IBAT Inhibitor A4250 for Cholestatic Pruritus
|Study Design:||Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
|Official Title:||An Exploratory, Phase IIa Study to Demonstrate the Safety and Efficacy of A4250 in Patients With Primary Biliary Cirrhosis and Cholestatic Pruritus|
- •Safety and Tolerability assessed by the occurrence of treatment-emergent SAEs during the four weeks of treatment with A4250 [ Time Frame: 4 weeks ]
- Safety laboratory measurements [ Time Frame: 4 weeks ]Changes in safety laboratory test results (including hematology, clinical chemistry and urinalysis) from baseline to Day 28 of A4250 treatment
- VAS-Itch [ Time Frame: 4 weeks ]Change in VAS-Itch (most severe itch during last 24 hrs) during the fourth treatment week of A4250
- Itching scale [ Time Frame: Four weeks ]Change in PBC40
- Bile acid evaluation [ Time Frame: Four weeks ]Change in serum and fecal bile acids (BAs)
- Liver biochemistry [ Time Frame: Four weeks ]Change in ALP
|Study Start Date:||January 2015|
|Study Completion Date:||October 2016|
|Primary Completion Date:||October 2016 (Final data collection date for primary outcome measure)|
A4250 once daily
A4250 once daily
Other Name: IBAT Inhibitor A4250
A4250PBCpruritus (EudraCT 2014-004070-42) is an open-label exploratory study.
The primary objective of this study is to assess the safety and tolerability of A4250, 1.5 - 3 mg orally during a four-week treatment period, in patients with PBC and cholestatic pruritus, as determined by the occurrence of treatment-emergent serious adverse events (SAEs).
Other safety objectives of this study include assessment of the safety and tolerability of A4250 during a four-week treatment period, as determined by the occurrence of treatment-emergent adverse events (AEs) and changes in other safety parameters including liver and kidney function tests and vital signs.
Exploratory efficacy objectives of this study are to demonstrate the efficacy of A4250 orally on pruritus variables and on QoL and lysophosphatidic acid formation as well as evaluation of changes in pharmacodynamic parameters of bile acid metabolism such as serum and fecal bile acids, C4 and fibroblast growth factor 19 (FGF19) assessments and assessment of surrogate markers of cholestatic liver disease such as alkaline phosphatase, transaminases and bilirubin.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02360852
|Principal Investigator:||Hanns-Ulrich Marschall, MD||Sahlgrenska Academy, Institute of Medicine,|