Study to Evaluate ALIS (Amikacin Liposome Inhalation Suspension) in Participants With Nontuberculous Mycobacterial Lung Infection Caused by Mycobacterium Avium Complex

NCT ID: NCT04677569

Last Updated: 2025-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

425 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-04-01

Study Completion Date

2026-01-15

Brief Summary

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The main objective of this study is to evaluate the efficacy of ALIS (amikacin liposome inhalation suspension) + background regimen (azithromycin \[AZI\] + ethambutol \[ETH\]) compared to the ELC (empty liposome control) + background regimen on participant-reported respiratory symptoms at Month 13.

Detailed Description

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Conditions

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Mycobacterium Infections, Nontuberculous

Keywords

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Nontuberculous Mycobacterial Lung Infection Mycobacterium avium complex Arikayce®

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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ALIS + Background Regimen

Participants will be administered 590 mg of ALIS (amikacin liposome inhalation suspension) once daily. Participants will also be administered azithromycin 250 mg and ethambutol 15 mg/kg, once daily.

Group Type EXPERIMENTAL

ALIS

Intervention Type DRUG

Inhalation via nebulization over approximately 6 to 15 minutes

Azithromycin

Intervention Type DRUG

Oral tablet

Ethambutol

Intervention Type DRUG

Oral tablet

ELC + Background Regimen

Participants will be administered ELC (empty liposome control), a visually matching placebo to ALIS (amikacin liposome inhalation suspension), once daily. Participants will also be administered azithromycin 250 mg and ETH (ethambutol) 15 mg/kg, once daily.

Group Type PLACEBO_COMPARATOR

Azithromycin

Intervention Type DRUG

Oral tablet

Ethambutol

Intervention Type DRUG

Oral tablet

ELC (matching placebo for ALIS)

Intervention Type DRUG

Inhalation via nebulization over approximately 6 to 15 minutes

Interventions

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ALIS

Inhalation via nebulization over approximately 6 to 15 minutes

Intervention Type DRUG

Azithromycin

Oral tablet

Intervention Type DRUG

Ethambutol

Oral tablet

Intervention Type DRUG

ELC (matching placebo for ALIS)

Inhalation via nebulization over approximately 6 to 15 minutes

Intervention Type DRUG

Other Intervention Names

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Amikacin liposome inhalation suspension ARIKAYCE® AZI Zithromax ETH Myambutol Empty liposome control

Eligibility Criteria

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Inclusion Criteria

* Male or female, ≥ 18 years of age (19 years or older in South Korea, 20 years or older in Japan).
* Current diagnosis of MAC lung infection. MAC or mixed infection with MAC as the dominant species is allowed, with MAC as the intended organism for treatment.
* A chest computerized tomography (CT) scan, read locally, within 6 months prior to Screening to determine presence and size of pulmonary cavities. Participants who do not have a chest CT scan within 6 months prior to Screening will be required to obtain a chest CT scan, read locally, during Screening.
* Willingness and ability to adhere to prescribed study treatment during the study.
* Women of childbearing potential (WOCBP) (ie, fertile following menarche and until becoming postmenopausal unless permanently sterile) and fertile men (ie, all men after puberty unless permanently sterile by bilateral orchidectomy) agree to practice a highly effective method of birth control from Day 1 to at least 90 days after the last dose. Examples of such birth controls are:

* true abstinence (refraining from heterosexual intercourse during the entire study),
* copper intrauterine device (IUD),
* hormonal methods (levonorgestrel-releasing intrauterine system, progestogen implant, combined oral contraceptive pill \[combined with barrier method\]),
* exclusive homosexual relationship, or
* sole male partner who has undergone surgical sterilization with confirmation of azoospermia at least 3 months post procedure.
* Provide signed informed consent prior to administration of study drugs or performing any study-related procedure.
* Be able to comply with study drugs use, study visits, and study procedures as defined by the protocol.
* Men with partners who are WOCBP (pregnant or non-pregnant) agree to use condoms and non-pregnant partners should practice a highly effective method of birth control

Exclusion Criteria

* Diagnosis of cystic fibrosis (CF).
* History of more than 3 MAC lung infections (a 4th MAC lung infection is not eligible)
* Received any mycobacterial antibiotic treatment for current MAC lung infection
* Refractory MAC lung infection, defined as having positive MAC cultures while being treated with a multidrug mycobacterial antibiotic treatment regimen for a minimum of 6 consecutive months and no documented successful treatment, defined as negative sputum culture for MAC and cessation of treatment
* Relapse of prior MAC lung infection, defined as positive sputum culture for MAC ≤6 months of cessation of prior successful treatment
* Evidence of any pulmonary cavity ≥ 2 cm in diameter, as determined by chest CT scan, read locally, during Screening or within 6 months prior to Screening
* Radiographic finding of new lobar consolidation, atelectasis, significant pleural effusion, or pneumothorax during routine clinical care within 2 months prior to Screening
* Active pulmonary malignancy (primary or metastatic) or any malignancy requiring chemotherapy or radiation therapy within 1 year prior to Screening or anticipated during the study
* Acute pulmonary exacerbation (eg, chronic obstructive pulmonary disease (COPD) or bronchiectasis) requiring treatment with antibiotics, or corticosteroids (intravenous \[IV\] or oral), within 4 weeks prior to and during Screening
* Current smoker
* History of lung transplantation
* Prior exposure to ALIS (including clinical study)
* Known hypersensitivity or contraindications to use to ALIS, aminoglycosides, or any of their excipients
* Disseminated MAC infection
* Administration of any investigational drug within 8 weeks prior to Screening
* Known or suspected acquired immunodeficiency syndromes (Human Immunodeficiency Virus (HIV) -positive, regardless of CD4 counts). Other immunodeficiency syndromes that may interfere with study participation in the opinion of the Investigator
* Current alcohol, medication, or illicit drug abuse
* Known and active COVID-19 infection
* Known hypersensitivity or contraindications to use of ethambutol, azithromycin (including other macrolides or ketolides), or any of their excipients per local labeling guidance.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Insmed Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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USA008

Birmingham, Alabama, United States

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USA062

Fresno, California, United States

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USA060

Los Angeles, California, United States

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USA090

Newport Beach, California, United States

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USA058

Sacramento, California, United States

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USA048

San Diego, California, United States

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USA081

San Francisco, California, United States

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USA055

Santa Barbara, California, United States

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USA050

Stanford, California, United States

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USA001

Denver, Colorado, United States

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USA023

Washington D.C., District of Columbia, United States

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USA080

Bradenton, Florida, United States

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USA028

Brandon, Florida, United States

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USA003

Clearwater, Florida, United States

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USA043

Jacksonville, Florida, United States

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USA072

Jacksonville, Florida, United States

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USA085

Jacksonville, Florida, United States

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USA047

Jacksonville, Florida, United States

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USA042

Kissimmee, Florida, United States

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USA019

Leesburg, Florida, United States

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USA039

Naples, Florida, United States

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USA096

Naples, Florida, United States

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USA097

Naples, Florida, United States

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USA046

New Port Richey, Florida, United States

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USA054

Sarasota, Florida, United States

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USA084

Sebastian, Florida, United States

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USA026

Sebring, Florida, United States

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USA070

St. Petersburg, Florida, United States

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USA014

St. Petersburg, Florida, United States

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USA067

Tampa, Florida, United States

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USA068

Tampa, Florida, United States

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USA053

Weston, Florida, United States

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USA038

Atlanta, Georgia, United States

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USA066

Augusta, Georgia, United States

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USA004

Lawrenceville, Georgia, United States

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USA029

Rincon, Georgia, United States

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USA079

Coeur d'Alene, Idaho, United States

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USA031

Chicago, Illinois, United States

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USA073

Springfield, Illinois, United States

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USA030

Springfield, Illinois, United States

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USA074

Iowa City, Iowa, United States

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USA037

Kansas City, Kansas, United States

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USA002

New Orleans, Louisiana, United States

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USA017

Baltimore, Maryland, United States

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USA099

Lexington, Massachusetts, United States

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USA077

Springfield, Massachusetts, United States

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USA013

Rochester, Minnesota, United States

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USA082

Columbia, Missouri, United States

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USA061

St Louis, Missouri, United States

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USA045

Omaha, Nebraska, United States

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USA009

Omaha, Nebraska, United States

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USA069

Hillsborough, New Jersey, United States

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USA032

Toms River, New Jersey, United States

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USA065

Bayside, New York, United States

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USA088

East Syracuse, New York, United States

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USA011

New York, New York, United States

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USA059

The Bronx, New York, United States

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USA076

Charlotte, North Carolina, United States

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USA035

Charlotte, North Carolina, United States

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USA033

Charlotte, North Carolina, United States

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USA051

Durham, North Carolina, United States

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USA034

Mooresville, North Carolina, United States

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USA007

Winston-Salem, North Carolina, United States

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USA075

Cincinnati, Ohio, United States

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USA064

Cincinnati, Ohio, United States

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USA018

Cleveland, Ohio, United States

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USA063

Columbus, Ohio, United States

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USA071

Portland, Oregon, United States

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USA025

Portland, Oregon, United States

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USA015

DuBois, Pennsylvania, United States

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USA040

Philadelphia, Pennsylvania, United States

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USA093

Philadelphia, Pennsylvania, United States

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USA092

Providence, Rhode Island, United States

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USA044

Anderson, South Carolina, United States

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USA024

Charleston, South Carolina, United States

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USA020

Charleston, South Carolina, United States

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USA012

Rock Hill, South Carolina, United States

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USA022

Franklin, Tennessee, United States

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USA041

Hendersonville, Tennessee, United States

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USA078

Nashville, Tennessee, United States

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USA016

Dallas, Texas, United States

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USA100

Dallas, Texas, United States

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USA091

Denison, Texas, United States

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USA086

Houston, Texas, United States

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USA021

McKinney, Texas, United States

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USA006

San Antonio, Texas, United States

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USA089

Temple, Texas, United States

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USA052

Tyler, Texas, United States

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USA083

Charlottesville, Virginia, United States

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USA087

Madison, Wisconsin, United States

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ARG004

Ciudad autAnoma de Buenos Aires, Buenos Aires, Argentina

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ARG005

Quilmes, Buenos Aires, Argentina

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ARG006

Villa Vatteone, Buenos Aires, Argentina

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ARG003

CArdoba, CArdoba, Argentina

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ARG002

Córdoba, CArdoba, Argentina

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ARG001

Rosario, Santa Fe Province, Argentina

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ARG007

San Miguel de Tucumán, Tucumán Province, Argentina

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AUS006

Concord, New South Wales, Australia

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AUS004

New Lambton Heights, New South Wales, Australia

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AUS002

Westmead, New South Wales, Australia

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AUS012

Birtinya, Queensland, Australia

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AUS010

Chermside, Queensland, Australia

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AUS013

Douglas, Queensland, Australia

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AUS003

Greenslopes, Queensland, Australia

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AUS016

Herston, Queensland, Australia

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AUS014

Meadowbrook, Queensland, Australia

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AUS001

South Brisbane, Queensland, Australia

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AUS011

Woolloongabba, Queensland, Australia

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AUS008

Adelaide, South Australia, Australia

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AUS007

Clayton, Victoria, Australia

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AUS009

Melbourne, Victoria, Australia

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AUS015

Spearwood, Western Australia, Australia

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AUS005

Perth, , Australia

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AUT003

Wels, OberAsterreich, Austria

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AUT001

Linz, Upper Austria, Austria

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BEL003

Brussels, Brussels Capital, Belgium

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BEL002

Ghent, , Belgium

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BEL001

Leuven, , Belgium

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CAN003

Winnepeg, Manitoba, Canada

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CAN002

Toronto, Ontario, Canada

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CAN004

Windsor, Ontario, Canada

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CHL001

Curicó, Maule Region, Chile

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CHL002

Quillota, , Chile

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CHL004

Talca, , Chile

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CHL003

Valparaíso, , Chile

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DNK005

Vejle, Region Syddanmark, Denmark

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DNK001

Roskilde, Zeeland, Denmark

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DNK004

Aalborg, , Denmark

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DNK002

Aarhus N, , Denmark

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DNK003

Odensa C, , Denmark

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FRA012

Nice, Alpes-Maritimes, France

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FRA016

Strasbourg, Bas-Rhin, France

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FRA015

Pessac, Gironde, France

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FRA010

Suresnes, Hauts-de-Seine, France

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FRA008

Nantes, Loire-Atlantique, France

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FRA006

Amiens, , France

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FRA005

Brest, , France

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FRA007

Caen, , France

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FRA002

Créteil, , France

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FRA001

Marseille, , France

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FRA014

Paris, , France

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FRA009

Paris, , France

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FRA013

Paris, , France

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FRA004

Saint-Denis, , France

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FRA003

Saint-Pierre, , France

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FRA011

Toulouse, , France

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GER001

Donaustauf, Bavaria, Germany

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GER013

Gauting, Bavaria, Germany

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GER005

München, Bavaria, Germany

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GER012

Potsdam, Brandenburg, Germany

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GER010

Immenhausen, Hesse, Germany

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GER003

Borstel, Lower Saxony, Germany

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GER014

Cologne, North Rhine-Westphalia, Germany

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GER011

Cologne, North Rhine-Westphalia, Germany

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GER008

Berlin, , Germany

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GER006

Dresden, , Germany

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GER009

Essen, , Germany

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GER002

Frankfurt, , Germany

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GER004

Freiburg im Breisgau, , Germany

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GER007

Hanover, , Germany

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GRC002

Athens, Attica, Greece

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GRC003

Alexandroupoli, , Greece

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GRC001

Heraklion, , Greece

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HUN002

Budapest, , Hungary

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HUN001

Törökbálint, , Hungary

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ISR008

Tel Aviv, Tel Aviv, Israel

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ISR001

Ashkelon, , Israel

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ISR002

Beer-Yaakov, , Israel

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ISR007

Haifa, , Israel

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ISR005

Jerusalem, , Israel

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ISR003

Petah Tikva, , Israel

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ISR004

Ramat Gan, , Israel

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ITA006

Modena, Emilia-Romagna, Italy

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ITA011

Reggio Emilia, Emilia-Romagna, Italy

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ITA010

Genoa, Liguria, Italy

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ITA002

Milan, Milano, Italy

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ITA008

Siena, Tuscany, Italy

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ITA004

Florence, , Italy

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ITA001

Milan, , Italy

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ITA009

Milan, , Italy

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ITA007

Monza, , Italy

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ITA003

Pavia, , Italy

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ITA005

Roma, , Italy

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JPN011

Nagoya, Aiti, Japan

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JPN020

Kita Kyushu-shi, Hukuoka, Japan

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JPN022

Yokohama-Shi Tsuzuki-Ku, Kanagawa, Japan

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JPN017

Matsusaka-Shi, Mie-ken, Japan

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JPN018

Nagasaki, Nagasaki, Japan

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JPN023

Kurashiki-Shi, Okayama-ken, Japan

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JPN019

Izumo-Shi, Shimane, Japan

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JPN014

Hamamatsu, Shizuoka, Japan

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JPN015

Kiyose-Shi, Tokyo, Japan

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JPN021

Ōta-ku, Tokyo, Japan

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JPN008

Shinjuku-ku, Tokyo, Japan

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JPN001

Fukui, , Japan

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JPN004

Fukuoka, , Japan

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JPN003

Jonan-ku, , Japan

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JPN010

Kiyose, , Japan

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JPN006

Nagano, , Japan

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JPN012

Naka-gun, , Japan

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JPN002

Sakaishi, , Japan

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JPN009

Shimonoseki, , Japan

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JPN005

Tokyo, , Japan

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JPN016

Toyonaka-Shi, Ôsaka, Japan

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NZL003

Hastings, Hawkes's Bay, New Zealand

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NZL002

Hamilton, Waikato Region, New Zealand

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NZL004

Auckland, , New Zealand

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NZL001

Christchurch, , New Zealand

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POL003

Kielce, Świętokrzyskie Voivodeship, Poland

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PRT002

Braga, , Portugal

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PRT003

Lisbon, , Portugal

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KOR008

Ansan-si, Gyeonggido, South Korea

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KOR005

Seongnam-si, Gyeonggido, South Korea

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KOR007

Seongbuk-Gu, Seoul Teugbyeolsi, South Korea

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KOR006

Incheon, , South Korea

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KOR004

Seoul, , South Korea

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KOR002

Seoul, , South Korea

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KOR009

Seoul, , South Korea

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KOR003

Seoul, , South Korea

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KOR001

Seoul, , South Korea

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ESP009

L'Hospitalet de Llobregat, Barcelona, Spain

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ESP007

Vigo, Pontevedra, Spain

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ESP002

Barcelona, , Spain

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ESP008

Barcelona, , Spain

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ESP003

Barcelona, , Spain

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ESP005

Girona, , Spain

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ESP010

Madrid, , Spain

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ESP001

Madrid, , Spain

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ESP004

Madrid, , Spain

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ESP011

Pamplona, , Spain

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ESP006

Pontevedra, , Spain

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ESP012

Seville, , Spain

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TWN004

Chiayi City, Chiayi, Taiwan

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TWN001

Douliu, , Taiwan

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TWN012

Hsinchu, , Taiwan

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TWN005

Kaohsiung City, , Taiwan

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TWN003

Kaohsiung City, , Taiwan

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TWN007

New Taipei City, , Taiwan

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TWN006

Taipei, , Taiwan

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TWN011

Taipei, , Taiwan

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TWN010

Taipei, , Taiwan

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TWN008

Taipei, , Taiwan

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TWN009

Taipei, , Taiwan

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TWN002

Taipei, , Taiwan

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TUR004

Maltepe, Istanbul, Turkey (Türkiye)

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TUR003

Izmir, , Turkey (Türkiye)

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TUR002

Kocaeli, , Turkey (Türkiye)

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GBR009

Plymouth, Devon, United Kingdom

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GBR012

Salford, Lancashire, United Kingdom

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GBR013

Swansea, West Glamorgan, United Kingdom

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GBR008

Birmingham, West Midlands, United Kingdom

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GBR005

Aberdeen, , United Kingdom

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GBR001

Cambridge, , United Kingdom

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GBR002

Dundee, , United Kingdom

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GBR004

Liverpool, , United Kingdom

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GBR007

London, , United Kingdom

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GBR010

London, , United Kingdom

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GBR003

London, , United Kingdom

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Countries

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United States Argentina Australia Austria Belgium Canada Chile Denmark France Germany Greece Hungary Israel Italy Japan New Zealand Poland Portugal South Korea Spain Taiwan Turkey (Türkiye) United Kingdom

Other Identifiers

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2020-003079-16

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

2023-505273-33-00

Identifier Type: OTHER

Identifier Source: secondary_id

INS-416

Identifier Type: -

Identifier Source: org_study_id