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Design and Endpoints for Clinical Trials in Primary Sclerosing Cholangitis
Cyriel Y Ponsioen1, Keith D Lindor2, Ruby Mehta3
1Department of Gastroenterology & Hepatology, Academic Medical Center, Amsterdam, The Netherlands.
Hepatology (Baltimore, Md.)
|March 26, 2018
Summary
Primary sclerosing cholangitis (PSC) is a rare liver disease lacking effective treatments. This review examines endpoints for clinical trials to better measure treatment success in PSC patients.
Area of Science:
- Hepatology
- Gastroenterology
- Clinical Trial Design
Background:
- Primary sclerosing cholangitis (PSC) is a rare, chronic cholestatic liver disease.
- Currently, no approved effective therapies exist for PSC.
- There is a critical need for standardized outcome measures in PSC clinical trials.
Purpose of the Study:
- To review regulatory perspectives on clinical trial endpoints for PSC.
- To evaluate the natural history data relevant to endpoint selection.
- To identify and discuss potential clinical and surrogate endpoints for evaluating therapeutic interventions in PSC.
Main Methods:
- Comprehensive literature search of PubMed and regulatory agency websites.
- Review of published natural history studies of PSC.
- Analysis of proposed endpoints in existing and ongoing PSC clinical trials.
Main Results:
- Regulatory guidance emphasizes meaningful clinical benefit but lacks specific PSC endpoints.
- Natural history data show significant variability, complicating endpoint validation.
- Several candidate endpoints, including liver biochemistry, imaging, and patient-reported outcomes, are discussed.
Conclusions:
- Standardized and validated endpoints are crucial for advancing PSC therapeutic development.
- A combination of clinical and surrogate endpoints may be necessary to capture treatment effects.
- Further research and consensus are needed to establish optimal endpoints for PSC trials.
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