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

Neurofibromatosis 1 Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06300502
• Other study ID #: 2023P003620
• Status: Not yet recruiting
• Phase: Phase 1
• Start date: June 2024
• Est. completion date: December 2025

Study information

• Verified date: March 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 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 10
• Est. completion date: December 2025
• Est. primary completion date: December 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 85 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:

• 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.

Related Conditions & MeSH terms

• Neurofibroma
• Neurofibromatoses
• Neurofibromatosis 1

Intervention

Drug:
• Kybella
Injection into the cutaneous Neurofibromas lesion.
• Asclera
Injection into the cutaneous Neurofibromas lesion.

Device:
• 755nm Alexandrite Laser
Pulse laser at a wavelength of 755nm to the cutaneous Neurofibromas lesion.
• 1064nm Nd:YAG Laser
Pulse laser at a wavelength of 1064nm 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 clinically via photography completed by a member of the study team at baseline, 1 month, 2 month, 5 month and if applicable, 6 month, 7 month and 10 month post-treatment.	Baseline, 1 month, 2 month, 5 month and if applicable, 6 month, 7 month and 10 month.
Other	cNF appearance (volume)	Clinically completed 2D and 3D photography Cherry Imaging. Change from baseline in volume of cNFs.	Baseline, 1 month, 2 month, 5 month and if applicable, 6 month, 7 month and 10 month.
Other	cNF appearance (height)	Clinically completed 2D and 3D photography Cherry Imaging. Change from baseline in height of cNFs.	Baseline, 1 month, 2 month, 5 month and if applicable, 6 month, 7 month and 10 month.
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.	Time Frame: 3 months after treatment
Secondary	Numeric Rating Scale (NRS-11)/Patient Satisfaction	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).	Baseline, 5 month and if applicable, 10 month.
Secondary	Skindex 16	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).	Baseline, 5 month and if applicable, 10 month.
Secondary	Patient Reported Outcomes	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).	Baseline, 5 month and if applicable, 10 month.
Secondary	Clinician Reported Outcomes	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).	Baseline, 5 month and if applicable, 10 month.