Safety, Tolerability and Pharmacodynamic Effect of ARO-AAT (SEQUOIA)
Safety, Tolerability and Pharmacodynamic Effect of ARO-AAT (SEQUOIA)
Brief Summary:
The purpose of AROAAT2001 (SEQUOIA) is to evaluate the safety, efficacy and tolerability of multiple doses of the investigational product, ARO-AAT, administered subcutaneously to participants with alpha-1 antitrypsin deficiency (AATD).
Detailed Description:
Participants will be enrolled to receive multiple subcutaneous injections of ARO-AAT or placebo. Eligible participants will require a pre-dose biopsy completed as part of the study within the screening window. However, any participant with a biopsy result within 1 year of screening showing no fibrosis does not require a pre-dose biopsy. Only participants who have liver fibrosis will undergo a post-dose biopsy and may continue treatment in an open-label extension.
Study Type: Interventional (Clinical Trial)
Actual Enrollment: 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Placebo-Controlled, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Pharmacodynamic Effect of ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA]
Actual Study Start Date: August 7, 2019
Estimated Primary Completion Date: July 13, 2022
Estimated Study Completion Date: July 13, 2022
Arms:
- Experimental: ARO-AAT
- Placebo Comparator: Placebo
| Category | Value |
|---|---|
| Study type(s) | Interventional |
| Actual enrolment | 40 |
| Actual Study start date | 07 August 2019 |
| Estimated Study Completion Date | 13 July 2022 |