A First in Class Disease Modifying Therapy to Treat Alpha-1 Antitrypsin Deficiency a Genetically Linked Orphan Disease

Trial Information
Who is this study for?Adult patients with Alpha-1 Antitrypsin Deficiency
What treatments are being studied?Alvelestat
Status: Recruiting
Location: See all (9) locations...
Intervention Type: Drug, Other
Study Type: Interventional
Study Phase: Phase 2
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.

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This content was sourced from clinicaltrials.gov