Clinical Trials List
Protocol NumberINS-415
NCT Number(ClinicalTrials.gov Identfier)NCT04677543
2021-02-01 - 2022-06-16
Phase III
Not yet recruiting6
Recruiting1
ICD-10A31.0
Pulmonary mycobacterial infection
ICD-9031.0
Pulmonary diseases due to other mycobacteria
ARISE - A Randomized, Double-Blind, Placebo-Controlled, Active Comparator, Multicenter Study to Validate Patient-Reported Outcome Instruments in Adult Subjects With Newly Diagnosed Nontuberculous Mycobacterial (NTM) Lung Infection Caused by Mycobacterium Avium Complex (MAC)
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Sponsor
Insmed Incorporated
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
- 鄭孟軒 無
- Hung-Ling Huang 未分科
- Hung-Ling Huang 無
- 陳家閔 無
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Nontuberculous Mycobacterial (NTM) Lung Infection Caused by Mycobacterium Avium Complex (MAC)
Objectives
The primary objective of this study is to generate evidence demonstrating the domain specification via modern psychometric methods, reliability, validity, and responsiveness of Quality of Life - Bronchiectasis (QoL-B) and Patient-Reported Outcome Measurement Information System - Fatigue-Short Form (PROMIS F-SF 7a) in participants with newly diagnosed nontuberculous mycobacterial (NTM) lung infection caused by Myobaxterium avium Complex (MAC).
Test Drug
Amikacin liposome inhalation suspension (ALIS)
Active Ingredient
Amikacin (Amikacin Sulfate)
Dosage Form
liposome inhalation suspension
liposome inhalation suspension
liposome inhalation suspension
Dosage
70mg/ml
70mg/ml
70mg/ml
Endpoints
Primary Outcome Measures :
Change from Baseline Score on the Quality of Life - Bronchiectasis (QoL-B) Questionnaire [ Time Frame: Baseline to Month 7 ]
Change from Baseline Score on the Patient-Reported Outcome Measurement Information System - Fatigue-Short Form 7a (PROMIS F-SF 7a) Questionnaire [ Time Frame: Baseline to Month 7 ]
Secondary Outcome Measures :
Percentage of Participants Achieving Culture Conversion for Myobacterium avium Complex (MAC) [ Time Frame: By Month 6 ]
Change from Baseline in Respiratory Symptoms as Assessed by the Quality of Life - Bronchiectasis (QoL-B) Questionnaire After 1 Month Off Treatment [ Time Frame: Baseline to Month 7 ]
Change from Baseline in Fatigue Symptoms as Assessed by the Patient-Reported Outcome Measurement Information System - Fatigue-Short Form 7a (PROMIS F-SF 7a) Questionnaire After 1 Month Off Treatment [ Time Frame: Baseline to Month 7 ]
Time to Culture Conversion for Myobacterium avium Complex (MAC) [ Time Frame: Baseline up to Month 6 ]
Time to culture conversion is defined as the time from baseline to the first two consecutive negative cultures for Myobacterium avium Complex (MAC)
Time to First Negative Culture [ Time Frame: Baseline up to Month 6 ]
Time to first negative culture is defined as the time from baseline to the first negative culture
Percentage of Participants Who Develop a Mycobacterium avium Complex (MAC) Isolate with ALIS [ Time Frame: Up to Month 7 ]
Percentage of Participants Achieving Culture Conversion and Subsequently Have a Mycobacterium avium Complex (MAC) Matching the Baseline Culture [ Time Frame: Baseline to Month 7 ]
Percentage of Participants Achieving Culture Conversion and Subsequently Have a Mycobacterium avium Complex (MAC) Positive Culture Different from the Baseline Culture [ Time Frame: Baseline to Month 7 ]
Number of Participants Who Experience an Adverse Event of Special Interest [ Time Frame: Baseline to Month 7 ]
Change from Baseline Score on the Quality of Life - Bronchiectasis (QoL-B) Questionnaire [ Time Frame: Baseline to Month 7 ]
Change from Baseline Score on the Patient-Reported Outcome Measurement Information System - Fatigue-Short Form 7a (PROMIS F-SF 7a) Questionnaire [ Time Frame: Baseline to Month 7 ]
Secondary Outcome Measures :
Percentage of Participants Achieving Culture Conversion for Myobacterium avium Complex (MAC) [ Time Frame: By Month 6 ]
Change from Baseline in Respiratory Symptoms as Assessed by the Quality of Life - Bronchiectasis (QoL-B) Questionnaire After 1 Month Off Treatment [ Time Frame: Baseline to Month 7 ]
Change from Baseline in Fatigue Symptoms as Assessed by the Patient-Reported Outcome Measurement Information System - Fatigue-Short Form 7a (PROMIS F-SF 7a) Questionnaire After 1 Month Off Treatment [ Time Frame: Baseline to Month 7 ]
Time to Culture Conversion for Myobacterium avium Complex (MAC) [ Time Frame: Baseline up to Month 6 ]
Time to culture conversion is defined as the time from baseline to the first two consecutive negative cultures for Myobacterium avium Complex (MAC)
Time to First Negative Culture [ Time Frame: Baseline up to Month 6 ]
Time to first negative culture is defined as the time from baseline to the first negative culture
Percentage of Participants Who Develop a Mycobacterium avium Complex (MAC) Isolate with ALIS [ Time Frame: Up to Month 7 ]
Percentage of Participants Achieving Culture Conversion and Subsequently Have a Mycobacterium avium Complex (MAC) Matching the Baseline Culture [ Time Frame: Baseline to Month 7 ]
Percentage of Participants Achieving Culture Conversion and Subsequently Have a Mycobacterium avium Complex (MAC) Positive Culture Different from the Baseline Culture [ Time Frame: Baseline to Month 7 ]
Number of Participants Who Experience an Adverse Event of Special Interest [ Time Frame: Baseline to Month 7 ]
Inclution Criteria
Inclusion Criteria:
Male or female, ≥ 18 years of age (20 years or older in Japan)
Current diagnosis of Myobacterium avium Complex (MAC) lung infection (initial, second, or third infection event)
Positive sputum culture for MAC within 6 months prior to screening
A high-resolution chest computerized tomography (CT) scan or chest CT scan with contrast, read locally, within 6 months prior to Screening and provided to the Sponsor. 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
Ability to produce (spontaneously or with induction) approximately 2mL of sputum for mycobacteriology at screening
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
Be able to comply with study drug use, study visits, and study procedures as defined by the protocol
Male or female, ≥ 18 years of age (20 years or older in Japan)
Current diagnosis of Myobacterium avium Complex (MAC) lung infection (initial, second, or third infection event)
Positive sputum culture for MAC within 6 months prior to screening
A high-resolution chest computerized tomography (CT) scan or chest CT scan with contrast, read locally, within 6 months prior to Screening and provided to the Sponsor. 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
Ability to produce (spontaneously or with induction) approximately 2mL of sputum for mycobacteriology at screening
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
Be able to comply with study drug use, study visits, and study procedures as defined by the protocol
Exclusion Criteria
Exclusion Criteria:
Diagnosis of cystic fibrosis (CF)
History of 3 or more 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 amikacin liposome inhalation suspension (ALIS) (including clinical study)
Known hypersensitivity to aminoglycosides
Disseminated MAC infection
Positive pregnancy test or lactation at Screening. All WOCBP will be tested. Women not of childbearing potential are defined as postmenopausal (ie, amenorrheic for 12 months without an alternative medical cause), or naturally or surgically sterile through bilateral oophorectomy, hysterectomy, or bilateral salpingectomy. For women under the age of 45, confirmatory testing with follicle stimulating hormone should be considered.)
Administration of any investigational drug within 8 weeks prior to Screening
Acquired and primary immunodeficiency syndromes (eg, HIV-positive, regardless of CD4 counts)
Current alcohol, medication, or illicit drug abuse
Diagnosis of cystic fibrosis (CF)
History of 3 or more 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 amikacin liposome inhalation suspension (ALIS) (including clinical study)
Known hypersensitivity to aminoglycosides
Disseminated MAC infection
Positive pregnancy test or lactation at Screening. All WOCBP will be tested. Women not of childbearing potential are defined as postmenopausal (ie, amenorrheic for 12 months without an alternative medical cause), or naturally or surgically sterile through bilateral oophorectomy, hysterectomy, or bilateral salpingectomy. For women under the age of 45, confirmatory testing with follicle stimulating hormone should be considered.)
Administration of any investigational drug within 8 weeks prior to Screening
Acquired and primary immunodeficiency syndromes (eg, HIV-positive, regardless of CD4 counts)
Current alcohol, medication, or illicit drug abuse
The Estimated Number of Participants
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Taiwan
15 participants
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Global
250 participants
