CCSJ137X2201
Research type
Research Study
Full title
A first-in-human, two-part (open label, and randomized/double blind/placebo controlled), single dose study of CSJ137 in erythropoietin-treated chronic hemodialysis patients with functional iron-deficiency anaemia.
IRAS ID
191830
Contact name
Iain MacDougall
Contact email
Sponsor organisation
Novartis Pharma Services AG
Eudract number
2015-002475-13
Duration of Study in the UK
1 years, 6 months, 1 days
Research summary
This is a first-in-human, two-part, single-dose, non-confirmatory study of CSJ137 to assess safety, tolerability, PK, PD and efficacy in a chronic hemodialysis patient population. Part 1 is a first-in-human, single-dose, open-label dose-finding study. Part 2 is a randomized, double-blind, placebo-controlled, single-dose study that will compare two dose levels of CSJ137. The purpose of this study is to evaluate whether CSJ137 warrants further clinical development in anemia associated with chronic kidney disease.
Part 1
The aims of Part 1 are (a) to evaluate single-dose safety, PK, PD, and tolerability, and (b) to determine the minimum PAD of CSJ137, defined as the lowest dose tested in Part 1 that results in an increase in Hgb (median change from baseline ~ 0.5 g/dL) at 29 days post-dose. patients will be assigned to one of up to 6 open label dose cohorts of up to 6 patients each. The minimum PAD of CSJ137, as defined above, will be the lower dose arm selected for Part 2.Part 2
The aims of Part 2 are (a) to evaluate safety, PK, PD, and tolerability and (b) to determine efficacy based on Hgb changes in response to single dose CSJ137 vs. placebo. Part 2 will include up to three arms: Up to two CSJ137 dose arms and a placebo arm. The two CSJ137 dose arms will be derived from data generated in Part 1. Part 2 will include approximately 60 patients with a randomization of 1:1:1 to the three arms.REC name
London - West London & GTAC Research Ethics Committee
REC reference
16/LO/0048
Date of REC Opinion
27 Jan 2016
REC opinion
Unfavourable Opinion