Systemic Sclerosis MedDRA version: 6.1 Level: PT Classification code 10042953
Conditions
Sponsors
AZIENDA OSPEDALIERO UNIVERSITARIA OSPEDALI RIUNITI UMBERTO I - G.M.LANCISI - G.SALESI
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. SSc as defined above 2. Presence of active interstitial alveolitis as defined above 3. Resistance to conventional immunosuppressive treatment as defined above 4. Age 18-80 years 5. Ability to give an informed consent 6. Use of an acceptable method of birth control (if applicable). Pregnancy will be ruled out before study beginning. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Connective tissue diseases other than SSc 2. Smoking habit 3. Pregnancy or lactation 4. HBV or HCV infection 5. Hepatic Disease (ALT or ALP >1.5 fold above normal levels) 6. Moderate or Severe Renal Failure (creatinina clearance <59ml/min) 7. Severe Heart Failure, with Ejection Fraction ≤35% measured by echocardiography 8. Thyroid disease with clinical evidence of hyperthyroidism or hypothyroidism treated with substitutive therapy
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The main objective of this study is to verify efficacy and tolerability of a new therapy on pulmonary and skin fibrosis in patients affected by systemic sclerosis;Secondary Objective: The proposed action has the following other objectives: 1. strengthening of a national network composed of centres trained in the use of experimental drugs for this disease, with a special focus on the development of uniform criteria for outcome evaluation, enforcement of communication channels, creation of strategic synergies for the future, through ideas, projects and data exchange; 2. training of young investigators; 3. sharing of advanced experimental techniques and ?rare? biological samples to improve knowledge about pathogenesis of scleroderma, avoiding as well costly overlap.;Primary end point(s): The primary outcomes are: 1. the improvement of the pulmonary interstitial disease. A positive (good) response is defined as follow: increase ≥15% in FVC rate and/or TLC and/or increase ≥15% of Diffusing Lung Carbon Oxide (DLCO) AND PaO2 >90% of initial value AND HR CT-scan pattern defined as ?unchanged? or ?improved? (see below). The effect will be measured through the percentage of subjects with a positive response as defined above; 2. the evaluation of drug tolerability. | — |
Countries
Italy
Outcome results
None listed