Sign in
Enrolling by invitationPhase 1

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

Key facts

Who runs it
Massachusetts General Hospital
Where
Boston, United StatesSee the site
Trial code
Status
Enrolling by invitation
Phase
Phase 1
People taking part
15 people, planned
Started
8 Apr 2025
Expected to end
Feb 2027
Type of study
Interventional, 2 groupsOne group gets an existing treatment, for comparison.
Treatments tested
  • Kybella
Record updated
22 April 2026

Who can take part

Age
18 years to 85 years
Sex
Women and men
Healthy volunteers
Cannot take part

The registry criteria, word for word

Show all 13 criteriaShow less

Who can take part

  • Participant is an adult ≥18 years of age
  • 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 dysplasia of long bones, often in the lower leg,; Tumor on the optic nerve that may interfere with vision
  • Participant is seeking treatment for cNF
  • Participant has ≥ 6 paired cNF that are visible and measure 2 mm or more in size. The target treatment area must be amenable to both deoxycholate injections and surveillance with digital and 3D photography. Preferred locations are trunk (back or chest), arms and legs
  • Participant is able and willing to comply with all visit, treatment and evaluation schedules and requirements
  • Participant is able to understand and provide written informed consent
  • Participant has no known allergy to deoxycholate
  • Participant has no concurrent injury or wound in the target area

Who cannot take part

  • Participant has a known allergy to deoxycholic acid
  • Participant has any condition which, in the Investigator's opinion, would make it unsafe (for the participant or study personnel) to treat the participant as part of this research study
  • Participant cannot give informed consent or adhere to study schedule
  • Participant is actively tanning during the course of the study
  • For female participants: those who are pregnant

Check if you might fit

Answer for yourself. This is not a decision: only the study team can say whether you can take part.

  • Are you between 18 and 85 years old?

Where in United States

  • Wellman Center for PhotomedicineBoston, United StatesEnrolling by invitation

About the trial

What the trial wants to find out

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:

Read full original descriptionShow less

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.

The 2 groups in the trial

  • Treated cutaneous neurofibromas (cNFs)Existing treatment, for comparison

    Injection into up to 50 cutaneous Neurofibromas (for at total maximum of 10 mL injected) with Kybella (1% deoxycholic acid).

    Treatment: Kybella

  • Control cutaneous neurofibromas (cNFs)No study treatment, for comparison

    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.

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
Assessing the Efficacy of Repeat, Monthly Treatments of Cutaneous Neurofibromas (cNFs)
Fit questions
Built from NCT06300502, who can take part
Record updated
22 April 2026