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Purpose

The purpose of AROAAT2001 (SEQUOIA) is to evaluate the safety, tolerability and effect on liver histologic parameters with administration of the investigational product, ARO-AAT, in participants with alpha-1 antitrypsin deficiency (AATD). Participants will receive multiple subcutaneous doses of ARO-AAT.

Condition

Eligibility

Eligible Ages
Between 18 Years and 75 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Diagnosis of AATD
  • Liver biopsy at Screening indicating liver fibrosis
  • 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

Study Design

Phase
Phase 2/Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
ARO-AAT 		Part A: administered on Days 1, 29 and 113 and every 84 days thereafter until dose selected for Part B Part B: minimum of 6, maximum of 9 doses
  • Drug: ARO-AAT Injection
    solution for subcutaneous (sc) injection
Placebo Comparator
Placebo 		Part A: administered on Days 1, 29 and 113 and every 84 days thereafter until dose selected for Part B Part B: minimum of 6, maximum of 9 doses
  • Other: Placebo
    sterile normal saline (0.9% NaCl), calculated to match active comparator, for sc injection

Recruiting Locations

University of Alabama at Birmingham Medical Center
Birmingham, Alabama 35233
Contact:
Rakesha Garner
205-934-1224
rgarner@uabmc.edu

More Details

NCT ID
NCT03945292
Status
Recruiting
Sponsor
Arrowhead Pharmaceuticals

Study Contact

Medical Monitor
626-304-3400
medicalmonitor@arrowheadpharma.com

Detailed Description

In Part A, participants will be enrolled to receive multiple doses of ARO-AAT or placebo at varying dose levels. Once Part A is complete, a single dose level for Part B will be selected based on safety and pharmacodynamic parameters from Part A. Patients enrolled into Part A will roll over to the Part B dose level or continue to receive placebo.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.