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Trial record 1 of 1 for:    INS415 | Nontuberculous Mycobacterial (NTM) Lung Infection Caused by Mycobacterium avium Complex (MAC)
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Validation of Patient Reported Outcome Measures in Participants With Nontuberculous Mycobacterial Lung Infection Caused by Mycobacterium Avium Complex (ARISE)

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ClinicalTrials.gov Identifier: NCT04677543
Recruitment Status : Recruiting
First Posted : December 21, 2020
Last Update Posted : July 19, 2021
Sponsor:
Information provided by (Responsible Party):
Insmed Incorporated

Brief Summary:
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).

Condition or disease Intervention/treatment Phase
Mycobacterium Infections, Nontuberculous Drug: ALIS Drug: Azithromycin Drug: Ethambutol Drug: ELC Phase 3

Detailed Description:
Participants will also complete the EXAcerbations of Chronic Pulmonary Disease Tool (EXACT), EXACT Respiratory Symptoms (EXACT-RS), St. George Respiratory Questionnaire (SGRQ), Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-Fatigue), Patient Global Impression of Severity - Respiratory (PGIS-Respiratory),and Patient Global Impression of Severity - Fatigue (PGIS-Fatigue) at baseline and throughout the study as anchors for the validation of the QoL-B and PROMIS F-SF 7a.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Other
Official Title: 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)
Actual Study Start Date : December 22, 2020
Estimated Primary Completion Date : February 28, 2022
Estimated Study Completion Date : February 28, 2022


Arm Intervention/treatment
Active Comparator: 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 tablets orally, 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 Name: AZI

Drug: Ethambutol
Oral tablet
Other Name: ETH

Placebo Comparator: ELC + Azithromycin + Ethambutol
Participants will be administered ELC (empty liposome control), a matching placebo to ALIS, once daily. Participants will also be administered azithromycin 250 mg and ethambutol 15 mg/kg tablets orally, once daily.
Drug: Azithromycin
Oral tablet
Other Name: AZI

Drug: Ethambutol
Oral tablet
Other Name: ETH

Drug: ELC
Inhalation via nebulization over approximately 6 to 15 minutes.
Other Name: Empty liposome control




Primary Outcome Measures :
  1. Change from Baseline Score on the Quality of Life - Bronchiectasis (QoL-B) Questionnaire [ Time Frame: Baseline to Month 7 ]
  2. 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 :
  1. Percentage of Participants Achieving Culture Conversion for Myobacterium avium Complex (MAC) [ Time Frame: By Month 6 ]
  2. 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 ]
  3. 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 ]
  4. 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)

  5. 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

  6. Percentage of Participants Who Develop a Mycobacterium avium Complex (MAC) Isolate with ALIS [ Time Frame: Up to Month 7 ]
  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 ]
  8. 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 ]
  9. Number of Participants Who Experience an Adverse Event of Special Interest [ Time Frame: Baseline to Month 7 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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 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:

  • 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

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): NCT04677543


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: NCT04677543    
Other Study ID Numbers: INS-415
2020-002545-42 ( EudraCT Number )
First Posted: December 21, 2020    Key Record Dates
Last Update Posted: July 19, 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
Psychometric validation
Patient-reported outcome
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