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

NCT06132165 | Completed Phase 1 FDA Drug FDA Device

• 1 other identifier: 2023P002152
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

Brief Summary

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..

Conditions

Neurofibromatosis 1

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Patient Report Outcomes

  For the 12 months after treatment

• Clinician Reported Outcomes

  For the 12 months after treatment

Other Outcomes (2)

• Rate of healing

  Baseline, 3 months, 6 months, and 12 months, post-treatment

• cNF Appearance

  Baseline, 3 months, 6 months, and 12 months, post-treatment

Study Arms (4)

Kybella Injection

ACTIVE COMPARATOR

Drug: Deoxycholic Acid

Asclera Injection

ACTIVE COMPARATOR

Drug: Polidocanol

1064nm laser

ACTIVE COMPARATOR

Device: 1064nm Nd:YAG laser

755nm laser

ACTIVE COMPARATOR

Device: 755nm Alexandrite Laser

Interventions

Deoxycholic Acid DRUG

Injection into the cutaneous Neurofibromas lesion.

Also known as: Kybella

Kybella Injection

Polidocanol DRUG

Injection into the cutaneous Neurofibromas lesion.

Also known as: Asclera

Asclera Injection

1064nm Nd:YAG laser DEVICE

Pulse laser at a wavelength of 1064nm to the cutaneous Neurofibromas lesion.

1064nm laser

755nm Alexandrite Laser DEVICE

Pulse laser at a wavelength of 755nm to the cutaneous Neurofibromas lesion

755nm laser

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adult ≥18 years of age
• 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
• Patients must be seeking treatment for cNF
• 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.
• cNF must be located on the trunk, arms or legs of the patient
• Able and willing to comply with all visit, treatment and evaluation schedules and requirements
• Able to understand and provide written informed consent

You may not qualify if:

• Individuals who cannot give informed consent or adhere to study schedule.
• Actively tanning during the course of the study.
• 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;
• Known allergy to injectable anesthetics, deoxycholic acid or polidocanol.
• Women who are pregnant.
• Those with acute thromboembolic diseases.
• Those with bleeding abnormalities or those who are currently being treated with antiplatelet or anticoagulant therapy.
• Those with dysphagia.
• 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.

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• Johns Hopkins University: collaborator

Study Sites (1)

Wellman Center for Photomedicine

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Neurofibromatosis 1

Interventions

Deoxycholic Acid; Polidocanol; Lasers, Solid-State

Condition Hierarchy (Ancestors)

Neurofibromatoses; Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Cholic Acids; Bile Acids and Salts; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Cholanes; Polyethylene Glycols; Ethylene Glycols; Glycols; Alcohols; Organic Chemicals; Polymers; Macromolecular Substances; Biomedical and Dental Materials; Manufactured Materials; Technology, Industry, and Agriculture; Lasers; Optical Devices; Equipment and Supplies; Radiation Equipment and Supplies

Study Officials

• Richard R Anderson, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Wellman Center for Photomedicine

Study Record Dates

• First Submitted: November 1, 2023
• First Posted: November 15, 2023
• Study Start: March 1, 2024
• Primary Completion: July 31, 2025
• Study Completion: July 31, 2025
• Last Updated: November 21, 2025

Record last verified: 2025-11

Locations

US (1)