Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)

Ki vehet részt

• Participant is an adult ≥18 years of age
• Participant has 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
• Participant is seeking treatment for cNF
• Participant has ≥ 6 paired cNF that are visible and measure 2 mm or more in size. The target treatment area must be amenable to both deoxycholate injections and surveillance with digital and 3D photography. Preferred locations are trunk (back or chest), arms and legs
• Participant is able and willing to comply with all visit, treatment and evaluation schedules and requirements
• Participant is able to understand and provide written informed consent
• Participant has no known allergy to deoxycholate
• Participant has no concurrent injury or wound in the target area

Ki nem vehet részt

• Participant has a known allergy to deoxycholic acid
• Participant has 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
• Participant cannot give informed consent or adhere to study schedule
• Participant is actively tanning during the course of the study
• For female participants: those who are pregnant