drugset / Trial / NCT03679598
Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency
Phase 2
Completed
63 enrolled
University of Alabama at Birmingham
Mereo BioPharma · collabNational Center for Advancing Translational Sciences (NCATS) · collabNational Institutes of Health (NIH) · collab
RandomizedParallel-groupTriple-blindTreatment
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.
Timeline
- Start
- 2019-04-08
- Primary completion
- 2023-11-30
- Completion
- 2023-12-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Comparator | Alvelestat | Small molecule | 120 mg | Oral |