NCT06300502

Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)

Assessing the Efficacy of Repeat, Monthly Treatments of Cutaneous Neurofibromas (cNFs)

• Status: Enrolling By Invitation
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Massachusetts General Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Neurofibromas, Cutaneous
• Neurofibromatosis 1

Interventions

Type	Name	Description
DRUG	Kybella	Kybella is sterile 1% deoxycholic acid provided in a 2 mL single-use vial.

Timeline

• Start date: 2025-04-08
• Primary completion: 2026-02-01
• Completion: 2026-02-01
• First posted: 2024-03-08
• Last updated: 2025-11-20

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study