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

Arrowhead Pharmaceuticals

Status and phase: Completed; Phase 2; Phase 1

Conditions: Non-alcoholic Steatohepatitis

Treatments: Drug: sterile normal saline (0.9% NaCl); Drug: ARO-HSD Injection

Study type: Interventional

Funder types: Industry

Identifiers

NCT04202354

AROHSD1001

Details and patient eligibility

About

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.

Enrollment: 50 patients

Sex: All

Ages: 19 to 65 years old

Volunteers: Accepts Healthy Volunteers

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

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Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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50 participants in 11 patient groups, including a placebo group

Cohort 1: ARO-HSD 25 mg

Experimental group

Description:

Normal healthy volunteers randomized to double blind ARO-HSD 25 mg on Day 1 only.

Treatment:

Drug: ARO-HSD Injection

Cohort 1: Placebo

Placebo Comparator group

Description:

Normal healthy volunteers randomized to double blind placebo on Day 1 only.

Treatment:

Drug: sterile normal saline (0.9% NaCl)

Cohort 2: ARO-HSD 50 mg

Experimental group

Description:

Normal healthy volunteers randomized to double blind ARO-HSD 50 mg on Day 1 only.

Treatment:

Drug: ARO-HSD Injection

Cohort 2: Placebo

Placebo Comparator group

Description:

Normal healthy volunteers randomized to double blind placebo on Day 1 only.

Treatment:

Drug: sterile normal saline (0.9% NaCl)

Cohort 3: ARO-HSD 100 mg

Experimental group

Description:

Normal healthy volunteers randomized to double blind ARO-HSD 100 mg on Day 1 only.

Treatment:

Drug: ARO-HSD Injection

Cohort 3: Placebo

Placebo Comparator group

Description:

Normal healthy volunteers randomized to double blind placebo on Day 1 only.

Treatment:

Drug: sterile normal saline (0.9% NaCl)

Cohort 4: ARO-HSD 200 mg

Experimental group

Description:

Normal healthy volunteers randomized to double blind ARO-HSD 200 mg on Day 1 only.

Treatment:

Drug: ARO-HSD Injection

Cohort 4: Placebo

Placebo Comparator group

Description:

Normal healthy volunteers randomized to double blind placebo on Day 1 only.

Treatment:

Drug: sterile normal saline (0.9% NaCl)

Cohort 1b: ARO-HSD 25 mg

Experimental group

Description:

Participants with suspected non-alcoholic steatohepatitis (NASH) receive open-label ARO-HSD 25 mg on Days 1 and 29.

Treatment:

Drug: ARO-HSD Injection

Cohort 3b: ARO-HSD 100 mg

Experimental group

Description:

Participants with suspected NASH receive open-label ARO-HSD 100 mg on Days 1 and 29.

Treatment:

Drug: ARO-HSD Injection

Cohort 4b: ARO-HSD 200 mg

Experimental group

Description:

Participants with suspected NASH receive open-label ARO-HSD 200 mg on Days 1 and 29.

Treatment:

Drug: ARO-HSD Injection