Massachusetts General Hospital
The goal of this clinical trial is to evaluate the tolerability and effectiveness of multiple treatments of an FDA-approved drug in those with Neurofibromatosis Type 1 (NF1) Cutaneous Neurofibromas (cNFs). The main questions it aims to answer are: Will performing: * Up to 6 months treatment sessions * A minimum of 30 days apart * With up to 50 injections of deoxycholate into a maximum of 50 cNFs in a single region of the body (for a maximum total dose of 10 mL per monthly treatment session) result in tolerable local skin reactions and reduction in both individual cNF size by \>50% as well as improved cNF appearance in the treated field? Researchers will compare treated tumors and control tumors to see if the treatment is effective. Participants will: * Receive up to 6 monthly treatments with Kybella (deoxycholate). Treatment for a given tumor will be stopped when the tumor is assessed as clear clinically. * Complete surveys asking about pain during and after treatments. * Complete surveys asking about satisfaction with the treatments. * Undergo 2D photography and 3D imaging of treatment areas. * Optionally, receive biopsies of up to 6 treated lesions to investigate characteristics of tumors that respond well to treatment as well as non-respondent tumors.
Inclusion Criteria: 1. Participant is an adult ≥18 years of age. 2. Participant has a diagnosis of NF1 based on germline genetic testing or by meeting ≥ 2 the following criteria: * Family history of NF1, * Six or more light brown ("cafe-au-lait") spots on the skin, * Presence of two or more neurofibromas of any type, or one or more plexiform neurofibromas, * Freckling under the arms or in the groin area, * Two or more pigmented, benign bumps on the eye's iris (Lisch nodules), * A distinctive bony lesion: dysplasia (abnormal growth) of the sphenoid bone behind the eye, or dy…
Kybella is sterile 1% deoxycholic acid provided in a 2 mL single-use vial.