Rational clinical trial design in PSC is challenging for several reasons including the lack of standardized definitions for diagnosis, disease staging, validated surrogate markers, and effective therapeutic study endpoints. Nonetheless, clinical trials for PSC patients are increasingly performed around the world reflecting the rising focus on this disease by investigators, industry, regulators and patient advocacy organizations.
The IPSCSG aims to meaningfully impact this area of need through the Clinical Trial Design Working Group. The roles of this working group are to:
1. Develop a PSC Clinical Trials Toolkit to assist in the design of clinical trials
2. Promote discussion of the anticipated PSC trial endpoint document by the FDA and develop subsequent iterations over time
3. Facilitate coordination of investigator initiated clinical trials within the IPSCSG
The development of a PSC Clinical Trial Toolkit will provide guidance in the design of Phase 1, 2 and 3 studies through identification of relevant clinical and mechanistic endpoints, subject selection based on relevant disease sub-phenotyping, appropriate trial duration, and data analysis from completed trials.
Please contact David.Assis@yale.edu for questions or to become involved in this working group.