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

Trial Summary

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 data supports the effectiveness of the treatment for neurofibromatosis using photodynamic therapy with aminolevulinic acid?

Photodynamic therapy using aminolevulinic acid has shown promise in treating skin conditions like actinic keratoses and basal cell carcinoma by effectively targeting and reducing tumors. In a clinical trial for neurofibromatosis, this treatment specifically targeted neurofibromas, suggesting it may offer a less invasive option compared to surgery. ^{1 2 3 4 5}

Is photodynamic therapy using aminolevulinic acid safe for humans?

Photodynamic therapy using aminolevulinic acid (ALA) is generally considered safe for humans, with studies showing it is well tolerated and has minor side effects when used for skin conditions like actinic keratoses and basal cell carcinoma. ^{1 2 3 4 5}

How is the treatment for neurofibromatosis using photodynamic therapy with aminolevulinic acid unique?

Photodynamic therapy with aminolevulinic acid (ALA) is unique because it uses a light-activated process to target and treat skin conditions, which is different from traditional treatments that may involve surgery or medication. ALA is applied topically and activated by light to destroy abnormal cells, offering a non-invasive option with good cosmetic results. ^{2 3 4 5 6}

What is the purpose of this trial?

The investigators wish to determine the time to disease progression for benign neurofibromas treated with Levulan Kerastick topical photosensitizer and red light photodynamic therapy (PDT) in patients with neurofibromatosis type 1 (NF1). The investigators also wish to measure tumor size for control and treatment tumors in order to gain insights into tumor growth rates.

Research Team

Donald G Basel, MD

Principal Investigator

Medical College of Wisconsin

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

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]

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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Photodynamic Therapy for Benign Cutaneous Neurofibromas Using Aminolevulinic Acid Topical Application and 633 nm Red Light Illumination. [2021]

2. United States 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. [2013]

3. Canada pubmed.ncbi.nlm.nih.gov

Aminolevulinic acid (Levulan) in photodynamic therapy of actinic keratoses. [2017]

4. United States pubmed.ncbi.nlm.nih.gov

Photodynamic therapy of actinic keratoses with topical aminolevulinic acid hydrochloride and fluorescent blue light. [2013]

5. Netherlands pubmed.ncbi.nlm.nih.gov

Topical 5-aminolevulinic acid-mediated photodynamic therapy for basal cell carcinoma. [2021]

6. England 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. [2016]