Cyclosporine in Interstitial Cystitis: Efficacy, Safety and Mechanism of Action

NCT01990898 | Completed Phase 2 Results DMC

• 1 other identifier: 13-1271
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 1

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.

Conditions

Interstitial Cystitis

Outcome Measures

Primary Outcomes (1)

• 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.

  3 Months

Study Arms (1)

Cyclosporine

EXPERIMENTAL

Drug: Cyclosporine, Pill form, dosage calculated upon patient study visit, frequency - twice daily, duration - 3 months.

Drug: Cyclosporine

Interventions

Cyclosporine DRUG

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.

Also known as: Gengraf Cyclosporine

Cyclosporine

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• age \> 18
• able to give consent
• commitment to return for follow up appointments
• agree to all parts of the study, including pain sensation testing
• total Interstitial Cystitis Symptom Index (ICSI) score \>9

You may not qualify if:

• active cancer
• history of pelvic radiation
• history of previous urological malignancy
• serum Cr \> 1.5 mg/dl
• diagnosis of diabetes mellitus types I or II
• untreated hypertension or blood pressure on treatment \> 140/90
• proteinuria at enrollment
• current or previous urinary diversion or bladder augmentation
• chronic use of a medication class with significant impact on Cyclosporine A (CyA) blood levels (eg. macrolide antibiotics, phenytoin, oral antifungals, calcium channel blockers)
• untreated urinary tract infection
• pregnant or breast feeding
• neurological impairment or spinal cord injury
• known hypersensitivity to CyA
• concurrent use of another immunosuppressive drug (eg. oral corticosteroids, tacrolimus, mycophenolate mofetil)

Sponsors & Collaborators

• The Cleveland Clinic: lead

Study Sites (1)

Cleveland Clinic - Main Campus Only

Cleveland, Ohio, 44195, United States

Location

MeSH Terms

Conditions

Cystitis, Interstitial

Interventions

Cyclosporine

Condition Hierarchy (Ancestors)

Cystitis; Urinary Bladder Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases

Intervention Hierarchy (Ancestors)

Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Dr. Daniel Shoskes
• Organization: Cleveland Clinic

dshoskes@gmail.com

Study Officials

• Daniel Shoskes, M.D.

  The Cleveland Clinic

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 18, 2013
• First Posted: November 25, 2013
• Study Start: November 1, 2013
• Primary Completion: October 1, 2015
• Study Completion: April 1, 2016
• Last Updated: April 19, 2017
• Results First Posted: April 19, 2017

Record last verified: 2016-10

Locations

US (1)