A Phase 1 Study to Evaluate ALN-HSD in HV and Adult Patients with NASH
Research type
Research Study
Full title
A Phase 1, Randomized, Double-Blind, Placebo-Controlled, 2-Part Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single Dose ALN-HSD in Healthy Adult Subjects and Multiple Dose ALN-HSD in Adult Patients with Nonalcoholic Steatohepatitis (NASH)
IRAS ID
286227
Contact name
Jorg Taubel
Contact email
Sponsor organisation
Alnylam UK Limited
Eudract number
2020-000847-29
Duration of Study in the UK
0 years, 10 months, 17 days
Research summary
Non-alcoholic fatty liver disease (NAFLD) is the most rapidly growing cause of liver-related mortality and morbidity, affecting 25% of the global adult population [Paik 2020; Younossi 2019a]. The prevalence of non-alcoholic steatohepatitis (NASH) in the United States (US), Europe, and other developed countries is between 1.5% and 6.5%, and it is estimated that 7% to 30% of NAFLD patients progress to NASH [Farrell and Larter 2006; Williams 2011; Younossi 2016]. There are currently no approved pharmacological treatments for NASH, despite the rising burden and increasing NASH prevalence.
ALN-HSD is a novel synthetic RNA interference (RNAi) therapeutic in development for the treatment of NASH. ALN-HSD contains a small interfering RNA (siRNA) targeting hydroxysteroid 17β dehydrogenase 13 messenger RNA (HSD17β13 mRNA). Based on the mechanism of RNA interference, ALN-HSD is specifically designed to reduce the liver synthesis of HSD17B13 protein. By doing this it is anticipated that ALN-HSD will halt and/or reduce liver injury, fibrosis, and inflammation that occur in NASH.
This study will be conducted in 2 parts: Part A; a single ascending dose part in healthy adult subjects, and Part B; a multiple dosing part in adult patients with NASH.
REC name
London - London Bridge Research Ethics Committee
REC reference
20/LO/0891
Date of REC Opinion
17 Sep 2020
REC opinion
Further Information Favourable Opinion