Assessment of Changes in a Activity Scale in Response to ARO-AAT
Research type
Research Study
Full title
A Pilot Open Label, Multi-dose, Phase 2 Study to Assess the Safety and Efficacy of ARO-AAT in Patients with Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)
IRAS ID
262227
Contact name
Dr. William James Houghton Griffiths, BA (Hons) MB BChir PhD FRCP
Contact email
Sponsor organisation
Arrowhead Pharmaceuticals, Inc
Eudract number
2019-000068-86
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 3 months, 1 days
Research summary
An open label, multi-center, multi-dose Phase 2 study will be conducted to see how safe and well tolerated the study drug, ARO-AAT, is in the treatment to decrease Alpha-1 Antitrypsin in the blood and in the liver. Participants who have signed an IRB/EC approved informed consent and have met all the protocol eligibility criteria during Screening, will receive either 5 or 7 subcutaneous doses of ARO-AAT. Participants who complete their cohort, or group of scheduled doses may choose to take part in an extension study. All subjects will require a pre-dose biopsy completed within the Screening phase of the study and a post-dose biopsy completed 90 days after the last dose of study drug.
REC name
West Midlands - Coventry & Warwickshire Research Ethics Committee
REC reference
19/WM/0186
Date of REC Opinion
1 Jul 2019
REC opinion
Favourable Opinion