A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo.

Phase 2

Completed

• Conditions: Emphysema; COPD; Alpha 1-Antitrypsin Deficiency
• Interventions: Drug: Placebo Oral Tablet; Drug: Alvelestat oral tablet - dose 1; Drug: Alvelestat oral tablet - dose 2

• Registration Number: NCT03636347
• Lead Sponsor: Mereo BioPharma

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-06-20
• Study First Submitted QC: 2018-08-15
• Study First Posted: 2018-08-17
• Study Start: 2018-10-29
• Primary Completion: 2022-03-02
• Study Completion: 2022-03-30
• Status Verified: 2022-04
• Last Update Submitted: 2022-04-06
• Last Update Posted: 2022-04-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 99

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo oral tablet	Placebo Oral Tablet	-
Alvelestat oral tablet - dose 1	Alvelestat oral tablet - dose 1	MPH966
Alvelestat oral tablet - dose 2	Alvelestat oral tablet - dose 2	MPH966

Primary Outcome Measures

Name	Time	Method
Change from baseline on blood biomarkers of neutrophil elastase activity compared to baseline and placebo	12 weeks	Within-individual change from baseline up to end of treatment in: * Blood neutrophil elastase activity; * Blood Aα-Val 360 levels; * Plasma desmosine/isodesmosine levels

Secondary Outcome Measures

Name	Time	Method
Change from baseline on other blood biomarkers of neutrophil elastase activity	12 weeks	Frequency of neutrophil elastase levels below the limit of detection from baseline to end of treatment

Trial Locations

Locations (26)

University of Alabama, Birmingham (UAB); 🇺🇸 Birmingham, Alabama, United States

UCLA Medical Center; 🇺🇸 Los Angeles, California, United States

UC Davis Medical Centre; 🇺🇸 Sacramento, California, United States

PMG Research of Wilmington; 🇺🇸 Wilmington, North Carolina, United States

UZ Gent; 🇧🇪 Gent, Belgium

UZ Leuven; 🇧🇪 Leuven, Belgium

The University Lung Clinic; 🇨🇦 Edmonton, Alberta, Canada

Centre for Heart Lung Innovation, St Pauls Hospital; 🇨🇦 Vancouver, British Columbia, Canada

Inspiration Research Ltd; 🇨🇦 Toronto, Ontario, Canada

University of Saskatchewan Royal University Hospital; 🇨🇦 Saskatoon, Saskatchewan, Canada

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