SEQUOIA
Research type
Research Study
Full 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]
IRAS ID
251881
Contact name
Alice Turner
Contact email
Sponsor organisation
Arrowhead Pharmaceuticals, Inc
Eudract number
2018-003385-14
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 6 months, 31 days
Research summary
A multi-center multi-dose placebo-controlled Phase 2/3 study will be conducted to evaluate the safety, tolerability and effect on intrahepatic and circulating AAT levels of the investigational product, ARO-AAT, administered subcutaneously to patients with Alpha-1 Antitrypsin Deficiency. Participants who have signed an IRB/EC approved informed consent and have met all the protocol eligibility criteria during Screening, will receive multiple subcutaneous doses of ARO-AAT or placebo.
All patients will require a pre-dose biopsy as per Inclusion criteria #3.
• Patients with a fibrosis score of F0 will be eligible to participate in Part A for the dose selection phase of the study. The F0 participants will not be eligible for Part B.
• Patients with a fibrosis score of F1-F3 (or equivalent) are eligible for Parts A and B. All subjects will require a pre-dose biopsy completed within the Screening window and a post-dose biopsy completed at the End of Study or at Early Termination as applicable.
After the first 36 patients have completed their Day 113 visit, an analysis of Part A will be performed to select a single dose level (25, 100 or 200 mg) for Part B.
Participation in the study will be approximately 72 to 108 weeks, including a 60 day screening period and a telephone call 90 days after the last dose.REC name
East Midlands - Leicester Central Research Ethics Committee
REC reference
20/EM/0206
Date of REC Opinion
19 Oct 2020
REC opinion
Further Information Favourable Opinion