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

Visirna Therapeutics

Status and phase: Not yet enrolling; Phase 2

Conditions: Nash

Treatments: Drug: Placebo; Drug: VSA006

Study type: Interventional

Funder types: Industry

Identifiers

NCT06322628

VSA006-2001

Details and patient eligibility

About

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.

Enrollment: 45 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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

Exclusion criteria

• 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;

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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45 participants in 4 patient groups, including a placebo group

VSA006 low dose

Experimental group

Treatment:

Drug: VSA006

VSA006 low dose comparator

Placebo Comparator group

Treatment:

Drug: Placebo

VSA006 high dose

Experimental group

Treatment:

Drug: VSA006

VSA006 high dose comparator

Placebo Comparator group

Treatment:

Drug: Placebo