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

Mass General Brigham

Status and phase: Not yet enrolling; Phase 1

Conditions: Neurofibromatosis 1; Neurofibromas, Cutaneous

Treatments: Device: 1064nm Nd:YAG Laser; Drug: Kybella; Device: 755nm Alexandrite Laser; Drug: Asclera

Study type: Interventional

Funder types: Other

Identifiers

NCT06300502

2023P003620

Details and patient eligibility

About

This study will evaluate the tolerability and effectiveness of four FDA-approved treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas. These treatments are: a 1064nm laser, a 755nm laser, and Kybella and Polidocanol injections. Each patient will have a treatment and a control site.

This study is designed with the goal of improved efficacy/tumor reduction via multiple treatment visits. If there is minimal to no clinical improvement in tumor size with one treatment after three treatment visits, the subject will be given the option of crossover treatment with the most effective of the four treatments. Three treatment visits with the crossover treatment will then take place.

Enrollment: 10 estimated patients

Sex: All

Ages: 18 to 85 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Adult ≥18 years of age

2. Have 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

3. Patients must be seeking treatment for cNF

4. Patients must have ≥ 6 paired cNF (3 to be treated and 3 untreated) that are visible and measure between 2-8mm in size. These must be in areas amenable to treatment and surveillance with digital photography

5. cNF must be located on the trunk, arms or legs of the patient

6. Able and willing to comply with all visit, treatment and evaluation schedules and requirements

7. Able to understand and provide written informed consent

Exclusion criteria

1. Individuals who cannot give informed consent or adhere to study schedule.
2. Actively tanning during the course of the study.
3. Known allergy to deoxycholic acid or polidocanol.
4. Women who are pregnant.
5. Those with acute thromboembolic diseases.
6. Those with bleeding abnormalities or those who are currently being treated with antiplatelet or anticoagulant therapy.
7. 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.

Trial design

Primary purpose: Other

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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10 participants in 4 patient groups

Kybella Injection

Active Comparator group

Description:

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

Treatment:

Drug: Kybella

Asclera Injection

Active Comparator group

Description:

Asclera is sterile 1% polidocanol provided in a 2 mL single-use vial.

Treatment:

Drug: Asclera

1064nm laser

Active Comparator group

Description:

The laser used is the GentleMax Pro laser (755/1064 nm wavelength) which targets hemoglobin.

Treatment:

Device: 1064nm Nd:YAG Laser

755nm laser

Active Comparator group

Description:

The laser used is the GentleMax Pro laser (755/1064 nm wavelength) which targets hemoglobin.

Treatment:

Device: 755nm Alexandrite Laser