Phase 2 N=50 Randomized Quadruple-blind Treatment

Study of ARO-HSD in Healthy Volunteers and Patients With Non-Alcoholic Steatohepatitis (NASH) or Suspected NASH

Non-alcoholic Steatohepatitis

Bottom Line

View on ClinicalTrials.gov: NCT04202354 ↗

Enrolled (actual)

Serious AEs

2.0%

Results posted

Oct 2025

Primary outcome: Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Possibly or Probably Related to Treatment — 0; 0; 1; 3 participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: ARO-HSD Injection (Drug); sterile normal saline (0.9% NaCl) (Drug)
Age: Adult, Older Adult · 19+ yrs
Sex: All
Sponsor: Arrowhead Pharmaceuticals
Primary completion: Sep 2021

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Possibly or Probably Related to Treatment	0; 0; 1; 3; 0; 1	—
SECONDARY Pharmacokinetics (PK) of ARO-HSD: Plasma Concentrations	0.0000; 0.0000; 0.0000; 0.0000; 0.0000; 0.0000	—
SECONDARY PK of ARO-HSD in Normal Healthy Volunteers: Maximum Observed Plasma Concentration (Cmax)	74.0; 184; 294; 644	—
SECONDARY PK of ARO-HSD in Normal Healthy Volunteers: Time to Reach Cmax (Tmax)	2.5; 3.0; 8.0; 3.0	—
SECONDARY PK of ARO-HSD in Normal Healthy Volunteers: Terminal Elimination Half-Life (t1/2)	3.3; 2.5; 3.7; 4.5	—
SECONDARY PK of ARO-HSD in Normal Healthy Volunteers: Area Under the Concentration-Time Curve From Dosing (Time 0) to the Time of the Last Measured Concentration (AUClast)	788; 1830; 3670; 8450	—
SECONDARY PK of ARO-HSD in Normal Healthy Volunteers: Area Under the Curve From Time 0 to Infinity (AUCinf)	806; 1840; 3790; 8500	—
SECONDARY Secondary: PK of ARO-HSD in Normal Healthy Volunteers: Oral Clearance (CL/F)	31.1; 27.9; 26.8; 23.6	—
SECONDARY PK of ARO-HSD in Normal Healthy Volunteers: Apparent Volume of Distribution During the Terminal-Phase (Vz/F)	148; 99.9; 141; 153	—
SECONDARY Urine PK of ARO-HSD in Normal Healthy Volunteers: Amount of Unchanged Drug Recovered in Urine Over 0-24 Hours Postdose (Ae0-24h)	1.56; 3.64; 6.67; 45.8	—
SECONDARY Urine PK of ARO-HSD in Normal Healthy Volunteers: Percentage of the Administrated Drug Recovered in Urine Over 0-24 Hours (Fe0-24h)	6.23; 7.29; 6.67; 22.9	—
SECONDARY Urine PK of ARO-HSD in Normal Healthy Volunteers: Renal Clearance (CLr)	1.97; 1.99; 1.82; 5.75	—

Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple doses of ARO-HSD in healthy adult volunteers and in patients with NASH or suspected NASH.

Eligibility Criteria

Inclusion Criteria

Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding and must be willing to use contraception
Willing to provide written informed consent and to comply with study requirements
On a stable diet for at least 4 weeks with no plans to significantly alter diet or weight over course of study
Normal electrocardiogram (ECG) at Screening
No abnormal finding of clinical relevance (other than NASH, suspected NASH in patients) at Screening that could adversely impact subject safety during the study or adversely impact study results.

Exclusion Criteria

Clinically significant health concerns (other than NASH, suspected NASH in patients)
Human immunodeficiency virus (HIV) infection, seropositive for Hepatitis B Virus (HBV), seropositive for Hepatitis C Virus (HCV)
Uncontrolled hypertension
Excessive use of alcohol within three months prior to Screening
Use of illicit drugs within 1 year prior to Screening, or positive urine drug screen at Screening
Use of an investigational agent or device within 30 days prior to dosing or current participation in an investigational study

NOTE: additional inclusion/exclusion criteria may apply, per protocol

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04202354). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.