Safety, Tolerability and Effect on Liver Histologic Parameters of 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
Principal Investigator
by Philip Rosenthal

Description

Summary

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.

Official Title

A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA]

Details

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.

Keywords

Alpha 1-Antitrypsin Deficiency ARO-AAT Injection ARO-AAT

Eligibility

You can join if…

Open to people ages 18-75

  • 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

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 accepting new patients
    San Francisco California 94158 United States
  • Stanford Health Care accepting new patients
    Stanford California 94305 United States

Lead Scientist at UCSF

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Arrowhead Pharmaceuticals
ID
NCT03945292
Phase
Phase 2/3
Study Type
Interventional
Last Updated
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