Protocol Summary

Protocol No.: OCR21902

Sponsor Protocol No.: AROAAT2001

Protocol Title.: Safety, Tolerability and Pharmacodynamic Effect of ARO-AAT

Principal Investigator: Clark, Virginia

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

Description: Participants will be enrolled to receive multiple subcutaneous injections of ARO-AAT 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 extension.

Phase: Phase II

Age Group: Adult

Age: 18 Years - 75 Years

Gender: All

Treatment:

Experimental: ARO-AAT
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 15 total doses.

Placebo Comparator: Placebo
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 15 total doses.

Detailed Eligibility:

Inclusion Criteria: - 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 Exclusion Criteria: - 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

Applicable Conditions:

  • Alpha-1 Antitrypsin Deficiency (AATD)
  • Participation Institution:

  • No UF Health MRN
  • UF Gainesville
  • UF Jacksonville
  • More Information: View study listing on ClinicialTrials.gov http://www.clinicaltrials.gov/ct2/show/NCT03945292