Mycobacterium Infections, NontuberculousPhase 2NCT03038178
Liposomal Amikacin for Inhalation (LAI) in the Treatment of Mycobacterium Abscessus Lung Disease
A Phase 2 study testing LAI plus multi-drug regimen in people with Mycobacterium Infections, Nontuberculous and Mycobacteria, Atypical, run by Kevin Winthrop at 2 sites in United States.
Trial summary
The proposed study will assess the efficacy, safety and tolerability of once daily dosing of Liposomal-Amikacin for Inhalation (LAI) 590 mg for 12 months plus standard of care (SOC) mycobacterial multi-drug regimen in accordance with the 2007 ATS/ IDSA guidelines, for treatment of mycobacterium abscessus lung disease. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Mycobacterium Infections, Nontuberculous, Mycobacteria, Atypical
- What is being tested?
- LAI plus multi-drug regimen
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 2 sitesUnited States
- Study dates
- Oct 2016to 31 Dec 2019
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female patients 12 years and older
- Diagnosis of M. abscessus, including all subspecies, abscessus, bolleti, and massiliense lung disease according to the 2007 ATS/IDSA criteria
- Both newly diagnosed and currently on treatment or previously treated patients will be included
- Culture positive (either sputum or bronchoscopy) for M. abscessus at time of screening
- Willingness to adhere to a treatment regimen, study visits, and study procedures during the course of the study.
- Ability to produce at approximately 3.0 mL of sputum or be willing to undergo an induction that produces approximately 3.0 mL of sputum for culture collection
- Female of childbearing potential agrees to practice an acceptable method of birth control (e.g., abstinence, hormonal or barrier methods, partner sterilization, or IUD)
- Written informed consent or assent obtained from the patient, parent or legal guardian prior to the performance of any study related procedures
Who cannot join
- Active pulmonary tuberculosis requiring treatment at screening
- Treatment with inhaled or intravenous Amikacin within 14 days prior to baseline
- Known hypersensitivity to aminoglycosides
- Aspartate aminotransferase or alanine aminotransferase ≥ 3 times the upper limit of normal or total bilirubin ≥ 2 times the upper limit of normal (ULN) at screening
- Current addiction to alcohol or illicit drug abuse
- Any condition which in the opinion of the Investigator interferes with ability to adhere to study requirements
Show all 12Show fewer
- Primary immunodeficiency syndromes and acquired immunodeficiency syndromes (e.g., HIV-positive patients regardless of CD4 counts)
- Absolute neutrophil count ≤500/μL at Screening
- Significant (as determined by the investigator) hearing loss, vestibular dysfunction, neuromuscular weakness or a diagnosis of myasthenia gravis, where the potential risk of aminoglycoside toxicity outweighs the potential benefit
- Serum creatinine >2 times ULN at Screening
- History of lung transplantation
- Any condition that, in the judgment of the Investigator, would compromise the ability of the subject to complete the study
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Oregon Health & Science University
Portland, Oregon, 97239
University of Texas Health Science Center
Tyler, Texas, 75708
About this study
Official title: An Open-label Study of Efficacy, Safety and Tolerability of Liposomal Amikacin for Inhalation (LAI) Once Daily in Addition to Standard Multi-antibiotic Therapy in the Treatment of Mycobacterium Abscessus Lung Disease
The proposed study will assess the efficacy, safety and tolerability of once daily dosing of Liposomal-Amikacin for Inhalation (LAI) 590 mg for 12 months plus standard of care (SOC) mycobacterial multi-drug regimen in accordance with the 2007 ATS/ IDSA guidelines, for treatment of mycobacterium abscessus lung disease.
Read the detailed description
This is an open-label study of efficacy, safety and tolerability of once daily dosing of Liposomal-Amikacin for Inhalation (LAI), in addition to a standard multi-drug antibiotic therapy in accordance with the 2007 ATS/ IDSA guidelines, in patients with Mycobacterium abscessus lung disease. The multi-drug therapy is determined at the discretion of the Investigator. After screening, all eligible patients will enter the trial and will receive LAI 590 mg once daily for 12 months. If deemed necessary by the investigator, dose may be adjusted from a minimum of three times per week up to daily, in cases of adverse events relating to tolerability.
All patients who enter the study will have subsequent study visits at Months 1,2,4,6,9, at End-of-Study or Month 12, and at 1 and 3 months post study drug discontinuation. At each visit (including screening), review of concomitant medications, review of adverse events, and physical exam, will be performed. Chest CT scan will be performed at baseline, 6 months, and 12 months, unless a chest CT scan has already been performed within 6 months on this time point. The 6 minute walk test and QOL-B-NTM will be performed at baseline, 6 months, 12 months, and 3 months post study drug discontinuation. All patients will have inducted sputum collected at each study visit, and patients will self-collect expectorated sputum during intervening monthly time-points until study completion to determine changes in mycobacterial smear and culture status.
Unscheduled visits will occur as needed should subjects' symptoms worsen between visits.
- Being tested
- LAI plus multi-drug regimen
- Keywords
- Mycobacterium Infections, Nontuberculous, Mycobacteria, Atypical, Amikacin, Inhalation
- Medical subject headings
- Mycobacterium Infections, Nontuberculous, Mycobacterium Infections, Respiratory Aspiration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LAI plus multi-drug regimenDrug
Also called ARIKAYCE™
Liposomal Amikacin for Inhalation (LAI) is the experimental treatment which, in this single arm will be taken in conjunction with standard of care multi-drug treatment regimen
| Study group | Role | What they receive |
|---|---|---|
| LAI plus multi-drug regimen | Experimental | once daily dosing of Liposomal-Amikacin for Inhalation (LAI) 590 mg for 12 months plus standard of care (SOC) mycobacterial multi-drug regimen in accordance with the 2007 ATS/ IDSA guidelines Receives: Drug: LAI plus multi-drug regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline sputum culture at 12 months
Measured: Sputum examined for culture change from Baseline at 12 months
Secondary outcomes (6)
Change from Baseline 6-minute Walk Test at 6 months
Measured: 6-minute Walk Test results examined for change from Baseline at 6 months
Change from Baseline 6-minute Walk Test at 12 months
Measured: 6-minute Walk Test results examined for change from Baseline at 12 months
Change from End of Treatment (EOT) sputum culture at 3 months post EOT
Measured: Sputum examined for culture change from EOT at 3 months post EOT
Number of Hospitalizations for pulmonary exacerbations
Measured: Number of Hospitalizations for pulmonary exacerbations that occur between Baseline and 12 months
Number of Adverse Events
Measured: Number of Patient-reported and Investigator-reported Adverse Events at 12 months
Number of subjects discontinuing study drug due to Adverse Event
Measured: Number of subjects who discontinue study drug before 12 months due to Adverse Event will be examined at 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2017 (estimated)
- Study startOct 2016
- Primary completion31 Oct 2019
- Study completion31 Dec 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Mycobacterium Infections, NontuberculousStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kevin Winthrop
- Sponsor type
- University, hospital or other
- Collaborators
- Insmed Incorporated, The University of Texas Health Science Center at Tyler
- Responsible party
- Sponsor-investigator, Kevin Winthrop, Oregon Health and Science University
- Investigators
- Kevin L Winthrop, MD, MPH, Principal investigator, Oregon Health and Science University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03038178
- Sponsor's protocol ID
- LAI/INS-IIR-01
- Status last verified
- Mar 2020
- First submitted
- 3 Jan 2017
- Last updated
- 4 Mar 2020
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Siegel SAR, Griffith DE, Philley JV, Brown-Elliott BA, Brunton AE, Sullivan PE, Fuss C, Strnad L, Wallace RJ Jr, Winthrop KL. Open-Label Trial of Amikacin Liposome Inhalation Suspension in Mycobacterium abscessus Lung Disease. Chest. 2023 Oct;164(4):846-859. doi: 10.1016/j.chest.2023.05.036. Epub 2023 Jun 17.
PubMed 37419144
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Study of Efficacy, Safety and Tolerability of Liposomal Amikacin for Inhalation (LAI) Once Daily in Addition to Standard Multi-antibiotic Therapy in the Treatment of Mycobacterium Abscessus Lung Disease
- ClinicalTrials.gov ID
- NCT03038178
- Acronym / sponsor ID
- LAI/INS-IIR-01
- Keywords
- Mycobacterium Infections, Nontuberculous, Mycobacteria, Atypical, Amikacin, Inhalation
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Kevin WinthropUniversity, hospital or other
- Collaborators
- Insmed Incorporated, The University of Texas Health Science Center at Tyler
- Study start
- Oct 2016 (actual)
- Primary completion
- 31 Oct 2019 (actual)
- Study completion
- 31 Dec 2019 (actual)
- First posted
- 31 Jan 2017 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The proposed study will assess the efficacy, safety and tolerability of once daily dosing of Liposomal-Amikacin for Inhalation (LAI) 590 mg for 12 months plus standard of care (SOC) mycobacterial multi-drug regimen in accordance with the 2007 ATS/ IDSA guidelines, for treatment of mycobacterium abscessus lung disease.
Detailed description
This is an open-label study of efficacy, safety and tolerability of once daily dosing of Liposomal-Amikacin for Inhalation (LAI), in addition to a standard multi-drug antibiotic therapy in accordance with the 2007 ATS/ IDSA guidelines, in patients with Mycobacterium abscessus lung disease. The multi-drug therapy is determined at the discretion of the Investigator. After screening, all eligible patients will enter the trial and will receive LAI 590 mg once daily for 12 months. If deemed necessary by the investigator, dose may be adjusted from a minimum of three times per week up to daily, in cases of adverse events relating to tolerability.
All patients who enter the study will have subsequent study visits at Months 1,2,4,6,9, at End-of-Study or Month 12, and at 1 and 3 months post study drug discontinuation. At each visit (including screening), review of concomitant medications, review of adverse events, and physical exam, will be performed. Chest CT scan will be performed at baseline, 6 months, and 12 months, unless a chest CT scan has already been performed within 6 months on this time point. The 6 minute walk test and QOL-B-NTM will be performed at baseline, 6 months, 12 months, and 3 months post study drug discontinuation. All patients will have inducted sputum collected at each study visit, and patients will self-collect expectorated sputum during intervening monthly time-points until study completion to determine changes in mycobacterial smear and culture status.
Unscheduled visits will occur as needed should subjects' symptoms worsen between visits.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LAI plus multi-drug regimen | Experimental | Drug: LAI plus multi-drug regimenonce daily dosing of Liposomal-Amikacin for Inhalation (LAI) 590 mg for 12 months plus standard of care (SOC) mycobacterial multi-drug regimen in accordance with the 2007 ATS/ IDSA guidelines |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Male or female patients 12 years and older
- Diagnosis of M. abscessus, including all subspecies, abscessus, bolleti, and massiliense lung disease according to the 2007 ATS/IDSA criteria
- Both newly diagnosed and currently on treatment or previously treated patients will be included
- Culture positive (either sputum or bronchoscopy) for M. abscessus at time of screening
- Willingness to adhere to a treatment regimen, study visits, and study procedures during the course of the study.
Key exclusion criteria (12 listed)
- Active pulmonary tuberculosis requiring treatment at screening
- Treatment with inhaled or intravenous Amikacin within 14 days prior to baseline
- Known hypersensitivity to aminoglycosides
- Aspartate aminotransferase or alanine aminotransferase ≥ 3 times the upper limit of normal or total bilirubin ≥ 2 times the upper limit of normal (ULN) at screening
- Current addiction to alcohol or illicit drug abuse
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LAI plus multi-drug regimenAlso: ARIKAYCE™Liposomal Amikacin for Inhalation (LAI) is the experimental treatment which, in this single arm will be taken in conjunction with standard of care multi-drug treatment regimen | Drug | LAI plus multi-drug regimen |
Primary outcome
Change from Baseline sputum culture at 12 months
Time frame: Sputum examined for culture change from Baseline at 12 months
6 secondary outcomes
Change from Baseline 6-minute Walk Test at 6 months
Time frame: 6-minute Walk Test results examined for change from Baseline at 6 months
Change from Baseline 6-minute Walk Test at 12 months
Time frame: 6-minute Walk Test results examined for change from Baseline at 12 months
Change from End of Treatment (EOT) sputum culture at 3 months post EOT
Time frame: Sputum examined for culture change from EOT at 3 months post EOT
Number of Hospitalizations for pulmonary exacerbations
Time frame: Number of Hospitalizations for pulmonary exacerbations that occur between Baseline and 12 months
Number of Adverse Events
Time frame: Number of Patient-reported and Investigator-reported Adverse Events at 12 months
Number of subjects discontinuing study drug due to Adverse Event
Time frame: Number of subjects who discontinue study drug before 12 months due to Adverse Event will be examined at 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2020.
- Derived from this study: PubMed 37419144Siegel SAR, Griffith DE, Philley JV, Brown-Elliott BA, Brunton AE, Sullivan PE, Fuss C, Strnad L, Wallace RJ Jr, Winthrop KL. Open-Label Trial of Amikacin Liposome Inhalation Suspension in Mycobacterium abscessus Lung Disease. Chest. 2023 Oct;164(4):846-859. doi: 10.1016/j.chest.2023.05.036. Epub 2023 Jun 17.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kevin L Winthrop, MD, MPH principal investigator
Oregon Health and Science University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LAI plus multi-drug regimen in people with Mycobacterium Infections, Nontuberculous and Mycobacteria, Atypical, run by Kevin Winthrop at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from Baseline sputum culture at 12 months (time frame: Sputum examined for culture change from Baseline at 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03038178, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Mar 2020.