Pulmonary Involvement in Scleroderma: A Clinical Study of the Safety and Efficacy of Mycophenolate Mofetil in Scleroderma Patients With Lung Involvement
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Pulmonary Involvement in Scleroderma: Safety and Efficacy of Mycophenolate Mofetil in Scleroderma Patients|
- Mean Change From Baseline in Forced Vital Capacity (FVC) [ Time Frame: Baseline, 12 months ] [ Designated as safety issue: No ]compare pre- and post-therapy FVC (post- minus pre-). Forced vital capacity (FVC) is the volume of air (liters) that can forcibly be blown out after full inspiration.
- Mean Change in Bronchoalveolar Lavage (BAL) Components (Neutrophils, Eosinophils) [ Time Frame: Baseline, 12 months ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: No ]Participants reported frequency of shortness of breath experienced with exertion
- Mean Change in Six Minute Walk Distance [ Time Frame: 12 months ] [ Designated as safety issue: No ]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 ] [ Designated as safety issue: No ]DLCO was measured before beginning and after completion of study therapy
|Study Start Date:||May 2006|
|Study Completion Date:||January 2009|
|Primary Completion Date:||January 2009 (Final data collection date for primary outcome measure)|
|Drug: Mycophenolate mofetil|
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00333437
|United States, California|
|UCSF, 400 Parnassus Ave|
|San Francisco, California, United States, 94143|
|Principal Investigator:||Jeffrey A Golden, MD||University of California, San Francisco|