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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

About this trial

This is an interventional other trial for Cutaneous Neurofibroma focused on measuring Neurofibromatosis Type 1

Eligibility Criteria

18 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

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

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

wellmancrc@partners.org

First Name & Middle Initial & Last Name or Official Title & Degree

Beverly Dammin

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

bdammin@mgh.harvard.edu

First Name & Middle Initial & Last Name & Degree

Fernanda Sakamoto

Phone

617-724-5329

fsakamoto@mgh.harvard.edu

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas

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