Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas

NCT04730583 | Completed Phase 1 FDA Drug FDA Device

• 1 other identifier: 2020P004137
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 1

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

Keywords

Neurofibromatosis Type 1

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Patient Report Outcomes

  For the 12 months after treatment

• Clinician Reported Outcomes

  For the 12 months after treatment

Study Arms (3)

Kybella Injection

ACTIVE COMPARATOR

Drug: Kybella

1064nm laser

ACTIVE COMPARATOR

Device: 1064nm laser

755nm laser

ACTIVE COMPARATOR

Device: 755nm Alexandrite Laser

Interventions

Kybella DRUG

Injection into the cutaneous Neurofibromas lesion

Kybella Injection

1064nm laser DEVICE

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

1064nm laser

755nm Alexandrite Laser DEVICE

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

755nm laser

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• Johns Hopkins University: collaborator

Study Sites (1)

Wellman Center for Photomedicine, Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Neurofibromatosis 1

Interventions

Deoxycholic Acid; Lasers; Lasers, Solid-State

Condition Hierarchy (Ancestors)

Neurofibromatoses; Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Cholic Acids; Bile Acids and Salts; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Cholanes; Optical Devices; Equipment and Supplies; Radiation Equipment and Supplies

Study Officials

• R. Rox Anderson, MD

  Wellman Center for Photomedicine, Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principle Investigator

Study Record Dates

• First Submitted: January 12, 2021
• First Posted: January 29, 2021
• Study Start: June 2, 2021
• Primary Completion: April 19, 2024
• Study Completion: April 19, 2024
• Last Updated: February 21, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)