DDT-BMQ-000146 Assessing clinically meaningful change in PSC as determined by quantitative MRCP (MRCP+) measured number of strictures - Abstract
MRCP+ metrics are quantitative markers of the biliary tree which can be used to inform
biliary tree health. These markers have shown promise as dynamic biomarkers which
can be used to support disease monitoring and, by extension, support the assessment
of the effects of pharmacotherapies. In this project, Perspectum Inc. seeks to complete
the necessary evidence to qualify the MRCP+ metric number of strictures as a
monitoring Drug Development Tool for PSC. It is envisioned that this MRCP+ metric
will be used in conjunction with clinical assessments, to identify patients who are more
likely to meet endpoints (including biochemical and imaging endpoints) in clinical trials
signifying appropriate response to treatments.
With the funding from this Drug Development Tools Research Grant, and leveraging
the close collaboration with the FDA, we will perform a retrospective multi-center
longitudinal observational study in 150 participants.
Primary objective:
1. To collect and analyze MRCP data to generate 3D models and
associated MRCP+ metrics which will be used to determine the change
in the MRCP+ metric ‘number of strictures’ in relation to disease
progression.
Secondary Objectives:
1. To collect clinical data which can be used to determine the
prognostic utility of change in the MRCP+ metric ‘number of strictures’
for predicting clinical outcomes (including cholangitis, requirement for
ERCP, cirrhosis, hepatic decompensation, hepatobiliary malignancies,
liver transplant or death).
2. To collect clinical data which can be used to determine whether
change in predicts incidence free transplant free survival.
The study will be performed in collaboration with members of the CALiD registry who
will include a representative sample of their patient population. Patients must have at
least two time points of interim duration of 6 months or more with associated clinical
data.
