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

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
Massachusetts General Hospital
Study ID
NCT06300502
Phase
PHASE1
Status
Enrolling By Invitation

Conditions

  • Neurofibromas, Cutaneous
  • Neurofibromatosis 1

Eligibility Criteria

Sex
ALL
Age
18 Years - 85 Years
Healthy Volunteers
Not accepted

Interventions

  • Kybella — DRUG
    Kybella is sterile 1% deoxycholic acid provided in a 2 mL single-use vial.

Study Details

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.

Key Dates

Start date
Apr 8, 2025
Status verified
Apr 2026
Primary completion
Feb 28, 2027
Completion
Feb 28, 2027

Study Design

Enrollment
15 participants (estimated)
Allocation
RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
OTHER

Arms

  • Active Comparator: Treated cutaneous neurofibromas (cNFs)
    Injection into up to 50 cutaneous Neurofibromas (for at total maximum of 10 mL injected) with Kybella (1% deoxycholic acid).
  • No Intervention: Control cutaneous neurofibromas (cNFs)
    A complementary region of cNFs of similar characteristics to the treatment area in the same body region will be selected to serve as an untreated control group.

Primary Outcome Measure

Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: 3 months after treatment ]

Locations (1)

FacilityCityStateZIPSite coordinators
Wellman Center for PhotomedicineBostonMassachusetts02114-

Find similar trials in Boston, MA

By research site
Wellman Center for Photomedicine· Boston, MA

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