Interstitial Cystitis
Conditions
Brief 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.
Detailed description
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.
Interventions
DRUGCyclosporine
Drug will be provided to patient's at study visit. Drug is to be taken twice daily. Dosage will be calculated at study visit and provided to patient.
Sponsors
The Cleveland Clinic
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
1. age \> 18 2. able to give consent 3. commitment to return for follow up appointments 4. agree to all parts of the study, including pain sensation testing 5. total Interstitial Cystitis Symptom Index (ICSI) score \>9
Exclusion criteria
1. active cancer 2. history of pelvic radiation 3. history of previous urological malignancy 4. serum Cr \> 1.5 mg/dl 5. diagnosis of diabetes mellitus types I or II 6. untreated hypertension or blood pressure on treatment \> 140/90 7. proteinuria at enrollment 8. current or previous urinary diversion or bladder augmentation 9. chronic use of a medication class with significant impact on Cyclosporine A (CyA) blood levels (eg. macrolide antibiotics, phenytoin, oral antifungals, calcium channel blockers) 10. untreated urinary tract infection 11. pregnant or breast feeding 12. neurological impairment or spinal cord injury 13. known hypersensitivity to CyA 14. concurrent use of another immunosuppressive drug (eg. oral corticosteroids, tacrolimus, mycophenolate mofetil)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Symptom Improvement of Interstitial Cystitis | 3 Months | Number of participants with \> 30% Improved Interstitial Cystitis Symptoms Index (ICSI) which is measured on a scale from 0 - 19 where the higher numbers are worse. No additional analyses have been done. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Treatment Cyclosporine | 26 |
| Total | 26 |
Baseline characteristics
| Characteristic | Treatment |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 6 Participants |
| Age, Categorical Between 18 and 65 years | 20 Participants |
| Age, Continuous | 50 years |
| Region of Enrollment United States | 26 participants |
| Sex: Female, Male Female | 14 Participants |
| Sex: Female, Male Male | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 2 / 26 |
| serious Total, serious adverse events | 0 / 26 |
Outcome results
Primary
Symptom Improvement of Interstitial Cystitis
Number of participants with \> 30% Improved Interstitial Cystitis Symptoms Index (ICSI) which is measured on a scale from 0 - 19 where the higher numbers are worse. No additional analyses have been done.
Time frame: 3 Months
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Treatment | Symptom Improvement of Interstitial Cystitis | 9 Participants |