A randomized, double-blind, placebo controlled trial of adalimumab for interstitial cystitis/bladder pain syndrome

Abstract

Purpose: The efficacy of adalimumab for the treatment of interstitial cystitis/bladder pain syndrome was investigated in a phase III, randomized, double-blind, placebo controlled, proof of concept study.

Materials and methods: Patients with interstitial cystitis/bladder pain syndrome were randomized to receive a loading dose of 80 mg subcutaneous adalimumab followed by 40 mg every 2 weeks or subcutaneous placebo for 12 weeks, and outcome measures were assessed. The incidence of adverse events was also assessed.

Results: Of a total of 43 patients 21 received adalimumab and 22 received placebo. Of the patients who received adalimumab, there was a statistically significant improvement demonstrated in the O'Leary-Sant Interstitial Cystitis Symptom and Problem Indexes (p = 0.0002), Interstitial Cystitis Symptom Index (p = 0.0011), Interstitial Cystitis Problem Index (p = 0.0002), and Pelvic Pain, Urgency, Frequency Symptom Scale (p = 0.0017) at 12 weeks compared to baseline. At 12 weeks 11 of 21 (53%) patients in the adalimumab group had a 50% or greater improvement in global response assessment (p ≤ 0.0001). There was not a statistically significant improvement in any outcome measure in patients receiving adalimumab compared to placebo. There were no significant adverse events.

Conclusions: Adalimumab treatment resulted in a statistically significant improvement in outcome measures compared to baseline in patients with moderate to severe interstitial cystitis/bladder pain syndrome. Adalimumab failed to demonstrate positive proof of concept compared to placebo due to a significant placebo effect.

Trial registration: ClinicalTrials.gov NCT01295814.

Keywords: GRA; IC/BPS; ICPI; ICSI; Interstitial Cystitis Problem Index; Interstitial Cystitis Symptom Index; O'Leary-Sant Interstitial Cystitis Symptom Index and Problem Index; OSPI; PUF; Pelvic Pain, Urgency, Frequency Symptom Scale; TNF-α; adalimumab; autoimmune diseases; cystitis; global response assessment; interstitial; interstitial cystitis/bladder pain syndrome; tumor necrosis factor-alpha; tumor necrosis factor-α.