URACYST® For the Treatment of GAG Deficient Interstitial Cystitis

Study Overview
=================
Brief Summary
-----------------
This protocol describes a multi-centre, community based open label study designed to assess the efficacy and safety of intravesical sodium chrondroitin sulfate (Uracyst®) in the treatment of patients with a clinical diagnosis of interstitial cystitis (IC). The safety of the study product will be evaluated through the incidence of adverse events and from results of physical examinations and laboratory tests.

Detailed Description
-----------------
The primary efficacy endpoint will be the percent responders to treatment as indicated by improvement on a seven-point Patient Global Assessment scale at week 10 (after 6 treatments) compared to baseline. The patient evaluates the overall change in their condition as markedly improved, moderately improved, slightly improved, no change, slightly worse, moderately worse or markedly worse. Secondary efficacy objectives will be as follows: Improvement in individual IC symptoms during the treatment period and at week 10 (after 6 treatments) compared to baseline. Pain and urgency scores (0-10 cm VAS) will be obtained using patient on-site questionnaires prior to first treatment (baseline), and again for weeks 4, 6, 10, 14, 18, 22 and 24. Change in Patient Symptom/Problem Index scores over the course of the treatment until the end of study (week 24) compared to baseline. The validated O'Leary Symptom Problem/Index will be completed prior to first treatment (baseline) and again for weeks 4, 6, 10, 14, 18, 22 and 24. The validated PUF questionnaire will be completed prior to first treatment (baseline) and again for weeks 4, 6, 10, 14, 18, 22 and 24. Change in patient condition every month throughout the therapy and treatment follow-up. In addition to measuring the change in patient condition at week 14, the Patient Global Assessment will be completed at weeks 4, 6, 10, 14, 18, 22 and 24.

Official Title
-----------------
URACYST® For the Treatment of GAG Deficient Interstitial Cystitis

Conditions
-----------------
Painful Bladder Syndrome, Interstitial Cystitis

Intervention / Treatment
-----------------
* Device: Uracyst


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Patients must meet the following eligibility criteria in order to be enrolled in this study. Clinical diagnosis of interstitial cystitis Legally majority female capable and willing to provide informed consent Negative blood test for pregnancy at baseline or assurance of previous surgery, condition or state rendering conception impossible A sterile bacterial urine culture no more than thirty (30) days prior to first treatment An average urinary frequency of at least 11 times per 24-hour day An average pain/discomfort score of 4 or greater on a 0-10cm VAS scale Available for the duration of the study including treatment and follow-up (4 months) Exclusion Criteria: Pregnant or lactating Currently receiving or having received investigational drugs thirty (30) days or less prior to screening Currently receiving or having had prior therapy with intravesical treatment (eg. Uracyst, Cystistat®, heparin or BCG) Receiving therapy for less than three months with antidepressants, antihistaminics, hormonal agonists or antagonists; hence patient not stabilized on therapy. (Stable therapy defined as continuous treatment for at least three months.) Currently receiving or having received prior therapy with oral pentosanpolysulfate (Elmiron) 3 months or less prior to screening IC symptoms relieved by antimicrobials, anticholinergics or antispasmodics Bladder capacity of greater than 500 ml on awake cystometry using liquid filling medium Neurologic disease affecting bladder function; any previous surgery or procedure having affected bladder function Current urinary tract infection (must be treated and have a negative culture before study entry) Current diagnosis of chemical, tuberculous or radiation cystitis History of bladder or lower ureteral calculi History of cancer within the last five years other than adequately treated non-melanoma skin cancers Active sexual transmitted disease Current vaginitis Endometriosis Any condition/disease which in the opinion of the investigator could interfere with patient compliance and/ or interfere with the interpretation of the treatment results

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
Female

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Uracyst
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br>Uracyst® single arm open label Treatment Group | Device: Uracyst<br>* 2% weekly for 6 weeks, monthly for 4 months<br>* Other names: chondroitin sulphate;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Responders to Treatment | indicated by a marked or moderate improvement on a seven-point patient Global Response Assessment (GRA) scale compared with baseline | Week 10 (4 weeks after the initial six treatments | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Likert Pain Score From Baseline at 10 Weeks | Scale of 0-10; subjects report 0=no pain and 10 pain as bad as you can imagine. | Baseline and 10 Weeks | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
PBS, Painful Bladder Syndrome, Interstitial Cystitis, chrondroitin sulfate