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

NCT ID: NCT00428883

Last Updated: 2007-01-30

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 45 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-01-31
• Study Completion Date: 2009-02-28

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

Conditions

Scleroderma, Diffuse

Keywords

Scleroderma, Systemic; Scleroderma, Diffuse

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Interventions

N-acetylcysteine (NAC)

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Università Politecnica delle Marche

OTHER

Sponsor Role: lead

Principal Investigators

Armando Gabrielli, MD,professor

Role: PRINCIPAL_INVESTIGATOR

Università Politecnica delle Marche

Locations

Università politecnica delle marche

Ancona, , Italy

Site Status: RECRUITING

Università di Firenze

Florence, , Italy

Site Status: RECRUITING

Università de L'Aquila

L’Aquila, , Italy

Site Status: RECRUITING

Seconda Università di Napoli

Napoli, , Italy

Site Status: RECRUITING

Catholic University of the Sacred

Roma, , Italy

Site Status: RECRUITING

Countries

Italy

Central Contacts

Armando Gabrielli, MD,Professor

Role: CONTACT

Phone: +390712206101

Email: a.gabrielli@univpm.it

Giovanni Pomponio, MD

Role: CONTACT

Phone: +390715964209

Email: g.pomponio@ao-umbertoprimo.marche.it

Facility Contacts

Armando Gabrielli, MD,professor

Role: primary

Giovanni Pomponio, MD

Role: backup

Marco Matucci-Cerinic, MD,professor

Role: primary

Roberto Giacomelli, Ph

Role: primary

Gabriele Valentini, MD,professor

Role: primary

Gianfranco Ferraccioli, MD,professor

Role: primary

Other Identifiers

FARM5X8AWM

Identifier Type: -

Identifier Source: org_study_id