Study of ARO-AAT in Normal Adult Volunteers

NCT03362242 | Completed Phase 1 FDA Drug

• 1 other identifier: AROAAT1001
• Study Type: interventional
• Target: 45
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-AAT in healthy adult volunteers.

Conditions

Alpha 1-Antitrypsin Deficiency

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events (AEs) Possibly or Probably Related to Treatment

  Part A (single-ascending dose [SAD] phase): up to 29 (+/- 2) days post-dose; Part B (multiple-ascending dose [MAD] phase): up to 113 (+/- 2) days post-dose

Secondary Outcomes (7)

• Pharmacokinetics (PK) of ARO-AAT: Maximum Observed Plasma Concentration (Cmax)

  Part A (single-ascending dose [SAD] phase): up to 48 hours post-dose; Part B (multiple-ascending dose [MAD] phase): up to 48 hours post-dose

• PK of ARO-AAT: Time to Maximum Plasma Concentration (Tmax)

  Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose

• PK of ARO-AAT: Terminal Elimination Half-Life (t½)

  Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose

• PK of ARO-AAT: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24)

  Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose

• PK of ARO-AAT: Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUCinf)

  Part A (SAD phase): up to 48 hours post-dose; Part B (MAD phase): up to 48 hours post-dose

Study Arms (2)

ARO-AAT

ACTIVE COMPARATOR

Drug: ARO-AAT Injection

Placebo

PLACEBO COMPARATOR

Other: Sterile Normal Saline (0.9% NaCl)

Interventions

ARO-AAT Injection DRUG

Single or multiple doses of ARO-AAT by subcutaneous (sc) injections

ARO-AAT

Sterile Normal Saline (0.9% NaCl) OTHER

Calculated volume to match active comparator

Placebo

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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
• Non-smoker for at least one year
• Normal lung function
• No abnormal finding of clinical relevance at Screening
• Normal AAT level at Screening visit

You may not qualify if:

• Clinically significant health concerns
• Regular use of alcohol within one month prior to Screening
• Use of an investigational agent or device within 30 days prior to dosing or current participation in an investigational study
• Recent use of illicit drugs
• Use of any drugs or dietary/herbal supplements know to interfere with liver metabolism

Sponsors & Collaborators

• Arrowhead Pharmaceuticals: lead

Study Sites (1)

Research Site 1

Grafton, Auckland, 1010, New Zealand

Location

MeSH Terms

Conditions

alpha 1-Antitrypsin Deficiency

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Emphysema; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 30, 2017
• First Posted: December 5, 2017
• Study Start: March 12, 2018
• Primary Completion: October 23, 2018
• Study Completion: March 21, 2020
• Last Updated: December 24, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

NZ (1)