Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous Neurofibromas

Phase 1

Recruiting

• Conditions: Neurofibromatosis 1
• Interventions: Drug: Deoxycholic Acid; Drug: Polidocanol; Device: 755nm Alexandrite Laser

• Registration Number: NCT06132165
• Lead Sponsor: Massachusetts General Hospital

Brief Summary

This study will evaluate the effectiveness of skin cooling in increasing tolerability of four treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas. These treatments are: a 980nm laser, a 755nm laser, radio-frequency injection, and a Kybella injection. Each patient will have a treatment and a control site..

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-11-01
• Study First Submitted QC: 2023-11-13
• Study First Posted: 2023-11-15
• Status Verified: 2024-02
• Last Update Submitted: 2024-02-07
• Last Update Posted: 2024-02-09
• Study Start: 2024-03-01
• Primary Completion: 2024-10-31
• Study Completion: 2024-10-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 30

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:

   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 (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. 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;
4. Known allergy to injectable anesthetics, deoxycholic acid or polidocanol.
5. Women who are pregnant.
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. 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: PARALLEL

Arm && Interventions

Group	Intervention	Description
Kybella Injection	Deoxycholic Acid	-
Asclera Injection	Polidocanol	-
755nm laser	755nm Alexandrite Laser	-

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

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