Clinical trial NCT01829126: Development and Prevention of Severe Heart Disease in Systemic Sclerosis
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Systemic Sclerosis
Cardiac Diseases
Heart Block
Cardiac Arrhythmia
Congestive Heart Failure
Lead sponsor: University of Campania Luigi Vanvitelli
Sponsor class: OTHER
Collaborators: name: European Union
name: University of Giessen
name: University of Zurich
name: University of Paris 5 - Rene Descartes
name: University of Florence
name: University of Basel
name: University College, London
name: Charite University, Berlin, Germany
name: University of Pecs
name: University of Leeds
name: Schoen Klinik Hamburg Eilbek
First posted: 2013-04-11
Enrollment: 765
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: The study population are adult and juvenile SSc patients from the EUSTAR cohort (MEDSonline database) and the jSScWG cohort

Brief summary: Systemic sclerosis is an orphan, multiorgan disease affecting the connective tissue of the skin and all internal organs. Cardiac involvement, mainly characterised by small intramyocardial coronary artery involvement and myocardial fibrosis, can cause the development of impaired diastolic ventricular filling, cardiac blocks and ventricular arrhythmias, and can ensue in congestive heart failure and sudden death. Until now, no drug has been proven to have a therapeutic effect on SSc myocardial disease on an evidence-based level. Short-term trials and retrospective studies have suggested a favourable and protective effect of calcium channel blockers and angiotensin converting enzyme inhibitors in patients with myocardial involvement. However, no data are presently available on the prevention and treatment of severe heart disease.

This observational trial is part of the collaborative project "DeSScipher", one out of five observational trials to decipher the optimal management of systemic sclerosis. Aim of this observational trial is to assess the efficacy and safety of calcium channel blockers and angiotensin converting enzyme inhibitors in asymptomatic SSc patients with cardiac involvement.


Eligibility criteria: Inclusion Criteria:

* Juvenile and adult systemic sclerosis patients, with diagnosis according to the SSc ACR/EULAR criteria or the PRES/ACR/EULAR juvenile SSc criteria respectively
* Asymptomatic (for cardiac disease) systemic sclerosis patients at risk for severe heart disease with at least one of the following risk factors: male sex and/or DLCO lower than 80% and/or sPAP \> 30 mmHg and/or synovitis and/or joint contractures and/or digital ulcers and/or proteinuria.

Asymptomatic for cardiac disease is defined by patients without dyspnea NYHA \>/= II, without palpitations and without bilateral leg edema.

Exclusion Criteria:

* Any significant pulmonary parenchymal (FVC \< 70% and/or DLCO \< 70%), pulmonary vascular (estimated systolic PAP \> 40 mmHg), gastrointestinal (malabsorption syndrome or paralytic ileus) or renal (serum creatinine level \>1.2 mg/dl, dialysis or previous scleroderma renal crisis) involvement
* Patients with dyspnea class NYHA \>/= II
* Patients with palpitations
* Patients with bilateral leg edema.

Primary outcomes:
measure: Cumulative incidence of CB, VA, pacemaker implantation, congestive heart failure and sudden death
timeFrame: 1 years
Secondary outcomes:
Other outcomes:
measure: Incidence of drug-related adverse events, incidence of withdrawal from treatment due to drug-related adverse events
timeFrame: 1 year
Locations:
city: Paris
country: France
facility: Université Paris Descartes, Hôpital Cochin, Service de Rhumatologie A & INSERM 1016
city: Bad Nauheim
country: Germany
facility: Justus-Liebig-University Gießen, Kerckhoff Clinic, Departement of Rheumatology and Clinical Immunology
city: Berlin
country: Germany
facility: Charité Universitätsmedizin Berlin, Charité Centrum 12 für Innere Medizin und Dermatologie, Medizinische Klinik mit Schwerpunkt Rheumatologie und Klinische Immunologie
city: Hamburg
country: Germany
facility: Centre for Pediatric Rheumatology, Klinikum Eilbek
city: Pecs
country: Hungary
facility: Pecsi Tudomanyegyetem - University of Pecs
city: Firenze
country: Italy
facility: University of Florence, Denothe Centre, Division of Rheumatology AOUC, Department of Biomedicine
city: Napoli-Italia
country: Italy
facility: Policlinico, Via Pansini
city: Basel
country: Switzerland
facility: Felix-Platter Spital, University of Basel
city: Zurich
country: Switzerland
facility: University of Zurich, Department of Rheumatology
city: Leeds
country: United Kingdom
facility: The Universitiy of Leeds, Division of Rheumatic and Musculoskeletal Disease, St James's University Hospital
city: London
country: United Kingdom
facility: Royal Free Hospital, University College London
Study officials:
affiliation: Justus-Liebig-University Gießen, Kerckhoff Clinic, Departement of Rheumatology and Clinical Immunology
name: Ulf Müller-Ladner, Prof.
role: STUDY_CHAIR
affiliation: Policlinico, Via Pansini, Napoli-Italia
name: Gabriele Valentini, Prof.
role: PRINCIPAL_INVESTIGATOR
References: