Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous Neurofibromas

This study will evaluate the effectiveness of skin cooling in increasing tolerability of four treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas. These treatments are: a 980nm laser, a 755nm laser, radio-frequency injection, and a Kybella injection. Each patient will have a treatment and a control site..

Study Overview

• Status: Completed
• Conditions: Neurofibromatosis 1
• Intervention / Treatment: Device: 755nm Alexandrite Laser; Drug: Deoxycholic Acid; Drug: Polidocanol; Device: 1064nm Nd:YAG laser

• Study Type: Interventional
• Enrollment (Actual): 13
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Wellman Center for Photomedicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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:

   1. Family history of NF1
   2. Six or more light brown ("cafe-au-lait") spots on the skin
   3. Presence of two or more neurofibromas of any type, or one or more plexiform neurofibromas
   4. Freckling under the arms or in the groin area
   5. Two or more pigmented, benign bumps on the eye's iris (Lisch nodules)
   6. A distinctive bony lesion: dysplasia (abnormal growth) of the sphenoid bone behind the eye, or dysplasia of long bones, often in the lower leg
   7. 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. Adverse reactions to compounds of any external agent (e.g., gels, lotions or anesthetic creams) required for use in the study, if no alternative to the said agent exists;
4. Known allergy to injectable anesthetics, deoxycholic acid or polidocanol.
5. Women who are pregnant.
6. Those with acute thromboembolic diseases.
7. Those with bleeding abnormalities or those who are currently being treated with antiplatelet or anticoagulant therapy.
8. Those with dysphagia.
9. 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Kybella Injection	Drug: Deoxycholic Acid; Injection into the cutaneous Neurofibromas lesion.; Other Names: Kybella
Active Comparator: 755nm laser	Device: 755nm Alexandrite Laser; Pulse laser at a wavelength of 755nm to the cutaneous Neurofibromas lesion
Active Comparator: 1064nm laser	Device: 1064nm Nd:YAG laser; Pulse laser at a wavelength of 1064nm to the cutaneous Neurofibromas lesion.
Active Comparator: Asclera Injection	Drug: Polidocanol; Injection into the cutaneous Neurofibromas lesion.; Other Names: Asclera

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]	Device based treatment will be considered tolerable if <40% of participants treated have a >grade 2 adverse event (AE). A grade 2 AE is defined as an event that requires treatment.	3 months after treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patient Report Outcomes	Using questionnaires, we will determine the patients' reported outcomes.	For the 12 months after treatment
Clinician Reported Outcomes	Using questionnaires we will determine the clinicians' reported outcomes.	For the 12 months after treatment

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of healing	Measured by photography completed by member at medical team	Baseline, 3 months, 6 months, and 12 months, post-treatment
cNF Appearance	Measured via clinically completed 2D and 3D photography	Baseline, 3 months, 6 months, and 12 months, post-treatment

Collaborators and Investigators

• Sponsor: Massachusetts General Hospital
• Collaborators: Johns Hopkins University
• Investigators: Principal Investigator: Richard R Anderson, MD, Massachusetts General Hospital

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2024
• Primary Completion (Actual): July 31, 2025
• Study Completion (Actual): July 31, 2025

Study Registration Dates

• First Submitted: November 1, 2023
• First Submitted That Met QC Criteria: November 13, 2023
• First Posted (Actual): November 15, 2023

Study Record Updates

• Last Update Posted (Actual): November 21, 2025
• Last Update Submitted That Met QC Criteria: November 18, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms; Neuromuscular Diseases; Genetic Diseases, Inborn; Peripheral Nervous System Diseases; Neoplasms by Histologic Type; Neurodegenerative Diseases; Neoplasms, Nerve Tissue; Heredodegenerative Disorders, Nervous System; Nerve Sheath Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Neurofibroma; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurofibromatoses; Neurofibromatosis 1; Organic Chemicals; Polycyclic Compounds; Equipment and Supplies; Lasers; Optical Devices; Radiation Equipment and Supplies; Steroids; Fused-Ring Compounds; Polymers; Macromolecular Substances; Biomedical and Dental Materials; Manufactured Materials; Technology, Industry, and Agriculture; Alcohols; Glycols; Cholic Acids; Bile Acids and Salts; Cholanes; Ethylene Glycols; Polyethylene Glycols; Polidocanol; Deoxycholic Acid; Lasers, Solid-State
• Other Study ID Numbers: 2023P002152

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: Yes
• product manufactured in and exported from the U.S.: Yes