NCT04669158

Study of Oral Idebenone to Treat Non-Alcoholic Steatohepatitis

A Pilot Study to Treat Adults With Non-Alcoholic Steatohepatitis With Oral Idebenone

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 53 (actual)
• Sponsor: Stanford University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a randomized, placebo-controlled phase 1/2a study to investigate the safety and tolerability of Idebenone in patients 18 years of age or older with non-alcoholic steatohepatitis, with stage 1-3 fibrosis. As secondary end point target engagement and fibrosis improvement will be assessed.

Detailed description

This is a prospective phase 1/2a, randomized, double-blinded, placebo-controlled, single center study of safety and efficacy of oral idebenone in adults 18 years of age or older with non-alcoholic steatohepatitis (NASH). Following IRB approval and written informed consent, 45 participants will be enrolled in the study and randomized in a 1:2 ratio on REDCap data capturing software. Participants will be randomized to two groups and receive the following tablets: placebo, idebenone at escalating doses of 200mg by mouth (P.O.) once a day for 2 weeks, then 200 mg twice a day for 2 weeks, then 3 times per day for the remainder of the study (up to 48 weeks). Monitoring and safety evaluation will continue for 12 weeks after the final dose. The study will investigate the safety and tolerability as primary end point and assess target engagement and fibrosis improvement as secondary end points.

Conditions

• Non Alcoholic Steatohepatitis
• Fibrosis

Interventions

Type	Name	Description
DRUG	Idebenone	Idebenone, initially 200mg by mouth (P.O.) once a day for 2 weeks, then 200 mg twice a day for 2 weeks, then 200 mg three times per day for the remainder of the study will be used.
DRUG	Placebo	Placebo to match Idebenone once a day for 2 weeks, then twice a day for 2 weeks, then three times per day for the remainder of the study will be used.

Timeline

• Start date: 2021-07-30
• Primary completion: 2023-07-05
• Completion: 2023-07-05
• First posted: 2020-12-16
• Last updated: 2026-03-27
• Results posted: 2026-03-27

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study