Assessing the Efficacy of Repeat, Monthly Treatments of Cutaneous Neurofibromas (cNFs)

Phase 1

Not yet recruiting

• Conditions: Neurofibromatosis 1; Neurofibromas, Cutaneous
• Interventions: Drug: Asclera; Device: 755nm Alexandrite Laser; Drug: Kybella

• Registration Number: NCT06300502
• Lead Sponsor: Massachusetts General Hospital

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-02-13
• Status Verified: 2024-03
• Study First Submitted QC: 2024-03-07
• Last Update Submitted: 2024-03-07
• Study First Posted: 2024-03-08
• Last Update Posted: 2024-03-08
• Study Start: 2024-06
• Primary Completion: 2025-12
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 10

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Asclera Injection	Asclera	Asclera is sterile 1% polidocanol provided in a 2 mL single-use vial.
755nm laser	755nm Alexandrite Laser	The laser used is the GentleMax Pro laser (755/1064 nm wavelength) which targets hemoglobin.
Kybella Injection	Kybella	Kybella is sterile 1% deoxycholic acid provided in a 2 mL single-use vial.

Primary Outcome Measures

Name	Time	Method
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]	Time Frame: 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 Outcome Measures

Name	Time	Method
Numeric Rating Scale (NRS-11)/Patient Satisfaction	Baseline, 5 month and if applicable, 10 month.	Patient rates degree of pain experienced prior to, during, and after treatments on a scale of 0-10. Satisfaction with each treatment modality is rated from 0 (very unsatisfied) to 5 (very satisfied).
Skindex 16	Baseline, 5 month and if applicable, 10 month.	Validated measure assessing of the effects of skin diseases on patients' quality of life on a scale of 0 (never bothered) to 5 (always bothered).
Clinician Reported Outcomes	Baseline, 5 month and if applicable, 10 month.	Using questionnaires we will determine the clinicians' reported outcomes. Physician Global Assessment: Clinician rates degree of change of treated and control cNFs on a scale from 1 (no change) to 7 (very large improvement).
Patient Reported Outcomes	Baseline, 5 month and if applicable, 10 month.	Using questionnaires we will determine the patients' reported outcomes. Patient Global Assessment: Patient rates degree of change of treated and control cNFs on a scale from 1 (no change) to 7 (very large improvement).

Trial Locations

Locations (1)

Wellman Center for Photomedicine; 🇺🇸 Boston, Massachusetts, United States