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| Name | Class |
|---|---|
| Johns Hopkins University | OTHER |
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This study will evaluate the tolerability and effectiveness of two treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas. These treatments are: Kybella and Asclera 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 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Kybella | 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 adverse event that requires treatment. | 3 months after treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Treatment specific patient reported outcomes (PRO) | NRS11, modality specific satisfaction assessment | Baseline, 1 day after treatment, 1 week after treatment, 1 month after treatment, 2 months after treatment, 3 months after treatment, 6 months after treatment, 12 months after treatment |
| Clinician reported outcomes (ClinRO) |
| Measure | Description | Time Frame |
|---|---|---|
| Rate of healing | Measured clinically via photography completed by a member of the study team at baseline, 3-month, 6-month, and 12-month post-treatment. | Baseline, 3 months after treatment, 6 months after treatment, and 12 months after treatment |
| cNF appearance |
Inclusion Criteria:
Adult males and females ≥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 per modality (3 treated and 3 untreated). cNF should be 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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| ID | Term |
|---|---|
| D009456 | Neurofibromatosis 1 |
| ID | Term |
|---|---|
| D017253 | Neurofibromatoses |
| D009455 | Neurofibroma |
| D018317 | Nerve Sheath Neoplasms |
| D009380 | Neoplasms, Nerve Tissue |
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| ID | Term |
|---|---|
| D003840 | Deoxycholic Acid |
| D000077423 | Polidocanol |
| ID | Term |
|---|---|
| D002793 | Cholic Acids |
| D001647 | Bile Acids and Salts |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
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| Asclera |
| Drug |
Injection into the cutaneous Neurofibromas lesion. |
|
Clinician assessment of cNF via questionnaire. Physician rates degree of change of treated and control cNFs on a scale from -3 (no change) to 3 (very large improvement). |
| Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment |
| Modified SkinDex for cNF | Health-related quality of life measure. Asks how much participants have been bothered by cNFs over the past week from 0 (never bothered) to 5 (always bothered). | Baseline, 3 months after treatment, 6 months after treatment, 12 months after treatment |
Clinically completed 2D and 3D photography Cherry Imaging. |
| Baseline, 3 months after treatment, 6 months after treatment, and 12 months after treatment |
| Biologic effect | Degree of tissue necrosis on skin lesion biopsy at 3 months as assessed by review of area of necrosis in histology slides. | 3 months after treatment |
| 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 |
| 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 |