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 ]

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.


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