Study to Compare the Efficacy of Mycophenolate Mofetil in Systemic Sclerosis Related Early Interstitial Lung Disease

Phase 3

Completed

• Conditions: Systemic Sclerosis; Scleroderma; Interstitial Lung Disease
• Interventions: Drug: Placebo; Drug: Mycophenolate mofetil

• Registration Number: NCT02896205
• Lead Sponsor: Post Graduate Institute of Medical Education and Research, Chandigarh

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.

Study Timeline

• Study First Submitted: 2016-08-27
• Study First Submitted QC: 2016-09-06
• Study First Posted: 2016-09-12
• Study Start: 2016-10
• Primary Completion: 2017-07-01
• Study Completion: 2017-07-01
• Status Verified: 2018-06
• Last Update Submitted: 2018-06-05
• Last Update Posted: 2018-06-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 41

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Subjects in this arm will be given matching placebo, made of lactulose, starting at two tablets per day and increased by one tablet every 2 weeks to a target of 4 tablets per day.
Mycophenolate mofetil	Mycophenolate mofetil	Subjects will be started on Mycophenolate Mofetil 500mg twice a day and increased by 500mg every 2 weeks, if tolerated, to a target dose of 2gram per day.

Primary Outcome Measures

Name	Time	Method
Change from baseline in Forced vital capacity (FVC) at 6 months, after treatment with oral mycophenolate mofetil or placebo	6 months

Secondary Outcome Measures

Name	Time	Method
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
Change from baseline in Mahler Dyspnoea Index (MDI) at 6 months	6 months
Change from baseline in Quality of Life (QoL) score by Medical Outcome Short Form 36 (SF-36v2) at 6 months	6 months

Trial Locations

Locations (1)

PGIMER; 🇮🇳 Chandigarh, India