High Dose Intravenous N-Acetylcysteine Versus Iloprost for Early, Rapidly Progressive Diffuse Systemic Sclerosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00428883
Recruitment Status : Unknown
Verified January 2007 by Università Politecnica delle Marche.
Recruitment status was:  Recruiting
First Posted : January 30, 2007
Last Update Posted : January 30, 2007
Information provided by:
Università Politecnica delle Marche

Brief Summary:
  • Systemic sclerosis (scleroderma; SSc) is a rare, disfiguring systemic disorder characterized by fibrosis of the skin and visceral organs that alters every aspect of an individual life
  • Although some features of scleroderma phenotype are well established and represent the hallmarks of the disease, the primary cause is not fully delineated, though both endothelial cell damage, immunological abnormalities and excessive extracellular matrix production are well-documented
  • Recently, excessive oxidative stress has been implicated in the pathogenesis of scleroderma
  • N-acetylcysteine (NAC) exhibits direct and indirect antioxidant properties. Its free thiol group is capable of interacting with the electrophilic groups of ROS. This interaction with ROS leads to intermediate formation of NAC thiol, with NAC disulphide as a major end product. The net result is a decrease of the concentrations of OH-, H2O2, and HOCl. In addition, NAC exerts an indirect antioxidant effect related to its role as a glutathione (GSH) precursor. It serves as a central factor in protecting against internal toxic agents.
  • In view of these considerations we expect that NAC can confer substantial benefit in patients with scleroderma reducing skin fibrosis in view of its antioxidant properties, and we have decided to conduct a double blind, multicenter trial to establish whether NAC could ameliorate skin fibrosis in scleroderma patients

Condition or disease Intervention/treatment Phase
Scleroderma, Diffuse Drug: N-acetylcysteine (NAC) Phase 2 Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 45 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Double
Primary Purpose: Treatment
Official Title: Rare Disease With Microvascular Involvement: High Dose Intravenous N-Acetylcysteine Versus Iloprost for Early, Rapidly Progressive Diffuse Systemic Sclerosis
Study Start Date : January 2007
Estimated Study Completion Date : February 2009

Primary Outcome Measures :
  1. The primary outcome is the reduction of skin thickness
  2. Evaluated by the modified Rodnan skin score.

Secondary Outcome Measures :
  1. scleroderma disease activity assessed as established
  2. patient physical and emotional well-being (VAS, HAQ, SF36)
  3. laboratory evidence of skin fibroblast activation;
  4. the levels of Glutathione and of oxidized glutathione (GSSG).

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Clinical diagnosis of early diffuse scleroderma
  • ability to give an informed consent
  • use of an acceptable method of birth control (if women in childbearing age). Pregnancy will be ruled out before study beginning.

Exclusion Criteria:

  • connective tissue diseases or other autoimmune diseases other than SSc;
  • history of intolerance to the study drugs;
  • 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;
  • resting PaO2 <60mm/hg
  • creatinine clearance below 90ml/h
  • severe hepatic failure
  • bronchial asthma h. hemorrhagic diathesis i. pregnancy or lactation

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00428883

Contact: Armando Gabrielli, MD,Professor +390712206101
Contact: Giovanni Pomponio, MD +390715964209

Università politecnica delle marche Recruiting
Ancona, Italy, 60020
Contact: Armando Gabrielli, MD,professor    +390712206104   
Contact: Giovanni Pomponio, MD    +390715964205   
Principal Investigator: Armando Gabrielli, MD,professor         
Università de L’Aquila Recruiting
Aquila, Italy
Contact: Roberto Giacomelli, Ph   
Principal Investigator: Roberto Giacomelli, MD,professor         
Università di Firenze Recruiting
Firenze, Italy
Contact: Marco Matucci-Cerinic, MD,professor   
Principal Investigator: Marco Matucci-Cerinic, MD,professor         
Seconda Università di Napoli Recruiting
Napoli, Italy
Contact: Gabriele Valentini, MD,professor   
Principal Investigator: Gabriele Valentini, MD,professor         
Catholic University of the Sacred Recruiting
Roma, Italy
Contact: Gianfranco Ferraccioli, MD,professor         
Principal Investigator: Gianfranco Ferraccioli, MD,professor         
Sponsors and Collaborators
Università Politecnica delle Marche
Principal Investigator: Armando Gabrielli, MD,professor Università Politecnica delle Marche Identifier: NCT00428883     History of Changes
Other Study ID Numbers: FARM5X8AWM
First Posted: January 30, 2007    Key Record Dates
Last Update Posted: January 30, 2007
Last Verified: January 2007

Keywords provided by Università Politecnica delle Marche:
Scleroderma, Systemic
Scleroderma, Diffuse

Additional relevant MeSH terms:
Scleroderma, Systemic
Scleroderma, Diffuse
Pathologic Processes
Connective Tissue Diseases
Skin Diseases
Antiviral Agents
Anti-Infective Agents
Respiratory System Agents
Free Radical Scavengers
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Platelet Aggregation Inhibitors
Vasodilator Agents