NCT03945292

Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)

A Placebo-Controlled, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA]

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 40 (actual)
• Sponsor: Arrowhead Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

The purpose of AROAAT2001 (SEQUOIA) is to evaluate the safety, efficacy and tolerability of multiple doses of the investigational product, Fazirsiran Injection, administered subcutaneously to participants with alpha-1 antitrypsin deficiency (AATD).

Detailed description

Participants will be enrolled to receive multiple subcutaneous injections of Fazirsiran Injection (also referred to as TAK-999 Injection or ARO-AAT Injection) 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 phase.

Conditions

• Alpha 1-Antitrypsin Deficiency

Interventions

Type	Name	Description
DRUG	Fazisiran Injection	solution for subcutaneous (sc) injection
OTHER	Placebo	sterile normal saline (0.9% NaCl), calculated to match active comparator, for sc injection

Timeline

• Start date: 2019-08-07
• Primary completion: 2021-11-08
• Completion: 2023-09-18
• First posted: 2019-05-10
• Last updated: 2025-11-04
• Results posted: 2024-12-30

Locations

21 sites across 6 countries: United States, Germany, Italy, Netherlands, Portugal, Spain

Regulatory

• FDA-regulated drug study