Pulmonary Involvement in Scleroderma: A Clinical Study of the Safety and Efficacy of Mycophenolate Mofetil in Scleroderma Patients With Lung Involvement
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00333437|
Recruitment Status : Completed
First Posted : June 5, 2006
Results First Posted : May 17, 2013
Last Update Posted : November 13, 2013
|Condition or disease||Intervention/treatment|
|Scleroderma, Systemic||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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||7 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Pulmonary Involvement in Scleroderma: Safety and Efficacy of Mycophenolate Mofetil in Scleroderma Patients|
|Study Start Date :||May 2006|
|Primary Completion Date :||January 2009|
|Study Completion Date :||January 2009|
|Drug: Mycophenolate mofetil|
- Mean Change From Baseline in Forced Vital Capacity (FVC) [ Time Frame: Baseline, 12 months ]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 ]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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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|