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Table 2 Primary, secondary and exploratory outcome measures of the BAT117213 study

From: BAT117213: Ileal bile acid transporter (IBAT) inhibition as a treatment for pruritus in primary biliary cirrhosis: study protocol for a randomised controlled trial

1. Primary outcome measures:
• Safety assessment following repeat doses of oral GSK2330672
Safety will be assessed using clinical haematology, clinical chemistry, urinalysis, single 12-lead electrocardiograms (ECGs), vital sign measurements including systolic and diastolic blood pressure (BP) and pulse rate.
• Tolerability assessment using Gastrointestinal Symptom Rating Scale (GSRS)
Subjects will be asked to complete GSRS, a validated scale and the scale will be used to assess symptoms experienced by subject over the preceding 5 to 7 days
• Faecal occult blood (FOB) testing
FOB monitoring for symptomatic or visible gastrointestinal bleeding or asymptomatic occult bleeding
2. Secondary outcome measures:
• Subject reported outcomes-daily pruritus 0 to 10 point scale
This scale will be implemented to measure symptoms of itching as well as other associated symptoms twice daily in the morning and evening (approximately the time of drug dosing). The severity of itching symptoms from “0” (no itching) to “10” (worst possible itching) will be recorded
• Subject reported outcomes-5D-itch scale
The 5-D itch scale covers five dimensions of itching experienced by subjects including duration, degree, direction, disability and distribution
• Subject reported outcomes-PBC-40 quality of life (QoL) scale
The PBC-40 QoL scale has six domains; cognitive, itch, fatigue, social, emotional and (other) symptoms
• Measurement of serum profiles of total bile acid concentrations and 7-alpha hydroxy-4-cholesten-3-one (C4). C4 is the first committed step of bile acid synthesis from cholesterol
• Steady-state pharmacokinetics (PK) assessment of UDCA and its taurine and glycine conjugates taurodeoxycholic acid (TUDCA) and glycoursodeoxcholic acid (GUDCA).
Blood sample will be collected for measurements of steady state PK parameters of UDCA and its metabolites including maximum observed plasma concentration (Cmax), time to Cmax (tmax) and terminal phase half-life (t1/2).
3. Exploratory outcome measures:
• Markers of disease progression: ALT/AST, AP, GGT, bilirubin, albumin, PT/INR
• An exit interview conducted at end of follow-up phase to assess subject’s experience of benefits and disadvantages with GSK2330672
• Pharmacogenomics for genes related to pruritus and GSK2330672 response
• Metabonomics to study serum bile acid species, serum autotaxin and FGF-19 before and after treatment with GSK2330672
• Microbiomics to study gut microbiota in PBC patients with pruritus