Study of ARO-AAT in Normal Adult Volunteers

Alpha 1-Antitrypsin Deficiency Clinical Trial

Official 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03362242
• Other study ID #: AROAAT1001
• Status: Completed
• Phase: Phase 1
• Start date: March 12, 2018
• Est. completion date: March 21, 2020

Study information

• Verified date: August 2020
• Source: Arrowhead Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 45
• Est. completion date: March 21, 2020
• Est. primary completion date: October 23, 2018
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 55 Years

Eligibility

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

Related Conditions & MeSH terms

• Alpha 1-Antitrypsin Deficiency

Intervention

Drug:
• ARO-AAT Injection
Single or multiple doses of ARO-AAT by subcutaneous (sc) injections

Other:
• Sterile Normal Saline (0.9% NaCl)
Calculated volume to match active comparator

Locations

Country	Name	City	State
New Zealand	Auckland Clinical Studies Limited	Grafton	Auckland

Sponsors (1)

Lead Sponsor	Collaborator
Arrowhead Pharmaceuticals

Country where clinical trial is conducted

New Zealand, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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