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

NCT03945292 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2025-11-04

Study results available
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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).

Conditions

Interventions

DRUG

Fazisiran Injection

solution for subcutaneous (sc) injection

OTHER

Placebo

sterile normal saline (0.9% NaCl), calculated to match active comparator, for sc injection

Sponsors & Collaborators

  • Arrowhead Pharmaceuticals

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-08-07
Primary Completion
2021-11-08
Completion
2023-09-18
FDA Drug
Yes

Countries

  • United States
  • Germany
  • Italy
  • Netherlands
  • Portugal
  • Spain

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03945292 on ClinicalTrials.gov