1580. Clofazimine Use in Patients with Mycobacterial Infections under Single Patient Investigational New Drug (SPIND)
Session: Poster Abstract Session: Mycobacterial Infections
Saturday, October 20, 2012
Room: SDCC Poster Hall F-H

Background: The U.S. Food and Drug Administration (FDA) may permit an investigational drug to be used for the treatment of a patient by a physician under SPIND. Clofazimine (Lamprene) is approved for the treatment of lepromatous leprosy and available under SPIND for treatment of other mycobacterial infections in patients failing therapy. A retrospective study of SPIND reports in patients with mycobacterial infections was performed to evaluate the adverse events (AE) reported for various clofazimine-containing regimens.


Methods: Annual SPIND reports (2005-2011) for 209 patients with positive sputum or skin mycobacterial cultures at diagnosis were reviewed. De-identified information on demographics and AE based on investigator reports were compiled in an EXCEL database. Qualitative and quantitative analyses of AE were performed and presented as descriptive summary statistics.


Results: The study population included 144 females, 56 males, and 9 unreported genders, HIV-negative, with a median age of 60 years. Patients were mostly infected with Mycobacterium avium complex (79%), multi-drug resistant tuberculosis (11%), and Mycobacterium abscessus (4%). The duration of clofazimine (50mg or 100mg once or twice daily) treatment was 12 to 24 months. Of 209 patients, 54 (26%) completed treatment and 77 (37%) were ongoing, and 35 (17%) discontinued due to AE or ineffective treatment. There were 43 (20%) deaths which were reported to be unrelated to clofazimine. Follow-up sputum cultures were available for 53 (25%) patients and conversion to negative culture occurred in 34/53 (64%). Adverse events reported in 64/201 (32%) patients, included skin hyperpigmentation (36, 56%), gastrointestinal reactions (23, 36%), and other (5, 8%).

Conclusion: Adverse events associated with clofazimine were reported in one-third of patients. Conclusions cannot be drawn about the safety or efficacy of the drug for treatment of mycobacterial infections in this SPIND population. A randomized controlled trial with reliable clinical endpoints is warranted to evaluate the risk and benefit of clofazimine in the treatment of mycobacterial infections.

Pujita Vaidya, M.P.H., Elizabeth O'Shaughnessy, M.D., Ramou Mauer, B.A., Alison Rodgers, B.S., Maureen Dillon-Parker, B.S., Eileen Navarro Almario, M.D. and John Farley, M.D., M.P.H., U.S. Food and Drug Administration, Silver Spring, MD


P. Vaidya, None

E. O'Shaughnessy, None

R. Mauer, None

A. Rodgers, None

M. Dillon-Parker, None

E. Navarro Almario, None

J. Farley, None

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