Clinical Trial: Pulmonary Involvement in Scleroderma: A Clinical Study of the Safety and Efficacy of Mycophenolate Mofetil in Scleroderma Patients With Lung Involvement
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Pulmonary Involvement in Scleroderma: Safety and Efficacy of Mycophenolate Mofetil in Scleroderma Patients
Brief Summary: Researchers from the Division of Pulmonary and Critical Care Medicine at University of California, San Francisco (UCSF) are conducting a study to evaluate whether mycophenolate mofetil (an immunosuppressive medication, trade named CellCept) is safe and effective for preventing the lung damage from scleroderma from getting worse.
Detailed Summary:
The proposed study is designed to evaluate the safety and efficacy of mycophenolate mofetil (CellCept) for the treatment of symptomatic pulmonary alveolitis due to systemic sclerosis (SSc). This study utilizes a prospective, open-label, experimental design.
Primary Hypothesis: The alveolitis in patients with SSc, as defined by decreased forced vital capacity (FVC), bronchoalveolar lavage (BAL), and High Resolution Chest Tomography (HRCT) is responsive to 1 year of daily mycophenolate mofetil therapy.
Secondary Hypothesis: Quality of life, six-minute walk and single-breath diffusing capacity for carbon monoxide (DLCO) improve in patients with SSc mediated alveolitis after therapy with mycophenolate mofetil. This response to therapy is associated with a change in the inflammatory cytokine profile present in BAL fluid.
Sponsor: University of California, San Francisco
Current Primary Outcome: Mean Change From Baseline in Forced Vital Capacity (FVC) [ Time Frame: Baseline, 12 months ]
Original Primary Outcome:
- improvement in forced vital capacity (FVC), as % predicted
- Improvement in HRCT abnormalities
Current Secondary Outcome:
- Mean Change in Bronchoalveolar Lavage (BAL) Components (Neutrophils, Eosinophils) [ Time Frame: Baseline, 12 months ]BAL samples were colleected from the affected lobe (as determined by lung CT scans) before beginning and after completing study therapy.
- Change in Shortness of Breath (Self-reported) [ Time Frame: Baseline, 12 months ]Participants reported frequency of shortness of breath experienced with exertion
- Mean Change in Six Minute Walk Distance [ Time Frame: 12 months ]Comparison of 6-minute walk distance before beginning and after completing study therapy
- Mean Change in Diffusion Capacity of the Lung for Carbon Monoxide (DLCO) [ Time Frame: 12 months ]DLCO was measured before beginning and after completion of study therapy
Original Secondary Outcome:
- Improvement in diffusing capacity, DLCO
- Improvement in functional ability as assessed by six-minute walk test
- Improvement in quality of life as assessed by the Medical Outcomes Survey (SGRQ)
- Comparison of BAL fluid cell type and cytokine profile pre- and post-therapy.
Information By: University of California, San Francisco
Dates:
Date Received: June 2, 2006
Date Started: May 2006
Date Completion:
Last Updated: September 23, 2013
Last Verified: September 2013