Efficacy and safety of intravesical instillation of KRP-116D (50% dimethyl sulfoxide solution) for interstitial cystitis/bladder pain syndrome in Japanese patients: A multicenter, randomized, double-blind, placebo-controlled, clinical study.

Abstract

ObjectiveTo evaluate the efficacy and safety of intravesical KRP‐116D, 50% dimethyl sulfoxide solution compared with placebo, in interstitial cystitis/bladder pain syndrome patients.MethodsJapanese interstitial cystitis/bladder pain syndrome patients with an O’Leary‐Sant Interstitial Cystitis Symptom Index score of ≥9, who exhibited the bladder‐centric phenotype of interstitial cystitis/bladder pain syndrome diagnosed by cystoscopy and bladder‐derived pain, were enrolled. Patients were allocated to receive either KRP‐116D (n = 49) or placebo (n = 47). The study drug was intravesically administered every 2 weeks for 12 weeks.ResultsFor the primary endpoint, the change in the mean O’Leary‐Sant Interstitial Cystitis Symptom Index score from baseline to week 12 was −5.2 in the KRP‐116D group and −3.4 in the placebo group. The estimated difference between the KRP‐116D and placebo groups was −1.8 (95% confidence interval −3.3, −0.3; P = 0.0188). Statistically significant improvements for KRP‐116D were also observed in the secondary endpoints including O’Leary‐Sant Interstitial Cystitis Problem Index score, micturition episodes/24 h, voided volume/micturition, maximum voided volume/micturition, numerical rating scale score for bladder pain, and global response assessment score. The adverse drug reactions were mild to moderate, and manageable.ConclusionsThis first randomized, double‐blind, placebo‐controlled trial shows that KRP‐116D improves symptoms, voiding parameters, and global response assessment, compared with placebo, and has a well‐tolerated safety profile in interstitial cystitis/bladder pain syndrome patients with the bladder‐centric phenotype.