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siRNA

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 participants without fibrosis: pre-dose on days 1 & 29, and on days 113, 197 and 281; participants with fibrosis: pre-dose on all dosing visits (days 1, 29, 113, and every 12 weeks up to week 196)

Awards & highlights

SEQUOIA Trial Summary

This trial is testing a new product, ARO-AAT, to see if it is safe and effective for people with a certain lung disease.

SEQUOIA Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ participants without fibrosis: pre-dose on days 1 & 29, and on days 113, 197 and 281; participants with fibrosis: pre-dose on all dosing visits (days 1, 29, 113, and every 12 weeks up to week 196)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~participants without fibrosis: pre-dose on days 1 & 29, and on days 113, 197 and 281; participants with fibrosis: pre-dose on all dosing visits (days 1, 29, 113, and every 12 weeks up to week 196)

This trial's timeline: 3 weeks for screening, Varies for treatment, and participants without fibrosis: pre-dose on days 1 & 29, and on days 113, 197 and 281; participants with fibrosis: pre-dose on all dosing visits (days 1, 29, 113, and every 12 weeks up to week 196) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Percentage Change From Baseline in Serum Z-Alpha-1 Antitrypsin (Z-AAT)

Secondary outcome measures

Alanine Transaminase

Absolute Change from Baseline in Liver Function Tests: International Normalized Ratio (INR) at Week 16 and over time through EOS

Absolute Change from Baseline in Liver Function Tests: Total Bilirubin, Direct Bilirubin at Week 16 and over time through EOS

+19 more

SEQUOIA 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

Find a Location

Who is running the clinical trial?

Arrowhead PharmaceuticalsLead Sponsor

39 Previous Clinical Trials

4,918 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the geographic scope of this trial's implementation?

"This research is being conducted in various places, such as the University of Iowa Hospitals and Clinics situated in Iowa City, UCSF Medical Center Benioff Children's Hospital located in San Francisco, California, and also the University of Alabama at Birmingham Medical Center from Birmingham. There are a total 16 additional medical centres involved as well."

Answered by AI

Is the enrollment for this research restricted to adults over 25 years of age?

"This research project seeks out participants who are over the age of maturity and under 75 years old."

Answered by AI

Is it feasible for me to enroll in this research trial?

"Patient eligibility criteria for this trial consists of alpha 1-antitrypsin deficiency and a range between 18 to 75 years old. The total number of participants required is 40 individuals."

Answered by AI

Are there any vacant openings for participation in this medical experiment?

"Clinicaltrials.gov reveals that this medical trial, which was published on August 7th 2019 and last updated November 3rd 2022, is not currently recruiting patients. Nevertheless, 13 other research projects are actively seeking participants at the moment."

Answered by AI

Has the ARO-AAT Injection been given clearance by the FDA?

"The safety of ARO-AAT injection is rated at 2 on our team's scale, since this Phase 2 trial offers indications of it being safe yet no proof that the drug works."

Answered by AI

Recent research and studies

Current Opinion in PharmacologyJournal

Alpha1-antitrypsin Deficiency: New Therapies on the Horizon

Remih, Katharina, Samira Amzou, and Pavel Strnad. 2021. “Alpha1-antitrypsin Deficiency: New Therapies on the Horizon”. Current Opinion in Pharmacology. Elsevier BV. doi:10.1016/j.coph.2021.06.001.

clinicaltrials.govStudy

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

2019. "Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03945292.