Clinical Trial: Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Evaluation of Sirolimus for the Treatment of the Autoimmune Blistering Dermatosis Pemphigus

Brief Summary: The purpose of this research is to study alternative treatments for the skin disease pemphigus (a rare autoimmune blistering disorder of the skin) by using sirolimus, an immunosuppressive drug. Immunosuppressive drugs inhibit or prevent the activity of the immune system and are commonly used to treat autoimmune diseases, inflammatory diseases, and organ transplantation rejection.

Detailed Summary:

The purpose of this study is to explore a new medication for the skin disease termed pemphigus. Our specific aim is to determine whether the use of sirolimus will allow for a decrease in the dosage or possibly eliminate the need for corticosteroids, which so far is the only type of drug that can control this disease.

Pemphigus is an autoimmune disease characterized by blistering, caused by autoantibodies against certain cells in the skin. This disease most commonly occurs in individuals ages 50 and older, and it presents as painful shallow erosions and/or blisters in the mouth and/or skin. Pemphigus is very painful and uncomfortable, associated with impaired quality of life and significant morbidity. Severe or untreated cases of pemphigus can become fatal if the involved surface area becomes large enough to cause dehydration and/or infection. The first line of therapy, and the standard of care, for pemphigus remain to be systemic corticosteroids. However, corticosteroids have many known side effects, especially when used for a long time. Many cases of pemphigus are insufficiently controlled with corticosteroids alone and require the addition of other immunosuppressive agents, such as azathioprine, cyclophosphamide, mycophenolate mofetil, or a variety of other therapies used off-label. All of these treatments are not always successful and have undesirable side effects, including increased risk of malignancy and infections. Although these treatments can offer some relief from the disease, they are also often the cause of many side effects.

Sirolimus (formerly known as rapamycin) is a drug commonly used after renal transplants to prevent organ rejection. In this study, pemphigus subjects with active disease will begin taking sirolimus in conjunction with corticosteroids, using a similar regimen used for organ transplantation
Sponsor: University of California, Irvine

Current Primary Outcome: Improvement of ABSIS Score While Reducing Steroid Dosage [ Time Frame: Expected time line 24 months ]

Measurement of disease severity will be quantified using ABSIS (Autoimmune Bullous Skin Disorder Intensity Score). Improvement in disease control is quantified by the maintenance or improvement of ABSIS score while reducing steroid dosage.

No results as study has been terminated early by the investigator.



Original Primary Outcome: Improvement of ABSIS Score while reduing Steroid Dosage [ Time Frame: 12 Months ]

Measurement of disease severity will be quantified using ABSIS (Autoimmune Bullous Skin Disorder Intensity Score). Improvement in disease control is quantified by the maintenance or improvement of ABSIS score while reducing steroid dosage.


Current Secondary Outcome: Statistical Measures [ Time Frame: Intended assessment at 24 months ]

The statistical goal is to observe "success," an improvement in disease control while up-titrating sirolimus dosage. As there will be no control group, the subject or progress at the end of the study will be compared to their baseline at the beginning of the study. The subject and disease severity at the beginning of the study will be compared to the disease severity at each visit and be correlated with the dosage of sirolimus and corticosteroid. However, since no patient completed the study, the outcome and any data collected was not assessed.


Original Secondary Outcome: Statistical Measures [ Time Frame: 12 Months ]

The statistical goal is to observe "success," an improvement in disease control while uptitrating sirolimus dosage. As there will be no control group, the subject's progress at the end of the study will be compared to their baseline at the beginning of the study. The subject's disease severity at the beginning of the study will be compared to the disease severity at each visit and be correlated with the dosage of sirolimus and corticosteroid.


Information By: University of California, Irvine

Dates:
Date Received: March 10, 2011
Date Started: February 2011
Date Completion:
Last Updated: February 24, 2016
Last Verified: January 2016