Effectiveness and Safety Study of Pentosan Polysulfate Sodium for the Treatment of Interstitial Cystitis

Randomized, Double-Blind, Placebo-Controlled, Parallel Group Evaluation of the Efficacy and Tolerability of Two Different Doses of ELMIRON for the Treatment of Interstitial Cystitis

The purpose of this study is to evaluate the safety and effectiveness of pentosan polysulfate sodium 100 mg once a day, pentosan polysulfate sodium 100 mg three times a day, and placebo for 24 weeks for the relief of bladder pain or discomfort associated with interstitial cystitis.

Study Overview

• Status: Terminated
• Conditions: Interstitial Cystitis
• Intervention / Treatment: Drug: Pentosan polysulfate sodium 100 mg; Drug: Pentosan polysulfate sodium 100 mg; Drug: Placebo

Detailed Description

The purpose of this multi-center, double-blind (neither the patient nor the physician knows whether drug or placebo is being taken, or at what dosage), randomized (patients are assigned different treatments based on chance), parallel group trial is to evaluate the effectiveness and safety of two doses of pentosan polysulfate sodium (100 mg once a day and 100 mg three times a day) versus placebo for 24 weeks for the relief of bladder pain or discomfort associated with interstitial cystitis. The hypothesis of the study is that there is no treatment difference in the proportion of responders at study endpoint (Week 24). Effectiveness will be assessed based on the reduction in the O'Leary-Sant Interstitial Cystitis Symptom Index (ICSI) total score. Safety assessments include vital signs, laboratory tests, adverse events and physical exams. Patients will receive one of the following study treatments by mouth each day for 24 weeks: pentosan polysulfate sodium 100 mg once a day group - one pentosan polysulfate sodium 100 mg capsule in the morning, and one matching placebo capsule in the afternoon and evening; pentosan polysulfate sodium 100 mg capsule three times a day group - one pentosan polysulfate sodium 100 mg capsule in the morning, afternoon and evening; placebo group - one placebo capsule in the morning, afternoon and evening

• Study Type: Interventional
• Enrollment (Actual): 369
• Phase: Phase 4

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Edmonton, Alberta, Canada
  • British Columbia
    • Kelowna, British Columbia, Canada
    • Surrey, British Columbia, Canada
    • Victoria, British Columbia, Canada
  • New Brunswick
    • Fredericton, New Brunswick, Canada
  • Nova Scotia
    • Halifax, Nova Scotia, Canada
    • Kentville, Nova Scotia, Canada
  • Ontario
    • Barrie, Ontario, Canada
    • Burlington, Ontario, Canada
    • Kingston, Ontario, Canada
    • Kitchener, Ontario, Canada
    • Newmarket, Ontario, Canada
    • Toronto, Ontario, Canada
• United States
  • Alabama
    • Birmingham, Alabama, United States
    • Homewood, Alabama, United States
  • Alaska
    • Anchorage, Alaska, United States
  • California
    • San Bernardino, California, United States
    • San Carlos, California, United States
    • San Diego, California, United States
    • Torrance, California, United States
  • Colorado
    • Denver, Colorado, United States
    • Longmont, Colorado, United States
    • Wheat Ridge, Colorado, United States
  • Connecticut
    • Newington, Connecticut, United States
    • Norwalk, Connecticut, United States
  • Florida
    • Celebration, Florida, United States
    • Longwood, Florida, United States
    • Plantation, Florida, United States
    • Tampa, Florida, United States
    • Trinity, Florida, United States
    • Venice, Florida, United States
    • Weston, Florida, United States
  • Georgia
    • Atlanta, Georgia, United States
  • Illinois
    • Centralia, Illinois, United States
    • Evanston, Illinois, United States
    • Peoria, Illinois, United States
  • Indiana
    • Jeffersonville, Indiana, United States
  • Michigan
    • Dearborn, Michigan, United States
    • Grand Rapids, Michigan, United States
    • Kalamazoo, Michigan, United States
  • Missouri
    • Saint Louis, Missouri, United States
  • Nevada
    • Henderson, Nevada, United States
  • New Jersey
    • West Orange, New Jersey, United States
  • New York
    • Albany, New York, United States
  • North Carolina
    • Charlotte, North Carolina, United States
    • Concord, North Carolina, United States
    • Winston Salem, North Carolina, United States
  • Ohio
    • Lima, Ohio, United States
  • Oklahoma
    • Ponca City, Oklahoma, United States
    • Tulsa, Oklahoma, United States
  • Oregon
    • Medford, Oregon, United States
    • Portland, Oregon, United States
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States
  • Tennessee
    • Nashville, Tennessee, United States
  • Texas
    • Arlington, Texas, United States
    • Austin, Texas, United States
    • Carrollton, Texas, United States
    • Fort Worth, Texas, United States
    • San Antonio, Texas, United States
  • Utah
    • Ogden, Utah, United States
    • Salt Lake City, Utah, United States
  • Virginia
    • Richmond, Virginia, United States
  • Washington
    • Mountlake Terrace, Washington, United States
    • Seattle, Washington, United States
  • Wisconsin
    • Milwaukee, Wisconsin, United States

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Must have a total score of 8 or greater on the O'Leary-Sant Interstitial Cystitis Symptom Index (ICSI) and a score of > 0 on each of the 4 questions on the ICSI
• must have experienced bladder pain, urinary urgency and urinary frequency, each not related to a urinary tract infection, for at least 6 months prior to entry into the study
• must have averaged >= 10 voids per day (>=30 voids over 3 consecutive days) and averaged >= 1 void at night
• urine culture showing no evidence of urinary tract infection
• urine cytology showing no evidence of neoplastic cells

Exclusion Criteria:

• Scheduled for or use of intravesical therapy (eg, bladder distention, dimethyl sulfoxide) during or within 4 weeks prior to the study
• patients who have had cytoscopic evaluation within 4 weeks prior to the study
• patients who are currently (within last month) receiving other medications that may affect symptoms of interstitial cystitis (ie, antidepressants, antihistamines, antispasmodics, anticholinergics)
• patients who are chronic users of Schedule II narcotics or who are using any scheduled narcotics at the time of study entry
• patients taking coumadin, anticoagulants, heparin, or thrombolytic agents such as tissue plasminogen activator and streptokinase

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Pentosan polysulfate sodium 100 mg once a day; One 100 mg pentosan polysulfate sodium capsule in the morning and 1 matching placebo capsule in the afternoon and evening for 24 weeks	Drug: Pentosan polysulfate sodium 100 mg; One 100 mg pentosan polysulfate sodium capsule 3 times a day (morning, afternoon, and evening) for 24 weeks; Drug: Pentosan polysulfate sodium 100 mg; One 100 mg pentosan polysulfate sodium capsule in the morning, and 1 matching placebo capsule in the afternoon and evening for 24 weeks
Experimental: Pentosan polysulfate sodium 100 mg three times a day; One 100 mg pentosan polysulfate sodium capsule 3 times a day (morning afternoon and evening) for 24 weeks	Drug: Pentosan polysulfate sodium 100 mg; One 100 mg pentosan polysulfate sodium capsule 3 times a day (morning, afternoon, and evening) for 24 weeks; Drug: Pentosan polysulfate sodium 100 mg; One 100 mg pentosan polysulfate sodium capsule in the morning, and 1 matching placebo capsule in the afternoon and evening for 24 weeks
Placebo Comparator: Placebo; Placebo One placebo capsule 3 times a day (morning afternoon and evening) for 24 weeks	Drug: Placebo; One placebo capsule 3 times a day (morning, afternoon and evening) for 24 weeks

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Responders Defined as Having at Least a 30% Reduction in the O'Leary-Sant Interstitial Cystitis Symptom Index (ICSI) From Baseline to Study Endpoint	The ICSI is a four-item self-administered instrument developed for the evaluation and management of patients with interstitial cystitis. The index measures the presence and extent of symptoms including urinary urgency, urinary frequency, nocturia and pain/burning in the bladder. Each question in the ICSI is on a 0-5 scale, where each answer is given a specific rating. The sum of the individual question ratings is the score for the ICSI. The range of the test is a score of 0 to 20. A lower score indicates a better condition.	Baseline to Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Responders Defined as Having at Least a Four Point Reduction in the O'Leary-Sant Interstitial Cystitis Symptom Index (ICSI) From Baseline to Study Endpoint	The ICSI is a four-item self-administered instrument developed for the evaluation and management of patients with interstitial cystitis. The index measures the presence and extent of symptoms including urinary urgency, urinary frequency, nocturia and pain/burning in the bladder. Each question in the ICSI is on a 0-5 scale, where each answer is given a specific rating. The sum of the individual question ratings is the score for the ICSI. The range of the test is a score of 0 to 20. A lower score indicates a better condition.	Baseline to Week 24

Collaborators and Investigators

• Sponsor: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
• Investigators: Study Director: Johnson & Johnson Pharmaceutical Research & Development, L.L.C. Clinical Trial, Johnson & Johnson Pharmaceutical Research & Development, L.L.C.

Publications and helpful links

General Publications

• Imamura M, Scott NW, Wallace SA, Ogah JA, Ford AA, Dubos YA, Brazzelli M. Interventions for treating people with symptoms of bladder pain syndrome: a network meta-analysis. Cochrane Database Syst Rev. 2020 Jul 30;7(7):CD013325. doi: 10.1002/14651858.CD013325.pub2.

Study record dates

Study Major Dates

• Study Start: September 1, 2003
• Primary Completion (Actual): June 1, 2011
• Study Completion (Actual): June 1, 2011

Study Registration Dates

• First Submitted: July 7, 2004
• First Submitted That Met QC Criteria: July 8, 2004
• First Posted (Estimate): July 9, 2004

Study Record Updates

• Last Update Posted (Estimate): April 21, 2014
• Last Update Submitted That Met QC Criteria: April 2, 2014
• Last Verified: April 1, 2014

More Information

Terms related to this study

• Keywords: Interstitial Cystitis; Urinary Bladder Pain
• Additional Relevant MeSH Terms: Urologic Diseases; Urinary Bladder Diseases; Cystitis; Cystitis, Interstitial; Anticoagulants; Pentosan Sulfuric Polyester
• Other Study ID Numbers: CR004576; C-2002-036 (Other Identifier: Johnson & Johnson Pharmaceutical Research & Development, L.L.C.)