A Phase 2 Clinical Trial to Evaluate the Efficacy and Safety of VSA006 in Chinese NASH Patients

NCT06322628 | Active Not Recruiting Phase 2 DMC

• 1 other identifier: VSA006-2001
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 1

Brief Summary

Human genetic studies have shown that loss of function (LOF) mutations in HSD17β13 gene have a protective effect on the progression of alcohol-related and non-alcohol-related liver diseases, such as NASH, without significant adverse phenotypes. VSA006 is a siRNA drug targeting HSD17β13 mRNA in the liver and reduce the protein level of HSD17β13. Based on phase 1 study results in healthy volunteers and NASH/suspected NASH patients, this phase 2 study is designed to evaluate the efficacy, safety, PK profiles and immunogenicity of VSA006 in Chinese NASH patients.

Conditions

Nash

Keywords

efficacy; safety; HSD17β13

Outcome Measures

Primary Outcomes (2)

• Percentage of Participants Achieving ≥ 1 Stage Improvement in Histological Fibrosis with no Worsening of NASH

  No Worsening of NASH is defined as no increase in inflammation, ballooning, or steatosis scores in the NAS score.

  At week 52

• Percentage of Participants Achieving NASH Improvement with no Worsening of Fibrosis

  NASH Improvement indicates a reduction by at least 2 points in the NAS score, with at least one-point reduction in ballooning without increase in steatosis score.

  At week 52

Secondary Outcomes (10)

• Compared with placebo, the percentage change in serum alanine aminotransferase (ALT)

  At week 24, week 52 and week 82

• Compared with placebo, the change in liver fat fraction from baseline and liver fat percentage change from baseline

  At week 24 and week 52

• Compared with placebo, the percentage of participants with a > 30% decrease in liver fat fraction from baseline

  At week 24 and week 52

• Compared with placebo, the change and percentage change in noninvasive markers of fibrosis from baseline: FIB-4, NAFLD fibrosis score, and AST/PLT ratio index (APRI)

  At week 24, week 52 and week 82

• Percentage of Participants Achieving NASH Resolution with no Worsening of Fibrosis

  At week 52

Study Arms (4)

VSA006 low dose

EXPERIMENTAL

Drug: VSA006

VSA006 low dose comparator

PLACEBO COMPARATOR

Drug: Placebo

VSA006 high dose

EXPERIMENTAL

Drug: VSA006

VSA006 high dose comparator

PLACEBO COMPARATOR

Drug: Placebo

Interventions

VSA006 DRUG

every 12 weeks, subcutaneous injections

VSA006 high dose; VSA006 low dose

Placebo DRUG

every 12 weeks, subcutaneous injections

VSA006 high dose comparator; VSA006 low dose comparator

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• body mass index (BMI) of 24-35 kg/m2 ;
• NASH patients confirmed by liver histopathology: NAS score is ≥ 4 and CRN fibrosis is F2 or F3 ;
• At screening, ALT is \> ULN;
• At screening, the liver fat content measured by MRI-PDFF is ≥ 8%;
• Weight change \< 5% at least 3 months prior to screening;
• For patient with T2DM, the hypoglycemic agents and HbA1c is stable

You may not qualify if:

• Pregnant or lactating women;
• Previous diagnosis of alcoholic liver disease or hepatitis/liver disease due to other causes;
• Previous or current diagnosis of cirrhosis or decompensated cirrhosis;
• Previous or current diagnosis of hyperthyroidism, hypothyroidism, or other diseases that can lead to fatty degeneration of liver;
• Participants diagnosed with type 1 diabetes, or with unstable type 2 diabetes
• Participants who cannot receive an MRI examination;

Sponsors & Collaborators

• Visirna Therapeutics HK Limited: lead

Study Sites (1)

Beijing Tsinghua Changgeng Hospital

Beijing, Beijing Municipality, China

Location

MeSH Terms

Conditions

Non-alcoholic Fatty Liver Disease

Condition Hierarchy (Ancestors)

Fatty Liver; Liver Diseases; Digestive System Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 22, 2023
• First Posted: March 21, 2024
• Study Start: April 22, 2024
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): January 1, 2027
• Last Updated: March 12, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)