevant MeSH terms:
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Familial Primary Pulmonary Hypertension
Hypertension
Heart Diseases
Heart Defects, Congenital
Vascular Diseases
Cardiovascular Diseases
Hypertension, Pulmonary
Lung Diseases
Respiratory Tract Diseases
Cardiovascular Abnormalities
Congenital Abnormalities