# P2 Study of Clofazimine for Pulmonary M. avium Disease IND 125,266 (1/5/2015)

> **NIH FDA R01** · OREGON HEALTH & SCIENCE UNIVERSITY · 2020 · $492,780

## Abstract

PROJECT 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, we have developed a randomized, placebo-controlled clinical trial to
assess the clinical efficacy and safety of clofazimine. To be eligible, patients must have MAC lung disease,
positive sputum cultures for MAC, and not currently taking antibiotics for MAC. Eligible patients (102 total
enrolled) will be randomly given either clofazimine or placebo for 6 months, and followed closely by their
treating physician. The percentage of patients who become culture negative in each group will be compared,
as we suspect patients treated with clofazimine will be more likely to become culture negative. We will also
measure the safety of clofazimine as well as other potential benefits of the therapy including changes in lung
function and quality of life.

## Key facts

- **NIH application ID:** 9832617
- **Project number:** 5R01FD005401-03
- **Recipient organization:** OREGON HEALTH & SCIENCE UNIVERSITY
- **Principal Investigator:** Kevin Loring Winthrop
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2020
- **Award amount:** $492,780
- **Award type:** 5
- **Project period:** 2016-09-01 → 2023-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9832617

## Citation

> US National Institutes of Health, RePORTER application 9832617, P2 Study of Clofazimine for Pulmonary M. avium Disease IND 125,266 (1/5/2015) (5R01FD005401-03). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9832617. Licensed CC0.

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