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

NCT00428883 | Unknown Phase 2

• 1 other identifier: FARM5X8AWM
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 5

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

Outcome Measures

Primary Outcomes (2)

• The primary outcome is the reduction of skin thickness

• Evaluated by the modified Rodnan skin score.

Secondary Outcomes (4)

• scleroderma disease activity assessed as established

• patient physical and emotional well-being (VAS, HAQ, SF36)

• laboratory evidence of skin fibroblast activation;

• the levels of Glutathione and of oxidized glutathione (GSSG).

Interventions

N-acetylcysteine (NAC) DRUG

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Università Politecnica delle Marche: lead

Study Sites (5)

Università politecnica delle marche

Ancona, 60020, Italy

RECRUITING

Università di Firenze

Florence, Italy

RECRUITING

Università de L'Aquila

L’Aquila, Italy

RECRUITING

Seconda Università di Napoli

Napoli, Italy

RECRUITING

Catholic University of the Sacred

Roma, Italy

RECRUITING

MeSH Terms

Conditions

Scleroderma, Diffuse; Scleroderma, Systemic

Interventions

Acetylcysteine

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

Cysteine; Amino Acids, Sulfur; Sulfur Compounds; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Study Officials

• Armando Gabrielli, MD,professor

  Università Politecnica delle Marche

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Armando Gabrielli, MD,Professor

CONTACT

+390712206101; a.gabrielli@univpm.it

Giovanni Pomponio, MD

CONTACT

+390715964209; g.pomponio@ao-umbertoprimo.marche.it

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: January 29, 2007
• First Posted: January 30, 2007
• Study Start: January 1, 2007
• Study Completion: February 1, 2009
• Last Updated: January 30, 2007

Record last verified: 2007-01

Locations

IT (5)