Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous Neurofibromas
Study Details
Study Description
Brief Summary
This study will evaluate the effectiveness of skin cooling in increasing tolerability of four 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 |
|---|---|---|
|
Phase 1 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Active Comparator: Kybella Injection
|
Drug: Deoxycholic Acid
Injection into the cutaneous Neurofibromas lesion.
Other Names:
|
| Active Comparator: Asclera Injection
|
Drug: Polidocanol
Injection into the cutaneous Neurofibromas lesion.
Other Names:
|
| Active Comparator: 1064nm laser
|
Device: 1064nm Nd:YAG laser
Pulse laser at a wavelength of 1064nm 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
|
Outcome Measures
Primary Outcome Measures
- 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
- Patient Report Outcomes [For the 12 months after treatment]
Using questionnaires, we will determine the patients' reported outcomes.
- Clinician Reported Outcomes [For the 12 months after treatment]
Using questionnaires we will determine the clinicians' reported outcomes.
Other Outcome Measures
- Rate of healing [Baseline, 3 months, 6 months, and 12 months, post-treatment]
Measured by photography completed by member at medical team
- cNF Appearance [Baseline, 3 months, 6 months, and 12 months, post-treatment]
Measured via clinically completed 2D and 3D photography
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Adult ≥18 years of age
-
Have a diagnosis of NF1 based on germline genetic testing or by meeting ≥ 2 the following criteria:
-
Family history of NF1
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Six or more light brown ("cafe-au-lait") spots on the skin
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Presence of two or more neurofibromas of any type, or one or more plexiform neurofibromas
-
Freckling under the arms or in the groin area
-
Two or more pigmented, benign bumps on the eye's iris (Lisch nodules)
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A distinctive bony lesion: dysplasia (abnormal growth) of the sphenoid bone behind the eye, or dysplasia of long bones, often in the lower leg
-
Tumor on the optic nerve that may interfere with vision
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Patients must be seeking treatment for cNF
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Patients must have ≥ 6 paired cNF (3 to be treated and 3 untreated) that are visible and measure between 2-8mm in size. These must be in areas amenable to treatment and surveillance with digital photography.
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cNF must be located on the trunk, arms or legs of the patient
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Able and willing to comply with all visit, treatment and evaluation schedules and requirements
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Able to understand and provide written informed consent
Exclusion Criteria:
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Individuals who cannot give informed consent or adhere to study schedule.
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Actively tanning during the course of the study.
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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, deoxycholic acid or polidocanol.
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Women who are pregnant.
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Those with acute thromboembolic diseases.
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Those with bleeding abnormalities or those who are currently being treated with antiplatelet or anticoagulant therapy.
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Those with dysphagia.
-
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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Wellman Center for Photomedicine | Boston | Massachusetts | United States | 02114 |
Sponsors and Collaborators
- Massachusetts General Hospital
- Johns Hopkins University
Investigators
- Principal Investigator: Richard R Anderson, MD, Massachusetts General Hospital
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 2023P002152