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 : May 31, 2023
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|Condition or disease||Intervention/treatment||Phase|
|Mycobacterium Infections, Nontuberculous||Drug: ALIS Drug: Azithromycin Drug: Ethambutol Drug: ELC (matching placebo for ALIS)||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||250 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||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 Mycobact|
|Actual Study Start Date :||April 1, 2021|
|Estimated Primary Completion Date :||August 21, 2023|
|Estimated Study Completion Date :||August 21, 2023|
Experimental: 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.
Inhalation via nebulization over approximately 6 to 15 minutes
Placebo Comparator: 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.
Drug: ELC (matching placebo for ALIS)
Inhalation via nebulization over approximately 6 to 15 minutes
Other Name: Empty liposome control
- Change from Baseline in Respiratory Symptom Score at Month 13 [ Time Frame: Baseline to Month 13 ]
- Percentage of Participants Achieving Durable Culture Conversion at Month 15 [ Time Frame: Month 15 ]
- Change from Baseline in Fatigue Symptom Score at Month 13 [ Time Frame: Baseline to Month 13 ]
- Percentage of Participants Achieving Culture Conversion by Month 12 [ Time Frame: Month 12 ]
- Percentage of Participants Achieving Culture Conversion by Month 6 [ Time Frame: Month 6 ]
- Percentage of Participants Achieving Culture Conversion During Treatment [ Time Frame: Baseline to Month 12 ]
- Time to Culture Conversion [ Time Frame: Baseline to Month 12 ]
- Time to the First Negative Culture [ Time Frame: Baseline to Month 12 ]
- 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 ]
- Percentage of Participants Achieving Culture Conversion and a Subsequent at Least one MAC Positive Culture in Agar Media or Broth Media in at Least 2 Consecutive Visits With Matching Species and Genome at Screening/ Baseline [ Time Frame: Baseline to Month 15 ]
- Percentage of Participants Achieving Culture Conversion and a Subsequently have at least One MAC Positive Culture in Agar Media or Broth Media in at Least 2 Consecutive Visits With Differing Species and/or Genome at Screening/ Baseline [ Time Frame: Baseline to Month 15 ]
- Percentage of Participants Meeting the Within-subject Meaningful Change Threshold as Reflected in Patient Reported Outcome Changes Scores Computed from Baseline in Participant-reported Symptoms [ Time Frame: Baseline to Month 15 ]
- Number of Participants Who Experience an Adverse Event (AEs) [ Time Frame: Baseline to Month 15 ]
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|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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.
- Diagnosis of cystic fibrosis (CF).
- History of more than 3 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 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 to ethambutol, azithromycin (including other macrolides or ketolides), or any of their excipients per local labeling guidance.
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
|Contact: Insmed Medical Information||844-4-INSMEDemail@example.com|
|Responsible Party:||Insmed Incorporated|
|Other Study ID Numbers:||
2020-003079-16 ( EudraCT Number )
|First Posted:||December 21, 2020 Key Record Dates|
|Last Update Posted:||May 31, 2023|
|Last Verified:||May 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Nontuberculous Mycobacterial Lung Infection
Mycobacterium avium complex
Mycobacterium Infections, Nontuberculous
Gram-Positive Bacterial Infections
Bacterial Infections and Mycoses