This study is in progress, not accepting new patients

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

a study on Alpha-1 Antitrypsin Deficiency

Summary

Eligibility
for people ages 18-75 (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
estimated completion

Description

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).

Official Title

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]

Details

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.

Keywords

Alpha 1-Antitrypsin Deficiency, Fazisiran Injection

Eligibility

You can join if…

Open to people ages 18-75

  • Diagnosis of AATD
  • Liver biopsy at Screening indicating liver fibrosis (score less than F4); a patient with no fibrosis may participate based on a previous biopsy conducted within one year
  • Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception
  • Willing to provide written informed consent and to comply with study requirements
  • Non-smoker for at least 1 year
  • No abnormal finding of clinical relevance at Screening

You CAN'T join if...

  • Clinically significant health concerns other than AATD
  • Previous diagnosis or diagnosis at Screening of definitive liver cirrhosis
  • Previous lung or liver transplant due to AATD
  • Regular use of alcohol within one month prior to Screening
  • Use of an investigational agent or device within 30 days prior to dosing or current participation in an investigational study involving therapeutic intervention

  • Use of illicit drugs within 1 year prior to Screening

    NOTE: additional inclusion/exclusion criteria may apply, per protocol

Locations

  • UCSF Medical Center Benioff Children's Hospital
    San Francisco California 94158 United States
  • Stanford Health Care
    Stanford California 94305 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Arrowhead Pharmaceuticals
ID
NCT03945292
Phase
Phase 2 Alpha-1 Antitrypsin Deficiency Research Study
Study Type
Interventional
Participants
About 40 people participating
Last Updated