Interstitial Cystitis
Conditions
Keywords
Interstitial Cystitis, Urinary Bladder Pain
Brief summary
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.
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
Interventions
DRUGPentosan polysulfate sodium 100 mg
One 100 mg pentosan polysulfate sodium capsule 3 times a day (morning, afternoon, and evening) for 24 weeks
DRUGPlacebo
One placebo capsule 3 times a day (morning, afternoon and evening) for 24 weeks
Sponsors
Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 | Baseline to Week 24 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | Baseline to Week 24 | 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. |
Countries
Canada, United States
Participant flow
Pre-assignment details
Three hundred and sixty-nine patients enrolled in this study. One patient did not take any study drug. Therefore, only 368 patients were included in the primary analysis dataset.
Participants by arm
| Arm | Count |
|---|---|
| PLACEBO | 118 |
| Pentosan Polysulfate Sodium (ELMIRON) 100MG QD One 100 mg pentosan polysulfate sodium capsule QD (once daily) | 129 |
| Pentosan Polysulfate Sodium (ELMIRON) 100MG TID One 100 mg pentosan polysulfate sodium capsule TID (three times a day) | 122 |
| Total | 369 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Adverse Event | 12 | 17 | 14 |
| Overall Study | Lack of Efficacy | 6 | 6 | 5 |
| Overall Study | Lost to Follow-up | 15 | 8 | 10 |
| Overall Study | NONCOMPLIANCE | 5 | 7 | 6 |
| Overall Study | Other | 4 | 2 | 3 |
| Overall Study | Protocol Violation | 8 | 3 | 0 |
| Overall Study | STUDY TERMINATED BY SPONSOR | 0 | 1 | 0 |
| Overall Study | Withdrawal by Subject | 0 | 4 | 10 |
| Overall Study | WITHDRAWAL OF CONSENT | 5 | 7 | 5 |
Baseline characteristics
| Characteristic | PLACEBO | Pentosan Polysulfate Sodium (ELMIRON) 100MG QD | Pentosan Polysulfate Sodium (ELMIRON) 100MG TID | Total |
|---|---|---|---|---|
| AgeCategorical 18-<64 YEARS | 104 participants | 109 participants | 109 participants | 322 participants |
| AgeCategorical <18 YEARS | 0 participants | 0 participants | 0 participants | 0 participants |
| AgeCategorical 65-<74 YEARS | 10 participants | 17 participants | 11 participants | 38 participants |
| AgeCategorical >=75 YEARS | 4 participants | 3 participants | 2 participants | 9 participants |
| Age, Categorical <=18 years | 1 Participants | 1 Participants | 0 Participants | 2 Participants |
| Age, Categorical >=65 years | 14 Participants | 20 Participants | 13 Participants | 47 Participants |
| Age, Categorical Between 18 and 65 years | 103 Participants | 108 Participants | 109 Participants | 320 Participants |
| Age, Continuous | 44.6 years STANDARD_DEVIATION 14.58 | 45.6 years STANDARD_DEVIATION 15.68 | 42.7 years STANDARD_DEVIATION 15.71 | 44.3 years STANDARD_DEVIATION 15.35 |
| Baseline BMI | 27 kg/cm2 STANDARD_DEVIATION 6.5 | 26.8 kg/cm2 STANDARD_DEVIATION 9.42 | 27.1 kg/cm2 STANDARD_DEVIATION 11.02 | 26.9 kg/cm2 STANDARD_DEVIATION 9.18 |
| Sex: Female, Male Female | 101 Participants | 119 Participants | 113 Participants | 333 Participants |
| Sex: Female, Male Male | 17 Participants | 10 Participants | 9 Participants | 36 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 95 / 118 | 107 / 128 | 97 / 122 |
| serious Total, serious adverse events | 4 / 118 | 4 / 128 | 0 / 122 |
Outcome results
Primary
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.
Time frame: Baseline to Week 24
Population: Intent-to-Treat (ITT) Analysis set
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| PLACEBO | 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 | 48 participants |
| Pentosan Polysulfate Sodium (ELMIRON) 100MG QD | 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 | 51 participants |
| Pentosan Polysulfate Sodium (ELMIRON) 100MG TID | 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 | 52 participants |
Secondary
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.
Time frame: Baseline to Week 24
Population: Intent-to-Treat (ITT) Analysis set
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| PLACEBO | 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 | 55 participants |
| Pentosan Polysulfate Sodium (ELMIRON) 100MG QD | 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 | 55 participants |
| Pentosan Polysulfate Sodium (ELMIRON) 100MG TID | 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 | 60 participants |