Study of ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)
Study of ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)
Brief Summary:
The purpose of this study is to evaluate the the safety and efficacy of ARO-AAT Injection (also referred to as ARO-AAT) administered subcutaneously to patients with alpha-1 antitrypsin deficiency.
Detailed Description:
Participants will be enrolled to receive multiple subcutaneous injections of ARO-AAT. All eligible participants will require a pre-dose biopsy completed as part of the study within the screening window. All participants will undergo an End of Study (EOS) biopsy. Treated participants will be offered the opportunity to continue treatment in an open label extension during which they will undergo a final biopsy.3
Study Type: Interventional (Clinical Trial)
Actual Enrollment: 16 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Open Label, Multi-dose, Phase 2 Study to Assess the Safety and Efficacy of ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)
Actual Study Start Date: December 19, 2019
Estimated Primary Completion Date: August 23, 2024
Estimated Study Completion Date: August 23, 2024
Arms:
- Experimental: ARO-AAT Cohort 1
- Experimental: ARO-AAT Cohort 1b
- Experimental: ARO-AAT Cohort 2
| Category | Value |
|---|---|
| Study type(s) | Interventional |
| Actual enrolment | 16 |
| Actual Study start date | 19 December 2019 |
| Estimated Study Completion Date | 23 August 2024 |