Dosing and Tolerability of Deoxycholic Acid Versus Polidocanol in the Treatment of 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 two treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas. These treatments are: Kybella and Asclera injection. Each patient will have a treatment and a control site.

Official Title
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Dosing and Tolerability of Deoxycholic Acid Versus Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas

Conditions
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Neurofibromatosis 1

Intervention / Treatment
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* Drug: Kybella
* Drug: Asclera


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 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 per modality (3 treated and 3 untreated). cNF should be 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: 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, polidocanol or deoxycholic acid Those with acute thromboembolic diseases Those with bleeding abnormalities or those who are currently being treated with antiplatelet or anticoagulant therapy Those with dysphagia Women who are pregnant 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: 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: Asclera Injection<br> | Drug: Asclera<br>* Injection into 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 adverse event that requires treatment. | 3 months after treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Treatment specific patient reported outcomes (PRO) | NRS11, modality specific satisfaction assessment | Baseline, 1 day after treatment, 1 week after treatment, 1 month after treatment, 2 months after treatment, 3 months after treatment, 6 months after treatment, 12 months after treatment | 
| Clinician reported outcomes (ClinRO) | Clinician assessment of cNF via questionnaire | Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment | 
| Modified SkinDex for cNF | Health-related quality of life measure | Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment |