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Clinical trial

Study of ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)

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Last updated:3rd May 2022
Status: Active, not recruiting
Identifier: NCT03946449
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

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