Mycobacterium Avium ComplexPhase 2NCT02968212
Clofazimine in the Treatment of Pulmonary Mycobacterium Avium Complex (MAC)
A Phase 2 study testing Clofazimine and sugar pill in people with Mycobacterium Avium Complex, run by Oregon Health and Science University at 6 sites in United States.
Trial summary
The purpose of this study is to evaluate the clinical effectiveness and safety of clofazimine when used to treat Mycobacteria avium complex (MAC) lung disease. Funding Source - FDA OOPD Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Mycobacterium Avium Complex
- What is being tested?
- Clofazimine and sugar pill
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 102 participants
- Where
- 6 sitesUnited States
- Study dates
- 11 Apr 2017to Oct 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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
Who may be able to join
- At least 2 positive MAC sputum cultures in the last 12 months with at least one obtained within 12 weeks prior to randomization
- Meet ATS/IDSA 2007 pulmonary disease criteria
- Adult males and females age 18 or over
- Ability to provide informed consent for the use of study drug
Who cannot join
- Any patient who is unwilling or unable to provide consent or to comply with this protocol
- Cavitary NTM disease
- Patients who are currently taking or within the prior 12 weeks received any of the following: bedaquiline, or any component of ATS/IDSA multi-drug recommended therapy (macrolide, ethambutol, rifampin) for MAC
- Current usage of inhaled amikacin, tobramycin, or gentamicin
- In the judgment of the investigator, the patient is not a candidate for observation (e.g. severe symptoms, extensive disease burden) but rather should be treated with standard multi-drug therapy
- Prior use of clofazimine that has resulted in an allergy to clofazimine or a severe adverse reaction
Show all 15Show fewer
- Current usage of medications associated with QT prolongation (see Appendix C for full list of prohibited concomitant medications)
- Corrected QT (QTc) interval on electrocardiogram (ECG) > 470 ms for females or 450 ms for males, calculated using Fridericia's formula60,61
- Advanced lung disease (FEV<30%)
- HIV
- Active pulmonary tuberculosis requiring treatment at screening
- Active pulmonary malignancy or chemotherapy or radiation within 1 year of screening
- Use of chronic systemic corticosteroids at doses of 15 mg/day for more than 12 weeks
- Prior lung or other solid organ transplant
- Pregnancy, or breastfeeding that will continue during treatment
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Louisiana State University
Baton Rouge, Louisiana, 70803
University of Chicago
Chicago, Illinois, 60637
National Jewish Health
Denver, Colorado, 80206
Oregon Health & Science University
Portland, Oregon, 97239
University of South Florida
Tampa, Florida, 33620
University of Texas Health Science Center
Tyler, Texas, 75708
About this study
Official title: Phase 2 Study of Clofazimine for the Treatment of Pulmonary Mycobacterium Avium Disease
The purpose of this study is to evaluate the clinical effectiveness and safety of clofazimine when used to treat Mycobacteria avium complex (MAC) lung disease.
Funding Source - FDA OOPD
Read the detailed description
Clofazimine is an orphan antibiotic drug that is no longer available through pharmacies in the United States. It is approved for the treatment of Mycobacterium leprae (leprosy) infections. Clofazimine has been used for many years off-label against other Mycobacterium, including Mycobacteria avium complex (MAC) lung disease, an increasingly prevalent infection in older Americans. The U.S. Food and Drug Administration currently oversees clofazimine use to treat MAC lung disease through a special investigational drug access program. However, to date, there is little understanding of the benefits and risks of clofazimine when used to treat MAC lung disease. Accordingly, the investigators have developed a randomized, placebo-controlled clinical trial to assess the clinical efficacy and safety of clofazimine. To be eligible, participants must have MAC lung disease, positive sputum cultures for MAC, and not currently taking antibiotics for MAC. Eligible participants (102 total enrolled) will be randomly given either clofazimine or placebo for 6 months, and followed closely by their treating physician. The percentage of participants who become culture negative in each group will be compared, as it is suspected that participants treated with clofazimine will be more likely to become culture negative. The safety of clofazimine will be measured as well as other potential benefits of the therapy including changes in lung function and quality of life.
- Conditions
- Mycobacterium Avium Complex
- Being tested
- Clofazimine, sugar pill
- Keywords
- Mycobacterium avium Complex, Clofazimine
- Medical subject headings
- Mycobacterium avium-intracellulare Infection
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ClofazimineDrug
Also called Lamprene
All participants in the experimental/treatment arm on this protocol will take a loading dose of 200 mg daily in soft capsule form of clofazimine for 16 weeks, dropping to 100 mg daily for the next 8 weeks.
sugar pillOther
Also called placebo
All participants in the placebo arm on this protocol will take placebo in soft capsule form daily dropping to a smaller dose after 16 weeks to mirror the treatment arm dosing.
| Study group | Role | What they receive |
|---|---|---|
| clofazimine | Experimental | Participants receive lamprene Receives: Drug: Clofazimine |
| sugar pill | Placebo comparator | Participants receive placebo Receives: Other: sugar pill |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline sputum culture at 24 weeks
sputum will be processed for acid fast bacilli stain/acid fast bacterial culture. A semi-quantitative assessment will be made by colony count for patients.
Measured: Sputum examined for culture change from Baseline at 24 weeks
Secondary outcomes (13)
Change from Baseline 6 Minute Walk Test at 24 weeks
Walking distance achieved in 6 minutes is assessed
Measured: 6 Minute Walk Test results examined for change from Baseline at 24 weeks
Change from Baseline PROMIS Fatigue 7a short form questionnaire at 24 weeks
Self-administered questionnaire assessing a range of self-reported symptoms over the past seven days, from mild subjective feelings of tiredness to an overwhelming, debilitating, and sustained sense of exhaustion that likely decreases one's ability to execute daily activities and function normally in family or social roles. Fatigue is divided into the experience of fatigue (frequency, duration, and intensity) and the impact of fatigue on physical, mental, and social activities.
Measured: PROMIS Fatigue 7a short form results examined for change from Baseline at 24 weeks
Change from Baseline Quality of Life-Bronchiectasis (QOL-B) with NTM module at 24 weeks
Self-administered questionnaire measuring 8 separate domains: Physical Functioning, Role Functioning, Vitality, Emotional Functioning, Social Functioning, Treatment Burden, Health Perceptions, and Respiratory Symptoms.
Measured: QOL-B results examined for change from Baseline at 24 weeks
Change from Baseline CT scan at 24 weeks
CT scans will be computationally evaluated using custom software to provide volumetric assessment of NTM-associated abnormalities.
Measured: CT scan examined for change from Baseline at 24 weeks
Change from Baseline semi-quantitative sputum acid fast smear culture at 24 weeks
sputum will be processed for acid fast bacilli stain/acid fast bacterial culture. A semi-quantitative assessment will be made by colony count for patients.
Measured: semi-quantitative sputum acid fast smear culture examined for change from Baseline at 24 weeks
Change from Baseline Spirometry at 24 weeks
Mean change in pulmonary function parameters as measured by %predicted FEV1 and FVC
Measured: Spirometry with FEV1/FVC ratio examined for change from Baseline at 24 weeks
Change from Baseline Erythrocyte Sedimentation Rate at 24 weeks
Detecting change in Inflammatory markers
Measured: Erythrocyte Sedimentation Rate examined for change from Baseline at 24 weeks
Change from Baseline C-Reactive Protein levels at 24 weeks
Detecting change in Inflammatory markers
Measured: C-Reactive Protein levels examined for change from Baseline at 24 weeks
Number of Adverse Events
Comparison of experienced adverse events between the two study groups
Measured: Number of Patient-reported and Investigator-reported Adverse Events at 24 weeks
Change from Baseline QT interval at 24 weeks
A 12-lead ECG will be conducted, and the QT interval calculated using Fridericia's formula: QTC = QT / RR 1/3
Measured: QT interval examined for change from Baseline at 24 weeks
Change from Baseline blood serum chemistry at week 24
Detecting changes in liver ALT and AST levels
Measured: blood serum chemistry examined for change from Baseline at 24 weeks
Change from Baseline complete blood count at week 24
Detecting changes in complete blood count
Measured: complete blood count examined for change from Baseline at 24 weeks
Change from Baseline Minimal Inhibitory Concentration of MAC isolates in vitro at week 24
Detecting change in MAC isolates sensitivity to clofazimine
Measured: Minimal Inhibitory Concentration of MAC isolates in vitro examined for change from Baseline at 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2016 (estimated)
- Study start11 Apr 2017
- Primary completion29 Aug 2025
- Study completionOct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mycobacterium Avium ComplexStudies of the same condition, open and under way.
- More trials run by Oregon Health and Science UniversityThe 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 102 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oregon Health and Science University
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), National Jewish Health, The University of Texas Health Science Center at Tyler, National Institute of Allergy and Infectious Diseases (NIAID), University of Chicago, Temple University, University of South Florida
- Responsible party
- Principal investigator, Kevin Winthrop, Oregon Health and Science University
- Investigators
- Kevin Winthrop, MD, Principal investigator, Oregon Health and Science University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02968212
- Sponsor's protocol ID
- FD-R-5401
- Status last verified
- May 2026
- First submitted
- 10 Nov 2016
- Last updated
- 6 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Study of Clofazimine for the Treatment of Pulmonary Mycobacterium Avium Disease
- ClinicalTrials.gov ID
- NCT02968212
- Acronym / sponsor ID
- FD-R-5401
- Conditions
- Mycobacterium Avium Complex
- Keywords
- Mycobacterium avium Complex, Clofazimine
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 102 participants (actual)
- Lead sponsor
- Oregon Health and Science UniversityUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), National Jewish Health, The University of Texas Health Science Center at Tyler, National Institute of Allergy and Infectious Diseases (NIAID), University of Chicago, Temple University, University of South Florida
- Study start
- 11 Apr 2017 (actual)
- Primary completion
- 29 Aug 2025 (actual)
- Study completion
- Oct 2026 (estimated)
- First posted
- 18 Nov 2016 (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 purpose of this study is to evaluate the clinical effectiveness and safety of clofazimine when used to treat Mycobacteria avium complex (MAC) lung disease.
Funding Source - FDA OOPD
Detailed description
Clofazimine is an orphan antibiotic drug that is no longer available through pharmacies in the United States. It is approved for the treatment of Mycobacterium leprae (leprosy) infections. Clofazimine has been used for many years off-label against other Mycobacterium, including Mycobacteria avium complex (MAC) lung disease, an increasingly prevalent infection in older Americans. The U.S. Food and Drug Administration currently oversees clofazimine use to treat MAC lung disease through a special investigational drug access program. However, to date, there is little understanding of the benefits and risks of clofazimine when used to treat MAC lung disease. Accordingly, the investigators have developed a randomized, placebo-controlled clinical trial to assess the clinical efficacy and safety of clofazimine. To be eligible, participants must have MAC lung disease, positive sputum cultures for MAC, and not currently taking antibiotics for MAC. Eligible participants (102 total enrolled) will be randomly given either clofazimine or placebo for 6 months, and followed closely by their treating physician. The percentage of participants who become culture negative in each group will be compared, as it is suspected that participants treated with clofazimine will be more likely to become culture negative. The safety of clofazimine will be measured as well as other potential benefits of the therapy including changes in lung function and quality of life.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| clofazimine | Experimental | Drug: ClofazimineParticipants receive lamprene |
| sugar pill | Placebo comparator | Other: sugar pillParticipants receive placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- At least 2 positive MAC sputum cultures in the last 12 months with at least one obtained within 12 weeks prior to randomization
- Meet ATS/IDSA 2007 pulmonary disease criteria
- Adult males and females age 18 or over
- Ability to provide informed consent for the use of study drug
Key exclusion criteria (15 listed)
- Any patient who is unwilling or unable to provide consent or to comply with this protocol
- Cavitary NTM disease
- Patients who are currently taking or within the prior 12 weeks received any of the following: bedaquiline, or any component of ATS/IDSA multi-drug recommended therapy (macrolide, ethambutol, rifampin) for MAC
- Current usage of inhaled amikacin, tobramycin, or gentamicin
- In the judgment of the investigator, the patient is not a candidate for observation (e.g. severe symptoms, extensive disease burden) but rather should be treated with standard multi-drug therapy
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 |
|---|---|---|
| ClofazimineAlso: LampreneAll participants in the experimental/treatment arm on this protocol will take a loading dose of 200 mg daily in soft capsule form of clofazimine for 16 weeks, dropping to 100 mg daily for the next 8 weeks. | Drug | clofazimine |
| sugar pillAlso: placeboAll participants in the placebo arm on this protocol will take placebo in soft capsule form daily dropping to a smaller dose after 16 weeks to mirror the treatment arm dosing. | Other | sugar pill |
Primary outcome
Change from Baseline sputum culture at 24 weeks
Time frame: Sputum examined for culture change from Baseline at 24 weeks
sputum will be processed for acid fast bacilli stain/acid fast bacterial culture. A semi-quantitative assessment will be made by colony count for patients.
13 secondary outcomes
Change from Baseline 6 Minute Walk Test at 24 weeks
Time frame: 6 Minute Walk Test results examined for change from Baseline at 24 weeks
Change from Baseline PROMIS Fatigue 7a short form questionnaire at 24 weeks
Time frame: PROMIS Fatigue 7a short form results examined for change from Baseline at 24 weeks
Change from Baseline Quality of Life-Bronchiectasis (QOL-B) with NTM module at 24 weeks
Time frame: QOL-B results examined for change from Baseline at 24 weeks
Change from Baseline CT scan at 24 weeks
Time frame: CT scan examined for change from Baseline at 24 weeks
Change from Baseline semi-quantitative sputum acid fast smear culture at 24 weeks
Time frame: semi-quantitative sputum acid fast smear culture examined for change from Baseline at 24 weeks
Change from Baseline Spirometry at 24 weeks
Time frame: Spirometry with FEV1/FVC ratio examined for change from Baseline at 24 weeks
Change from Baseline Erythrocyte Sedimentation Rate at 24 weeks
Time frame: Erythrocyte Sedimentation Rate examined for change from Baseline at 24 weeks
Change from Baseline C-Reactive Protein levels at 24 weeks
Time frame: C-Reactive Protein levels examined for change from Baseline at 24 weeks
Number of Adverse Events
Time frame: Number of Patient-reported and Investigator-reported Adverse Events at 24 weeks
Change from Baseline QT interval at 24 weeks
Time frame: QT interval examined for change from Baseline at 24 weeks
Change from Baseline blood serum chemistry at week 24
Time frame: blood serum chemistry examined for change from Baseline at 24 weeks
Change from Baseline complete blood count at week 24
Time frame: complete blood count examined for change from Baseline at 24 weeks
Change from Baseline Minimal Inhibitory Concentration of MAC isolates in vitro at week 24
Time frame: Minimal Inhibitory Concentration of MAC isolates in vitro examined for change from Baseline at 24 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2026.
- 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 Winthrop, MD principal investigator
Oregon Health and Science University
6 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 Clofazimine and sugar pill in people with Mycobacterium Avium Complex, run by Oregon Health and Science University at 6 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 4 inclusion and 15 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: under way, not looking for participants, as of the sponsor's last update on 6 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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. Some participants in this study may receive a placebo. 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?
This study is still under way. Results are not available in the source record yet.
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 24 weeks (time frame: Sputum examined for culture change from Baseline at 24 weeks). It is what the study was designed to measure, not a finding.
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 6 May 2026.