Study of ARO-AAT in Normal Adult Volunteers

Arrowhead Pharmaceuticals

Status and phase: Completed; Phase 1

Conditions: Alpha 1-Antitrypsin Deficiency

Treatments: Drug: ARO-AAT Injection; Other: Sterile Normal Saline (0.9% NaCl)

Study type: Interventional

Funder types: Industry

Identifiers

NCT03362242

AROAAT1001

Details and patient eligibility

About

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.

Enrollment: 45 patients

Sex: All

Ages: 18 to 55 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
• 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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45 participants in 2 patient groups, including a placebo group

ARO-AAT

Active Comparator group

Treatment:

Drug: ARO-AAT Injection

Placebo

Placebo Comparator group

Treatment:

Other: Sterile Normal Saline (0.9% NaCl)