Brief Title
A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo.
Official Title
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 Antitrypsin Deficiency.
Brief Summary
The purpose of this study is to investigate the effect of alvelestat (an oral neutrophil
elastase inhibitor) on blood and sputum biomarkers in patients with PiZZ, null or rare
variant phenotype/genotype alpha-1 anti-trypsin deficient lung disease. Change in a number of
different blood and sputum biomarkers related to lung damage, inflammation and elastase
activity will be measured over a 12 week period. The effect on lung function and respiratory
symptoms will also be measured.
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
Change from baseline on blood biomarkers of neutrophil elastase activity compared to baseline and placebo
Secondary Outcome
Change from baseline on other blood biomarkers of neutrophil elastase activity
Condition
Alpha 1-Antitrypsin Deficiency
Intervention
Placebo Oral Tablet
Study Arms / Comparison Groups
Placebo oral tablet
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
99
Start Date
October 29, 2018
Completion Date
March 30, 2022
Primary Completion Date
March 2, 2022
Eligibility Criteria
Inclusion Criteria:
- Patients with a confirmed diagnosis of alpha-1-anti-trypsin deficiency and a PiZZ,
null or other rare geno/phenotype and serum anti-alpha1 antitrypsin levels of less
than 11uM
- FEV1 ≥20% predicted
- Computerised tomography (CT) scan evidence of emphysema
- Non-smokers
Exclusion Criteria:
- Primary diagnosis of bronchiectasis
- An ongoing acute exacerbation of the underlying lung disease
- Underlying liver disease or abnormal liver function tests
- Previous augmentation therapy within 6 months of dosing
Gender
All
Ages
18 Years - 75 Years
Accepts Healthy Volunteers
No
Contacts
Robert Stockley, Prof., ,
Location Countries
Belgium
Location Countries
Belgium
Administrative Informations
NCT ID
NCT03636347
Organization ID
MPH966-2-01
Secondary IDs
2018-001309-95
Responsible Party
Sponsor
Study Sponsor
Mereo BioPharma
Collaborators
Syneos Health
Study Sponsor
Robert Stockley, Prof., Principal Investigator, University of Birmingham
Verification Date
April 2022