Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas

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

Study Overview

• Status: Completed
• Conditions: Cutaneous Neurofibroma
• Intervention / Treatment: Drug: Kybella; Device: 755nm Alexandrite Laser; Device: 1064nm laser

• Study Type: Interventional
• Enrollment (Actual): 34
• Phase: Phase 1

Contacts and Locations

Study Locations

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

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Adult males and females ≥18 years of age
• Have a diagnosis of Neurofibromatosis Type 1
• Patients must be seeking treatment for cutaneous Neurofibromas
• Patients must have ≥ 6 paired cutaneous Neurofibromas (3 to be treated and 3 untreated) that are visible and measure between 2-4mm in size.
• Able and willing to comply with all visit, treatment and evaluation schedules and requirements
• Able to understand and provide written informed consent
• Access to a Smart Phone to be able to take and upload photographs to an application

Exclusion Criteria:

• Patients who are undergoing other treatment modalities or investigational agents for their cNF lesions
• 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 or deoxycholic acid
• 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;
• Pregnant females, due to possible discomfort with the procedure even though the procedure is localized and there is no new drug.

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

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Kybella Injection	Drug: Kybella; Injection into the cutaneous Neurofibromas lesion
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 laser; Pulse laser at a wavelength of 1064nm to the cutaneous Neurofibromas lesion

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

Collaborators and Investigators

• Sponsor: Massachusetts General Hospital
• Collaborators: Johns Hopkins University
• Investigators: Principal Investigator: R. Rox Anderson, MD, Wellman Center for Photomedicine, Massachusetts General Hospital

Study record dates

Study Major Dates

• Study Start (Actual): June 2, 2021
• Primary Completion (Actual): April 19, 2024
• Study Completion (Actual): April 19, 2024

Study Registration Dates

• First Submitted: January 12, 2021
• First Submitted That Met QC Criteria: January 26, 2021
• First Posted (Actual): January 29, 2021

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 19, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Keywords: Neurofibromatosis Type 1
• 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; Nervous System Neoplasms; Heredodegenerative Disorders, Nervous System; Nerve Sheath Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Peripheral Nervous System Neoplasms; Neurofibromatoses; Neurofibromatosis 1; Neurofibroma; Gastrointestinal Agents; Cholagogues and Choleretics; Deoxycholic Acid
• Other Study ID Numbers: 2020P004137

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

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