Pulmonary Involvement in Scleroderma: A Clinical Study of the Safety and Efficacy of Mycophenolate Mofetil in Scleroderma Patients With Lung Involvement
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.
Drug: Study subjects receive standard mycophenolate dosing.
|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 in Forced Vital Capacity (FVC) [ Time Frame: 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: 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.
- Improved Shortness of Breath (Self-reported) [ Time Frame: 12 months ] [ Designated as safety issue: No ]Participants reported whether they experienced less shortness of breath with exertion after 12 months of MMF therapy as compared to before beginning therapy
- 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)|
Study subjects receive standard mycophenolate dosing.
|Drug: Study subjects receive standard mycophenolate dosing.|
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.
|United States, California|
|UCSF, 400 Parnassus Ave|
|San Francisco, California, United States, 94143|
|Principal Investigator:||Jeffrey A Golden, MD||University of California, San Francisco|