Multiple Treatments for Skin Tumors

Overseen By Devon Gunter

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Massachusetts General Hospital

Must not be taking: Antiplatelets, Anticoagulants

Disqualifiers: Pregnancy, Thromboembolic diseases, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 6

Duration: 5 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to evaluate the effectiveness of four existing treatments in reducing skin tumors in individuals with Neurofibromatosis Type 1 (NF1). The treatments being tested include two lasers (1064nm Nd:YAG Laser and 755nm Alexandrite Laser) and two types of injections (Asclera and Kybella). Participants will receive treatments on some tumors, while others will remain untreated for comparison. If a treatment does not shrink the tumors after three visits, participants may switch to the most promising treatment. The trial seeks individuals with NF1 who have visible skin tumors that are easy to photograph and measure. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants a chance to contribute to early-stage medical research.

Do I need to stop my current medications for the trial?

The trial protocol does not specify whether you need to stop taking your current medications. However, if you are on antiplatelet or anticoagulant therapy, you may not be eligible to participate.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that the treatments in this study are generally safe. The 1064nm Nd:YAG laser is safe for various skin types, including darker ones, and often treats skin issues like blood vessel problems without damaging the skin's surface.

The 755nm Alexandrite laser is also considered safe. It addresses skin roughness and redness, though it might temporarily change skin color, such as darkening or lightening.

Asclera, known as polidocanol, has FDA approval for treating small varicose veins. It is mostly safe, but there is a small risk of serious allergic reactions, making medical supervision during treatment important.

Kybella, another FDA-approved treatment, is safe for reducing fat under the chin. Common side effects include swelling and bruising at the injection site. While approved for use under the chin, using it elsewhere might be riskier.

Overall, these treatments have been studied and are generally safe. However, monitoring for any side effects is important, as with any medical treatment. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about these treatments for skin tumors because they employ innovative methods to target and reduce tumors. The 1064nm and 755nm lasers use specific wavelengths to precisely target hemoglobin in the blood vessels of the tumors, potentially leading to more effective and less invasive treatment. Asclera and Kybella injections offer non-surgical options with unique active ingredients; Asclera uses polidocanol to sclerose blood vessels, while Kybella utilizes deoxycholic acid to break down fat cells. These approaches provide alternatives to traditional surgical and topical treatments, with the potential for faster recovery and fewer side effects.

What evidence suggests that this trial's treatments could be effective for Neurofibromatosis Type 1 Cutaneous Neurofibromas?

Research has shown that the 1064nm Nd:YAG laser, one of the treatments in this trial, holds promise for treating skin tumors. One study found that it completely removed nearly 40% of treated moles and almost completely cleared about 70% of cases. Another treatment option in this trial, the 755nm Alexandrite laser, effectively targets pigmented spots and surface-level skin cancers, significantly reducing skin lesion scores. Asclera, a polidocanol injection included in this trial, achieved total tumor disappearance in 95% of cases. Kybella, another treatment under study, is commonly used to reduce chin fat and has shown improvements in fat reduction, though its use for tumors is less documented. Overall, these treatments have demonstrated varying levels of effectiveness for skin-related conditions. ^{6 7 8 9 10}

Who Is on the Research Team?

Richard R Anderson, MD

Principal Investigator

Massachusetts General Hospital

Are You a Good Fit for This Trial?

This trial is for individuals with Neurofibromatosis Type 1 who have skin tumors known as Cutaneous Neurofibromas. Participants should be willing to undergo multiple treatment sessions and allow for a control site on their body to remain untreated for comparison.

Inclusion Criteria

I have at least 6 matching skin growths on my trunk, arms, or legs, each 2-8mm in size.

I have been diagnosed with NF1 either through genetic testing or because I have symptoms like skin spots, tumors, or freckles.

I am seeking treatment for cutaneous neurofibromatosis.

Exclusion Criteria

Actively tanning during the study

Known allergy to deoxycholic acid or polidocanol

Pregnant women

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive one of four FDA-approved treatments for Neurofibromatosis Type 1 Cutaneous Neurofibromas, with three treatment visits. If minimal improvement is observed, crossover treatment with the most effective option is offered, followed by three additional treatment visits.

5 months

6 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with assessments at 1, 2, 5, and if applicable, 6, 7, and 10 months post-treatment.

7 months

Multiple visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• 1064nm Nd:YAG Laser
• 755nm Alexandrite Laser
• Asclera
• Kybella

Trial Overview The study tests the effectiveness of four FDA-approved treatments: two types of lasers (1064nm Nd:YAG and 755nm Alexandrite) and injections (Kybella and Asclera). Patients will receive repeated monthly treatments, with an option to switch if there's no improvement after three visits.

How Is the Trial Designed?

Group I: Treated cutaneous neurofibromas (cNFs) Active Control 1 Intervention

Injection into up to 50 cutaneous Neurofibromas (for at total maximum of 10 mL injected) with Kybella (1% deoxycholic acid).

Group II: Control cutaneous neurofibromas (cNFs) Active Control 1 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.

Who Is Running the Clinical Trial?

Massachusetts General Hospital

Lead Sponsor

Trials

3,066

Recruited

13,430,000+

Johns Hopkins University

Collaborator

Trials

2,366

Recruited

15,160,000+

Neurofibromatosis Therapeutic Acceleration Program

Collaborator

Trials

3

Recruited

600+

Published Research Related to This Trial

Nd:YAG laser therapy was successfully used to treat 94 skin lesions in 51 patients, demonstrating its effectiveness for various skin tumors, including basal-cell carcinomas and actinic keratosis.

The treatment is advantageous as it can often be completed in a single outpatient procedure, making it a convenient option for patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Use of Nd:YAG laser for treatment of basal-cell carcinomas and some premalignant conditions]. Rubisz-Brzezińska, J., Bendkowski, W.[2007]

In a study involving 10 patients with basal cell carcinoma (BCC), treatment with a pulsed 1064 nm Nd:YAG laser resulted in a 92% rate of complete histologic clearance after just one session, indicating high efficacy for this non-surgical approach.

The laser treatment was well tolerated with no significant adverse events or scarring reported, suggesting it is a safe alternative to traditional surgical methods for treating BCC on the trunk and extremities.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

1064 nm long-pulsed Nd:YAG laser treatment of basal cell carcinoma. Ortiz, AE., Anderson, RR., Avram, MM.[2015]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11136083/

Efficacy of Long-Pulsed Nd:YAG Laser for Classic Kaposi's ... Our study demonstrated both dermoscopic and clinical evidence of the rapid, effective, and safe outcomes achieved with Nd:YAG laser treatment.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3346906/

Efficacy and Safety of 1064 nm Q-switched Nd:YAG Laser ... In this study, 821 of 2,604 nevi (39.8%) were completely removed, and 1,439 of 2,064 nevi (69.7%) were almost cleared (excellent-to-good results) with a single ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT02662244

Long-pulsed 1064 nm Nd:YAG Laser Treatment of Basal ... The primary outcome data collected during the study will include: Number of tumors that showed histologic complete regression.

4. nature.com nature.com/articles/s41598-025-85401-8

YAG laser and sclerotherapy in the treatment of pyogenic ... Effective: the tumor is eliminated by 21–80%, the volume is significantly reduced, but there is still tumor residue on the skin surface.

5. mdedge.com mdedge.com/hematology-oncology/article/260725/nonmelanoma-skin-cancer/long-pulsed-1064-nm-ndyag-nonaggressive

Long-pulsed 1064 nm Nd:YAG for nonaggressive BCC ' ... At a mean of 9 months, 100% of lesions remained clear as determined by clinical observation.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10971304/

Efficacy and Safety of Q-Switched 1064/532 nm Nd:YAG ... Particularly, the Q-switched 1064/532 nm Nd:YAG laser has proved an effective and safe technique for the treatment of pigmented skin lesions as ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT02662244

Long-pulsed 1064 nm Nd:YAG Laser Treatment of Basal ... It is an FDA approved device that enables the treatment of small superficial vessels to large vascular lesions. Only the 1064 nm wavelength will be used for ...

8. cureus.com cureus.com/articles/98935-evaluation-of-the-efficacy-and-safety-profile-of-long-pulsed-1064-neodymiumyttrium-aluminum-garnet-ndyag-laser-in-hemangioma-and-vascular-malformation-in-darker-skin-types

Evaluation of the Efficacy and Safety Profile of Long-Pulsed ... Long-pulsed 1064 Nd:YAG laser proves to be an effective treatment for hemangioma and vascular malformation even in darker skin patients with its ...

9. thedermdigest.com thedermdigest.com/medical-use-of-laser-devices/

Medical Use of Laser Devices Because the 1064 nm Nd:YAG has a longer wavelength, it bypasses epidermal melanin making it safe for all skin types, including types 5 and 6, ...

10. researchgate.net researchgate.net/publication/359062961_Effectiveness_and_Safety_of_Treatment_with_NeodymiumYAG_Laser_1064_nm_in_Patients_with_Classic_and_Epidemic_Kaposi_Sarcoma

(PDF) Effectiveness and Safety of Treatment with ... There is currently no standard treatment for KS; this study evaluated the efficacy and safety of Neodymium:YAG (Nd:YAG) laser 1064 nm treatment ...