Trial Information

Photodynamic Therapy for Benign Dermal Neurofibromas Using Levulan Kerastick For Topical Solution, Plus Illumination With Red Light

STUDY DESIGN This protocol is a Phase I light dose escalation pilot study to determine the
safety and, secondarily, the efficacy of PDT using Levulan and 630 nm light in the treatment
of benign dermal neurofibromas. This protocol represents the first two parts of a planned
three part study including both pediatric and adult subjects. Part 1 will consist of
studying the penetration and uptake of the PS in neurofibromas that are scheduled for
excision. These tumors will be excised for therapeutic reasons unrelated to this study, and
so this study will place no further burden on the subject other than a 3-24 hr incubation of
the Levulan on the tumor prior to excision. The primary hypothesis to be tested is whether
Levulan will accumulate, and be converted to PpIX, by the tumor tissue more than by the
surrounding normal tissue. Secondary hypotheses are that tumors incubated with Levulan will
show greater fluorescence than untreated tumors and tumors incubated with vehicle only
(placebo application).

As the Institutional Review Boards involved generally desire pilot data on adult populations
first, we will with then proceed with the adult clinical trial portion of this protocol as
part 2. Part 2 will use the optimum incubation time, if one has been identified in part 1,
and add a dose escalation study of the amount of red light used to activate the Levulan.
Part 3, with pediatric subjects, will commence at a future date, pending review of the
initial adult study results.