A two-part study to investigate the effects of two doses of golexanolone in adults with PBC
Research type
Research Study
Full title
A randomised, double-blind, placebo-controlled, two-part study to evaluate the pharmacokinetics, safety and tolerability, and preliminary efficacy of two dose levels of golexanolone in subjects with primary biliary cholangitis, fatigue, and cognitive dysfunction.
IRAS ID
1003871
Contact name
Magnus Doverskog
Contact email
Sponsor organisation
Umecrine Cognition AB
Eudract number
2022-000422-16
Research summary
Primary biliary cholangitis (PBC) is a serious chronic liver disease that harms the liver’s ability to function. Patients with PBC can experience cognitive dysfunction: inability to think, learn and remember clearly. Also they experience significant fatigue (tiredness).
The active investigational medicinal product - golexanolone – is a new drug that was developed for treatment of the spectrum of cognitive dysfunction associated with chronic liver disease. Based on research in animals and patients with cirrhosis, it is assumed that golexanolone may offer normalization of cognitive dysfunction and fatigue in patients with PBC.
Up to date, golexanolone has been investigated in research studies in more than 130 people, including healthy adults, patients with cirrhosis, and patients with idiopathic hypersomnia, in single and multiple doses up to 200 mg daily. In all human studies to date, golexanolone has been well tolerated.
The present study was designed to evaluate how safe and well tolerated golexanolone is, how golexanolone is absorbed, modified, and removed from the body, and evaluate the effects of golexanolone on your symptoms. The effects of golexanolone will be compared with placebo (inactive substance).
The study will be conducted in two parts, part A and part B, and in the United Kingdom, only part B will be conducted. The purpose of part B is to:
• evaluate how safe and well tolerated two different doses of golexanolone are when administered orally twice daily for 28 days
• evaluate the effects of golexanolone on your symptoms
In Part B participants who qualify will be randomly assigned in a 1:1:1 ratio to receive either golexanolone 40 mg or golexanolone 80 mg or placebo orally twice daily for 28 days.
Previously prescribed PBC therapy will be allowed as long as it is stable during the study.
The total duration of part B for a participant will be maximum 8 weeks, with 6 visits to a clinic and one phone call.REC name
East Midlands - Leicester Central Research Ethics Committee
REC reference
22/EM/0196
Date of REC Opinion
12 Oct 2022
REC opinion
Further Information Favourable Opinion