Photodynamic Therapy for Neurofibromatosis

Recruiting at 1 trial location

Overseen By Paula Engelking, BS, CRC

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Donald Basel

Must not be taking: Chemotherapy

Disqualifiers: Pregnancy, Porphyria, Photosensitivity, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 7

Duration: 1 day

30 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment for individuals with neurofibromatosis type 1 (NF1), a condition that causes benign tumors on the nerves. The treatment combines a topical drug, Aminolevulinic Acid (Levulan or ALA), with red light therapy to determine if it can slow tumor growth. Participants will have both treated and untreated tumors for comparison. Ideal candidates for the trial are those diagnosed with NF1 who have small, surface-level tumors on their skin, specifically on the trunk or limbs. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group, allowing participants to contribute to significant advancements in NF1 treatment.

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, if you have had chemotherapy within 6 weeks of the proposed treatment, you may not be eligible to participate.

What prior data suggests that this photodynamic therapy is safe for treating neurofibromas?

Research has shown that using aminolevulinic acid in light-based therapy is generally safe and well-tolerated for treating skin conditions. Studies have found that this treatment provides good cosmetic results with only minor side effects, such as temporary skin redness or swelling.

In past studies, patients reported positive experiences with aminolevulinic acid in this therapy, making it a promising option for people with neurofibromatosis type 1 (NF1). While the treatment remains under study, current evidence suggests it is a safe way to manage non-cancerous skin tumors. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for neurofibromatosis focus on symptom management and surgical removal of tumors. Unlike these options, photodynamic therapy with Aminolevulinic Acid offers a non-invasive approach that uses a combination of a topical solution and light to target neurofibromas. Researchers are excited about this treatment because it specifically activates the body's own processes to destroy tumor cells without the need for surgery. This method could potentially lead to fewer side effects and a more precise way to manage the growths associated with neurofibromatosis.

What evidence suggests that photodynamic therapy is effective for neurofibromatosis?

Research shows that photodynamic therapy (PDT) using aminolevulinic acid can effectively target and destroy tumor cells. In this trial, participants will receive either a placebo or Levulan Kerastick (aminolevulinic acid) topical application on matched sets of neurofibromas, serving as their own control. Studies have shown that this treatment completely removed tumor cells in people with neurofibromatosis type 1 (NF1), a condition that causes non-cancerous tumors to grow on nerves. This therapy specifically targets these tumors, known as neurofibromas, without harming normal tissue. Additionally, a case study demonstrated successful treatment of skin tumors using a similar PDT method with aminolevulinic acid. These findings suggest that PDT could be a promising option for managing non-cancerous tumors in NF1 patients. ^{1 2 3 6 7}

Who Is on the Research Team?

Donald G Basel, MD

Principal Investigator

Medical College of Wisconsin

Are You a Good Fit for This Trial?

This trial is for individuals aged 14 or older with neurofibromatosis type 1 (NF1), specifically those with superficial skin tumors on the trunk or limbs. Participants must not have other cancers, be pregnant, have a life expectancy under three years, or be sensitive to light used in therapy. They should not have had recent chemotherapy and must agree to follow-up requirements.

Inclusion Criteria

I have been diagnosed with NF1 according to specific guidelines.

I am 14 years old or older.

I am willing and able to follow the study's follow-up requirements.

Exclusion Criteria

I haven't had chemotherapy in the last 6 weeks.

I have been diagnosed with porphyria.

Life expectancy less than 3 years

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Levulan Kerastick topical photosensitizer and red light photodynamic therapy (PDT) for benign neurofibromas

1 day

1 visit (in-person)

Follow-up

Participants are monitored for tumor growth rate and time to disease progression using digital photography and ultrasound

3 years

Approximately every 6 months

What Are the Treatments Tested in This Trial?

Interventions

• Aminolevulinic Acid

Trial Overview

The study tests Levulan Kerastick topical photosensitizer combined with red light photodynamic therapy (PDT) on benign skin tumors in NF1 patients. It aims to observe how long before the treated tumors progress compared to untreated ones and understand tumor growth rates.

How Is the Trial Designed?

Group I: PDT Treatment Experimental Treatment 1 Intervention

Each subject will have either placebo or Levulan Kerastick topical application applied to matched sets of neurofibromas. Each subject will have both sets, in order to serve as his/her own control subject. 16 to 24 hours post study drug treatment, both sets of neurofibromas, Levulan and placebo treated, will be irradiated with red light (630 nm) from an Omnilux Revive light device at 100 mW/cm2 for 1000 seconds (16.7 minutes).

Aminolevulinic Acid is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Levulan Kerastick for:

• Actinic keratosis
• Moderate to severe acne vulgaris

Approved in European Union as Levulan for:

• Actinic keratosis
• Moderate to severe acne vulgaris

Approved in Canada as Levulan Kerastick for:

• Actinic keratosis

Who Is Running the Clinical Trial?

Donald Basel

Lead Sponsor

Trials

1

Recruited

30+

Harry T Whelan, MD

Lead Sponsor

Trials

4

Recruited

60+

Published Research Related to This Trial

Photodynamic therapy using Levulan Kerastick and blue light was effective in treating a 73-year-old man with nevoid basal cell carcinoma syndrome, leading to a reduction in the number and size of his basal cell carcinomas.

The treatment not only improved the appearance of surgical scars but also decreased the rate of new tumor development, suggesting a comprehensive benefit of this therapy for patients with multiple skin cancers.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Broad area photodynamic therapy for treatment of multiple basal cell carcinomas in a patient with nevoid basal cell carcinoma syndrome. Chapas, AM., Gilchrest, BA.[2013]

Photodynamic therapy (PDT) using aminolevulinic acid (ALA) has become a highly effective treatment for actinic keratoses, with the FDA approving Levulan Kerastick as the preferred topical solution.

Levulan is well tolerated by patients and provides excellent cosmetic outcomes with only minor side effects, making it a safe option for treating skin tumors.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Aminolevulinic acid (Levulan) in photodynamic therapy of actinic keratoses. Lang, K., Schulte, KW., Ruzicka, T., et al.[2017]

Topical photodynamic therapy using 5-aminolevulinic acid (Levulon) was effective in treating superficial basal cell carcinoma (BCC), achieving a complete response in 77 out of 82 patients, with excellent cosmetic results.

The treatment demonstrated a high overall recurrence-free survival (RFS) rate, with 92.1% at one year and 88.3% at three years, indicating both efficacy and safety for patients undergoing this therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Topical 5-aminolevulinic acid-mediated photodynamic therapy for basal cell carcinoma. Filonenko, E., Kaprin, A., Urlova, A., et al.[2021]

Topical photodynamic therapy (PDT) using aminolevulinic acid (ALA) was shown to effectively target cutaneous neurofibromas (cNF) in a phase I trial with 27 participants, demonstrating significant fluorescence in tumors and minimal impact on surrounding normal skin.

The treatment resulted in a notable increase in apoptotic cells in ALA-treated tumors compared to vehicle-treated tumors, indicating that PDT may provide a less invasive and tissue-sparing option for managing neurofibromatosis type 1.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Photodynamic Therapy for Benign Cutaneous Neurofibromas Using Aminolevulinic Acid Topical Application and 633 nm Red Light Illumination. Quirk, B., Olasz, E., Kumar, S., et al.[2021]

In a study involving 36 patients, topical application of 20% aminolevulinic acid (ALA) followed by exposure to blue light resulted in an 88% complete clearance of actinic keratoses (AKs) after 8 weeks, demonstrating high efficacy.

The treatment was conducted in a multicenter, randomized, vehicle-controlled design, confirming that photodynamic therapy with ALA and blue light is a safe and effective option for managing multiple AKs on the face and scalp.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Photodynamic therapy of actinic keratoses with topical aminolevulinic acid hydrochloride and fluorescent blue light. Jeffes, EW., McCullough, JL., Weinstein, GD., et al.[2013]

BF-200 ALA, a new gel formulation of 5-aminolaevulinic acid, demonstrated a significantly higher complete clearance rate for actinic keratosis (AK) lesions compared to placebo (78.2% vs. 17.1%) and was also more effective than the registered methyl-5-aminolaevulinate (MAL) cream (78.2% vs. 64.2%) in a study involving 600 patients.

The study highlighted that BF-200 ALA is a well-tolerated treatment option for AK, with varying efficacy and adverse events depending on the type of light source used during photodynamic therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Photodynamic therapy with BF-200 ALA for the treatment of actinic keratosis: results of a multicentre, randomized, observer-blind phase III study in comparison with a registered methyl-5-aminolaevulinate cream and placebo. Dirschka, T., Radny, P., Dominicus, R., et al.[2016]

Citations

1.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC8219184/

Photodynamic Therapy for Benign Cutaneous ...

Results showed that in vitro PDT resulted in complete ablation of various NF1 tumor cell cultures using Photofrin, talaporfin sodium, or aminolevulinic acid ( ...

2.

clinicaltrials.gov

clinicaltrials.gov/study/NCT01682811

Phase I Photodynamic Therapy (PDT) for Benign Dermal ...

GENERAL OBJECTIVE The general objective is to assess the safety and efficacy of photodynamic therapy (PDT) in the treatment of neurofibromatosis 1 (NF1) ...

3.

withpower.com

withpower.com/trial/phase-3-neurofibromatosis-1-7-2016-b784d

Photodynamic Therapy for Neurofibromatosis

Photodynamic therapy using Levulan Kerastick and blue light was effective in treating a 73-year-old man with nevoid basal cell carcinoma syndrome, leading to a ...

4.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/33470897/

Photodynamic Therapy for Benign Cutaneous ...

Conclusions: In the first reported clinical trial of PDT for NF1, PDT targeted neurofibromas specifically, and may offer a normal tissue-sparing ...

5.

skintherapyletter.com

skintherapyletter.com/actinic-keratosis/aminolevulinic-acid-levulan/

Aminolevulinic Acid (Levulan®): Actinic Keratoses ...

The maximal response (80%) was seen in patients treated with the maximal dose of light (10 J/cm2). Non-responding lesions were re-treated at 8 ...

6.

researchgate.net

researchgate.net/profile/Harry-Whelan/publication/348643209_Photodynamic_Therapy_for_Benign_Cutaneous_Neurofibromas_Using_Aminolevulinic_Acid_Topical_Application_and_633_nm_Red_Light_Illumination/links/60e36820299bf1ea9ee51651/Photodynamic-Therapy-for-Benign-Cutaneous-Neurofibromas-Using-Aminolevulinic-Acid-Topical-Application-and-633-nm-Red-Light-Illumination.pdf

Photodynamic-Therapy-for-Benign-Cutaneous- ...

Background: Neurofibromatosis type 1 (NF1) has no current effective treatments beyond surgery. Topical photodynamic therapy (PDT) has the ...

7.

sciencedirect.com

sciencedirect.com/science/article/pii/S0022202X23019619

Target Product Profile for Cutaneous Neurofibromas ...

Topical photodynamic therapy using aminolevulinic acid (ALA) and 633 nm red light has also been studied for cNFs (Dolmans et al., 2003; Quirk et al., 2021).