NCT04565717

A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH)

Terminated Phase 1 Last updated 17 May 2024

What this trial tests

Phase 1 trial testing ALN-HSD in Nonalcoholic Steatohepatitis in 6 participants. Terminated before completion.

Timeline

9 October 2020

Primary endpoint
6 January 2023

21 December 2023

Quick facts

Lead sponsor	Alnylam Pharmaceuticals
Phase	Phase 1
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	6
Start date	9 October 2020
Primary completion	6 January 2023
Estimated completion	21 December 2023
Sites	13 locations across Belgium, United Kingdom, Bulgaria, United States, Turkey (Türkiye)

Drugs / interventions tested

• ALN-HSD — full drug profile →
• Placebo

Conditions studied

• Nonalcoholic Steatohepatitis — all drugs for Nonalcoholic Steatohepatitis →
• NASH — all drugs for NASH →

Sponsor

Alnylam Pharmaceuticals — full company profile →

Who can join

Adults 18 to 65, any sex, with Nonalcoholic Steatohepatitis or NASH. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The purpose of this study is to evaluate the safety and tolerability of single ascending doses of ALN-HSD in healthy participants (Part A) and multiple doses of ALN-HSD in patients with NASH (Parts B and C).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Therapeutic siRNA: State-of-the-Art and Future Perspectives.
   Friedrich M, Aigner A. · · 2022 · cited 256× · PMID 35997897 · DOI 10.1007/s40259-022-00549-3
2. Targeted therapeutics and novel signaling pathways in non-alcohol-associated fatty liver/steatohepatitis (NAFL/NASH).
   Xu X, Poulsen KL, Wu L, Liu S, et al · · 2022 · cited 235× · PMID 35963848 · DOI 10.1038/s41392-022-01119-3
3. Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines.
   Liu M, Wang Y, Zhang Y, Hu D, et al · · 2025 · cited 62× · PMID 40059188 · DOI 10.1038/s41392-024-02112-8
4. Mouse models of nonalcoholic fatty liver disease (NAFLD): pathomechanisms and pharmacotherapies.
   Fang T, Wang H, Pan X, Little PJ, et al · · 2022 · cited 57× · PMID 36263163 · DOI 10.7150/ijbs.65044
5. Review article: the role of HSD17B13 on global epidemiology, natural history, pathogenesis and treatment of NAFLD.
   Amangurbanova M, Huang DQ, Loomba R. · · 2023 · cited 46× · PMID 36349732 · DOI 10.1111/apt.17292
6. HSD17B13: A Potential Therapeutic Target for NAFLD.
   Zhang HB, Su W, Xu H, Zhang XY, et al · · 2021 · cited 41× · PMID 35071330 · DOI 10.3389/fmolb.2021.824776
7. Advances in the genetics of nonalcoholic fatty liver disease.
   Ajmera V, Loomba R. · · 2023 · cited 30× · PMID 37144531 · DOI 10.1097/mog.0000000000000927
8. Hepatocyte targeting <i>via</i> the asialoglycoprotein receptor.
   Ramírez-Cortés F, Ménová P. · · 2025 · cited 22× · PMID 39628900 · DOI 10.1039/d4md00652f