ION224 for Non-alcoholic Fatty Liver Disease

No longer recruiting at 1 trial location
IP
SC
Overseen ByStudy Coordinator
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines how ION224 affects individuals with non-alcoholic steatohepatitis (NASH), a liver condition that can lead to serious health issues. Researchers aim to determine if ION224 can improve liver health and reduce liver fat through regular injections over 49 weeks. Participants will receive either the treatment or a placebo for comparison. Suitable candidates for this trial have NASH confirmed by a liver biopsy and liver fat of 10% or more, as determined by a special MRI scan. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of people.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that ION224 was safe and well-tolerated in earlier studies. Reports indicate that patients did not experience major side effects. Additionally, the treatment improved liver health in people with non-alcoholic fatty liver disease. This suggests that the treatment could be a safe option for those considering participation in a clinical trial.12345

Why do researchers think this study treatment might be promising for non-alcoholic fatty liver disease?

Unlike the standard treatments for non-alcoholic fatty liver disease, which often include lifestyle changes and medications to manage symptoms, ION224 takes a different approach. ION224 is unique because it is administered through a subcutaneous injection every four weeks, potentially offering a more convenient dosing schedule. Moreover, it works by targeting specific pathways involved in liver fat accumulation, which could lead to more direct and effective results. Researchers are excited about ION224 because it might provide a more targeted therapy, addressing the root causes of the disease rather than just alleviating its symptoms.

What evidence suggests that ION224 might be an effective treatment for non-alcoholic fatty liver disease?

Research has shown that the investigational treatment ION224, which participants in this trial may receive, may help treat non-alcoholic fatty liver disease. In earlier studies, ION224 significantly improved liver health by reducing liver inflammation and damage, as evidenced by a more than 2-point improvement in the Non-Alcoholic Fatty Liver Disease Activity Score (NAS), without worsening liver scarring. The treatment targets and reduces a protein that contributes to fat production in the liver. Early results suggest it could effectively lower liver fat and improve liver function.12345

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.
See 4 more

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?
2Treatment groups
Experimental Treatment
Placebo Group
Group I: ION224Experimental Treatment1 Intervention
Group II: PlaceboPlacebo Group1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Ionis Pharmaceuticals, Inc.

Lead Sponsor

Trials
151
Recruited
27,800+
Dr. Brett P. Monia profile image

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 profile image

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

Citations

1.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/40849139/
Antisense oligonucleotide DGAT-2 inhibitor, ION224, for ...The primary endpoint was ≥2-point reduction in Non-Alcoholic Fatty Liver Disease Activity Score Activity Score (NAS) with ≥1-point improvement ...
Ionis announces positive results from Phase 2 study of ...Significant improvement in steatohepatitis with >2 point improvement in NAS score without worsening fibrosis, the primary endpoint of the ...
Antisense oligonucleotide DGAT-2 inhibitor, ION224, for ...The primary endpoint was ≥2-point reduction in Non-Alcoholic Fatty Liver Disease Activity Score Activity Score (NAS) with ≥1-point improvement ...
NCT04932512 | A Study to Assess the Safety, Efficacy, and ...The purpose of this study is to assess the effect of multiple ION224 doses when administered by subcutaneous (SC) injection for 49 weeks (end of the ...
New Drug May Halt Fatty Liver DiseaseThe primary endpoint was ≥2-point reduction in Non-Alcoholic Fatty Liver Disease Activity Score (NAS) in addition to improvement in other ...
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