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Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas

Sponsor
Massachusetts General Hospital (Other)
Overall Status
Recruiting
CT.gov ID
NCT04730583
Collaborator
Johns Hopkins University (Other)
20
Enrollment
1
Location
4
Arms
30
Anticipated Duration (Months)
0.7
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study will evaluate the tolerability and effectiveness of four FDA-approved treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas. These treatments are: a 980nm laser, a 755nm laser, radio-frequency injection, and a Kybella injection. Each patient will have a treatment and a control site.

Condition or Disease Intervention/Treatment Phase
  • Drug: Kybella
  • Device: 980nm laser
  • Device: 755nm Alexandrite Laser
  • Device: Radiofrequency
 Phase 1

Study Design

Study Type:
Interventional
Anticipated Enrollment :
20 participants
Allocation:
Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Other
Official Title:
Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas
Actual Study Start Date :
Jun 2, 2021
Anticipated Primary Completion Date :
Jun 1, 2023
Anticipated Study Completion Date :
Dec 1, 2023

Arms and Interventions

Arm Intervention/Treatment
Active Comparator: Kybella Injection

Drug: Kybella
Injection into the cutaneous Neurofibromas lesion
Active Comparator: 980nm laser

Device: 980nm laser
Pulse laser at a wavelength of 980nm to the cutaneous Neurofibromas lesion
Active Comparator: 755nm laser

Device: 755nm Alexandrite Laser
Pulse laser at a wavelength of 755nm to the cutaneous Neurofibromas lesion
Active Comparator: Radiofrequency

Device: Radiofrequency
Using an insulated micro-needle, the radio-frequency will be delivered to the cutaneous Neurofibromas lesion

Outcome Measures

Primary Outcome Measures

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

  1. Patient Report Outcomes [For the 12 months after treatment]

    Using questionnaires we will determine the patients reported outcomes

  2. Clinician Reported Outcomes [For the 12 months after treatment]

    Using questionnaires we will determine the clinicians reported outcomes

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years to 85 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Wellman Center for Photomedicine, Massachusetts General Hospital Boston Massachusetts United States 02114

Sponsors and Collaborators

  • Massachusetts General Hospital
  • Johns Hopkins University

Investigators

  • Principal Investigator: R. Rox Anderson, MD, Wellman Center for Photomedicine, Massachusetts General Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Richard Rox Anderson, MD, Principle Investigator, Massachusetts General Hospital
ClinicalTrials.gov Identifier:
NCT04730583
Other Study ID Numbers:
  • 2020P004137
First Posted:
Jan 29, 2021
Last Update Posted:
Aug 12, 2022
Last Verified:
Aug 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
Yes
Product Manufactured in and Exported from the U.S.:
Yes
Keywords provided by Richard Rox Anderson, MD, Principle Investigator, Massachusetts General Hospital
Neurofibromatosis Type 1
Additional relevant MeSH terms:
Neurofibromatoses
Neurofibromatosis 1
Neurofibroma
Deoxycholic Acid

Study Results

No Results Posted as of Aug 12, 2022