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

NCT04677569 | Completed Phase 3 DMC FDA Drug

• 3 other identifiers: INS-4162020-003079-162023-505273-33-00
• Study Type: interventional
• Target: 425
• Locations: 22 countries
• Sites: 242

Brief Summary

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.

Conditions

Mycobacterium Infections, Nontuberculous

Keywords

Nontuberculous Mycobacterial Lung Infection; Mycobacterium avium complex; Arikayce®

Outcome Measures

Primary Outcomes (1)

• Change from Baseline in Respiratory Symptom Score at Month 13

  Baseline to Month 13

Secondary Outcomes (13)

• Percentage of Participants Achieving Durable Culture Conversion at Month 15

  Month 15

• Change from Baseline in Fatigue Symptom Score at Month 13

  Baseline to Month 13

• Percentage of Participants Achieving Culture Conversion by Month 6

  Month 6

• Percentage of Participants Achieving Culture Conversion by Month 12

  Month 12

• Percentage of Participants Achieving Culture Conversion by Month 13

  Month 13

Study Arms (2)

ALIS + Background Regimen

EXPERIMENTAL

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.

Drug: ALIS; Drug: Azithromycin; Drug: Ethambutol

ELC + Background Regimen

PLACEBO COMPARATOR

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.

Drug: Azithromycin; Drug: Ethambutol; Drug: ELC (matching placebo for ALIS)

Interventions

ALIS DRUG

Inhalation via nebulization over approximately 6 to 15 minutes

Also known as: Amikacin liposome inhalation suspension, ARIKAYCE®

ALIS + Background Regimen

Azithromycin DRUG

Oral tablet

Also known as: AZI, Zithromax

ALIS + Background Regimen; ELC + Background Regimen

Ethambutol DRUG

Oral tablet

Also known as: ETH, Myambutol

ALIS + Background Regimen; ELC + Background Regimen

ELC (matching placebo for ALIS) DRUG

Inhalation via nebulization over approximately 6 to 15 minutes

Also known as: Empty liposome control

ELC + Background Regimen

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Insmed Incorporated: lead

Study Sites (258)

USA008

Birmingham, Alabama, 35233-1711, United States

Location

USA062

Fresno, California, 93701, United States

Location

USA060

Los Angeles, California, 90033-5313, United States

Location

USA090

Newport Beach, California, 92663, United States

Location

USA058

Sacramento, California, 95817, United States

Location

USA048

San Diego, California, 92103, United States

Location

USA081

San Francisco, California, 94143, United States

Location

USA055

Santa Barbara, California, 93105, United States

Location

USA050

Stanford, California, 94305, United States

Location

USA001

Denver, Colorado, 80206, United States

Location

USA023

Washington D.C., District of Columbia, 20007, United States

Location

USA080

Bradenton, Florida, 34209, United States

Location

USA028

Brandon, Florida, 33511-5719, United States

Location

USA003

Clearwater, Florida, 33765, United States

Location

USA043

Jacksonville, Florida, 32204, United States

Location

USA072

Jacksonville, Florida, 32207-8567, United States

Location

USA085

Jacksonville, Florida, 32209-6511, United States

Location

USA047

Jacksonville, Florida, 32224, United States

Location

USA042

Kissimmee, Florida, 34746, United States

Location

USA019

Leesburg, Florida, 34748, United States

Location

USA039

Naples, Florida, 34102-5444, United States

Location

USA096

Naples, Florida, 34102, United States

Location

USA097

Naples, Florida, 34102, United States

Location

USA046

New Port Richey, Florida, 34652, United States

Location

USA054

Sarasota, Florida, 34239-2913, United States

Location

USA084

Sebastian, Florida, 32958, United States

Location

USA026

Sebring, Florida, 33870, United States

Location

USA070

St. Petersburg, Florida, 33704-2733, United States

Location

USA014

St. Petersburg, Florida, 33707, United States

Location

USA067

Tampa, Florida, 33606, United States

Location

USA068

Tampa, Florida, 33614-7101, United States

Location

USA053

Weston, Florida, 33331, United States

Location

USA038

Atlanta, Georgia, 30342-2147, United States

Location

USA066

Augusta, Georgia, 30912, United States

Location

USA004

Lawrenceville, Georgia, 30046-3325, United States

Location

USA029

Rincon, Georgia, 31326, United States

Location

USA079

Coeur d'Alene, Idaho, 83814, United States

Location

USA031

Chicago, Illinois, 60611, United States

Location

USA073

Springfield, Illinois, 62702-4968, United States

Location

USA030

Springfield, Illinois, 62702, United States

Location

USA074

Iowa City, Iowa, 52242-1081, United States

Location

USA037

Kansas City, Kansas, 66160, United States

Location

USA002

New Orleans, Louisiana, 70112, United States

Location

USA017

Baltimore, Maryland, 21224-3057, United States

Location

USA099

Lexington, Massachusetts, 02421, United States

Location

USA077

Springfield, Massachusetts, 01107, United States

Location

USA013

Rochester, Minnesota, 55905, United States

Location

USA082

Columbia, Missouri, 65212-1000, United States

Location

USA061

St Louis, Missouri, 63110, United States

Location

USA045

Omaha, Nebraska, 68124, United States

Location

USA009

Omaha, Nebraska, 68198, United States

Location

USA069

Hillsborough, New Jersey, 08844, United States

Location

USA032

Toms River, New Jersey, 08755-8000, United States

Location

USA065

Bayside, New York, 11361, United States

Location

USA088

East Syracuse, New York, 13057, United States

Location

USA011

New York, New York, 10017, United States

Location

USA059

The Bronx, New York, 10461, United States

Location

USA076

Charlotte, North Carolina, 28204, United States

Location

USA035

Charlotte, North Carolina, 28273, United States

Location

USA033

Charlotte, North Carolina, 28277-8915, United States

Location

USA051

Durham, North Carolina, 27710-0001, United States

Location

USA034

Mooresville, North Carolina, 28117, United States

Location

USA007

Winston-Salem, North Carolina, 27103, United States

Location

USA075

Cincinnati, Ohio, 45242-4402, United States

Location

USA064

Cincinnati, Ohio, 45267, United States

Location

USA018

Cleveland, Ohio, 44195, United States

Location

USA063

Columbus, Ohio, 43210-1240, United States

Location

USA071

Portland, Oregon, 97213-2982, United States

Location

USA025

Portland, Oregon, 97239, United States

Location

USA015

DuBois, Pennsylvania, 15801, United States

Location

USA040

Philadelphia, Pennsylvania, 19104, United States

Location

USA093

Philadelphia, Pennsylvania, 19107, United States

Location

USA092

Providence, Rhode Island, 02906, United States

Location

USA044

Anderson, South Carolina, 29621, United States

Location

USA024

Charleston, South Carolina, 29414, United States

Location

USA020

Charleston, South Carolina, 29425, United States

Location

USA012

Rock Hill, South Carolina, 29732, United States

Location

USA022

Franklin, Tennessee, 37067, United States

Location

USA041

Hendersonville, Tennessee, 37075, United States

Location

USA078

Nashville, Tennessee, 37232, United States

Location

USA016

Dallas, Texas, 75246, United States

Location

USA100

Dallas, Texas, 75246, United States

Location

USA091

Denison, Texas, 75020, United States

Location

USA086

Houston, Texas, 77070-1190, United States

Location

USA021

McKinney, Texas, 75069-8085, United States

Location

USA006

San Antonio, Texas, 78258, United States

Location

USA089

Temple, Texas, 76508, United States

Location

USA052

Tyler, Texas, 75708, United States

Location

USA083

Charlottesville, Virginia, 22903, United States

Location

USA087

Madison, Wisconsin, 53792-0001, United States

Location

ARG004

Ciudad autAnoma de Buenos Aires, Buenos Aires, C1426ABP, Argentina

Location

ARG005

Quilmes, Buenos Aires, B1878FNR, Argentina

Location

ARG006

Villa Vatteone, Buenos Aires, B1853AIK, Argentina

Location

ARG003

CArdoba, CArdoba, X5003DCE, Argentina

Location

ARG002

Córdoba, CArdoba, 5000, Argentina

Location

ARG001

Rosario, Santa Fe Province, S2000DTC, Argentina

Location

ARG007

San Miguel de Tucumán, Tucumán Province, 4000, Argentina

Location

AUS006

Concord, New South Wales, 2139, Australia

Location

AUS004

New Lambton Heights, New South Wales, 2305, Australia

Location

AUS002

Westmead, New South Wales, 2145, Australia

Location

AUS012

Birtinya, Queensland, 4575, Australia

Location

AUS010

Chermside, Queensland, 4032, Australia

Location

AUS013

Douglas, Queensland, 4814, Australia

Location

AUS003

Greenslopes, Queensland, 4120, Australia

Location

AUS016

Herston, Queensland, 4029, Australia

Location

AUS014

Meadowbrook, Queensland, 4131, Australia

Location

AUS001

South Brisbane, Queensland, 4101, Australia

Location

AUS011

Woolloongabba, Queensland, 4102, Australia

Location

AUS008

Adelaide, South Australia, 5000, Australia

Location

AUS007

Clayton, Victoria, 3168, Australia

Location

AUS009

Melbourne, Victoria, 3001, Australia

Location

AUS015

Spearwood, Western Australia, 6163, Australia

Location

AUS005

Perth, 5000, Australia

Location

AUT003

Wels, OberAsterreich, 4600, Austria

Location

AUT001

Linz, Upper Austria, 4040, Austria

Location

BEL003

Brussels, Brussels Capital, 1000, Belgium

Location

BEL002

Ghent, 9000, Belgium

Location

BEL001

Leuven, 3000, Belgium

Location

CAN003

Winnepeg, Manitoba, R2H 2A6, Canada

Location

CAN002

Toronto, Ontario, M5T 2S8, Canada

Location

CAN004

Windsor, Ontario, N8X-5A6, Canada

Location

CHL001

Curicó, Maule Region, 3440000, Chile

Location

CHL002

Quillota, 2260000, Chile

Location

CHL004

Talca, Chile

Location

CHL003

Valparaíso, 2340000, Chile

Location

DNK005

Vejle, Region Syddanmark, 7100, Denmark

Location

DNK001

Roskilde, Zeeland, 4000, Denmark

Location

DNK004

Aalborg, 9000, Denmark

Location

DNK002

Aarhus N, 8200, Denmark

Location

DNK003

Odensa C, 5000, Denmark

Location

FRA012

Nice, Alpes-Maritimes, 6000, France

Location

FRA016

Strasbourg, Bas-Rhin, 67091, France

Location

FRA015

Pessac, Gironde, 33064, France

Location

FRA010

Suresnes, Hauts-de-Seine, 92151, France

Location

FRA008

Nantes, Loire-Atlantique, 44000, France

Location

FRA006

Amiens, 80054, France

Location

FRA005

Brest, 29609, France

Location

FRA007

Caen, 14033, France

Location

FRA002

Créteil, 94000, France

Location

FRA001

Marseille, 13285, France

Location

FRA014

Paris, 75013, France

Location

FRA009

Paris, 75018, France

Location

FRA013

Paris, 75020, France

Location

FRA004

Saint-Denis, 97405, France

Location

FRA003

Saint-Pierre, 97448, France

Location

FRA011

Toulouse, 31400, France

Location

GER001

Donaustauf, Bavaria, 93093, Germany

Location

GER013

Gauting, Bavaria, 82131, Germany

Location

GER005

München, Bavaria, 80335, Germany

Location

GER012

Potsdam, Brandenburg, 14467, Germany

Location

GER010

Immenhausen, Hesse, 34376, Germany

Location

GER003

Borstel, Lower Saxony, 23845, Germany

Location

GER014

Cologne, North Rhine-Westphalia, 50678, Germany

Location

GER011

Cologne, North Rhine-Westphalia, 51109, Germany

Location

GER008

Berlin, 13125, Germany

Location

GER006

Dresden, 1307, Germany

Location

GER009

Essen, 45127, Germany

Location

GER002

Frankfurt, 69590, Germany

Location

GER004

Freiburg im Breisgau, 79106, Germany

Location

GER007

Hanover, 30625, Germany

Location

GRC002

Athens, Attica, 11527, Greece

Location

GRC003

Alexandroupoli, 681 00, Greece

Location

GRC001

Heraklion, 71110, Greece

Location

HUN002

Budapest, 1121, Hungary

Location

HUN001

Törökbálint, 2045, Hungary

Location

ISR008

Tel Aviv, Tel Aviv, 64239, Israel

Location

ISR001

Ashkelon, 78100, Israel

Location

ISR002

Beer-Yaakov, 70300, Israel

Location

ISR007

Haifa, 34362, Israel

Location

ISR005

Jerusalem, 91120, Israel

Location

ISR003

Petah Tikva, 4910000, Israel

Location

ISR004

Ramat Gan, 52621, Israel

Location

ITA006

Modena, Emilia-Romagna, 41124, Italy

Location

ITA011

Reggio Emilia, Emilia-Romagna, 42123, Italy

Location

ITA010

Genoa, Liguria, 16128, Italy

Location

ITA002

Milan, Milano, 20162, Italy

Location

ITA008

Siena, Tuscany, 53100, Italy

Location

ITA004

Florence, 50134, Italy

Location

ITA001

Milan, 20122, Italy

Location

ITA009

Milan, 20123, Italy

Location

ITA007

Monza, 20900, Italy

Location

ITA003

Pavia, 27100, Italy

Location

ITA005

Roma, 00168, Italy

Location

JPN011

Nagoya, Aiti, 465-0065, Japan

Location

JPN020

Kita Kyushu-shi, Hukuoka, 807-8556, Japan

Location

JPN022

Yokohama-Shi Tsuzuki-Ku, Kanagawa, 224-0032, Japan

Location

JPN017

Matsusaka-Shi, Mie-ken, 515-0073, Japan

Location

JPN018

Nagasaki, Nagasaki, 852-8501, Japan

Location

JPN023

Kurashiki-Shi, Okayama-ken, 710-0052, Japan

Location

JPN019

Izumo-Shi, Shimane, 693-0021, Japan

Location

JPN014

Hamamatsu, Shizuoka, 434-8511, Japan

Location

JPN015

Kiyose-Shi, Tokyo, 204-0023, Japan

Location

JPN021

Ōta-ku, Tokyo, 143-8541, Japan

Location

JPN008

Shinjuku-ku, Tokyo, 1628655, Japan

Location

JPN001

Fukui, 910-8526, Japan

Location

JPN004

Fukuoka, 819-8555, Japan

Location

JPN003

Jonan-ku, Japan

Location

JPN010

Kiyose, 204-8522, Japan

Location

JPN006

Nagano, 380-0928, Japan

Location

JPN012

Naka-gun, 319-1113, Japan

Location

JPN002

Sakaishi, 591-8555, Japan

Location

JPN009

Shimonoseki, 750-8520, Japan

Location

JPN005

Tokyo, Japan

Location

JPN016

Toyonaka-Shi, Ôsaka, 560-0045, Japan

Location

NZL003

Hastings, Hawkes's Bay, 4122, New Zealand

Location

NZL002

Hamilton, Waikato Region, 3204, New Zealand

Location

NZL004

Auckland, 1051, New Zealand

Location

NZL001

Christchurch, 8461, New Zealand

Location

POL003

Kielce, Świętokrzyskie Voivodeship, 25-751, Poland

Location

PRT002

Braga, 4710-243, Portugal

Location

PRT003

Lisbon, 1796-001, Portugal

Location

KOR008

Ansan-si, Gyeonggido, 15355, South Korea

Location

KOR005

Seongnam-si, Gyeonggido, 13620, South Korea

Location

KOR007

Seongbuk-Gu, Seoul Teugbyeolsi, 02841, South Korea

Location

KOR006

Incheon, 22332, South Korea

Location

KOR004

Seoul, 03722, South Korea

Location

KOR002

Seoul, 05505, South Korea

Location

KOR009

Seoul, 06591, South Korea

Location

KOR003

Seoul, 06973, South Korea

Location

KOR001

Seoul, South Korea

Location

ESP009

L'Hospitalet de Llobregat, Barcelona, 08907, Spain

Location

ESP007

Vigo, Pontevedra, 36312, Spain

Location

ESP002

Barcelona, 08003, Spain

Location

ESP008

Barcelona, 8025, Spain

Location

ESP003

Barcelona, 8035, Spain

Location

ESP005

Girona, 17007, Spain

Location

ESP010

Madrid, 28007, Spain

Location

ESP001

Madrid, 28034, Spain

Location

ESP004

Madrid, 28046, Spain

Location

ESP011

Pamplona, 31008, Spain

Location

ESP006

Pontevedra, 36001, Spain

Location

ESP012

Seville, 41013, Spain

Location

TWN004

Chiayi City, Chiayi, 613, Taiwan

Location

TWN001

Douliu, 640, Taiwan

Location

TWN012

Hsinchu, 300, Taiwan

Location

TWN005

Kaohsiung City, 807, Taiwan

Location

TWN003

Kaohsiung City, 813, Taiwan

Location

TWN007

New Taipei City, 23561, Taiwan

Location

TWN006

Taipei, 10002, Taiwan

Location

TWN011

Taipei, 10002, Taiwan

Location

TWN010

Taipei, 110, Taiwan

Location

TWN008

Taipei, 11217, Taiwan

Location

TWN009

Taipei, 11490, Taiwan

Location

TWN002

Taipei, 116, Taiwan

Location

TUR004

Maltepe, Istanbul, 34844, Turkey (Türkiye)

Location

TUR003

Izmir, 35110, Turkey (Türkiye)

Location

TUR002

Kocaeli, 41380, Turkey (Türkiye)

Location

GBR009

Plymouth, Devon, PL6 8BQ, United Kingdom

Location

GBR012

Salford, Lancashire, M6 8HD, United Kingdom

Location

GBR013

Swansea, West Glamorgan, SA6 6NL, United Kingdom

Location

GBR008

Birmingham, West Midlands, B9 5SS, United Kingdom

Location

GBR005

Aberdeen, AB25 2ZN, United Kingdom

Location

GBR001

Cambridge, CB2 OAY, United Kingdom

Location

GBR002

Dundee, DD1 9SY, United Kingdom

Location

GBR004

Liverpool, L14 3PE, United Kingdom

Location

GBR007

London, EC1A 7BE, United Kingdom

Location

GBR010

London, NW32QG, United Kingdom

Location

GBR003

London, SW3 6NP, United Kingdom

Location

MeSH Terms

Conditions

Mycobacterium Infections, Nontuberculous; Mycobacterium avium-intracellulare Infection

Interventions

Azithromycin; Ethambutol

Condition Hierarchy (Ancestors)

Mycobacterium Infections; Actinomycetales Infections; Gram-Positive Bacterial Infections; Bacterial Infections; Bacterial Infections and Mycoses; Infections

Intervention Hierarchy (Ancestors)

Erythromycin; Macrolides; Polyketides; Lactones; Organic Chemicals; Ethylenediamines; Diamines; Polyamines; Amines

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 16, 2020
• First Posted: December 21, 2020
• Study Start: April 1, 2021
• Primary Completion: November 21, 2025
• Study Completion: January 15, 2026
• Last Updated: February 3, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

NZ (4); GR (3); KR (9); US (90); HU (2); CL (4); PT (2); ES (12); FR (16); AU (2); JP (21); PL (1); DK (5); TU (3); GB (11); CA (3); BE (3); TW (12); IL (7); DE (14); IT (11); AR (7)