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

Systemic Sclerosis Clinical Trial

— MYILD  

Official title:

A Randomized Controlled Trial to Compare the Efficacy of Oral Mycophenolate Mofetil With Placebo in Patients With Systemic Sclerosis Related Early Interstitial Lung Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02896205
• Other study ID #: NK/2612/DM/10772
• Status: Completed
• Phase: Phase 3
• Start date: October 2016
• Est. completion date: July 1, 2017

Study information

• Verified date: June 2018
• Source: Postgraduate Institute of Medical Education and Research
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 41
• Est. completion date: July 1, 2017
• Est. primary completion date: July 1, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Interstitial Lung Disease
• Lung Diseases
• Lung Diseases, Interstitial
• Scleroderma
• Scleroderma, Diffuse
• Scleroderma, Systemic
• Sclerosis
• Systemic Sclerosis

Intervention

Drug:
• Mycophenolate 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
• Placebo
Subjects will be given matching placebo for 6 months

Locations

Country	Name	City	State
India	PGIMER	Chandigarh

Sponsors (1)

Lead Sponsor	Collaborator
Postgraduate Institute of Medical Education and Research

Country where clinical trial is conducted

India, 

References & Publications (3)

Hoyles RK, Ellis RW, Wellsbury J, Lees B, Newlands P, Goh NS, Roberts C, Desai S, Herrick AL, McHugh NJ, Foley NM, Pearson SB, Emery P, Veale DJ, Denton CP, Wells AU, Black CM, du Bois RM. A multicenter, prospective, randomized, double-blind, placebo-controlled trial of corticosteroids and intravenous cyclophosphamide followed by oral azathioprine for the treatment of pulmonary fibrosis in scleroderma. Arthritis Rheum. 2006 Dec;54(12):3962-70. — View Citation

Tashkin DP, Elashoff R, Clements PJ, Goldin J, Roth MD, Furst DE, Arriola E, Silver R, Strange C, Bolster M, Seibold JR, Riley DJ, Hsu VM, Varga J, Schraufnagel DE, Theodore A, Simms R, Wise R, Wigley F, White B, Steen V, Read C, Mayes M, Parsley E, Mubarak K, Connolly MK, Golden J, Olman M, Fessler B, Rothfield N, Metersky M; Scleroderma Lung Study Research Group. Cyclophosphamide versus placebo in scleroderma lung disease. N Engl J Med. 2006 Jun 22;354(25):2655-66. — View Citation

Tashkin DP, Roth MD, Clements PJ, Furst DE, Khanna D, Kleerup EC, Goldin J, Arriola E, Volkmann ER, Kafaja S, Silver R, Steen V, Strange C, Wise R, Wigley F, Mayes M, Riley DJ, Hussain S, Assassi S, Hsu VM, Patel B, Phillips K, Martinez F, Golden J, Connolly MK, Varga J, Dematte J, Hinchcliff ME, Fischer A, Swigris J, Meehan R, Theodore A, Simms R, Volkov S, Schraufnagel DE, Scholand MB, Frech T, Molitor JA, Highland K, Read CA, Fritzler MJ, Kim GHJ, Tseng CH, Elashoff RM; Sclerodema Lung Study II Investigators. Mycophenolate mofetil versus oral cyclophosphamide in scleroderma-related interstitial lung disease (SLS II): a randomised controlled, double-blind, parallel group trial. Lancet Respir Med. 2016 Sep;4(9):708-719. doi: 10.1016/S2213-2600(16)30152-7. Epub 2016 Jul 25. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in Forced vital capacity (FVC) at 6 months, after treatment with oral mycophenolate mofetil or placebo		6 months
Secondary	Change from baseline in Quality of Life (QoL) score by Medical Outcome Short Form 36 (SF-36v2) at 6 months		6 months
Secondary	Change from baseline in Mahler Dyspnoea Index (MDI) at 6 months		6 months
Secondary	Number of participants with serious and non seroius adverse events with mycophenolate mofetil (MMF) and placebo		6 months
Secondary	Change in Forced Vital Capacity (FVC) from baseline to 6 months according to antibody (anti-centromere and anti-topoisomerase1) profile		6 months