NCT03636347

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.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
99

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Oct 2018

Typical duration for phase_2

Geographic Reach
8 countries

26 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 20, 2018

Completed
2 months until next milestone

First Posted

Study publicly available on registry

August 17, 2018

Completed
2 months until next milestone

Study Start

First participant enrolled

October 29, 2018

Completed
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 2, 2022

Completed
28 days until next milestone

Study Completion

Last participant's last visit for all outcomes

March 30, 2022

Completed
Last Updated

April 14, 2022

Status Verified

April 1, 2022

Enrollment Period

3.3 years

First QC Date

June 20, 2018

Last Update Submit

April 6, 2022

Conditions

Keywords

PiZZ or NullNeutrophil elastase inhibitorAlpha-1AlvelestatAZD9668MPH966Rare variant genotypeAstraeusAATDAlpha 1-Antitrypsin DeficiencyLung Disease

Outcome Measures

Primary Outcomes (1)

  • Change from baseline on blood biomarkers of neutrophil elastase activity compared to baseline and placebo

    Within-individual change from baseline up to end of treatment in: * Blood neutrophil elastase activity * Blood Aα-Val 360 levels * Plasma desmosine/isodesmosine levels

    12 weeks

Secondary Outcomes (1)

  • Change from baseline on other blood biomarkers of neutrophil elastase activity

    12 weeks

Other Outcomes (2)

  • Change from baseline in St. George's Respiratory Questionnaire (SGRQ-C) to end of treatment

    12 weeks

  • Change from baseline in pulmonary function

    12 weeks

Study Arms (3)

Placebo oral tablet

PLACEBO COMPARATOR
Drug: Placebo Oral Tablet

Alvelestat oral tablet - dose 1

ACTIVE COMPARATOR

MPH966

Drug: Alvelestat oral tablet - dose 1

Alvelestat oral tablet - dose 2

ACTIVE COMPARATOR

MPH966

Drug: Alvelestat oral tablet - dose 2

Interventions

twice daily administration

Placebo oral tablet

twice daily administration

Also known as: MPH966
Alvelestat oral tablet - dose 1

twice daily administration

Also known as: MPH966
Alvelestat oral tablet - dose 2

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (26)

University of Alabama, Birmingham (UAB)

Birmingham, Alabama, 35294, United States

Location

UCLA Medical Center

Los Angeles, California, 90095, United States

Location

UC Davis Medical Centre

Sacramento, California, 95817, United States

Location

PMG Research of Wilmington

Wilmington, North Carolina, 28401, United States

Location

UZ Gent

Ghent, 9000, Belgium

Location

UZ Leuven

Leuven, 3000, Belgium

Location

The University Lung Clinic

Edmonton, Alberta, T6G 2C8, Canada

Location

Centre for Heart Lung Innovation, St Pauls Hospital

Vancouver, British Columbia, V6Z 1Y6, Canada

Location

Inspiration Research Ltd

Toronto, Ontario, M5T 3A9, Canada

Location

University of Saskatchewan Royal University Hospital

Saskatoon, Saskatchewan, S7N 0W8, Canada

Location

Aarhus Universitetshospital

Aarhus, 8200, Denmark

Location

Gentofte Hospital

Hellerup, 2900, Denmark

Location

Synddansk Universitet (SDU) - Odense University

Odense, 5000, Denmark

Location

Instytut Gruzilicy Chorob Pluc

Warsaw, 01-138, Poland

Location

Hospital Univ Clinico San Carlos

Madrid, Spain

Location

Hospital Sierrallana

Torrelavega, Spain

Location

CTC Gothia Forum Sahlgrenska University Hospital

Gothenburg, 41345, Sweden

Location

Lund University Hospital

Lund, 22185, Sweden

Location

University Hospitals Birmingham NHS Foundation Trust

Birmingham, B15 2GW, United Kingdom

Location

Cambridge University Hospitals Foundation NHS Trust

Cambridge, CB2 0QQ, United Kingdom

Location

University Hospital Coventry and Warwickshire

Coventry, CV2 2DX, United Kingdom

Location

Royal Infirmary of Edinburgh

Edinburgh, EH16 4TJ, United Kingdom

Location

Royal Devon and Exeter NHS Trust

Exeter, PhD, United Kingdom

Location

University Hospitals of Leicester NHS Trust

Leicester, LE1 7RH, United Kingdom

Location

Royal Brompton Hospital

London, SWP 6NP, United Kingdom

Location

Southampton General Hospital

Southampton, SO16 6YD, United Kingdom

Location

MeSH Terms

Conditions

alpha 1-Antitrypsin DeficiencyEmphysemaPulmonary Disease, Chronic ObstructiveLung Diseases

Condition Hierarchy (Ancestors)

Liver DiseasesDigestive System DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSubcutaneous EmphysemaPathologic ProcessesPathological Conditions, Signs and SymptomsLung Diseases, ObstructiveChronic DiseaseDisease Attributes

Study Officials

  • Robert Stockley, Prof.

    University of Birmingham

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Masking Details
Double-blind
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: placebo-controlled, dose ascending, sequential group
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 20, 2018

First Posted

August 17, 2018

Study Start

October 29, 2018

Primary Completion

March 2, 2022

Study Completion

March 30, 2022

Last Updated

April 14, 2022

Record last verified: 2022-04

Data Sharing

IPD Sharing
Will not share

Locations