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

Study Overview
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Brief Summary
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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..

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

Conditions
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Neurofibromatosis 1

Intervention / Treatment
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* Drug: Deoxycholic Acid
* Drug: Polidocanol
* Device: 1064nm Nd:YAG laser
* Device: 755nm Alexandrite Laser


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Exclusion Criteria: 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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 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: Deoxycholic Acid<br>* Injection into the cutaneous Neurofibromas lesion.<br>* Other names: Kybella;|
| Active Comparator: Asclera Injection<br> | Drug: Polidocanol<br>* Injection into the cutaneous Neurofibromas lesion.<br>* Other names: Asclera;|
| Active Comparator: 1064nm laser<br> | Device: 1064nm Nd:YAG 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 |