NCT04932512

A Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224

An Adaptive Two-Part Phase 2, Multi-Center, Randomized, Double Blind, Placebo-Controlled Study to Assess the Safety, Efficacy, and Pharmacokinetics of Multiple Doses of ION224 Administered Once Monthly in Adult Subjects With Confirmed Non-Alcoholic Steatohepatitis

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 160 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

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 treatment \[EOT\]) on non-alcoholic steatohepatitis (NASH) histologic improvement and to assess the effect on liver steatosis by magnetic resonance imaging-determined proton density fat fraction (MRI-PDFF), additional changes in NASH histologic features, liver biochemistry tests, and plasma lipid profile.

Detailed description

This is a Phase 2, double-blind, randomized, placebo-controlled study of ION224 in up to 150 participants. The study consists of 3 periods: 1) Screening Period: Week -8 to Week -1 (up to 8 weeks); 2) Treatment Period up to Week 49; and 3) Post-Treatment Period: Week 50 to Week 62 (12 weeks). Initially, 48 patients will be enrolled in three different dose cohorts to receive ION224 or placebo every four weeks and based on safety and effects on liver steatosis (assessed at Week 15), two dose cohorts will be selected to be expanded. After dose selection, an additional 102 patients will be enrolled in the 2 selected dose cohorts and will receive ION224 or placebo for up to 49 weeks. Participants in the 3rd cohort (not selected) will continue to complete up to 49 weeks of treatment without any cohort expansion.

Conditions

• Steatohepatitis, Nonalcoholic

Interventions

Type	Name	Description
DRUG	ION224	ION224 will be administered by SC injection.
OTHER	Placebo	ION224-matching placebo solution will be administered by SC injection.

Timeline

• Start date: 2021-06-17
• Primary completion: 2024-01-10
• Completion: 2024-02-28
• First posted: 2021-06-21
• Last updated: 2024-11-27

Locations

51 sites across 2 countries: United States, Puerto Rico

Regulatory

• FDA-regulated drug study