Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas

Phase 1

Completed

• Conditions: Cutaneous Neurofibroma
• Interventions: Device: 755nm Alexandrite Laser; Device: 1064nm laser; Drug: Kybella

• Registration Number: NCT04730583
• Lead Sponsor: Massachusetts General Hospital

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-01-12
• Study First Submitted QC: 2021-01-26
• Study First Posted: 2021-01-29
• Study Start: 2021-06-02
• Primary Completion: 2024-04-19
• Study Completion: 2024-04-19
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-19
• Last Update Posted: 2025-02-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 34

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
755nm laser	755nm Alexandrite Laser	-
1064nm laser	1064nm laser	-
Kybella Injection	Kybella	-

Primary Outcome Measures

Name	Time	Method
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]	3 months after treatment	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.

Secondary Outcome Measures

Name	Time	Method
Clinician Reported Outcomes	For the 12 months after treatment	Using questionnaires we will determine the clinicians reported outcomes
Patient Report Outcomes	For the 12 months after treatment	Using questionnaires we will determine the patients reported outcomes

Trial Locations

Locations (1)

Wellman Center for Photomedicine, Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States