Cutaneous Neurofibroma
Conditions
Keywords
Neurofibromatosis Type 1
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.
Interventions
DRUGKybella
Injection into the cutaneous Neurofibromas lesion
DEVICE1064nm laser
Pulse laser at a wavelength of 1064nm to the cutaneous Neurofibromas lesion
DEVICE755nm Alexandrite Laser
Pulse laser at a wavelength of 755nm to the cutaneous Neurofibromas lesion
Sponsors
Johns Hopkins University
Massachusetts General Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 85 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| Patient Report Outcomes | For the 12 months after treatment | Using questionnaires we will determine the patients reported outcomes |
| Clinician Reported Outcomes | For the 12 months after treatment | Using questionnaires we will determine the clinicians reported outcomes |
Countries
United States
Outcome results
None listed