Using light therapy to treat benign tumors in patients with neurofibromatosis type 1

Topical Photodynamic Therapy (PDT) With Levulan® Kerastick® for Benign Dermal Neurofibromas Phase II

This study is testing if a light therapy treatment can help young people with neurofibromatosis type 1 shrink their benign tumors and improve their quality of life.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 14 Years to 30 Years
• Sex: All
• Sponsor: Medical College of Wisconsin (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 1 site (Milwaukee, Wisconsin)
• Trial ID: NCT02728388 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to evaluate the effectiveness of photodynamic therapy (PDT) using a topical photosensitizer called Levulan Kerastick in treating benign dermal neurofibromas in patients with neurofibromatosis type 1 (NF1). Participants aged 14-30 will receive either the Levulan treatment or a placebo, followed by red light exposure to activate the drug. The primary goal is to measure the time to disease progression, defined as a 50% increase in tumor size, and to assess tumor growth rates over a three-year period. The study focuses on less established tumors to potentially reduce the psychosocial burden associated with these disfiguring growths.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 14-30 with a diagnosis of neurofibromatosis type 1 and superficial dermal neurofibromas.

Not a fit: Patients with a life expectancy of less than three years, those who are pregnant, or individuals with certain allergies or concurrent tumor therapies may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly slow the growth of neurofibromas, improving the quality of life for patients with NF1.

How similar studies have performed: While this approach is innovative, similar studies using photodynamic therapy for other conditions have shown promise, suggesting potential for success in this context.

Eligibility criteria

Inclusion Criteria:

1. Patient is 14 years or older.
2. Diagnosed NF1, determined by American Academy of Neurology Guidelines (see Diagnosis section).
3. Tumor Location: cutaneous, trunk, or limbs only.
4. Tumor Type: superficial dermal neurofibromas ≤4mm deep.
5. Patient has provided written informed consent.
6. Patient is willing to and can comply with study follow-up requirements.
7. Absence of any other malignancy.

Exclusion Criteria:

1. Life expectancy less than 3 years.
2. Pregnancy.
3. Cutaneous photosensitivity to the wavelengths used to active PDT.
4. A diagnosis of porphyria.
5. Allergy to aminolevulinic acid or any of the topical solution vehicle components.
6. Previous chemotherapy within 6 weeks of proposed PDT.
7. Other concurrent tumor therapy.

Where this trial is running

Milwaukee, Wisconsin

• Donald G Basel — Milwaukee, Wisconsin, United States (Recruiting)

Study contacts

• Study coordinator: Donald G Basel, MD

Conditions NEUROFIBROMATOSIS 1; NF1; Neurofibroma; Neurofibromatosis Type 1