Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas

Cutaneous Neurofibroma Clinical Trial

Official title:

Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04730583
• Other study ID #: 2020P004137
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: June 2, 2021
• Est. completion date: December 2024

Study information

• Verified date: November 2023
• Source: Massachusetts General Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 20
• Est. completion date: December 2024
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 85 Years

Eligibility

Inclusion Criteria:

• Adult males and females =18 years of age
• Have a diagnosis of Neurofibromatosis Type 1
• Patients must be seeking treatment for cutaneous Neurofibromas
• Patients must have = 6 paired cutaneous Neurofibromas (3 to be treated and 3 untreated) that are visible and measure between 2-4mm in size.
• Able and willing to comply with all visit, treatment and evaluation schedules and requirements
• Able to understand and provide written informed consent
• Access to a Smart Phone to be able to take and upload photographs to an application

Exclusion Criteria:

• Patients who are undergoing other treatment modalities or investigational agents for their cNF lesions
• Individuals who cannot give informed consent or adhere to study schedule
• Actively tanning during the course of the study
• 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;
• Known allergy to injectable anesthetics or deoxycholic acid
• 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;
• Pregnant females, due to possible discomfort with the procedure even though the procedure is localized and there is no new drug.

Related Conditions & MeSH terms

• Cutaneous Neurofibroma
• Neurofibroma
• Neurofibromatoses
• Neurofibromatosis 1

Intervention

Drug:
• Kybella
Injection into the cutaneous Neurofibromas lesion

Device:
• 1064nm 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, Massachusetts General Hospital	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
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