Clinical trial NCT06120036: Dosing and Tolerability of Deoxycholic Acid vs. Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas
Official title: Dosing and Tolerability of Deoxycholic Acid Versus Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Neurofibromatosis 1
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: Johns Hopkins University
First posted: 2023-11-07
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

1. Adult males and females ≥18 years of age
2. Have a diagnosis of NF1 based on germline genetic testing or by meeting ≥ 2 the following criteria:

   1. Family history of NF1
   2. Six or more light brown ("cafe-au-lait") spots on the skin
   3. Presence of two or more neurofibromas of any type, or one or more plexiform neurofibromas
   4. Freckling under the arms or in the groin area
   5. Two or more pigmented, benign bumps on the eye's iris (Lisch nodules)
   6. A distinctive bony lesion: dysplasia (abnormal growth) of the sphenoid bone behind the eye, or dysplasia of long bones, often in the lower leg
   7. Tumor on the optic nerve that may interfere with vision
3. Patients must be seeking treatment for cNF
4. 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.
5. cNF must be located on the trunk, arms or legs of the patient
6. Able and willing to comply with all visit, treatment and evaluation schedules and requirements
7. Able to understand and provide written informed consent

Exclusion Criteria:

1. Patients who are undergoing other treatment modalities or investigational agents for their cNF lesions
2. Individuals who cannot give informed consent or adhere to study schedule
3. Actively tanning during the course of the study
4. 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;
5. Known allergy to injectable anesthetics, polidocanol or deoxycholic acid
6. Those with acute thromboembolic diseases
7. Those with bleeding abnormalities or those who are currently being treated with antiplatelet or anticoagulant therapy
8. Those with dysphagia
9. Women who are pregnant
10. 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

Primary outcomes:
measure: Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
timeFrame: 3 months after treatment
Secondary outcomes:
measure: Treatment specific patient reported outcomes (PRO)
timeFrame: 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
measure: Clinician reported outcomes (ClinRO)
timeFrame: Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment
measure: Modified SkinDex for cNF
timeFrame: Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment
Other outcomes:
measure: Rate of healing
timeFrame: Baseline, 3 months after treatment, 6 months after treatment, and 12 months after treatment
measure: cNF appearance
timeFrame: Baseline, 3 months after treatment, 6 months after treatment, and 12 months after treatment
measure: Biologic effect
timeFrame: 3 months after treatment
Locations:
city: Boston
country: United States
facility: Wellman Center for Photomedicine
state: Massachusetts
Study officials:
affiliation: Massachusetts General Hospital
name: Richard R Anderson, MD
role: PRINCIPAL_INVESTIGATOR
References: