Phenotype Variability in Interstitial Cystitis Clinical Trial Recruitment

Area of Science:

• Urology
• Clinical Trials
• Patient Phenotyping

Background:

• Interstitial cystitis/bladder pain syndrome (IC/BPS) requires phenotype-specific therapies for effective treatment.
• Current clinical trial designs often lack patient phenotype stratification, leading to generalized treatment approaches.
• The American Urological Association (AUA) guidelines recommend phenotype recognition for IC/BPS.

Purpose of the Study:

• To characterize IC/BPS clinical trials that incorporate patient phenotype into eligibility criteria.
• To assess the alignment of IC/BPS trial design with AUA guidelines regarding phenotype recognition.
• To evaluate trends in phenotype inclusion based on funding, intervention type, and guideline updates.

Main Methods:

• Systematic review of registered IC/BPS clinical trials from ClinicalTrials.gov.
• Inclusion criteria: IC/BPS diagnosis and pain-related outcomes.
• Assessment of phenotype recognition, intervention type, funding, and registration date relative to 2015 AUA guidelines.

Main Results:

• Only 21.8% of 170 trials stratified by phenotype, primarily focusing on bladder-centric presentations.
• Industry-funded trials (40.5%) were more likely to include phenotypic stratification than non-industry funded trials (16.5%).
• Phenotype inclusion increased post-2015 AUA guidelines (26.6% vs. 15.8%), and was more common in physical therapy, psychosocial, and drug studies.

Conclusions:

• The limited incorporation of IC/BPS phenotypes in clinical trials impedes the evaluation of real-world treatment efficacy.
• A methodological gap exists in aligning trial design with patient symptom variability.
• Integrating phenotypic approaches into trial design is essential for developing targeted and effective IC/BPS therapies.