Scleroderma, DiffusePhase 2/3NCT00428883
High Dose Intravenous N-Acetylcysteine Versus Iloprost for Early, Rapidly Progressive Diffuse Systemic Sclerosis
A Phase 2/3 study testing N-acetylcysteine (NAC) in people with Scleroderma, Diffuse, run by Università Politecnica delle Marche at 5 sites in Italy.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Scleroderma, Diffuse
- What is being tested?
- N-acetylcysteine (NAC)
- How many people
- 45 participants
- Where
- 5 sitesItaly
- Study dates
- Jan 2007to Feb 2009
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
Italy5 sites, 5 recruiting
- Recruiting
Università politecnica delle marche
Ancona, 60020
Armando Gabrielli, MD,professor, +390712206104, a.gabrielli@univpm.it
Giovanni Pomponio, MD, +390715964205, g.pomponio@ao-umbertoprimo.marche.it
- Recruiting
Università di Firenze
Florence
Marco Matucci-Cerinic, MD,professor, cerinic@unifi.it
Marco Matucci-Cerinic, MD,professor
- Recruiting
Università de L'Aquila
L’Aquila
Roberto Giacomelli, Ph, roberto.giacomelli@cc.univaq.it
Roberto Giacomelli, MD,professor
- Recruiting
Seconda Università di Napoli
Naples
Gabriele Valentini, MD,professor, gabriele.valentini@unina2.it
Gabriele Valentini, MD,professor
- Recruiting
Catholic University of the Sacred
Roma
Gianfranco Ferraccioli, MD,professor
Gianfranco Ferraccioli, MD,professor
About this study
Official title: Rare Disease With Microvascular Involvement: High Dose Intravenous N-Acetylcysteine Versus Iloprost for Early, Rapidly Progressive Diffuse Systemic Sclerosis
- 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
- Being tested
- N-acetylcysteine (NAC)
- Keywords
- Scleroderma, Systemic, Scleroderma, Diffuse
- Medical subject headings
- Scleroderma, Diffuse, Scleroderma, Systemic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
N-acetylcysteine (NAC)Drug
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2007 (estimated)
- Study startJan 2007
- Study completionFeb 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Scleroderma, DiffuseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Double blind
- Enrolment
- 45 participants
Sponsor and investigators
- Lead sponsor
- Università Politecnica delle Marche
- Sponsor type
- University, hospital or other
- Investigators
- Armando Gabrielli, MD,professor, Principal investigator, Università Politecnica delle Marche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00428883
- Sponsor's protocol ID
- FARM5X8AWM
- Status last verified
- Jan 2007
- First submitted
- 29 Jan 2007
- Last updated
- 30 Jan 2007
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Rare Disease With Microvascular Involvement: High Dose Intravenous N-Acetylcysteine Versus Iloprost for Early, Rapidly Progressive Diffuse Systemic Sclerosis
- ClinicalTrials.gov ID
- NCT00428883
- Acronym / sponsor ID
- FARM5X8AWM
- Conditions
- Scleroderma, Diffuse
- Keywords
- Scleroderma, Systemic, Scleroderma, Diffuse
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 45 participants
- Lead sponsor
- Università Politecnica delle MarcheUniversity, hospital or other
- Study start
- Jan 2007
- Study completion
- Feb 2009
- First posted
- 30 Jan 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
- 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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Double blind
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| N-acetylcysteine (NAC) | Drug | – |
Primary outcome
The primary outcome is the reduction of skin thickness
Evaluated by the modified Rodnan skin score.
4 secondary outcomes
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).
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2007.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Armando Gabrielli, MD,professor principal investigator
Università Politecnica delle Marche
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing N-acetylcysteine (NAC) in people with Scleroderma, Diffuse, run by Università Politecnica delle Marche at 5 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 30 Jan 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Armando Gabrielli, MD,Professor (+390712206101, a.gabrielli@univpm.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The primary outcome is the reduction of skin thickness. The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jan 2007.