Systemic Scleroderma MedDRA version: 9.1 Level: LLT Classification code 10055953 Term: Scleroedema
Conditions
Interventions
Trade Name: HIDONAC*EV 1FL 5G 25ML
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Acetylcysteine
CAS Number: 616-91-1
Concentration unit: g gram(s)
Concentration type: equal
Concentra
Sponsors
AZIENDA OSPEDALIERO UNIVERSITARIA OSPEDALI RIUNITI UMBERTO I - G.M.LANCISI - G.SALESI
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: a. edSSc (ACR criteria); b. age 18-80 years; c. ability to give an informed consent; d. use of an acceptable method of birth control (if women in childbearing age). 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: a. connective tissue diseases other than SSc; b. history of intolerance to the study drugs; c. severe cardiac failure (NYHA >=3 or left ventricular ejection fraction <40%), recent (<6 months) history of myocardial infarction; symptomatic ischemic myocardial disease, ventricular tachyarrhythmia, atrial fibrillation; d. resting PaO2 <60mm/hg or Carbon Monoxide Diffusing Capacity <40% of predicted e. creatinina clearance below 90ml/h f. ALT or bilirubin above two times upper normal limits; g. bronchial asthma; h. hemorrhagic diathesis
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Specific objective of the present study is to treat scleroderma skin fibrosis using a therapeutic strategy based on pathogenetic mechanisms (N-Acetylcisteine). It is anticipated that the impact of the expected findings to health care may be considerable since they may be applied to more common organ-based fibrosis for which scleroderma remains a well known paradigm.;Secondary Objective: As for other values, the proposed action has the following objectives: - to improve communication between different Italian groups presently engaged in the research of pathogenesis and the ensuing possibilities for the specific therapy of chronic fibrotic disorders; 7 - to increase the collaboration in joint projects; - to avoid costly overlap; - to facilitate the contact between basic research and clinical studies as well as exchange of ideas, material and technology; - to allow comparison and pooling of data; - to study the safety and efficacy of a novel therapeutic approach in a subgroup of patients with a rare disease requiring collaboration of several research groups.;Primary end point(s): The primary outcome is the reduction of skin thickness evaluated by the modified Rodnan skin score. In patients with SSc the skin score assesses the skin fibrosis and correlates with both systemic involvement and progression of the disease | — |
Countries
Italy
Outcome results
None listed