B1871019 Phase 2 Study in Polycystic Kidney Disease
Research type
Research Study
Full title
A Phase 2, Multicentre, randomised, double -blind, placebo controlled study of the safety, clinical activity and pharmacokinetics of Bosutinib (PF-05208763) versus placebo in subjects with autosomal dominant polycystic kidney disease ( ADPKD)
IRAS ID
78732
Sponsor organisation
Pfizer Inc.
Eudract number
2010-023017-65
Clinicaltrials.gov Identifier
Research summary
This study will evaluate whether treatment with bosutinib can slow progression of autosomal dominant polycystic kidney disease (ADPKD). The primary population studied in this study is patients with a diagnosis of ADPKD, a total kidney volume =750 ml who have not experienced substantial decline in renal function [estimated glomerular (kidney) filtration rate (eGFR) = 60 ml/min]. This study will also evaluate effectiveness of bosutinib treatment in an exploratory population of patients with a total kidney volume =750 ml who have developed moderate renal impairment. Evaluation of the later, exploratory, population will not begin until a separate study demonstrates that bosutinib dose adjustment is not required in the setting of moderate renal impairment. This study will compare effectiveness of two different doses of bosutinib, 400mg/day and 200 mg/day with that of placebo. The primary measure of treatment effectiveness is the rate of kidney enlargement, as measured by MRI, following an initial 24 month treatment period. This study also incorporates secondary measures of treatment effectiveness which include the rate of decline in kidney function (eGFR) versus placebo following 24 months of treatment, frequency of onset or worsening of back or flank pain, frequency of onset or worsening of hypertension and measurements of study drug concentrations in the blood. If study results following 24 months of treatment show that bosutinib treatment is associated with a significant reduction in both the annualized rate of kidney enlargement and in the rate of decline of kidney function (eGFR) compared to placebo, then study drug treatment will continue for up to 46 additional months with a long term objective of determining if a bosutinib mediated reduction in the rate of kidney enlargement (versus placebo) over 24 months is predictive for achieving a longer term benefit of reduction in the cumulative decline in renal function (eGFR).
REC name
West of Scotland REC 1
REC reference
11/AL/0327
Date of REC Opinion
2 Aug 2011
REC opinion
Further Information Favourable Opinion