MedPath

Assessment of INS1007 in Participants With Non-Cystic Fibrosis Bronchiectasis

Phase 2
Completed
Conditions
Non-Cystic Fibrosis Bronchiectasis
Interventions
Registration Number
NCT03218917
Lead Sponsor
Insmed Incorporated
Brief Summary

The purpose of the study is to evaluate if INS1007 can reduce pulmonary exacerbations over a 24-week treatment period in participants with non-cystic fibrosis bronchiectasis.

Detailed Description

Phase 2 randomized, double-blind, placebo-controlled, parallel-group, multicenter, multi-national study to assess the efficacy, safety and tolerability, and pharmacokinetics (PK) of INS1007 administered once daily for 24 weeks in participants with non-cystic fibrosis bronchiectasis (NCFBE).

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
256
Inclusion Criteria
  1. Clinical history consistent with NCFBE (cough, chronic sputum production and/or recurrent respiratory infections)
  2. Are current sputum producers with a history of chronic expectoration and able to provide a sputum sample during Screening
  3. Have at least 2 documented pulmonary exacerbations in the past 12 months before Screening
Exclusion Criteria
  1. Have a primary diagnosis of chronic obstructive pulmonary disease (COPD) or asthma
  2. Have bronchiectasis due to cystic fibrosis (CF), hypogammaglobulinemia, common variable immunodeficiency, or alpha1-antitrypsin deficiency
  3. Are current smokers
  4. Are currently being treated for a nontuberculous mycobacterial lung infection, allergic bronchopulmonary aspergillosis, or tuberculosis
  5. Have any acute infections, (including respiratory infections)

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Brensocatib 25 mgBrensocatib 25 mgParticipants received brensocatib 25 mg QD before breakfast, for 24 weeks.
PlaceboPlaceboParticipants received the matching placebo QD before breakfast, for 24 weeks.
Brensocatib 10 mgBrensocatib 10 mgParticipants received brensocatib 10 mg once daily (QD) before breakfast, for 24 weeks.
Primary Outcome Measures
NameTimeMethod
Time to the First Pulmonary Exacerbation Over 24-Week Treatment PeriodBaseline (Day 1) to Week 24

Time to first pulmonary exacerbation was calculated as the number of days from the date of randomization to the date of first documentation of an exacerbation. Pulmonary exacerbation was defined as having 3 or more of the following symptoms for at least 48 hours resulting in a physician's decision to prescribe antibiotics: 1. Increased cough 2. Increased sputum volume or change in sputum consistency 3. Increased sputum purulence 4. Increased breathlessness and/or decreased exercise tolerance 5. Fatigue and/or malaise 6. Hemoptysis A minimum of 4 weeks must have occurred between one exacerbation onset and the next. Any exacerbation that occurred less than 4 weeks from the prior exacerbation was not considered a new exacerbation. The analysis was performed using the stratified log rank test and using Kaplan Meier (KM) curves.

Secondary Outcome Measures
NameTimeMethod
Change From Screening in Post-Bronchodilator Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Over 24-Week Treatment PeriodScreening (Days -42 to -1) to Week 24

FEV1 was used to assess lung function and is the maximum amount of air that can be forced out in one second after taking a deep breath. The percent predicted FEV1 was calculated by converting the spirometer reading to a percentage of what would be predicted as normal FEV1 based on a several personal factors (e.g. sex, age, etc.). Change from screening in percent predicted FEV1 to Week 24 was calculated as: percent predicted FEV1 value at Week 24 and percent predicted FEV1 value at screening. A positive percent change from screening indicates an improvement in lung function. The analysis was done using analysis of covariance (ANCOVA) with Pa colonization status and maintenance macrolide antibiotic use at Baseline as covariates.

Change From Baseline in Quality of Life Questionnaire - Bronchiectasis (QOL-B) Respiratory Symptoms Domain Score Over 24 Week Treatment PeriodBaseline (Day 1) to Week 24

The QOL-B is a validated, self-administered patient reported outcome (PRO) that assesses symptoms, functioning, and health-related (HR) QOL for participants with non-cystic fibrosis bronchiectasis (NCFBE). The QOL-B contains 37 items in 8 domains (Respiratory Symptoms, Physical Functioning, Role Functioning, Emotional Functioning, Social Functioning, Vitality, Health Perceptions and Treatment Burden). Each of the 37 items is scored from 1 to 4, and each of the 8 domains scale scores is standardized on a 0-100 point scale, with higher scores representing fewer symptoms or better functioning and HR QoL. A positive change from Baseline indicates improvement in symptoms. For this outcome measure, change in the respiratory symptoms domain score from Baseline was reported. The analysis was based on mixed model for repeated measures (MMRM) approach.

Change From Baseline in Concentration of Active Neutrophil Elastase (NE) in SputumBaseline (Day 1) to Week 24

The concentration of active NE in sputum, was measured by the difference between the pre-treatment concentration and on-treatment concentration. In bronchiectasis, activation of neutrophils in the airway leads to release of NE which leads to damaged airway walls, mucus hypersecretion, exacerbated inflammation, which in turn affects neutrophil and macrophage functions, increasing the risk of infection. Negative change from Baseline indicates improvement.

Number of Participants Who Experienced a Pulmonary ExacerbationBaseline (Day 1) to Week 24

Pulmonary exacerbation was defined as having 3 or more of the following symptoms for at least 48 hours resulting in a physician's decision to prescribe antibiotics. 1. Increased cough 2. Increased sputum volume or change in sputum consistency 3. Increased sputum purulence 4. Increased breathlessness and/or decreased exercise tolerance 5. Fatigue and/or malaise 6. Hemoptysis A minimum of 4 weeks must have occurred between one exacerbation onset and the next. Any exacerbation that occurred less than 4 weeks from the prior exacerbation was not considered a new exacerbation.

Trial Locations

Locations (108)

MHAT "Dr IvanSeliminski"-Sliven

🇧🇬

Sliven, Bulgaria

Cardioart Medical Center

🇧🇬

Stara Zagora, Bulgaria

Sjællands Universitetshospital, Roskilde

🇩🇰

Roskilde, Denmark

Klinikum der Universität München Medizinische Klinik V Pneumologie / Mukoviszidose-Zentrum für Erwachsene

🇩🇪

Munich, Bavaria, Germany

Krankenhaus Donaustauf

🇩🇪

Donaustauf, Bavaria, Germany

Universitätsklinikum Frankfurt

🇩🇪

Frankfurt, Hessen, Germany

Klinikum Nuernberg

🇩🇪

Nuernberg, Bayern, Germany

IKF Pneumologie Frankfurt

🇩🇪

Frankfurt, Hessen, Germany

Ruhrlandklinik Essen, University Essen

🇩🇪

Essen, NRW, Germany

Research Center for Medical Studies (RCMS)

🇩🇪

Berlin, Germany

Forschungszentrum Borstel

🇩🇪

Borstel, Schleswig-Holstein, Germany

Universitätsklinikum Jena

🇩🇪

Jena, Thuringen, Germany

Städt. Klinikum München GmbH, Bogenhausen

🇩🇪

München, Germany

Ospedale San Gerardo

🇮🇹

Monza, Monza E Brianza, Italy

Fondazione IRCCS Policlinico San Matteo

🇮🇹

Pavia, Italy

ICS Maugeri spa SB, IRCSS Telese

🇮🇹

Telese Terme, Italy

Gachon University Gil Medical Center

🇰🇷

Incheon, Namdong-gu, Korea, Republic of

Inha University Hospital

🇰🇷

Incheon, Jung-gu, Korea, Republic of

Chonnam National University Hospital

🇰🇷

Gwangju, Korea, Republic of

Seoul National University Hospital

🇰🇷

Seoul, Korea, Republic of

The Catholic University of Korea, St. Paul's Hospital

🇰🇷

Seoul, Korea, Republic of

The Catholic University of Korea Incheon St. Mary's Hospital

🇰🇷

Incheon, Korea, Republic of

Asan Medical Center

🇰🇷

Seoul, Korea, Republic of

Konkuk University Medical Center

🇰🇷

Seoul, Korea, Republic of

Noordwest Ziekenhuis Group

🇳🇱

Alkmaar, Netherlands

The Catholic University of Korea, Seoul St. Mary's Hospital

🇰🇷

Seoul, Korea, Republic of

Amphia Longresearch Astma/COPD Uni 52/5e etage

🇳🇱

Breda, Netherlands

RadboudUMC, location Dekkerswald

🇳🇱

Nijmegen, Netherlands

Zuyderland MC

🇳🇱

Sittard, Netherlands

P3 Research

🇳🇿

Tauranga, BOP, New Zealand

P3 Research (Hawke's Bay)

🇳🇿

Hastings, Hawkes Bay, New Zealand

Southern District health Board/Dunedin Hospital

🇳🇿

Dunedin, Otago, New Zealand

Waikato District Health Board

🇳🇿

Hamilton, Waikato, New Zealand

Auckland District Health Board, Greenlane clinical Centre

🇳🇿

Auckland, New Zealand

Centrum Badan Klinicznych, Piotr Napora Lekarze i Spolka Partnerska, Osrodek Badan Wczesnej Fazy

🇵🇱

Wrocław, Dolnośląskie, Poland

Grażyna Jasieniak Pinis Niepubliczny Zakład Opieki Zdrowotnej Atopia

🇵🇱

Kraków, Poland

NZOZ Krak-Medyk Sp. z o.o

🇵🇱

Kraków, Poland

Gabinet Lekarski Pediatryczno-Alergologiczny Zenon Bukowczan

🇵🇱

Sucha Beskidzka, Poland

Medical University of Lodz Poland

🇵🇱

Łódź, Poland

Singapore General Hospital

🇸🇬

Singapore, Singapore

Changi General Hospital

🇸🇬

Singapore, Singapore

Hospital Clinic de Barcelona

🇪🇸

Barcelona, Spain

Hospital Universitari de Bellvitge

🇪🇸

Barcelona, Spain

Hospital de la Santa Creu i Sant Pau

🇪🇸

Barcelona, Spain

Hospital Universiatrio y politecnico La Fe

🇪🇸

Valencia, Spain

Hospital Universitari de Girona Dr. Josep Trueta

🇪🇸

Girona, Spain

NHS Tayside

🇬🇧

Dundee, Angus, United Kingdom

Liverpool Heart and Chest Hospital NHS Foundation Trust

🇬🇧

Liverpool, Merseyside, United Kingdom

The Royal Wolverhampton NHS Trust

🇬🇧

Wolverhampton, West Midlands, United Kingdom

Royal Brompton & Harefield NHS Foundation Trust

🇬🇧

London, United Kingdom

Pennine Acute Hospitals NHS Trust

🇬🇧

Manchester, United Kingdom

University Hospital Southampton NHS Foundation Trust

🇬🇧

Southampton, United Kingdom

Institute for Respiratory Health

🇦🇺

Nedlands, Western Australia, Australia

University of Florida

🇺🇸

Gainesville, Florida, United States

University of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

NewportNativeMD

🇺🇸

Newport Beach, California, United States

National Jewish Heath

🇺🇸

Denver, Colorado, United States

St. Francis Medical Institute

🇺🇸

Clearwater, Florida, United States

San Marcus Research Clinic, Inc.

🇺🇸

Miami Lakes, Florida, United States

The Emory Clinic, Pulmonology

🇺🇸

Atlanta, Georgia, United States

University of Chicago

🇺🇸

Chicago, Illinois, United States

Johns Hopkins University, Division of Pulmonary and Critical Care Medicine

🇺🇸

Baltimore, Maryland, United States

University Hospitals Cleveland Medical Center

🇺🇸

Cleveland, Ohio, United States

Cleveland Clinic

🇺🇸

Cleveland, Ohio, United States

Oregon Health & Science University

🇺🇸

Portland, Oregon, United States

Temple University Hospital

🇺🇸

Philadelphia, Pennsylvania, United States

Respiratory Clinical Trials Unit, Royal Adelaide Hospital

🇦🇺

Adelaide, South Australia, Australia

Hvidovre Hospital

🇩🇰

Hvidovre, Capital Region, Denmark

Aarhus University Hospital

🇩🇰

Aarhus, Denmark

University of Milan, IRCCS Fondazione Ca' Granda Ospedale Maggiore Policlinico

🇮🇹

Milan, Lombardia, Italy

University Hospital of Pisa

🇮🇹

Pisa, Via Paradisa 2, Italy

Medical Center ReSpiro Ltd (Kiselov)

🇧🇬

Razgrad, Momina, Bulgaria

Phoenix Medical Group

🇺🇸

Peoria, Arizona, United States

UConn Health Center

🇺🇸

Farmington, Connecticut, United States

MedStar Georgetown University Hospital

🇺🇸

Washington, District of Columbia, United States

Box Hill Hospital, Eastern Clinical Research Unit

🇦🇺

Box Hill, Victoria, Australia

NHS Lothian

🇬🇧

Edinburgh, Scotland, United Kingdom

Papworth Hospital NHS Foundation Trust

🇬🇧

Cambridge, Cambridgeshire, United Kingdom

Ashford and St Peter's Hospitals NHS Foundation Trust

🇬🇧

Chertsey, Surrey, United Kingdom

UC Davis Medical Center

🇺🇸

Sacramento, California, United States

Palmtree Clinical Research

🇺🇸

Palm Springs, California, United States

Stanford University Medical Center

🇺🇸

Stanford, California, United States

Clinical Research Specialists, LLC

🇺🇸

Celebration, Florida, United States

University of Miami Miller School of Medicine

🇺🇸

Miami, Florida, United States

Sarasota Memorial Hospital Clinical Research Center

🇺🇸

Sarasota, Florida, United States

University of Iowa

🇺🇸

Iowa City, Iowa, United States

LSU Health Sciences Center

🇺🇸

New Orleans, Louisiana, United States

Pulmonary and Critical Care Associates of Baltimore

🇺🇸

Towson, Maryland, United States

Michigan Medicine

🇺🇸

Ann Arbor, Michigan, United States

The University of North Carolina at Chapel Hill

🇺🇸

Chapel Hill, North Carolina, United States

Columbia University Medical Center/ New York Presbyterian Hospital

🇺🇸

New York, New York, United States

Novant Health Pulmonary Medicine South

🇺🇸

Charlotte, North Carolina, United States

Mid Atlantic Pulmonary & Research Center

🇺🇸

Westminster, Maryland, United States

NYU Pulmonary & Critical Care Associates

🇺🇸

New York, New York, United States

University of Pennsylvania

🇺🇸

Philadelphia, Pennsylvania, United States

SMS Clinical Research, LLC

🇺🇸

Mesquite, Texas, United States

The University of Texas Health Science at Tyler

🇺🇸

Tyler, Texas, United States

MultiCare Institute for Research & Innovation

🇺🇸

Tacoma, Washington, United States

Concord Repatriation General Hospital

🇦🇺

Concord, New South Wales, Australia

Mater Misericordia Medical Centre

🇦🇺

Brisbane, Queensland, Australia

Gallipoli Medical Research Foundation

🇦🇺

Brisbane, Queensland, Australia

Metro North Hospital and Health Service (The Prince Charles Hospital)

🇦🇺

Chermside, Queensland, Australia

Respiratory Clinical Trials PTY LTD

🇦🇺

Kent Town, South Australia, Australia

Alfred Hospital

🇦🇺

Melbourne, Victoria, Australia

Medical Center UNIMED

🇧🇬

Plovdiv, Bulgaria

UZ Leuven

🇧🇪

Leuven, Vlaams Brabant, Belgium

Southeastern Research Center

🇺🇸

Winston-Salem, North Carolina, United States

Blackpool Teaching Hospitals NHS Foundation Trust

🇬🇧

Blackpool, Lancashire, United Kingdom

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