Trial Profile
A Phase 2, Proof-of-concept, Multicentre, Double-blind, Randomised, Dose-ascending, Sequential Group, Placebo-controlled Study to Evaluate the Mechanistic Effect, Safety, and Tolerability of 12 Weeks Twice Daily Oral Administration of Alvelestat (MPH966) in Participants With Alpha-1 (PiZZ or Null) Antitrypsin Deficiency
Status:
Completed
Phase of Trial:
Phase II
Latest Information Update: 14 Sep 2023
Price :
$35
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At a glance
- Drugs Alvelestat (Primary)
- Indications Alpha 1-antitrypsin deficiency
- Focus Biomarker; Pharmacodynamics; Proof of concept
- Acronyms ASTRAEUS
- Sponsors Mereo BioPharma
- 24 May 2023 Results presented at the 119th International Conference of the American Thoracic Society
- 24 May 2023 Results of an exploratory responder analysis assessing potential correlations between response of two blood biomarkers reflecting the pathogenic pathway of AATD-LD and a relevant clinical outcome presented at the 119th International Conference of the American Thoracic Society
- 23 May 2023 According to a Mereo Biopharma media release, findings from this study have informed recent and ongoing discussions with both the FDA and EMA, and validate the proposed design for our planned Phase 3 trial, which will be the first registrational study in AATD-LD to use both a patient-reported outcomes approach and clinical outcome measure as independent primary endpoints.