Clinical Trial Endpoints for Perianal Fistulizing Crohn's Disease

Area of Science:

• Gastroenterology
• Clinical Trial Design

Background:

• Perianal fistulizing Crohn's disease (pCD) causes severe symptoms and impacts quality of life.
• Lack of validated endpoints in randomized controlled trials (RCTs) hinders optimal treatment recommendations.

Purpose of the Study:

• To review considerations for pCD RCTs.
• To provide expert opinion on current endpoints for pCD clinical trials.

Main Methods:

• Analysis of primary endpoints in 10 pCD phase III/IV RCTs and post hoc analyses.
• Evaluation of fistula closure, clinical remission, and patient-reported outcomes.

Main Results:

• No universal definitions for clinical fistula response/remission exist.
• Current endpoints lack validation, reproducibility, and optimal effect sizes.
• Physical examination-based assessments are common but unvalidated.

Conclusions:

• Future pCD RCTs should employ co-primary clinical and radiologic assessments.
• Standardized, objective endpoints are crucial for evaluating novel therapies and improving patient outcomes.