Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas
Primary Purpose
Cutaneous Neurofibroma
Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Kybella
980nm laser
755nm Alexandrite Laser
Radiofrequency
Sponsored by
About this trial
This is an interventional other trial for Cutaneous Neurofibroma focused on measuring Neurofibromatosis Type 1
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.
Sites / Locations
- Wellman Center for Photomedicine, Massachusetts General HospitalRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Active Comparator
Active Comparator
Active Comparator
Active Comparator
Arm Label
Kybella Injection
980nm laser
755nm laser
Radiofrequency
Arm Description
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Patient Report Outcomes
Using questionnaires we will determine the patients reported outcomes
Clinician Reported Outcomes
Using questionnaires we will determine the clinicians reported outcomes
Full Information
NCT ID
NCT04730583
First Posted
January 12, 2021
Last Updated
August 10, 2022
Sponsor
Massachusetts General Hospital
Collaborators
Johns Hopkins University
1. Study Identification
Unique Protocol Identification Number
NCT04730583
Brief Title
Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas
Official Title
Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas
Study Type
Interventional
2. Study Status
Record Verification Date
August 2022
Overall Recruitment Status
Recruiting
Study Start Date
June 2, 2021 (Actual)
Primary Completion Date
June 2023 (Anticipated)
Study Completion Date
December 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Massachusetts General Hospital
Collaborators
Johns Hopkins University
4. Oversight
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
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cutaneous Neurofibroma
Keywords
Neurofibromatosis Type 1
7. Study Design
Primary Purpose
Other
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
20 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Kybella Injection
Arm Type
Active Comparator
Arm Title
980nm laser
Arm Type
Active Comparator
Arm Title
755nm laser
Arm Type
Active Comparator
Arm Title
Radiofrequency
Arm Type
Active Comparator
Intervention Type
Drug
Intervention Name(s)
Kybella
Intervention Description
Injection into the cutaneous Neurofibromas lesion
Intervention Type
Device
Intervention Name(s)
980nm laser
Intervention Description
Pulse laser at a wavelength of 980nm to the cutaneous Neurofibromas lesion
Intervention Type
Device
Intervention Name(s)
755nm Alexandrite Laser
Intervention Description
Pulse laser at a wavelength of 755nm to the cutaneous Neurofibromas lesion
Intervention Type
Device
Intervention Name(s)
Radiofrequency
Intervention Description
Using an insulated micro-needle, the radio-frequency will be delivered to the cutaneous Neurofibromas lesion
Primary Outcome Measure Information:
Title
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Description
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.
Time Frame
3 months after treatment
Secondary Outcome Measure Information:
Title
Patient Report Outcomes
Description
Using questionnaires we will determine the patients reported outcomes
Time Frame
For the 12 months after treatment
Title
Clinician Reported Outcomes
Description
Using questionnaires we will determine the clinicians reported outcomes
Time Frame
For the 12 months after treatment
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
85 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Margaret Funk
Phone
617-724-2168
Email
wellmancrc@partners.org
First Name & Middle Initial & Last Name or Official Title & Degree
Beverly Dammin
Email
bdammin@mgh.harvard.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
R. Rox Anderson, MD
Organizational Affiliation
Wellman Center for Photomedicine, Massachusetts General Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Wellman Center for Photomedicine, Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Beverly Dammin
Phone
617-726-3308
Email
bdammin@mgh.harvard.edu
First Name & Middle Initial & Last Name & Degree
Fernanda Sakamoto
Phone
617-724-5329
Email
fsakamoto@mgh.harvard.edu
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas
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