Interstitial Lung Disease, Scleroderma, Systemic Sclerosis
Conditions
Brief summary
Systemic sclerosis is a multisystem disease and can involve the lungs in the form of ILD. Lung involvement is the most common cause of death in these patients. The present study is performed to study the efficacy of oral mycophenolate mofetil in treating early and mild ILD in patients of SSc. The efficacy and side effects of mycophenolate mofetil will be compared with that of oral placebo.
Detailed description
Lung involvement is the leading cause of death among patients with systemic sclerosis (SSc). Treatment with immunosuppression drugs helps in retarding the progression of interstitial lung disease (ILD) and improves the morbidity and mortality among these patients. Presently, cyclophosphamide has been shown to be useful in stabilizing the lung functions among patients of systemic sclerosis with ILD. But use of cyclophosphamide is also associated with many adverse effects including infections, cytopenias, gonadal dysfunction and malignancies. Use of oral mycophenolate mofetil (MMF) in SSc-ILD in recent studies has been shown to be effective in retarding progression of ILD among these patients with a better side effect profile compared to cyclophosphamide. Contemporary expert opinion dictates that the treatment for SSc-ILD needs to be individualized. Generally, intense immunosuppression is required in patients with FVC \<70% of the predicted. In patients with FVC \>70% of the predicted, the need for high dose immunosuppression is not clear and varies from center-to-center. The present study is designed to determine the efficacy of oral MMF in patients with SSc related early ILD. The subjects in this study will be given either oral MMF or placebo and will be monitored for their response and adverse events. Informed consent will be taken from the subjects before including in the study.
Interventions
DRUGMycophenolate mofetil
Subjects will be given oral Mycophenolate Mofetil starting at 500mg twice a day and increased gradually to a target dose of 2gram per day for 6 months
DRUGPlacebo
Subjects will be given matching placebo for 6 months
Sponsors
Post Graduate Institute of Medical Education and Research, Chandigarh
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Patients of systemic sclerosis with presence of interstitial lung disease on High Resolution Computer Tomography (HRCT) chest 2. FVC ≥ 70% of predicted on pulmonary function tests 3. Age ≥18 years 4. Consenting for participating in study
Exclusion criteria
1. Received immunosuppression (except low dose steroids, prednisolone equivalent ≤10 mg/day) for ILD in the last 3 years 2. Persistent leucopenia or thrombocytopenia 3. Pregnant or breastfeeding females 4. Severe pulmonary arterial hypertension (mean pulmonary arterial pressure \>55mmHg) requiring drug therapy 5. Uncontrolled congestive heart failure 6. Any other abnormalities noted on chest X-ray or HRCT other than ILD 7. Active infection 8. Inflammatory myositis 9. Overlap syndrome 10. Mixed connective tissue disease 11. Other serious co-morbidities which could compromise patient's ability to complete the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in Forced vital capacity (FVC) at 6 months, after treatment with oral mycophenolate mofetil or placebo | 6 months |
Secondary
| Measure | Time frame |
|---|---|
| Change from baseline in Quality of Life (QoL) score by Medical Outcome Short Form 36 (SF-36v2) at 6 months | 6 months |
| Change from baseline in Mahler Dyspnoea Index (MDI) at 6 months | 6 months |
| Number of participants with serious and non seroius adverse events with mycophenolate mofetil (MMF) and placebo | 6 months |
| Change in Forced Vital Capacity (FVC) from baseline to 6 months according to antibody (anti-centromere and anti-topoisomerase1) profile | 6 months |
Countries
India
Outcome results
None listed