Clinical trial NCT06300502: Assessing the Efficacy of Repeat, Monthly Treatments of Cutaneous Neurofibromas (cNFs)
Official title: Assessing the Efficacy of Repeat, Monthly Treatments of Cutaneous Neurofibromas (cNFs)
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
Conditions: Neurofibromas, Cutaneous
Neurofibromatosis 1
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: Johns Hopkins University
First posted: 2024-03-08
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: OTHER

Brief summary: This study will evaluate the tolerability and effectiveness of four FDA-approved treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas. These treatments are: a 1064nm laser, a 755nm laser, and Kybella and Polidocanol injections. Each patient will have a treatment and a control site.

This study is designed with the goal of improved efficacy/tumor reduction via multiple treatment visits. If there is minimal to no clinical improvement in tumor size with one treatment after three treatment visits, the subject will be given the option of crossover treatment with the most effective of the four treatments. Three treatment visits with the crossover treatment will then take place.


Eligibility criteria: Inclusion Criteria:

1. Adult ≥18 years of age
2. 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
3. Patients must be seeking treatment for cNF
4. Patients must have ≥ 6 paired cNF (3 to be treated and 3 untreated) that are 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. Individuals who cannot give informed consent or adhere to study schedule.
2. Actively tanning during the course of the study.
3. Known allergy to deoxycholic acid or polidocanol.
4. Women who are pregnant.
5. Those with acute thromboembolic diseases.
6. Those with bleeding abnormalities or those who are currently being treated with antiplatelet or anticoagulant therapy.
7. 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: Time Frame: 3 months after treatment
Secondary outcomes:
measure: Numeric Rating Scale (NRS-11)/Patient Satisfaction
timeFrame: Baseline, 5 month and if applicable, 10 month.
measure: Skindex 16
timeFrame: Baseline, 5 month and if applicable, 10 month.
measure: Patient Reported Outcomes
timeFrame: Baseline, 5 month and if applicable, 10 month.
measure: Clinician Reported Outcomes
timeFrame: Baseline, 5 month and if applicable, 10 month.
Other outcomes:
measure: Rate of healing
timeFrame: Baseline, 1 month, 2 month, 5 month and if applicable, 6 month, 7 month and 10 month.
measure: cNF appearance (volume)
timeFrame: Baseline, 1 month, 2 month, 5 month and if applicable, 6 month, 7 month and 10 month.
measure: cNF appearance (height)
timeFrame: Baseline, 1 month, 2 month, 5 month and if applicable, 6 month, 7 month and 10 month.
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: