A randomized, multinational, noninferiority, phase III trial to evaluate the safety and efficacy of BF-200 aminolaevulinic acid gel vs. methyl aminolaevulinate cream in the treatment of nonaggressive basal cell carcinoma with photodynamic therapy

C. A. Morton (Lead / Corresponding author), R. Dominicus, P. Radny, T. Dirschka, A. Hauschild, U. Reinhold, R. Aschoff, M. Ulrich, S. Keohane, S. Ekanayake-Bohlig, S. Ibbotson, R. Ostendorf, C. Berking, D. Gröne, H. J. Schulze, H. M. Ockenfels, V. Jasnoch, H. Kurzen, M. Sebastian, H. StegeP. Staubach-Renz, F. Hübinger, F. Hübinger, I. Ziabreva, B. Schmitz, A. Gertzmann, H. Lübbert, R. M. Szeimies

Research output: Contribution to journalArticle

16 Citations (Scopus)
91 Downloads (Pure)

Abstract

Background: Basal cell carcinoma (BCC) represents the most common non-melanoma skin cancer worldwide affecting mainly adult, fair-skinned individuals. The WHO distinguishes aggressive and non-aggressive forms of which prototypical variants of the latter are primary nodular and superficial BCC.

Objectives: To demonstrate non-inferiority of BF-200 ALA (a nanoemulsion gel containing 5-aminolaevulinic acid) compared to MAL (a cream containing methyl-aminolevulinate) in the treatment of non-aggressive BCC with photodynamic therapy (PDT). Non-inferiority of the primary efficacy variable (overall patient complete response 12 weeks after last PDT) would be declared if the mean response for BF-200 ALA was no worse than that for MAL, within a statistical margin of Δ = -15%.

Patients/Methods: The study was a randomized, phase III trial performed in Germany and the UK with ongoing 5-year follow-up. Of 281 randomized patients, 138 were treated with BF-200 ALA, 143 with MAL. Patients received two PDT sessions one week apart. Remaining lesions 12 weeks after the second PDT were retreated. Illumination was performed with a red light source (635 nm, 37 J/cm2). Results shown include clinical endpoints as well as patients' reassessment 12 months after the last PDT.

Results: Of the BF-200 ALA-treated patients, 93.4% were complete responders compared to 91.8% in the MAL group. The difference of means was 1.6 with a one-sided 97.5% CI of -6.5, establishing non-inferiority (p<0.0001). Results for secondary efficacy parameters were in line with the primary outcome. Recurrence rates 12 months after the last treatment were ≤ 10%.

Conclusions: Treatment of non-aggressive BCC with BF-200 ALA-PDT is highly effective and well tolerated with proven non-inferiority to MAL-PDT and demonstrates low recurrence rates after 1-year follow-up.

Original languageEnglish
Pages (from-to)309-319
Number of pages11
JournalBritish Journal of Dermatology
Volume179
Issue number2
Early online date12 Feb 2018
DOIs
Publication statusPublished - 24 Aug 2018

Keywords

  • Journal Article
  • Basal cell carcinoma
  • 5 -aminolaevulinic acid
  • methyl -aminolevulinate
  • photodynamic therapy
  • BF -RhodoLED

Fingerprint Dive into the research topics of 'A randomized, multinational, noninferiority, phase III trial to evaluate the safety and efficacy of BF-200 aminolaevulinic acid gel vs. methyl aminolaevulinate cream in the treatment of nonaggressive basal cell carcinoma with photodynamic therapy'. Together they form a unique fingerprint.

  • Cite this

    Morton, C. A., Dominicus, R., Radny, P., Dirschka, T., Hauschild, A., Reinhold, U., Aschoff, R., Ulrich, M., Keohane, S., Ekanayake-Bohlig, S., Ibbotson, S., Ostendorf, R., Berking, C., Gröne, D., Schulze, H. J., Ockenfels, H. M., Jasnoch, V., Kurzen, H., Sebastian, M., ... Szeimies, R. M. (2018). A randomized, multinational, noninferiority, phase III trial to evaluate the safety and efficacy of BF-200 aminolaevulinic acid gel vs. methyl aminolaevulinate cream in the treatment of nonaggressive basal cell carcinoma with photodynamic therapy. British Journal of Dermatology, 179(2), 309-319. https://doi.org/10.1111/bjd.16441