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| Name | Class |
|---|---|
| Johns Hopkins University | OTHER |
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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..
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Kybella Injection | Active Comparator |
| |
| Asclera Injection | Active Comparator |
| |
| 1064nm laser | Active Comparator |
| |
| 755nm laser | Active Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Deoxycholic Acid | Drug | Injection into the cutaneous Neurofibromas lesion. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 3 months after treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Patient Report Outcomes | Using questionnaires, we will determine the patients' reported outcomes. | For the 12 months after treatment |
| Clinician Reported Outcomes | Using questionnaires we will determine the clinicians' reported outcomes. |
| Measure | Description | Time Frame |
|---|---|---|
| Rate of healing | Measured by photography completed by member at medical team | Baseline, 3 months, 6 months, and 12 months, post-treatment |
| cNF Appearance | Measured via clinically completed 2D and 3D photography |
Inclusion Criteria:
Adult ≥18 years of age
Have a diagnosis of NF1 based on germline genetic testing or by meeting ≥ 2 the following criteria:
Patients must be seeking treatment for cNF
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.
cNF must be located on the trunk, arms or legs of the patient
Able and willing to comply with all visit, treatment and evaluation schedules and requirements
Able to understand and provide written informed consent
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Richard R Anderson, MD | Massachusetts General Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Wellman Center for Photomedicine | Boston | Massachusetts | 02114 | United States |
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| Polidocanol | Drug | Injection into the cutaneous Neurofibromas lesion. |
|
|
| 1064nm Nd:YAG laser | Device | Pulse laser at a wavelength of 1064nm to the cutaneous Neurofibromas lesion. |
|
| 755nm Alexandrite Laser | Device | Pulse laser at a wavelength of 755nm to the cutaneous Neurofibromas lesion |
|
| For the 12 months after treatment |
| Baseline, 3 months, 6 months, and 12 months, post-treatment |
| ID | Term |
|---|---|
| D009456 | Neurofibromatosis 1 |
| ID | Term |
|---|---|
| D017253 | Neurofibromatoses |
| D009455 | Neurofibroma |
| D018317 | Nerve Sheath Neoplasms |
| D009380 | Neoplasms, Nerve Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009386 | Neoplastic Syndromes, Hereditary |
| D020752 | Neurocutaneous Syndromes |
| D009422 | Nervous System Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| D003840 | Deoxycholic Acid |
| D000077423 | Polidocanol |
| D053844 | Lasers, Solid-State |
| ID | Term |
|---|---|
| D002793 | Cholic Acids |
| D001647 | Bile Acids and Salts |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D002757 | Cholanes |
| D011092 | Polyethylene Glycols |
| D005026 | Ethylene Glycols |
| D006018 | Glycols |
| D000438 | Alcohols |
| D009930 | Organic Chemicals |
| D011108 | Polymers |
| D046911 | Macromolecular Substances |
| D001697 | Biomedical and Dental Materials |
| D008420 | Manufactured Materials |
| D013676 | Technology, Industry, and Agriculture |
| D007834 | Lasers |
| D055096 | Optical Devices |
| D004864 | Equipment and Supplies |
| D055618 | Radiation Equipment and Supplies |
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