In short
A groundbreaking clinical trial is underway to assess the effectiveness, safety, and tolerability of AZD2693 Sodium, a promising new drug for treating non-cirrhotic non-alcoholic steatohepatitis (NASH) with fibrosis. This study focuses on adult participants who carry a specific genetic marker, offering hope for a targeted treatment approach in NASH management.
At a glance
- Drug Name
- AZD2693 Sodium
- Condition Studied
- Non-cirrhotic non-alcoholic steatohepatitis (NASH) with fibrosis
- Study Type
- Randomized, double-blind, placebo-controlled, multi-centre Phase 2b
- Primary Objective
- Assess effect on histological resolution of NASH without worsening fibrosis
- Key Inclusion Criteria
- Adults 18-75, PNPLA3 rs738409 148M risk allele carriers, histological evidence of NASH
- Treatment Duration
- 52 weeks
- Administration Route
- Subcutaneous injection
- Primary Endpoint
- Resolution of NASH without worsening of liver fibrosis after 52 weeks
What is AZD2693 SODIUM?
AZD2693 SODIUM is an investigational drug being developed by AstraZeneca for the treatment of non-alcoholic steatohepatitis (NASH) with fibrosis. It is a synthetic small molecule that is administered as a solution for injection. The drug is specifically designed to target individuals who carry a specific genetic marker called PNPLA3 rs738409 148M risk allele.
Target Condition: Non-Alcoholic Steatohepatitis (NASH)
Non-alcoholic steatohepatitis (NASH) is a severe form of non-alcoholic fatty liver disease. It is characterized by fat accumulation in the liver, inflammation, and liver cell damage. NASH can lead to fibrosis (scarring) of the liver and potentially progress to cirrhosis if left untreated. The condition targeted by AZD2693 SODIUM is specifically non-cirrhotic NASH with fibrosis, meaning the liver damage has not yet progressed to cirrhosis.
Clinical Trial Overview
A Phase 2b clinical trial is being conducted to evaluate the efficacy, safety, and tolerability of AZD2693 SODIUM in adults with non-cirrhotic NASH and fibrosis who carry the PNPLA3 genetic marker. The study is:
- Randomized: Participants are randomly assigned to either the treatment or placebo group
- Double-blind: Neither the participants nor the researchers know who is receiving the actual drug or placebo
- Placebo-controlled: Some participants receive a placebo for comparison
- Multi-center: Conducted at multiple medical centers
Eligibility Criteria
To participate in this study, individuals must meet specific criteria, including:
Inclusion Criteria:
- Age: 18 to 75 years old
- Carry the PNPLA3 rs738409 148M risk allele (a specific genetic marker)
- Have histological evidence of NASH based on a liver biopsy, with a NAS (NAFLD Activity Score) ≥ 4
- Have liver fibrosis stage F2 or F3 (moderate to advanced fibrosis)
Exclusion Criteria:
- Other liver diseases (e.g., alcoholic steatohepatitis, viral hepatitis)
- History of cirrhosis or liver decompensation
- Severe kidney disease
- Certain abnormal liver function tests
Study Objectives
The main goal of this study is to assess how well AZD2693 SODIUM works compared to a placebo in treating NASH. Specifically, the researchers aim to:
- Evaluate the effect on histological resolution of NASH (improvement in liver tissue appearance) after 52 weeks of treatment
- Assess improvements in liver fibrosis
- Measure improvements in the NAS score
Study Endpoints
The study will measure several outcomes to determine the effectiveness of AZD2693 SODIUM:
Primary Endpoint:
Resolution of NASH without worsening of liver fibrosis after 52 weeks. This means:
- A ballooning score of 0 (no liver cell ballooning)
- An inflammation score of 0 to 1 (little to no inflammation)
- Any degree of steatosis (fat in the liver)
Secondary Endpoints:
- Improvement in liver fibrosis by at least one stage without worsening of NASH
- At least a 2-point improvement in the NAS score
Drug Administration
AZD2693 SODIUM is administered as a solution for injection via subcutaneous use (an injection under the skin). The treatment period in this study is 52 weeks, which is about one year. The exact dosage is not specified in the provided information.
It's important to note that this drug is still in the investigational stage, and more research is needed to fully understand its effectiveness and safety profile for treating NASH.