Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency
Status:
Recruiting
Trial end date:
2022-08-01
Target enrollment:
Participant gender:
Summary
This is a Phase 2, multicenter, double-blind, randomized (1:1), placebo-controlled, 12-week,
proof-of-concept study to evaluate the safety and tolerability as well as the mechanistic
effect of oral administration of alvelestat (MPH966) in subjects with confirmed AATD defined
as Pi*ZZ, Pi*SZ, Pi*null, or another rare phenotype/genotype known to be associated with
either low (serum AAT level <11 μM or <57.2 mg/dL) or functionally impaired AAT including "F"
or "I" mutations.
Phase:
Phase 2
Details
Lead Sponsor:
University of Alabama at Birmingham
Collaborators:
Mereo BioPharma National Institutes of Health (NIH)