E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 6.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10042953 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
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 |
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E.2.2 | Secondary objectives of the trial |
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. |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal 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. |
|
E.4 | Principal 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 |
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E.5 End points |
E.5.1 | 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. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Information not present in EudraCT |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 6 |