Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels
NCT02900183 · Status: WITHDRAWN · Phase: PHASE2 · Type: INTERVENTIONAL
Last updated 2025-12-18
Summary
Patients with Alpha-1 Antitrypsin Deficiency (AATD) will be enrolled to receive multiple doses of ARC-AAT Injection. All subjects will require a pre-dose biopsy and a biopsy post last dose.
Conditions
- Alpha-1 Antitrypsin Deficiency
Interventions
- DRUG
-
ARC-AAT Injection
RNA interference (RNAi)-based, liver-targeted therapeutic
Sponsors & Collaborators
-
Arrowhead Pharmaceuticals
lead INDUSTRY
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 18 Years
- Max Age
- 75 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2016-10-31
- Primary Completion
- 2016-11-30
- Completion
- 2016-11-30
Countries
- Canada
- Ireland
- Italy
- Sweden
Study Locations
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