Mycobacterium Avium Intracellulare Complex (MAC)MACNCT00826423
Mycobacterium Avium Intracellulare Complex (MAC) Study
An observational study of Mycobacterium Avium Intracellulare Complex (MAC), run by Mayo Clinic at 1 site in United States.
Trial summary
This study is short course (3 month) multiple drug antibiotic therapy. The purpose of this research study is to evaluate the clinical and radiology response to assess whether drug resistance develops and assess quality of life measures with MAC disease. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Mycobacterium Avium Intracellulare Complex (MAC)
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2008to Sep 2011
- Phase
- Observational
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Fifteen adult patients will be included in this pilot study. This sample size was chosen not on the basis of formal statistical considerations. However, data from 15 patients will be sufficient to provide rough preliminary estimates of parameters that may be needed for developing power statements for a follow-up larger (randomized) trial. If macrolide resistance occurs in 5% or less of patients in this population, with ≥83% probability only one or zero of the 15 patients will develop resistance. If two or more of the 15 patients develop resistance then further investigations of resistance will be warranted before moving on to randomized trials.
Who may be able to join
- All patients must meet diagnostic criteria for nontuberculous mycobacterial pulmonary disease as per ATS/IDSA Official Statement (AJRCCM Vol 175 pp 367-416, 2007)
- Confirmation both by a chest radiologist and the investigators of multifocal bronchiectasis and multiple small nodules on HRCT scanning of the chest
- Repeatedly positive sputum cultures and/or bronchial washings for MAC (in accordance with microbiologic diagnostic criteria outline by the American Thoracic Society)
- MAC must be proven to be macrolide (either Clarithromycin or Azithromycin) sensitive at the onset of the study
- Age greater than 18
- No active treatment for MAC lung disease within the past two years
- Patients and their physicians must be willing to discontinue other non-MAC antimicrobials which may have been used as part of their pre-study bronchial hygiene program.
Who cannot join
- History of Cystic Fibrosis or HIV disease
- Known allergy or intolerance to any of the proposed antibiotics
- Inability to return at three month intervals for testing over the six month study period
- Inability to complete quality of life questionnaires
- Pregnancy
- Presence of any abnormality on baseline ophthalmological examination which would preclude the use of ethambutol.
- Coexistence of non-tuberculous mycobacteria other than MAC
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Pilot Study to Assess Safety and Efficacy of Short Course Multiple Drug Therapy for Adult Patients With Mycobacterium Avium Intracellulare Complex (MAC) Infection Associated With Mulit Focal Bronchiectasis and Multiple Small Nodules
This study is short course (3 month) multiple drug antibiotic therapy. The purpose of this research study is to evaluate the clinical and radiology response to assess whether drug resistance develops and assess quality of life measures with MAC disease.
Read the detailed description
The goal of this pilot study is to assess the safety and efficacy of short course (3 months) multiple drug antimicrobial therapy in adults with MAC pulmonary disease associated with multifocal bronchiectasis and multiple small nodules. We propose to evaluate the clinical and radiographic response, assess whether macrolide (either Clarithromycin or Azithromycin) resistance develops, and assess quality of life measures.
No evidence of efficacy is required to proceed to longer term studies; however, we will need to confirm lack of development of macrolide resistance in this pilot study before proceeding to any additional studies to evaluate the efficacy of short course MAC treatment.
Once we have demonstrated the feasibility of short course therapy and confirm that resistance to macrolides does not develop, we hope to apply for external funding to support a longer term randomized controlled trial comparing "standard" MAC therapy (which usually consists of a period of eighteen to twenty-four months with at least three antimicrobials) to short course (three months) MAC antimicrobial treatment, alternating each year with nine months of non-MAC bronchial hygiene measures for two consecutive years. If a larger study confirms efficacy of this approach, we would then propose even larger multi-site studies to test the hypothesis that short course MAC therapy alternating every year with non-MAC bronchial hygiene therapy should be considered in all adult patients with MAC pulmonary disease associated with multifocal bronchiectasis and multiple small nodules throughout their lives.
The longer term goal of this research is to develop an optimal treatment strategy for these patients (in whom MAC will likely persist indefinitely) that will result in not only a better quality of life, but less evidence of long term lung damage, less risk of drug-related morbidity, and be better tolerated by the patients compared to current treatment strategies.
The specific aims of this pilot study are as follows:
Primary Aim:
- Confirm that macrolide (either Clarithromycin or Azithromycin) resistance does not develop as a result of short course treatment. Secondary Aims:
- Assess for change in quality of life metrics (St. George Respiratory Questionnaire and SF 12) from baseline to the conclusion of the study period (six months). We expect to see improvement in quality of life measures as a result of this short course treatment trial.
- Assess for change in pulmonary function from baseline to the conclusion of the study period. We expect to see improvement particularly in the FEV1, FEV1/FVC, and diffusing capacity for carbon monoxide as a result of this short course treatment trial.
- Assess for change in high resolution computerized tomography (HRCT) of the chest, evidence of MAC pulmonary disease (multifocal bronchiectasis associated with multiple small nodules) from baseline to the conclusion of the study period. We do not anticipate being able to demonstrate improvement in the HRCT evidence of MAC lung disease during the short period of this trial since these changes usually occur quite slowly.
- Keywords
- treatment with Triple antibiotic therapy
- Medical subject headings
- Mycobacterium avium-intracellulare Infection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jan 2009 (estimated)
- Study startJun 2008
- Primary completionJun 2011
- Study completionSep 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mycobacterium Avium Intracellulare Complex (MAC)Studies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Investigators
- Jack P Leventhal, MD, Principal investigator, Mayo Clinic Assistant Professor of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00826423
- Sponsor's protocol ID
- 07-004414
- Acronym
- MAC
- Status last verified
- Jul 2012
- First submitted
- 20 Jan 2009
- Last updated
- 30 Jul 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jul 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study to Assess Safety and Efficacy of Short Course Multiple Drug Therapy for Adult Patients With Mycobacterium Avium Intracellulare Complex (MAC) Infection Associated With Mulit Focal Bronchiectasis and Multiple Small Nodules
- ClinicalTrials.gov ID
- NCT00826423
- Acronym / sponsor ID
- MAC / 07-004414
- Keywords
- treatment with Triple antibiotic therapy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- Jun 2008
- Primary completion
- Jun 2011 (actual)
- Study completion
- Sep 2011 (actual)
- First posted
- 22 Jan 2009 (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.
This study is short course (3 month) multiple drug antibiotic therapy. The purpose of this research study is to evaluate the clinical and radiology response to assess whether drug resistance develops and assess quality of life measures with MAC disease.
Detailed description
The goal of this pilot study is to assess the safety and efficacy of short course (3 months) multiple drug antimicrobial therapy in adults with MAC pulmonary disease associated with multifocal bronchiectasis and multiple small nodules. We propose to evaluate the clinical and radiographic response, assess whether macrolide (either Clarithromycin or Azithromycin) resistance develops, and assess quality of life measures.
No evidence of efficacy is required to proceed to longer term studies; however, we will need to confirm lack of development of macrolide resistance in this pilot study before proceeding to any additional studies to evaluate the efficacy of short course MAC treatment.
Once we have demonstrated the feasibility of short course therapy and confirm that resistance to macrolides does not develop, we hope to apply for external funding to support a longer term randomized controlled trial comparing "standard" MAC therapy (which usually consists of a period of eighteen to twenty-four months with at least three antimicrobials) to short course (three months) MAC antimicrobial treatment, alternating each year with nine months of non-MAC bronchial hygiene measures for two consecutive years. If a larger study confirms efficacy of this approach, we would then propose even larger multi-site studies to test the hypothesis that short course MAC therapy alternating every year with non-MAC bronchial hygiene therapy should be considered in all adult patients with MAC pulmonary disease associated with multifocal bronchiectasis and multiple small nodules throughout their lives.
The longer term goal of this research is to develop an optimal treatment strategy for these patients (in whom MAC will likely persist indefinitely) that will result in not only a better quality of life, but less evidence of long term lung damage, less risk of drug-related morbidity, and be better tolerated by the patients compared to current treatment strategies.
The specific aims of this pilot study are as follows:
Primary Aim:
- Confirm that macrolide (either Clarithromycin or Azithromycin) resistance does not develop as a result of short course treatment. Secondary Aims:
- Assess for change in quality of life metrics (St. George Respiratory Questionnaire and SF 12) from baseline to the conclusion of the study period (six months). We expect to see improvement in quality of life measures as a result of this short course treatment trial.
- Assess for change in pulmonary function from baseline to the conclusion of the study period. We expect to see improvement particularly in the FEV1, FEV1/FVC, and diffusing capacity for carbon monoxide as a result of this short course treatment trial.
- Assess for change in high resolution computerized tomography (HRCT) of the chest, evidence of MAC pulmonary disease (multifocal bronchiectasis associated with multiple small nodules) from baseline to the conclusion of the study period. We do not anticipate being able to demonstrate improvement in the HRCT evidence of MAC lung disease during the short period of this trial since these changes usually occur quite slowly.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Fifteen adult patients will be included in this pilot study. This sample size was chosen not on the basis of formal statistical considerations. However, data from 15 patients will be sufficient to provide rough preliminary estimates of parameters that may be needed for developing power statements for a follow-up larger (randomized) trial. If macrolide resistance occurs in 5% or less of patients in this population, with ≥83% probability only one or zero of the 15 patients will develop resistance. If two or more of the 15 patients develop resistance then further investigations of resistance will be warranted before moving on to randomized trials.
Key inclusion criteria (7 listed)
- All patients must meet diagnostic criteria for nontuberculous mycobacterial pulmonary disease as per ATS/IDSA Official Statement (AJRCCM Vol 175 pp 367-416, 2007)
- Confirmation both by a chest radiologist and the investigators of multifocal bronchiectasis and multiple small nodules on HRCT scanning of the chest
- Repeatedly positive sputum cultures and/or bronchial washings for MAC (in accordance with microbiologic diagnostic criteria outline by the American Thoracic Society)
- MAC must be proven to be macrolide (either Clarithromycin or Azithromycin) sensitive at the onset of the study
- Age greater than 18
Key exclusion criteria (7 listed)
- History of Cystic Fibrosis or HIV disease
- Known allergy or intolerance to any of the proposed antibiotics
- Inability to return at three month intervals for testing over the six month study period
- Inability to complete quality of life questionnaires
- Pregnancy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Not reported in the source record.
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 30 Jul 2012.
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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.
Jack P Leventhal, MD principal investigator
Mayo Clinic Assistant Professor of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Mycobacterium Avium Intracellulare Complex (MAC), run by Mayo Clinic at 1 site 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 7 inclusion and 7 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 30 Jul 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
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.
Where can I read the original study information?
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 30 Jul 2012.