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A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD)

NCT02363946 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 65

Last updated 2026-01-12

Study results available
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Summary

The purpose of the study is to determine the safety and tolerability of escalating doses of ARC-AAT and to evaluate the pharmacokinetics of ARC-AAT and the effect of ARC-AAT on circulating levels of alpha-1 antitrypsin (AAT). The study will consist of two parts, Part A (conducted in healthy volunteers) and Part B (conducted in AATD patients) at up to 9 escalating dose levels with 6 participants per dose level.

Conditions

  • Alpha-1 Antitrypsin Deficiency

Interventions

DRUG

ARC-AAT Injection

RNA interference-based, liver-targeted therapeutic

OTHER

Placebo

0.9 % normal saline

DRUG

Diphenhydramine

Diphenhydramine 50 mg p.o. was administered 2 hours (±30 minutes) pre-dose as antihistamine pre-treatment.

Sponsors & Collaborators

  • Arrowhead Pharmaceuticals

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2015-02-28
Primary Completion
2016-11-30
Completion
2016-11-30

Countries

  • Australia
  • Germany
  • Netherlands
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02363946 on ClinicalTrials.gov