Protocol Summary

Protocol No.: OCR21902

Sponsor Protocol No.: AROAAT2001

Protocol Title.: Safety, Tolerability and Effect on Liver Histologic Parameters of ARO-AAT

Principal Investigator: Clark, Virginia

Objective: 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.

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.

Phase: Phase II

Age Group: Adult

Age: 18 Years - 75 Years

Gender: All

Treatment:

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

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

Detailed Eligibility:

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

Applicable Conditions:

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

  • No UF Health MRN : Angela Bauer
  • UF Gainesville : Angela Bauer
  • UF Jacksonville : Angela Bauer
  • Contact:
    Angela Bauer
    Phone: +1 352-273-9512
    Email: bauera@medicine.ufl.edu

    More Information: View study listing on ClinicialTrials.gov http://www.clinicaltrials.gov/ct2/show/NCT03945292