Purpose

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

Condition

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • 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

Exclusion Criteria

  • 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 - 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

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Triple (Participant, Care Provider, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
clofazimine
Participants receive lamprene
  • Drug: Clofazimine
    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.
    Other names:
    • Lamprene
Placebo Comparator
sugar pill
Participants receive placebo
  • Other: sugar pill
    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.
    Other names:
    • placebo

Recruiting Locations

More Details

Status
Recruiting
Sponsor
Oregon Health and Science University

Study Contact

Amanda Brunton, MPH
503-494-6327
brunton@ohsu.edu

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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.