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OT 5 – Development and prevention of severe heart disease

Cardiac involvement is often a silent disease manifestation that is asymptomatic in up to three out of four patients. However, in some cases it can progress to a more severe level. This trial focuses on the early stage of heart disease and the potential of different medications to prevent cardiac events in patients with Systemic Sclerosis.

The following protocol gives you a brief overview of observational trial 5:

 

PI: Gabriele VALENTINI (UNINA2)

Objectives

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.

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

Primary endpoint

Cumulative incidence of CB, VA, pacemaker implantation, congestive heart failure and sudden death (Time Frame: 1 year)

Safety endpoints
  • Incidence of drug-related adverse events
  • Incidence of withdrawal from treatment due to drug-related adverse events

 

Study Population

The study population are adult and juvenile SSc patients from the EUSTAR cohort (MEDSonline database) and the jSScWG cohort.

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

Trial design

Observational trial with 2 treatment arms

Treatment arms

Two different groups will be observed for analysis:
(1) Patients receiving CCB monotherapy, ACEi monotherapy, or CCB + ACEi combination therapy

(2) Patients receiving no CCB or ACEi therapy 

Follow-up

 

Patients will be assessed every 3 months by history, clinical examination and ECG, and every 6 months by Holter ECG and B-mode echocardiography until the end of the 1-year follow-up
ClinicalTrials.gov Identifier:  NCT01829126

For further Information:
http://clinicaltrials.gov/ct2/show/NCT01829126?term=desscipher&rank=3