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Clinical Trial Details — Status: Not yet recruiting

Administrative data

NCT number NCT06300502
Other study ID # 2023P003620
Secondary ID
Status Not yet recruiting
Phase Phase 1
First received
Last updated
Start date June 2024
Est. completion date December 2025

Study information

Verified date March 2024
Source Massachusetts General Hospital
Contact Devon Gunter
Phone 617-724-2168
Email wellmancrc@partners.org
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This study will evaluate the tolerability and effectiveness of four FDA-approved treatments in Neurofibromatosis Type 1 Cutaneous Neurofibromas. These treatments are: a 1064nm laser, a 755nm laser, and Kybella and Polidocanol injections. Each patient will have a treatment and a control site. This study is designed with the goal of improved efficacy/tumor reduction via multiple treatment visits. If there is minimal to no clinical improvement in tumor size with one treatment after three treatment visits, the subject will be given the option of crossover treatment with the most effective of the four treatments. Three treatment visits with the crossover treatment will then take place.


Recruitment information / eligibility

Status Not yet recruiting
Enrollment 10
Est. completion date December 2025
Est. primary completion date December 2025
Accepts healthy volunteers No
Gender All
Age group 18 Years to 85 Years
Eligibility Inclusion Criteria: 1. Adult =18 years of age 2. Have a diagnosis of NF1 based on germline genetic testing or by meeting = 2 the following criteria: - Family history of NF1 - Six or more light brown ("cafe-au-lait") spots on the skin - 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) - 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 3. Patients must be seeking treatment for cNF 4. 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 5. cNF must be located on the trunk, arms or legs of the patient 6. Able and willing to comply with all visit, treatment and evaluation schedules and requirements 7. Able to understand and provide written informed consent Exclusion Criteria: 1. Individuals who cannot give informed consent or adhere to study schedule. 2. Actively tanning during the course of the study. 3. Known allergy to deoxycholic acid or polidocanol. 4. Women who are pregnant. 5. Those with acute thromboembolic diseases. 6. Those with bleeding abnormalities or those who are currently being treated with antiplatelet or anticoagulant therapy. 7. 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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Kybella
Injection into the cutaneous Neurofibromas lesion.
Asclera
Injection into the cutaneous Neurofibromas lesion.
Device:
755nm Alexandrite Laser
Pulse laser at a wavelength of 755nm to the cutaneous Neurofibromas lesion.
1064nm Nd:YAG Laser
Pulse laser at a wavelength of 1064nm to the cutaneous Neurofibromas lesion

Locations

Country Name City State
United States Wellman Center for Photomedicine Boston Massachusetts

Sponsors (2)

Lead Sponsor Collaborator
Massachusetts General Hospital Johns Hopkins University

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Other Rate of healing Measured clinically via photography completed by a member of the study team at baseline, 1 month, 2 month, 5 month and if applicable, 6 month, 7 month and 10 month post-treatment. Baseline, 1 month, 2 month, 5 month and if applicable, 6 month, 7 month and 10 month.
Other cNF appearance (volume) Clinically completed 2D and 3D photography Cherry Imaging. Change from baseline in volume of cNFs. Baseline, 1 month, 2 month, 5 month and if applicable, 6 month, 7 month and 10 month.
Other cNF appearance (height) Clinically completed 2D and 3D photography Cherry Imaging. Change from baseline in height of cNFs. Baseline, 1 month, 2 month, 5 month and if applicable, 6 month, 7 month and 10 month.
Primary 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. Time Frame: 3 months after treatment
Secondary Numeric Rating Scale (NRS-11)/Patient Satisfaction Patient rates degree of pain experienced prior to, during, and after treatments on a scale of 0-10. Satisfaction with each treatment modality is rated from 0 (very unsatisfied) to 5 (very satisfied). Baseline, 5 month and if applicable, 10 month.
Secondary Skindex 16 Validated measure assessing of the effects of skin diseases on patients' quality of life on a scale of 0 (never bothered) to 5 (always bothered). Baseline, 5 month and if applicable, 10 month.
Secondary Patient Reported Outcomes Using questionnaires we will determine the patients' reported outcomes. Patient Global Assessment: Patient rates degree of change of treated and control cNFs on a scale from 1 (no change) to 7 (very large improvement). Baseline, 5 month and if applicable, 10 month.
Secondary Clinician Reported Outcomes Using questionnaires we will determine the clinicians' reported outcomes. Physician Global Assessment: Clinician rates degree of change of treated and control cNFs on a scale from 1 (no change) to 7 (very large improvement). Baseline, 5 month and if applicable, 10 month.
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