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Completed

NCT Number: NCT03362242

Study of ARO-AAT in Normal Adult Volunteers

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.

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Key information

Conditions

Alpha 1-Antitrypsin Deficiency Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Emphysema Genetic Diseases, Inborn Liver Diseases Lung Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Respiratory Tract Diseases Subcutaneous Emphysema

Age range

18 year–55 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Research Site 1

Grafton, Auckland, 1010, New Zealand

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Exclusion criteria

  • 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

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

Treatment and study plan

ARO-AAT Injection

Drug

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

Sterile Normal Saline (0.9% NaCl)

Other

Calculated volume to match active comparator

Primary outcomes

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

    Time frame: 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

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

    Time frame: 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

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

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

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

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

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

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

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

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

  6. Percent Change in Serum Alpha-1 Antitrypsin (AAT) Levels From Day 1 Pre-Dose Baseline to Nadir

    Time frame: Part A (SAD phase): up to 29 (+/- 2) days; Part B (MAD phase): up to 113 (+/- 2) days

  7. Duration of Response of Serum AAT levels From Nadir Back to Above 20% of Baseline or Above 90 mg/dL

    Time frame: Part A (SAD phase): up to 29 (+/- 2) days; Part B (MAD phase): up to 113 (+/- 2) days

Sponsors and collaborators

Lead sponsor

Arrowhead Pharmaceuticals

Industry

Registry information

Official study title

A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Effect of ARO-AAT on Serum Alpha-1 Antitrypsin Levels in Normal Adult Volunteers

Important dates

Study start
2018
Primary completion
2018
Study completion
2020
First posted
Dec 5, 2017
Registry last updated
Dec 24, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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