Clofazimine in the Treatment of Pulmonary Mycobacterium Avium Complex (MAC)

NCT02968212 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: FD-R-5401
• Study Type: interventional
• Target: 102
• Locations: 1 country
• Sites: 6

Brief 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

Conditions

Mycobacterium Avium Complex

Keywords

Mycobacterium avium Complex; Clofazimine

Outcome Measures

Primary Outcomes (1)

• 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.

  Sputum examined for culture change from Baseline at 24 weeks

Secondary Outcomes (13)

• Change from Baseline 6 Minute Walk Test at 24 weeks

  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

  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

  QOL-B results examined for change from Baseline at 24 weeks

• Change from Baseline CT scan at 24 weeks

  CT scan examined for change from Baseline at 24 weeks

• Change from Baseline semi-quantitative sputum acid fast smear culture at 24 weeks

  semi-quantitative sputum acid fast smear culture examined for change from Baseline at 24 weeks

Study Arms (2)

clofazimine

EXPERIMENTAL

Participants receive lamprene

Drug: Clofazimine

sugar pill

PLACEBO COMPARATOR

Participants receive placebo

Other: sugar pill

Interventions

Clofazimine DRUG

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.

Also known as: Lamprene

clofazimine

sugar pill OTHER

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.

Also known as: placebo

sugar pill

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Oregon Health and Science University: lead
• National Heart, Lung, and Blood Institute (NHLBI): collaborator
• National Jewish Health: collaborator
• The University of Texas Health Science Center at Tyler: collaborator
• National Institute of Allergy and Infectious Diseases (NIAID): collaborator
• University of Chicago: collaborator
• Temple University: collaborator
• University of South Florida: collaborator

Study Sites (6)

National Jewish Health

Denver, Colorado, 80206, United States

Location

University of South Florida

Tampa, Florida, 33620, United States

Location

University of Chicago

Chicago, Illinois, 60637, United States

Location

Louisiana State University

Baton Rouge, Louisiana, 70803, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

University of Texas Health Science Center

Tyler, Texas, 75708, United States

Location

MeSH Terms

Conditions

Mycobacterium avium-intracellulare Infection

Interventions

Clofazimine; Sugars

Condition Hierarchy (Ancestors)

Mycobacterium Infections, Nontuberculous; Mycobacterium Infections; Actinomycetales Infections; Gram-Positive Bacterial Infections; Bacterial Infections; Bacterial Infections and Mycoses; Infections

Intervention Hierarchy (Ancestors)

Phenazines; Heterocyclic Compounds, 3-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Carbohydrates

Study Officials

• Kevin Winthrop, MD

  Oregon Health and Science University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: November 10, 2016
• First Posted: November 18, 2016
• Study Start: April 11, 2017
• Primary Completion: August 29, 2025
• Study Completion (Estimated): October 1, 2026
• Last Updated: May 6, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)