An Open-Label, Phase 1 Study of INBRX-101 in Adults with AATD
Research type
Research Study
Full title
An Open-Label, Multicenter, Phase 1 Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Ascending Intravenous Doses of Inhibrx rhAAT-Fc (INBRX-101) in Adults with Alpha-1 Antitrypsin Deficiency (AATD)
IRAS ID
254206
Contact name
Alice Turner
Contact email
Sponsor organisation
Inhibrx 101 LP
Eudract number
2018-004326-29
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 12 months, 0 days
Research summary
Patients with alpha-1 antitrypsin deficiency (AATD) lack a protective enzyme inhibitor called alpha-1 antitrypsin protein (AAT). This makes these patients more vulnerable to the effects of inhaling smoke or materials like dust or chemicals. AAT is an enzyme inhibitor which means it controls enzymes in your body. Enzymes are proteins that make chemical processes happen in your body and do lots of different jobs.
AAT is mainly produced by the liver and the low levels of AAT in the blood occur because the AAT is abnormal and cannot be released from the liver at the normal rate. This leads to a build-up of abnormal AAT in the liver that can cause liver disease and a decrease of AAT in the blood that can lead to lung disease. Current medicines that treat AATD contain AAT collected from human donors.
This is a Phase 1, first in human study and the aim of the study is to see how safe and well tolerated INBRX-101 (study drug) is and to find the maximum tolerated dose and the optimal dose for future studies. The pharmacokinetics (what the body of participants with AATD does to the drug) will also be tested.
There will be approximately 30 participants in this study. The study is being carried out in centres in the United States of America and the United Kingdom. Participants will be seen in a hospital setting.REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
19/WM/0115
Date of REC Opinion
5 Jun 2019
REC opinion
Further Information Favourable Opinion