Arcaat-1001. Dose escalation of ARC-AAT in patients with AATD
Research type
Research Study
Full title
A Double-Blind, Placebo-Controlled, Dose-Escalating, Phase 1 Study to Determine the Safety, Tolerability, Pharmacokinetics and Effect on Circulating Alpha-1 Antitrypsin Levels of ARC-AAT in Healthy Volunteer Subjects and in Patients with Alpha-1 Antitrypsin Deficiency (AATD)
IRAS ID
179516
Contact name
Alice Turner
Contact email
Sponsor organisation
Arrowhead Research Corporation
Eudract number
2015-001147-36
Duration of Study in the UK
0 years, 6 months, 15 days
Research summary
Alpha-1 antitrypsin deficiency (AATD) is an inherited genetic condition where the patient has inherited two abnormal Alpha-1 antitrypsin genes from their parents. Around 1 in 3000 people have AATD and this leads to liver disease through PiZZ protein accumulation in some patients. Lung disease also occurs due to lack of protection from noxious stimuli, such as cigarette smoke. The current treatment for AATD is AAT replacement therapy however this does not help treat liver disease which occurs due to this accumulation of proteins.
This study is being conducted to look at how safe and well tolerated a new drug called ARC-AAT which aims to reduce the PiZZ protein production (thus slowing the rate of accumulation and liver disease). Our aim of this study is to see how safe and well tolerated the study drug is and to find the maximum tolerated dose The pharmacokinetics (the way the body processes the drug) will also be tested.
This is a 2 part study and Part A is being conducted in healthy patients whilst Part B will start once part A has been completed. Part B is being conducted in patients with AATD and will use the maximum tolerated dose found in part A. The UK will participate in Part B of this study only.REC name
Scotland A: Adults with Incapacity only
REC reference
15/SS/0109
Date of REC Opinion
20 Jul 2015
REC opinion
Further Information Favourable Opinion