Withdrawal Period study of LUM001 in patients with Alagille Syndrome
Research type
Research Study
Full title
Long-Term, Open-Label Study with a Double-Blind, Placebo Controlled, Randomized Drug Withdrawal Period of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Alagille Syndrome
IRAS ID
173548
Contact name
Noelia Martinez Lopez
Contact email
Sponsor organisation
Lumena Pharmaceuticals LLC
Eudract number
2013-005373-43
Duration of Study in the UK
1 years, 1 months, 30 days
Research summary
This is a Long-Term, Open-Label Study with a Double-Blind, Placebo Controlled, Randomized Drug Withdrawal Period of LUM001, an Apical Sodium-Dependent Bile Acid Transporter Inhibitor (ASBTi), in Patients with Alagille Syndrome. Approximately 30 subjects will be enrolled in this study.The study is divided into 4 parts: a 6-week open-label, dose escalation period at doses up to 400 μg/kg/day, a 12-week stable dosing period, a 4-week randomized, double-blind, placebo-controlled drug withdrawal period, and a 26-week long-term stable dosing period at doses up to 400 μg/kg/day. The primary efficacy evaluation will be the mean change in fasting serum bile acid levels from Week 18 to Week 22 for those who responded to LUM001 treatment, which is defined as subjects who had a >50% reduction in serum bile acid levels from baseline to week 12.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
15/LO/0342
Date of REC Opinion
21 Apr 2015
REC opinion
Further Information Favourable Opinion