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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
OTHER
Masking
NONE
Enrollment
20
Wellman Center for Photomedicine
Boston, Massachusetts, United States
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.
Time frame: 3 months after treatment
Treatment specific patient reported outcomes (PRO)
NRS11, modality specific satisfaction assessment
Time frame: 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. Physician rates degree of change of treated and control cNFs on a scale from -3 (no change) to 3 (very large improvement).
Time frame: Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment
Modified SkinDex for cNF
Health-related quality of life measure. Asks how much participants have been bothered by cNFs over the past week from 0 (never bothered) to 5 (always bothered).
Time frame: Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment
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