Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas

NCT ID: NCT04730583

Last Updated: 2025-02-21

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 34 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-06-02
• Study Completion Date: 2024-04-19

Brief 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.

Conditions

Cutaneous Neurofibroma

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: OTHER
• Blinding Strategy: NONE

Study Groups

Kybella Injection

Group Type: ACTIVE_COMPARATOR

Kybella

Intervention Type: DRUG

Injection into the cutaneous Neurofibromas lesion

1064nm laser

Group Type: ACTIVE_COMPARATOR

1064nm laser

Intervention Type: DEVICE

Pulse laser at a wavelength of 1064nm to the cutaneous Neurofibromas lesion

755nm laser

Group Type: ACTIVE_COMPARATOR

755nm Alexandrite Laser

Intervention Type: DEVICE

Pulse laser at a wavelength of 755nm to the cutaneous Neurofibromas lesion

Interventions

Kybella

Injection into the cutaneous Neurofibromas lesion

Intervention Type: DRUG

1064nm laser

Pulse laser at a wavelength of 1064nm to the cutaneous Neurofibromas lesion

Intervention Type: DEVICE

755nm Alexandrite Laser

Pulse laser at a wavelength of 755nm to the cutaneous Neurofibromas lesion

Intervention Type: DEVICE

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Johns Hopkins University

OTHER

Sponsor Role: collaborator

Massachusetts General Hospital

OTHER

Sponsor Role: lead

Responsible Party

Richard Rox Anderson, MD

Principle Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

R. Rox Anderson, MD

Role: PRINCIPAL_INVESTIGATOR

Wellman Center for Photomedicine, Massachusetts General Hospital

Locations

Wellman Center for Photomedicine, Massachusetts General Hospital

Boston, Massachusetts, United States

Countries

United States

Other Identifiers

2020P004137

Identifier Type: -

Identifier Source: org_study_id