Systemic Sclerosis
Conditions
Brief summary
This study is to evaluate the efficacy and safety of an antifibrotic agent, pirfenidone as treatment of systemic sclerosis. The primary outcome of this study is improvement of skin fibrosis.
Detailed description
All patients enrolled should suffer from moderate to severe skin fibrosis at the screening stage. The study period is 52 week. The study contains two stages, a 24-week blind stage following with open stage for another 24-week period of time. Subjects will receive pirfenidone or placebo randomized in the first 24-week and will all receive pirfenidone treatment in the second 24-week, with of combination of low dose steroids. The secondary outcomes of this study include but not limit to safety, improvement of lung fibrosis and digital ulcer burden.
Interventions
DRUGPirfenidone
A chemical synthesized agent showing anti-fibrotic effects in idiopathic pulmonary fibrosis.
DRUGPlacebo oral capsule
Placebo for pirfenidone capsule
DRUGSteroids
Low dose of glucocorticoids, a basic treatment for patients with systemic sclerosis
Sponsors
RenJi Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
* Fulfill EULAR/ACR classification criteria (2013) for systemic sclerosis (SSc). * Subjects must have mRSS≥10 at screening stage * Subjects must have signs of fibrosis in their chest CT at screening stage * If a subject has received treatments before, it should be stable at least 3 months until screening and with no significant efficacy (ΔmRSS≤-2)
Exclusion criteria
* Subjects with other connective tissue diseases overlapping with SSc * Subjects with skin atrophy as the main cutaneous manifestation * Subjects with active digital ulcers or gangrene * Active severe SSc-driven renal disease or heart dysfunction at screening * Subjects with significant hematologic abnormalities * Abnormal liver function test at screening (ALT, AST or total bilirubin over 2 fold of upper normal level * Clinically significant active infection including ongoing and chronic infections * History of human immunodeficiency virus (HIV) * Confirmed Positive tests for hepatitis B or positive test for hepatitis C * Active tuberculosis * Live or attenuated vaccine within 4 weeks prior to screening * Subjects with significant hematologic abnormalities
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Modified Rodnan Skin Score (mRSS） | Week 52 | A semi-quantitative score for skin fibrosis of every subjects |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Assessment of chest CT | Week 52 | Semi-quantitative scale for fibrotic changes in chest CT images from an individual patient |
| Forced vital capacity | Week 52 | A marker for lung function that may decrease with pulmonary fibrosis |
| Diffusing capacity | Week 52 | Another marker for lung function that may decrease with pulmonary fibrosis |
| 6 minute walk test | Week 52 | A simple, well-accepted and quantitative clinical exam to test heart and lung function |
| Modified Rodnan Skin Score | Week 24 | A semi-quantitative score for skin fibrosis of every subjects |
| Proportion of subjects with increased mRSS | Week 52 | Proportion of subjects with ∆mRSS≥5 |
| Quality of life | Week 52 | Assessed by the health assessment questionnaire disability index |
| Safety and Tolerability | Week 52 | Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 |
| Hand function assessment | Week 52 | Hand function will be measured by Cochin Hand Function Scale |
Countries
China
Outcome results
None listed