Phase II Study Efficacy and Safety of Two Dosing Regimens of MN-001 in Patients With Interstitial Cystitis

Study Overview
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Brief Summary
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To evaluate the safety and efficacy of 8 weeks of treatment with MN-001 at 500 mg bid, 500 mg once daily vs. placebo in patients with Interstitial Cystitis.

Detailed Description
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This is a randomized, double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of two dosing regimens of MN-001 in patients with Interstitial Cystitis (IC). Patients will be screened for study eligibility within seven to nine days of randomization. Eligible patients will be randomized in a 1:1:1 ratio to receive either 500 mg MN-001 bid, 500 mg MN-001 once daily or placebo. Patients will be dispensed study drug beginning at Baseline (Visit 2) and will return to the study center for Visit 3 (28 days ± 2 days after Baseline), and Visit 4 (56 days ± 2 days after Baseline), at end of study for safety and efficacy assessments. The patient will be contacted by telephone at Week 6 (42 days ± 2 days after Baseline) for an interim follow up. Study drug will be dispensed at Visits 2 and 3. Safety assessments will include adverse events, physical examinations, clinical laboratory testing, and changes in vital signs. Efficacy assessments include percentage of patients at least moderately improved for each treatment group using the patient reported Global Response Assessment (GRA) (see Appendix 1). Secondary assessments include a decrease in bladder pain/urgency based on change in the patient rating from baseline to endpoint using the GRA (see Appendix 1), modified Pelvic Pain and Urgency/Frequency (PUF) Patient Symptom Scale (see Appendix 2) and the O'Leary Sant IC Symptom and Problem Index.

Official Title
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A Phase II, Randomized, Double-blind, Placebo-controlled, Multi-center Study to Evaluate the Efficacy and Safety of Two Dosing Regimens of MN-001 in Patients With Interstitial Cystitis

Conditions
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Interstitial Cystitis

Intervention / Treatment
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* Drug: MN-001 BID
* Drug: MN-001
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female ≥ 18 years of age with a diagnosis of moderate to severe IC; Bladder pain ≥ 6 months prior to baseline; Urinary frequency of ≥ 8 ≤ 30 micturitions within 24 hours while awake; Nocturia ≥ 2x/night; Males and females of child-bearing potential (not surgically sterile or post-menopausal) must be abstinent or agree to use an study-accepted contraceptive regimens throughout the study: Female patients of child bearing age must have a negative urine pregnancy test at screening; Must provide a signed informed consent. Exclusion Criteria: Male or females < 18 years of age; Initiation of new IC medication ≤ 30 days prior to baseline; Treatment with Elmiron ≤ 120 days prior to baseline; Treatment with bladder hydro-distention ≤ 6 months prior to baseline; Treatment with intravesical therapy ≤ 60 days prior to baseline; History of previous procedure(s) (e.g., augmentation cytoplasty, cystectomy or cystolysis) that has affected bladder function; Active genital herpes or vaginitis ≤ 90 days prior to baseline; Urinary tract or prostatic infection ≤ 90 days prior to baseline; History of urethral diverticulum; History of bladder or ureteral calculi; History of cyclophosphamide or chemical cystitis, urinary tuberculosis or radiation cystitis; History of bladder tumors; History of uterine, cervical, vaginal, prostatic or urethral cancer ≤ 5 years prior to baseline; Patient is currently pregnant, lactating or likely to become pregnant during the study; Participated in another clinical study with an investigational drug or device ≤ 30 days prior to baseline.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: MN-001<br> | Drug: MN-001 BID<br>* Eligible patients received 500 mg MN-001 bid<br>Drug: MN-001<br>* Eligible patients received 500 mg MN-001 once daily (qd)<br>|
| Placebo Comparator: MN-001 once daily<br>placebo tablets | Drug: Placebo<br>* Eligible patients received placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number Subjects at Least Moderately Improved for Each Treatment Group in Patient Reported Global Response Assessment (GRA) | The primary endpoint was the GRA overall change in their condition at Week 8. Each patient completed the questionnaire that rated the improvement in their IC symptoms based on responses to the GRA questions. Each question asked the patient to describe the OVERALL CHANGE in pain, urgency, frequency or overall change in their problem compared to the status before taking the study medication. Each parameter was rated on a 7 point scale: markedly worse, moderately worse, mildly worse, same, mildly improved, moderately improved and markedly improved. | 8 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Responders for GRA Assessment in Their Condition at Week 4. | Responders were defined as patients who were 'moderately improved' or 'markedly improved' and non-responders were defined as patients who were 'markedly worse', 'moderately worse', 'mildly worse', no change, or 'mildly improved' on the GRA assessments. | 4 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Interstitial Cystitis, urgency, frequency, MN-001, Global Response Assessment, bladder pain/urgency, O'Leary Sant IC Symptom and Problem Index