Cyclosporine in Interstitial Cystitis: Efficacy, Safety and Mechanism of Action
Status:
Completed
Trial end date:
2016-04-01
Target enrollment:
Participant gender:
Summary
This is a study for patients that have a condition called Interstitial Cystitis, also known
as Painful Bladder Syndrome. Patients would have tried at least two different therapies,
unsuccessfully,(eg. medications, pelvic floor physical therapy)
The purpose of this research project is to study the immunosuppressive drug Cyclosporine in
patients with Interstitial Cystitis to assess how well it works, what the side effects are
and what is its mechanism of action. Cyclosporine is a drug that is FDA-approved to prevent
organ rejection after kidney, liver, and heart transplant but is not approved by the FDA for
Interstitial Cystitis. Several research studies have been published showing that Cyclosporine
can improve the symptoms of Interstitial Cystitis, that it works better than a placebo
("sugar pill" or inert treatment) and that it works more effectively than Pentosan
Polysulfate (Elmiron), which is an FDA approved therapy. The dose of Cyclosporine used in
these studies on Interstitial Cystitis are much lower that the doses used to prevent
rejection in transplant patients.
Based on these studies, the American Urological Association has recently published treatment
guidelines that recommend Cyclosporine therapy for Interstitial Cystitis after the failure of
other more conservative therapies and medications. Nevertheless, much is not known about
using Cyclosporine therapy for Interstitial Cystitis including the mechanism of action, the
ideal dose, how best to monitor for side effects and in particular whether kidney damage can
occur at the low doses used in these studies. In this study, to examine the mechanism of
action, blood and urine samples will be collected before, during and after therapy to look at
"biomarkers", chemical substances that can be associated with inflammation and tissue injury.
We will also test effects of therapy on skin sensation and pain perception because the
molecule which Cyclosporine binds to (calcineurin) is also found in nerves that conduct pain
signals.
This study is only being done at the Cleveland Clinic and will involve about 30 patients with
Interstitial Cystitis.