Working… Menu

Mycophenolate for Pulmonary Sarcoidosis

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. Identifier: NCT00262132
Recruitment Status : Terminated
First Posted : December 6, 2005
Last Update Posted : June 18, 2018
Hoffmann-La Roche
Information provided by:
Medical University of South Carolina

Brief Summary:
Corticosteroids are presently the drug of choice for the treatment of pulmonary sarcoidosis. However, corticosteroids are associated with many significant side effects. For this reason, it would be beneficial to find an alternative agent to corticosteroids for the treatment of pulmonary sarcoidosis. This study is an open label trial of mycophenolate for new onset pulmonary sarcoidosis. Patients are candidates for this study if they have biopsy proven pulmonary sarcoidosis and a vital capacity or FEV1 less than 80% of predicted. Patients must undergo bronchoscopy where not only is the diagnosis of pulmonary sarcoidosis required, but in addition, cells are obtained from bronchoalveolar lavage. If the patients are diagnosed with pulmonary sarcoidosis, they are placed on an initial dose of 500mg BID of mycophenolate for 1 week. If their blood counts are not affected on this dose and they have no significant symptoms that are thought to be drug related, then their dose is escalated to 1g BID for the remaining 9 weeks of the study (the total study drug therapy time is 10 weeks). The patients are followed with multiple study visits. At these visits blood tests are drawn to make sure that there are no significant side effects from mycophenolate. In addition, the patients have a history and physical performed to evaluate the clinical state of their sarcoidosis and to detect mycophenolate side effects. On completion of 10 weeks of mycophenolate therapy, the patients undergo a second bronchoscopy with bronchoalveolar lavage to obtain cells for analysis. The patients are evaluated with spirometry, measurements of shortness of breath (dyspnea), and a quality of life scale (SF36) at serial visits during their study. The primary endpoint of the study is improved, unchanged or worse FVC. It is hoped that this pilot study will suggest that mycophenolate is a reasonable treatment option for new onset pulmonary sarcoidosis.

Condition or disease Intervention/treatment Phase
Pulmonary Sarcoidosis Drug: Mycophenolate Phase 3

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Official Title: Mycophenolate for Pulmonary Sarcoidosis
Study Start Date : September 2003
Actual Primary Completion Date : August 2006
Actual Study Completion Date : August 2006

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion criteria:

New acute pulmonary sarcoidosis defined as a FVC or FEV1 less than 80 percent of predicted plus symptoms of worsening dyspnea, cough, chest pain, or wheezing in patients without a prior history of pulmonary sarcoidosis. All such patients will also require bronchoscopy with transbronchial biopsy that demonstrates noncaseating granulomas of unknown cause. Such patients are usually treated for acute pulmonary sarcoidosis. The transbronchial biopsy specimens must have negative stains for mycobacteria and fungi.

> 18 years of age

Signing a written informed consent form.

Exclusion Criteria

  1. Previous history of an adverse reaction to mycophenolate.
  2. Current use of another immunosuppressive medication.
  3. History of active hepatitis or another significant liver disease with the exception of proven or presumed sarcoidosis of the liver.
  4. Previous history of skin cancer
  5. Active peptic ulcer disease
  6. Pregnant and/ or lactating female
  7. Serum creatinine > 2.0 mg/dl
  8. Serum bilirubin > 3.0 mg/dl
  9. WBC < 4,000/cu mm3
  10. Has demonstrated non-compliance in the past
  11. Current alcohol or drug abuse
  12. Evidence of significant sarcoidosis in extrapulmonary organs that requires therapy such that the mycophenolate doses could not be tapered if the pulmonary sarcoidosis was stable or improved.
  13. History of previous severe digestive system disease
  14. Patients taking azathioprine
  15. Patients taking cholestyramine or other drugs that affect enterohepatic recirculation
  16. Patients with Lesch-Nyhan or Kelley-Seegmiller syndrome
  17. Patients be willing not to receive live vaccines during the study
  18. Patients with phenylketonuria
  19. Patients with elevated serum transaminases or total bilirubin at baseline

Information from the National Library of Medicine

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 identifier (NCT number): NCT00262132

Layout table for location information
United States, South Carolina
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Sponsors and Collaborators
Medical University of South Carolina
Hoffmann-La Roche
Layout table for investigator information
Principal Investigator: Marc A Judson, MD Medical University of South Carolina
Study Chair: Don C Rockey, MD Medical University of South Carolina
Layout table for additonal information Identifier: NCT00262132    
Other Study ID Numbers: CEL401
HR 11031
First Posted: December 6, 2005    Key Record Dates
Last Update Posted: June 18, 2018
Last Verified: April 2018
Additional relevant MeSH terms:
Layout table for MeSH terms
Sarcoidosis, Pulmonary
Lymphoproliferative Disorders
Lymphatic Diseases
Lung Diseases, Interstitial
Lung Diseases
Respiratory Tract Diseases
Mycophenolic Acid
Antibiotics, Antineoplastic
Antineoplastic Agents
Antibiotics, Antitubercular
Antitubercular Agents
Anti-Bacterial Agents
Anti-Infective Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action