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OpenTrials
Completed

NCT Number: NCT04730583

Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas

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.

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Key information

Conditions

Cutaneous Neurofibroma Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Neoplasms Neoplasms by Histologic Type Neoplasms, Nerve Tissue Neoplastic Syndromes, Hereditary Nerve Sheath Neoplasms Nervous System Diseases Neurocutaneous Syndromes Neurodegenerative Diseases Neurofibroma Neurofibromatoses Neurofibromatosis 1 Neuromuscular Diseases Peripheral Nervous System Diseases

Age range

18 year–85 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Wellman Center for Photomedicine, Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Kybella

Drug

Injection into the cutaneous Neurofibromas lesion

1064nm Laser

Device

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

755nm Alexandrite Laser

Device

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

Primary outcomes

  1. Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

    Time frame: 3 months after treatment

    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.

Secondary outcomes

  1. Patient Report Outcomes

    Time frame: For the 12 months after treatment

    Using questionnaires we will determine the patients reported outcomes

  2. Clinician Reported Outcomes

    Time frame: For the 12 months after treatment

    Using questionnaires we will determine the clinicians reported outcomes

Sponsors and collaborators

Lead sponsor

Massachusetts General Hospital

Other

Collaborators

  • Johns Hopkins University

Registry information

Important dates

Study start
2021
Primary completion
2024
Study completion
2024
First posted
Jan 29, 2021
Registry last updated
Feb 21, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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