The purpose of this study is to investigate the safety and effectiveness of a medication called CellCept in treating refractory (has not responded to other treatments) interstitial cystitis.
CellCept belongs to a class of medications called immuno-suppressants. Immuno-suppressants work in the body by reducing the immune system's ability to produce certain reactions that can cause inflammation. In some people, the inflammation produced by their immune system can damage healthy tissues and cause symptoms of pain and discomfort. CellCept is approved by the U.S. Food and Drug Administration (FDA) for use in patients who have had an organ transplant. When used in combination with other drugs, CellCept helps to prevent the rejection of the transplanted organ and is used widely in patients who have received kidney, liver and heart transplants. CellCept is also frequently used but not FDA approved for the treatment of severe rheumatoid arthritis which is a disease caused when the body's immune system acts against healthy tissues in the joints.
Due to its special activity, CellCept may be useful in treating certain inflammatory diseases or conditions like interstitial cystitis.
Primary Outcome Measures:
- To compare CellCept 2 grams daily to placebo for effects on overall IC symptoms and well being in patients with refractory PBS/IC. [ Time Frame: 12 Weeks ] [ Designated as safety issue: Yes ]
- To assess the safety profile of CellCept in the treatment of refractory PBS/IC. [ Time Frame: 12 Weeks ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- To investigate the association between clinical subgroups, characterized by differences in baseline characteristics (such as presence of ulcers, duration of symptoms, significant co-morbid diseases, serological abnormalities), and efficacy of CellCept. [ Time Frame: 12 Weeks ] [ Designated as safety issue: No ]
- To assess the patterns of patient expectations, associations with symptom severity, and the potential impact of patient expectations on response to treatment. [ Time Frame: 12 Weeks ] [ Designated as safety issue: No ]
- To assess patterns of treatment goals and goal achievement in this study population, as well as baseline characteristics and factors related to goal selection and achievement. [ Time Frame: 12 Weeks ] [ Designated as safety issue: No ]
- To assess impact of study medication on pain medication use. [ Time Frame: 12 Weeks ] [ Designated as safety issue: No ]
- To assess the frequency and mechanism of un-blinding on study results and assess how the patient's perception of which treatment they received changes over time and influences ultimate outcome. [ Time Frame: 12 Weeks ] [ Designated as safety issue: No ]
- To assess the rate of detectable immune disorders in patients with PBS/IC refractory to standard treatment using CellCept. [ Time Frame: 12 Weeks ] [ Designated as safety issue: No ]
Enrollment: |
210 |
Study Start Date: |
March 2007 |
Estimated Study Completion Date: |
April 2008 |
Primary Completion Date: |
February 2008 (Final data collection date for primary outcome measure) |
A: Active Comparator
2000 mg per day of CellCept (MMF) divided into 2 equal doses.
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Drug: Mycophenolate Mofetil
Drug: Mycofenolate Mofetil (MMF)
2000 mg per day divided into 2 equal doses.
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B: Placebo Comparator
Placebo
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Drug: Placebo
Placebo
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Interstitial Cystitis (IC) is a bladder syndrome characterized as painful, debilitating and chronic, with no universally successful treatment option currently available. Characteristic symptoms include pain with bladder filling, and marked urinary frequency (to relieve pain). The only FDA-approved oral medication for treatment of IC is pentosan polysulfate (Elmiron), recently demonstrated by our collaborative research network to perform with little more efficacy than placebo (ref), and which is expensive and has associated side effects. Current clinical treatment protocols are empiric and usually aimed at relieving pain. There is a pressing need for an effective oral medication for treatment of IC. The presentation of symptoms can be quite variable among patients, suggesting that IC is a multi-factorial syndrome with several proposed etiologies, some of which may be interrelated.