Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas

Study Overview
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Brief Summary
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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.

Official Title
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Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas

Conditions
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Cutaneous Neurofibroma

Intervention / Treatment
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* Drug: Kybella
* Device: 1064nm laser
* Device: 755nm Alexandrite Laser


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 85 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Kybella Injection<br> | Drug: Kybella<br>* Injection into the cutaneous Neurofibromas lesion<br>|
| Active Comparator: 1064nm laser<br> | Device: 1064nm laser<br>* Pulse laser at a wavelength of 1064nm to the cutaneous Neurofibromas lesion<br>|
| Active Comparator: 755nm laser<br> | Device: 755nm Alexandrite Laser<br>* Pulse laser at a wavelength of 755nm to the cutaneous Neurofibromas lesion<br>|

What is the study measuring?
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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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Neurofibromatosis Type 1