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

Neurofibromatosis 1 Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06132165
• Other study ID #: 2023P002152
• Status: Recruiting
• Phase: Phase 1
• Start date: March 1, 2024
• Est. completion date: October 31, 2024

Study information

• Verified date: February 2024
• Source: Massachusetts General Hospital
• Contact: Devon Gunter
• Phone: 617-724-2168
• Email: wellmancrc[@]partners.org
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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..

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: October 31, 2024
• Est. primary completion date: October 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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.

Related Conditions & MeSH terms

• Neurofibroma
• Neurofibromatoses
• Neurofibromatosis 1

Intervention

Drug:
• Deoxycholic Acid
Injection into the cutaneous Neurofibromas lesion.
• Polidocanol
Injection into the cutaneous Neurofibromas lesion.

Device:
• 1064nm Nd:YAG laser
Pulse laser at a wavelength of 1064nm to the cutaneous Neurofibromas lesion.
• 755nm Alexandrite Laser
Pulse laser at a wavelength of 755nm to the cutaneous Neurofibromas lesion

Locations

Country	Name	City	State
United States	Wellman Center for Photomedicine	Boston	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
Massachusetts General Hospital	Johns Hopkins University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Rate of healing	Measured by photography completed by member at medical team	Baseline, 3 months, 6 months, and 12 months, post-treatment
Other	cNF Appearance	Measured via clinically completed 2D and 3D photography	Baseline, 3 months, 6 months, and 12 months, post-treatment
Primary	Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]	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.	3 months after treatment
Secondary	Patient Report Outcomes	Using questionnaires, we will determine the patients' reported outcomes.	For the 12 months after treatment
Secondary	Clinician Reported Outcomes	Using questionnaires we will determine the clinicians' reported outcomes.	For the 12 months after treatment