Study of ARO-AAT in Normal Adult Volunteers

Arrowhead Pharmaceuticals45 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Alpha 1-Antitrypsin Deficiency

Interventions

ARO-AAT InjectionDRUG

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

Sterile Normal Saline (0.9% NaCl)OTHER

Calculated volume to match active comparator

Eligibility

Sex: ALLMin age: 18 YearsMax age: 55 YearsHealthy volunteers:

Medical Language ↔ Plain English

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

Locations (1)

Research Site 1

Grafton, Auckland, New Zealand

Outcomes

Primary Outcomes

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

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

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

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

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

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

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

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

Data from ClinicalTrials.gov

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