DPCP to Treat Cutaneous Neurofibromas Associated With NF1
Status:
Not yet recruiting
Trial end date:
2023-12-01
Target enrollment:
Participant gender:
Summary
Neurofibromatosis type 1 (NF1) is the most common genetic tumor predisposition syndrome,
affecting up to 1 in 2500 individuals. Cutaneous neurofibromas are benign with self-limited
growth; however, tumor burden may be excessive, tumors do not regress, and they can be
disfiguring, painful, and itchy. Currently, the only treatment is surgery or laser ablation;
however, outcomes are limited by the number of tumors that can be simultaneously removed,
operating room availability, and painful recovery, with significant risk of regrowth. There
is a strong need for noninvasive topical treatments for cutaneous neurofibromas.
Diphencyprone (DPCP) is a "hapten" medication, a small molecule that activates the immune
system when applied topically, which has been investigated as a cutaneous immunotherapy for
other skin conditions.
This is an open label Phase I study looking at safety and tolerability of this treatment as a
primary endpoint, and tumor treatment as a secondary endpoint. Approximately 30 subjects will
be enrolled at a single center within the US. Subjects with a clinical diagnosis of NF1 who
have measurable disease and at least 4 cutaneous neurofibromas, will have DPCP applied
topically to their neurofibromas once weekly for 10 weeks.