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Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04730583
Recruitment Status : Recruiting
First Posted : January 29, 2021
Last Update Posted : August 12, 2022
Sponsor:
Collaborator:
Johns Hopkins University
Information provided by (Responsible Party):
Richard Rox Anderson, MD, Massachusetts General Hospital

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
Cutaneous Neurofibroma Drug: Kybella Device: 980nm laser Device: 755nm Alexandrite Laser Device: Radiofrequency Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated 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 : June 2, 2021
Estimated Primary Completion Date : June 2023
Estimated Study Completion Date : December 2023


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




Primary Outcome Measures :
  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 Outcome Measures :
  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



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Ages Eligible for Study:   18 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04730583


Contacts
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Contact: Margaret Funk 617-724-2168 wellmancrc@partners.org
Contact: Beverly Dammin bdammin@mgh.harvard.edu

Locations
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United States, Massachusetts
Wellman Center for Photomedicine, Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Beverly Dammin    617-726-3308    bdammin@mgh.harvard.edu   
Contact: Fernanda Sakamoto    617-724-5329    fsakamoto@mgh.harvard.edu   
Sponsors and Collaborators
Massachusetts General Hospital
Johns Hopkins University
Investigators
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Principal Investigator: R. Rox Anderson, MD Wellman Center for Photomedicine, Massachusetts General Hospital
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Responsible Party: Richard Rox Anderson, MD, Principle Investigator, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT04730583    
Other Study ID Numbers: 2020P004137
First Posted: January 29, 2021    Key Record Dates
Last Update Posted: August 12, 2022
Last Verified: August 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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, Massachusetts General Hospital:
Neurofibromatosis Type 1
Additional relevant MeSH terms:
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Neurofibromatoses
Neurofibromatosis 1
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
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neuromuscular Diseases
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Deoxycholic Acid
Cholagogues and Choleretics
Gastrointestinal Agents