A phase I study of BMN 349 in PiZZ and PiMZ/MASH Adult Participants
Research type
Research Study
Full title
A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Oral Dose Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 349 in PiZZ and PiMZ/MASH Adult Participants
IRAS ID
1010596
Contact name
Upasana Sachar
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Clinicaltrials.gov Identifier
Research summary
Alpha-1 antitrypsin deficiency (AATD) is an inherited condition which affects the liver and lungs and may cause them to not function properly over time. AATD results from several possible genetic mutations to the SERPINIA1 gene, leading to a reduction in the amount of functioning Alpa-1 antitrypsin (AAT) protein in the blood. A functional protein is important to the proper function of the liver and lungs.
This research study will investigate the safety and initial activity of a potential new medicine (BMN 349). BMN 349 has been developed by BioMarin Pharmaceutical Inc. (the Sponsor) who is funding and overseeing this clinical trial. This is a phase I study and is the first time BMN 349 will be given to people with AATD. BMN 349 has already been tested in another study with healthy volunteers, and no serious side effects were seen.
Approximately 12 people with genetically confirmed AATD will participate in this study. They will receive a single dose of BMN 349 and will be monitored for any side effects. Blood samples will be taken to look at how BMN 349 is distributed and metabolised within the body and to look at what effects it has.
This study is running at a number of hospitals within the UK, as well as the USA. The results of this study will support the development of BMN 349 as a possible treatment for those with AATD.
REC name
London - Central Research Ethics Committee
REC reference
24/LO/0637
Date of REC Opinion
23 Sep 2024
REC opinion
Further Information Favourable Opinion