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Trial record 1 of 1 for:    INS416 | Amikacin Liposome Inhalation Suspension (ALIS)-Based Regimen in Adult Subjects with Newly Diagnosed Nontuberculous Mycobacterial (NTM) Lung Infection Caused by Mycobacterium avium Complex (MAC)
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Study to Evaluate ALIS (Amikacin Liposome Inhalation Suspension) in Participants With Nontuberculous Mycobacterial Lung Infection Caused by Mycobacterium Avium Complex (ENCORE)

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ClinicalTrials.gov Identifier: NCT04677569
Recruitment Status : Recruiting
First Posted : December 21, 2020
Last Update Posted : July 26, 2021
Sponsor:
Information provided by (Responsible Party):
Insmed Incorporated

Brief Summary:
The main objective of this study is to evaluate the efficacy of ALIS (amikacin liposome inhalation suspension) + Azithromycin + Ethambutol compared to the ELC (empty liposome control) + Azithromycin + Ethambutol on participant reported respiratory symptoms at Month 13.

Condition or disease Intervention/treatment Phase
Mycobacterium Infections, Nontuberculous Drug: ALIS Drug: Azithromycin Drug: Ethambutol Drug: ELC (matching placebo for ALIS) Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 250 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: ENCORE - A Randomized, Double-Blind, Placebo-Controlled, Active Comparator, Multicenter Study to Evaluate the Efficacy and Safety of an Amikacin Liposome Inhalation Suspension (ALIS)-Based Regimen in Adult Subjects With Newly Diagnosed Nontuberculous Mycobacterial (NTM) Lung Infection Caused by Mycobacterium Avium Complex (MAC)
Actual Study Start Date : April 1, 2021
Estimated Primary Completion Date : August 21, 2023
Estimated Study Completion Date : August 21, 2023


Arm Intervention/treatment
Experimental: ALIS + Azithromycin + Ethambutol
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
Inhalation via nebulization over approximately 6 to 15 minutes
Other Names:
  • Amikacin liposome inhalation suspension
  • ARIKAYCE®

Drug: Azithromycin
Oral tablet
Other Names:
  • AZI
  • Zithromax

Drug: Ethambutol
Oral tablet
Other Names:
  • ETH
  • Myambutol

Placebo Comparator: ELC + Azithromycin + Ethambutol
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
Oral tablet
Other Names:
  • AZI
  • Zithromax

Drug: Ethambutol
Oral tablet
Other Names:
  • ETH
  • Myambutol

Drug: ELC (matching placebo for ALIS)
Inhalation via nebulization over approximately 6 to 15 minutes
Other Name: Empty liposome control




Primary Outcome Measures :
  1. Change from Baseline in Respiratory Symptom Score at Month 13 [ Time Frame: Baseline to Month 13 ]

Secondary Outcome Measures :
  1. Percentage of Participants Achieving Durable Culture Conversion at Month 15 [ Time Frame: Month 15 ]
  2. Change from Baseline in Fatigue Symptom Score at Month 13 [ Time Frame: Baseline to Month 13 ]
  3. Percentage of Participants Achieving Culture Conversion at Month 12 [ Time Frame: Month 12 ]
  4. Percentage of Participants Achieving Culture Conversion at Month 6 [ Time Frame: Month 6 ]
  5. Percentage of Participants Achieving Culture Conversion During Treatment [ Time Frame: Baseline to Month 12 ]
  6. Time to Culture Conversion [ Time Frame: Baseline to Month 12 ]
  7. Time to the First Negative Culture [ Time Frame: Baseline to Month 12 ]
  8. Percentage of Participants Who Develop a Mycobacterium avium Complex (MAC) Isolate with Amikacin Minimum Inhibitory Concentration (MIC) ≥ 128 μg/mL More Than One Time [ Time Frame: Baseline to Month 15 ]
  9. Percentage of Participants Achieving Culture Conversion and a Subsequent Positive Mycobacterium avium Complex (MAC) Culture With Matching Species and Genome to Baseline [ Time Frame: Baseline to Month 15 ]
  10. Percentage of Participants Achieving Culture Conversion and a Subsequent Positive Mycobacterium avium Complex (MAC) Culture With Differing Species and Genome to Baseline [ Time Frame: Baseline to Month 15 ]
    The MAC positive culture can be a different species or the same species but a different genome.

  11. Number of Participants Who Experience an Adverse Event of Special Interest [ Time Frame: Baseline to Month 15 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female, ≥ 18 years of age (20 years or older in Japan).
  • Current diagnosis of Mybacterial avium Complex (MAC) lung infection. MAC or mixed infection with MAC as the dominant species allowed, with MAC as the intended organism for treatment.
  • A high-resolution chest computerized tomography (CT) scan or chest CT scan with contrast, read locally, within 6 months prior to Screening. Participants who do not have a chest CT scan within 6 months prior to Screening will be required to obtain a high resolution chest CT scan or chest CT scan with contrast, read locally, during Screening.
  • Adherence to a predefined multidrug antimycobacterial regimen during the study.
  • Women of child bearing potential (WOCBP) agree to practice an acceptable method of birth control (eg, 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] or double barrier method plus a spermicidal agent, exclusive homosexual relationship, or sole male partner who has undergone surgical sterilization with confirmation of azoospermia at least 3 months post procedure) while participating in the study.
  • Provide signed informed consent prior to administration of study drug or performing any study related procedure.
  • Ability to comply with study drug use, study visits, and study procedures as defined by the protocol.

Exclusion Criteria:

  • Diagnosis of cystic fibrosis (CF).
  • History of more than 3 prior MAC lung infections.
  • 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, 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 to aminoglycosides.
  • Disseminated MAC infection.
  • Administration of any investigational drug within 8 weeks prior to Screening.
  • Acquired and primary immunodeficiency syndromes (eg, Human Immunodeficiency Virus (HIV)-positive, regardless of CD4 counts).
  • Current alcohol, medication, or illicit drug abuse.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04677569


Contacts
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Contact: Insmed Medical Information 844-4-INSMED medicalinformation@insmed.com

Locations
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Sponsors and Collaborators
Insmed Incorporated
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Responsible Party: Insmed Incorporated
ClinicalTrials.gov Identifier: NCT04677569    
Other Study ID Numbers: INS-416
2020-003079-16 ( EudraCT Number )
First Posted: December 21, 2020    Key Record Dates
Last Update Posted: July 26, 2021
Last Verified: July 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Insmed Incorporated:
Nontuberculous Myobacterial Lung Infection
Myobacterium avium complex
Arikayce®
Additional relevant MeSH terms:
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Infection
Communicable Diseases
Mycobacterium Infections
Mycobacterium avium-intracellulare Infection
Mycobacterium Infections, Nontuberculous
Actinomycetales Infections
Gram-Positive Bacterial Infections
Bacterial Infections
Azithromycin
Amikacin
Ethambutol
Anti-Bacterial Agents
Anti-Infective Agents
Antitubercular Agents