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

NCT02896205 | Completed Phase 3

• 1 other identifier: NK/2612/DM/10772
• Study Type: interventional
• Target: 41
• Locations: 1 country
• Sites: 1

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.

Conditions

Systemic Sclerosis; Scleroderma; Interstitial Lung Disease

Outcome Measures

Primary Outcomes (1)

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

  6 months

Secondary Outcomes (4)

• 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

Study Arms (2)

Mycophenolate mofetil

EXPERIMENTAL

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.

Drug: Mycophenolate mofetil

Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo

Interventions

Mycophenolate mofetil DRUG

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

Also known as: MMF

Mycophenolate mofetil

Placebo DRUG

Subjects will be given matching placebo for 6 months

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients of systemic sclerosis with presence of interstitial lung disease on High Resolution Computer Tomography (HRCT) chest
• FVC ≥ 70% of predicted on pulmonary function tests
• Age ≥18 years
• Consenting for participating in study

You may not qualify if:

• Received immunosuppression (except low dose steroids, prednisolone equivalent ≤10 mg/day) for ILD in the last 3 years
• Persistent leucopenia or thrombocytopenia
• Pregnant or breastfeeding females
• Severe pulmonary arterial hypertension (mean pulmonary arterial pressure \>55mmHg) requiring drug therapy
• Uncontrolled congestive heart failure
• Any other abnormalities noted on chest X-ray or HRCT other than ILD
• Active infection
• Inflammatory myositis
• Overlap syndrome
• Mixed connective tissue disease
• Other serious co-morbidities which could compromise patient's ability to complete the study

Sponsors & Collaborators

• Post Graduate Institute of Medical Education and Research, Chandigarh: lead

Study Sites (1)

PGIMER

Chandigarh, 160012, India

Location

Related Publications (4)

• 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. doi: 10.1056/NEJMoa055120.

  PMID: 16790698 BACKGROUND

• 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. doi: 10.1002/art.22204.

  PMID: 17133610 BACKGROUND

• 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.

  PMID: 27469583 BACKGROUND

• Naidu GSRSNK, Sharma SK, Adarsh MB, Dhir V, Sinha A, Dhooria S, Jain S. Effect of mycophenolate mofetil (MMF) on systemic sclerosis-related interstitial lung disease with mildly impaired lung function: a double-blind, placebo-controlled, randomized trial. Rheumatol Int. 2020 Feb;40(2):207-216. doi: 10.1007/s00296-019-04481-8. Epub 2019 Dec 7.

  PMID: 31813058 DERIVED

MeSH Terms

Conditions

Scleroderma, Systemic; Scleroderma, Diffuse; Lung Diseases, Interstitial

Interventions

Mycophenolic Acid

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Lung Diseases; Respiratory Tract Diseases

Intervention Hierarchy (Ancestors)

Caproates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids; Lipids

Study Officials

• GSRSNK Naidu, MD

  Post Graduate Institute of Medical Education and Research, Chandigarh

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: SENIOR RESIDENT

Study Record Dates

• First Submitted: August 27, 2016
• First Posted: September 12, 2016
• Study Start: October 1, 2016
• Primary Completion: July 1, 2017
• Study Completion: July 1, 2017
• Last Updated: June 6, 2018

Record last verified: 2018-06

Data Sharing

• IPD Sharing: Will not share

Locations

IN (1)