Purpose

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.

Conditions

Eligibility

Eligible Ages
Between 18 Years and 75 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Patients with a confirmed diagnosis of alpha-1-anti-trypsin deficiency and a PiZZ or null geno/phenotype and serum anti-alpha1 antitrypsin levels of less than 11uM
  • FEV1 ≥25% 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

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
placebo-controlled, dose ascending, sequential group
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description
Double-blind

Arm Groups

ArmDescriptionAssigned Intervention
Placebo Comparator
Placebo oral tablet
  • Drug: Placebo Oral Tablet
    twice daily administration
Active Comparator
Alvelestat oral tablet - dose 1
MPH966
  • Drug: Alvelestat oral tablet - dose 1
    twice daily administration
    Other names:
    • MPH966
Active Comparator
Alvelestat oral tablet - dose 2
MPH966
  • Drug: Alvelestat oral tablet - dose 2
    twice daily administration
    Other names:
    • MPH966

Recruiting Locations

University of Alabama, Birmingham (UAB)
Birmingham, Alabama 35294
Contact:
Ian Hodgson, PhD
enquiries@mereobiopharma.com

More Details

Status
Recruiting
Sponsor
Mereo BioPharma

Study Contact

Ian Hodgson, PhD
+44 (0)333 0237 300
enquiries@mereobiopharma.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.