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Study is currently not funded at this time.
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| Name | Class |
|---|---|
| Johns Hopkins University | OTHER |
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The goal of this clinical trial is to evaluate the tolerability and effectiveness of multiple treatments of Alexandrite (755 nm) Laser in those with Neurofibromatosis Type 1 (NF1) Cutaneous Neurofibromas (cNFs). The main questions it aims to answer are:
Will performing up to 6 months of treatment sessions with alexandrite laser will result in tolerable local skin reactions and reduction in both individual cNF size by >50% as well as improved cNF appearance in the treated field? If there is a comparison group: Researchers will compare laser treatment with cooling to both laser treatment without cooling and an untreated control see if laser treatments are effective and if cooling makes treatment more tolerable.
Participants will:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treated cutaneous neurofibromas (cNFs) | Active Comparator | Participants will receive treatment with laser at a wavelength of 755 nm to the cutaneous Neurofibromas lesion. |
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| Control cutaneous neurofibromas (cNFs) | No Intervention | A complementary region of cNFs of similar characteristics to the treatment area in the same body region will be selected to serve as an untreated control group. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GentleMax Pro (with skin cooling) | Device | Treatment will be performed with the GentleMax Pro laser (755 nm wavelength), which targets hemoglobin. A negative pressure hand piece provides temporary, mild suction (~1/2 atm) before laser exposure. A pulse fluence of 60-100 J/cm2 (single or double pulse) will be used with 8 mm diameter exposure spot size, and 3 ms pulse duration. The laser system includes a dynamic cryogen spray cooling device (DCD) for epidermal protection. A spurt of cryogen cools the epidermis just before arrival of the treatment laser pulse. |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability) | Treatment will be considered tolerable if <40% of participants treated have a >Grade 2 CTCAE v5 adverse event (AE). | Anytime from initiation of treatment through study completion, an average of 8 months. |
| Measure | Description | Time Frame |
|---|---|---|
| Patient Global Assessment of cNF Improvement (Likert Scale) | Patient rates degree of change of treated cNFs on a scale from -3 (very much worse) to 3 (very large improvement). | Baseline, Day 90, and 30 days after final treatment (up to Day 240). |
| Clinician Global Assessment of cNF Improvement (Likert Scale) |
| Measure | Description | Time Frame |
|---|---|---|
| Biologic Effect/Histology | A subset of 2-6 cNFs from the treatment region will be assessed after the final treatment session. | 3 months after final treatment session. |
Inclusion Criteria:
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, Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
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| GentleMax Pro (without skin cooling) | Device | Treatment will be performed with the GentleMax Pro laser (755 nm wavelength), which targets hemoglobin. A negative pressure hand piece provides temporary, mild suction (~1/2 atm) before laser exposure. A pulse fluence of 60-100 J/cm2 (single or double pulse) will be used with 8 mm diameter exposure spot size, and 3 ms pulse duration. |
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Clinician rates degree of change of treated cNFs on a scale from -3 (very much worse) to 3 (very large improvement). |
| Baseline, Day 90, and 30 days after final treatment (up to Day 240). |
| VAS Pain Scale | Participants will be asked to rate their pain using a visual analog scale (0 to 10) at each treatment visit and 1 week after each treatment. | Baseline, every 30 days (up to Day 240). |
| Patient Satisfaction | Satisfaction with each treatment modality is rated from 1 (very unsatisfied) to 5 (very satisfied). | Baseline, Day 90, and 30 days after final treatment (up to Day 240). |
| Rate of Healing | Measured clinically via photography completed by a member of the study team at baseline, and all in-person visits. | Baseline, every 30 days (up to Day 240). |
| cNF Appearance (Height) | Clinically completed 3D Cherry Imaging. Change from baseline in height of cNFs. | Baseline, Day 90, and 30 days after final treatment (up to Day 240). |
| cNF Appearance (Volume) | Clinically completed 3D Cherry Imaging. Change from baseline in volume of cNFs. | Baseline, Day 90, and 30 days after final treatment (up to Day 240). |
| ID | Term |
|---|---|
| D009456 | Neurofibromatosis 1 |
| D017253 | Neurofibromatoses |
| ID | Term |
|---|---|
| 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 |
|---|---|
| D053844 | Lasers, Solid-State |
| D007834 | Lasers |
| ID | Term |
|---|---|
| D055096 | Optical Devices |
| D004864 | Equipment and Supplies |
| D055618 | Radiation Equipment and Supplies |
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