A Study to Evaluate the Efficacy and Safety of ALN-HSD in Adult Participants With Non-alcoholic Steatohepatitis (NASH) With Fibrosis With Genetic Risk Factors

• Status: Not yet recruiting
• Trial end date: 2026-08-17
• Target enrollment: 0
• Participant gender: All

Summary

The primary objective of Study 1 and Study 2 is to evaluate the effect of ALN-HSD on histopathologic assessment of liver fibrosis.

The secondary objectives of Study 1 and Study 2 are:

• To evaluate the effect of ALN-HSD on histopathologic assessment of steatohepatitis

• To evaluate the effect of ALN-HSD on serum biomarkers of hepatocellular injury

• To evaluate the effect of ALN-HSD on serum biomarkers of fibrosis

• To evaluate the effect of ALN-HSD on HSD17B13 expression

• To evaluate the effect of ALN-HSD on hepatocellular apoptosis

• To evaluate the effect of ALN-HSD on slowing progression of liver disease

• To evaluate safety and tolerability of ALN-HSD

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Regeneron Pharmaceuticals

Criteria

Key Inclusion Criteria:

1. A diagnosis of NASH with fibrosis (F) stage 2 or 3

2. NAS score ≥4 according to the NASH CRN histological scoring system

3. Meets genotype criteria for study enrollment in either Study 1 or Study 2

Key Exclusion Criteria:

1. Evidence of other forms of known chronic liver disease

2. Known history of alcohol or other substance abuse within the last year

3. History of Type 1 diabetes

4. Bariatric surgery within prior approximately 5 years or planned during the study period

5. Prior exposure to any investigational drug targeting HSD17B13 or PNPLA3 (eg, ALNHSD or ARO-HSD, AZD2693)

Note: Other protocol-defined Inclusion/Exclusion Criteria apply