Who is sponsoring NCT03362242?

NCT03362242 is sponsored by Arrowhead Pharmaceuticals.

What drugs are being tested in NCT03362242?

Interventions in NCT03362242: ARO-AAT Injection, Sterile Normal Saline (0.9% NaCl).

What condition does NCT03362242 study?

NCT03362242 studies Alpha 1-Antitrypsin Deficiency.

Is NCT03362242 still recruiting?

NCT03362242 is currently completed. Last updated 24 December 2025.

Last reviewed 2025-12-24 · How we verify

NCT03362242

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Study of ARO-AAT in Normal Adult Volunteers

Completed Phase 1 Last updated 24 December 2025

What this trial tests

Phase 1 trial testing ARO-AAT Injection in Alpha 1-Antitrypsin Deficiency in 45 participants. Completed in 21 March 2020.

Timeline

12 March 2018

Primary endpoint
23 October 2018

21 March 2020

Quick facts

Lead sponsor	Arrowhead Pharmaceuticals
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	double
Primary purpose	treatment
Enrollment	45
Start date	12 March 2018
Primary completion	23 October 2018
Estimated completion	21 March 2020
Sites	1 location across New Zealand

Drugs / interventions tested

ARO-AAT Injection — full drug profile →
Sterile Normal Saline (0.9% NaCl) — full drug profile →

Conditions studied

Alpha 1-Antitrypsin Deficiency — all drugs for Alpha 1-Antitrypsin Deficiency →

Sponsor

Arrowhead Pharmaceuticals — full company profile →

Who can join

Adults 18 to 55, any sex, with Alpha 1-Antitrypsin Deficiency. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Therapeutic siRNA: state of the art.
Hu B, Zhong L, Weng Y, Peng L, et al · · 2020 · cited 991× · PMID 32561705 · DOI 10.1038/s41392-020-0207-x
The landscape of nanoparticle-based siRNA delivery and therapeutic development.
Moazzam M, Zhang M, Hussain A, Yu X, et al · · 2024 · cited 116× · PMID 38204162 · DOI 10.1016/j.ymthe.2024.01.005
RNA delivery biomaterials for the treatment of genetic and rare diseases.
Zhao W, Hou X, Vick OG, Dong Y. · · 2019 · cited 65× · PMID 31255978 · DOI 10.1016/j.biomaterials.2019.119291
Therapeutic RNA Delivery for COVID and Other Diseases.
Dobrowolski C, Paunovska K, Hatit MZC, Lokugamage MP, et al · · 2021 · cited 46× · PMID 33661555 · DOI 10.1002/adhm.202002022
Development of an RNAi therapeutic for alpha-1-antitrypsin liver disease.
Wooddell CI, Blomenkamp K, Peterson RM, Subbotin VM, et al · · 2020 · cited 45× · PMID 32379724 · DOI 10.1172/jci.insight.135348
Hepatocyte targeting <i>via</i> the asialoglycoprotein receptor.
Ramírez-Cortés F, Ménová P. · · 2025 · cited 22× · PMID 39628900 · DOI 10.1039/d4md00652f
Development, opportunities, and challenges of siRNA nucleic acid drugs.
Xiao B, Wang S, Pan Y, Zhi W, et al · · 2025 · cited 19× · PMID 39897581 · DOI 10.1016/j.omtn.2024.102437
Targeting the Liver with Nucleic Acid Therapeutics for the Treatment of Systemic Diseases of Liver Origin.
Gogate A, Belcourt J, Shah M, Wang AZ, et al · · 2023 · cited 17× · PMID 37696583 · DOI 10.1124/pharmrev.123.000815

Verify or expand the search:

Other recruiting trials for Alpha 1-Antitrypsin Deficiency

Currently open trials in the same condition.

NCT06996756 — Gene Therapy for Alpha 1- Antitrypsin Deficiency · Phase 1 · recruiting
NCT05897424 — Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema · Phase 2 · active not recruiting
NCT04157049 — Alpha-1 Research Registry · recruiting

Other Arrowhead Pharmaceuticals trials

Trials by the same sponsor.

NCT07037771 — A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE) · Phase 3 · recruiting
NCT05537025 — Study of ARO-MMP7 Inhalation Solution in Healthy Subjects and Patients With Idiopathic Pulmonary Fibrosis · Phase 1, PHASE2 · completed
NCT05533294 — Study of ARO-RAGE in Healthy Subjects · Phase 1 · completed
NCT05413135 — Study of ARO-APOC3 in Adults With Dyslipidemia · Phase 2 · completed
NCT05276570 — Study of ARO-RAGE in Healthy Subjects and Patients With Inflammatory Lung Disease · Phase 1, PHASE2 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT03362242 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Arrowhead Pharmaceuticals
Last refreshed: 24 December 2025

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03362242.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing