Fazirsiran (also referred to as TAK-999 or ARO-AAT) for Alpha-1 Antitrypsin Deficiency (SEQUOIA Trial)

Phase 2

Waitlist Available

Research Sponsored by Arrowhead Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

This trial tests if Fazirsiran Injection, given under the skin, can help patients with alpha-1 antitrypsin deficiency by protecting their liver from damage. Fazirsiran is a treatment aimed at addressing liver damage in patients with alpha-1 antitrypsin deficiency, a condition where the liver is harmed due to abnormal protein accumulation.

Eligible Conditions

Alpha-1 Antitrypsin Deficiency

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Open-Label Fazisiran (also referred to as TAK-999 or ARO-AAT)Experimental Treatment1 Intervention

After all enrolled participants completed the Week 16 visit, an interim analysis was performed to select a single dose level (25, 100 or 200 mg) for the open-label phase of the study. Fazirsiran 200 mg was the selected dose and all participants with fibrosis at Screening who completed the post-dose liver biopsy at Week 48 (or Week 72 or 96) receive this dose every 12 weeks for the duration of the study (open-label phase).

Group II: Fazirsiran (also referred to as TAK-999 or ARO-AAT)Experimental Treatment1 Intervention

Participants with no fibrosis: Administered on Day 1 and Week 4 Participants with fibrosis: Administered on Day 1, Week 4, and Week 16, then every 12 weeks up to 18 total doses.

Group III: PlaceboPlacebo Group1 Intervention

Participants with no fibrosis: Administered on Day 1 and Week 4 Participants with fibrosis: Administered on Day 1, Week 4, and Week 16, then every 12 weeks up to 18 total doses.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Fazisiran Injection

2019

Completed Phase 2

~40

Do I qualify?Apply below to find out