Study of ARO-AAT in Normal Adult Volunteers

Phase 1

Completed

• Conditions: Alpha 1-Antitrypsin Deficiency
• Interventions: Other: Sterile Normal Saline (0.9% NaCl); Drug: ARO-AAT Injection

• Registration Number: NCT03362242
• Lead Sponsor: Arrowhead Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2017-11-30
• Study First Submitted QC: 2017-11-30
• Study First Posted: 2017-12-05
• Study Start: 2018-03-12
• Primary Completion: 2018-10-23
• Study Completion: 2020-03-21
• Status Verified: 2020-08
• Last Update Submitted: 2020-08-17
• Last Update Posted: 2020-08-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 45

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Sterile Normal Saline (0.9% NaCl)	-
ARO-AAT	ARO-AAT Injection	-

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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
Percent Change in Serum Alpha-1 Antitrypsin (AAT) Levels From Day 1 Pre-Dose Baseline to Nadir	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	Part A (SAD phase): up to 29 (+/- 2) days; Part B (MAD phase): up to 113 (+/- 2) days

Trial Locations

Locations (1)

Auckland Clinical Studies Limited; 🇳🇿 Grafton, Auckland, New Zealand