ION224 for Non-alcoholic Fatty Liver Disease

Overseen By Study Coordinator

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Ionis Pharmaceuticals, Inc.

Must not be taking: Obeticholic acid, Ursodeoxycholic acid

Disqualifiers: Bariatric surgery, Organ transplant, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 49 weeks

160 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests if ION224 injections can help patients with NASH by reducing liver fat and improving liver health. The study will monitor changes in liver condition using imaging and blood tests over several months.

Will I have to stop taking my current medications?

You may need to stop taking certain medications, especially if they are associated with non-alcoholic fatty liver disease or if you are not on a stable dose. It's best to discuss your specific medications with the trial team to see if any changes are needed.

What makes the drug ION224 unique for treating non-alcoholic fatty liver disease?

The drug ION224 is unique for treating non-alcoholic fatty liver disease because there are currently no standard approved therapies for this condition, making any new treatment option potentially novel and significant. ^{1 2 3 4 5}

Are You a Good Fit for This Trial?

Inclusion Criteria

Your liver enzyme levels (ALT and AST) should be below 200 units per liter and remain stable.

You have not had a liver biopsy in the last 6 months.

Your total bilirubin level must be below 1.3 mg/dL and not changing over time.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

8 weeks

Treatment

Participants receive ION224 or placebo by subcutaneous injection every 4 weeks for up to 49 weeks

49 weeks

Post-Treatment

Participants are monitored for safety and effectiveness after treatment

12 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ION224
• Placebo

How Is the Trial Designed?

Group I: ION224 Experimental Treatment 1 Intervention

Multiple doses of ION224 will be administered by SC injection once every 4 weeks for up to 49 weeks.

Group II: Placebo Placebo Group 1 Intervention

Multiple doses of matching placebo will be administered by SC injection once every 4 weeks for up to 49 weeks.

Who Is Running the Clinical Trial?

Ionis Pharmaceuticals, Inc.

Lead Sponsor

Trials

151

Recruited

27,800+

Dr. Brett P. Monia

Ionis Pharmaceuticals, Inc.

Chief Executive Officer since 2020

PhD in Pharmacology from the University of Pennsylvania, BSc in Molecular Biology and Analytical Chemistry from Stockton State College

Dr. Eric Bastings

Ionis Pharmaceuticals, Inc.

Chief Medical Officer

MD

Published Research Related to This Trial

In a study of 187 participants with nonalcoholic steatohepatitis (NASH) receiving placebo treatment, 20% showed a significant reduction in liver fat content (PDFF) after 12 weeks, highlighting a notable 'placebo effect' that can impact trial outcomes.

The analysis revealed an average decrease of 2.3 units in absolute PDFF values after 24 weeks of placebo treatment, suggesting that future clinical trials should account for this effect when calculating sample sizes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

MRI Quantification of Placebo Effect in Nonalcoholic Steatohepatitis Clinical Trials. Nedrud, MA., Chaudhry, M., Middleton, MS., et al.[2023]

Citations

1. United States pubmed.ncbi.nlm.nih.gov

MRI Quantification of Placebo Effect in Nonalcoholic Steatohepatitis Clinical Trials. [2023]

2. Netherlands pubmed.ncbi.nlm.nih.gov

Impact of sodium glucose cotransporter 2 inhibitor on histological features and glucose metabolism of non-alcoholic fatty liver disease complicated by diabetes mellitus. [2020]

3. United States pubmed.ncbi.nlm.nih.gov

Pathogenesis and therapeutic approaches for non-alcoholic fatty liver disease. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Non-alcoholic fatty liver disease. [2022]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Efficacy of probiotics in non-alcoholic fatty liver disease in adult and children: A meta-analysis of randomized controlled trials. [2020]