Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II

NEUROFIBROMATOSIS 1 Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02728388
• Other study ID #: PRO00026795
• Status: Recruiting
• Phase: Phase 2
• Start date: August 2016
• Est. completion date: December 2025

Study information

• Verified date: March 2024
• Source: Medical College of Wisconsin
• Contact: Donald G Basel, MD
• Phone: 414-266-4921
• Email: dbasel[@]mcw.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Description:

Neurofibromas contain a large quantity of fibrous matter, and it is not anticipated that significant reductions in tumor size can be achieved in large, long established, tumors. Cutaneous neurofibromas, which do not usually become apparent until puberty, continue to increase in size and number throughout adulthood. The psychosocial burden of these disfiguring tumors is significant, and the targeted age group (14-30) is at a life period associated with an acceleration in tumor growth. It is for these reasons that the investigators hope to affect the growth rate of less established tumors, in order to prevent or lessen this burden as the patient progresses further into adulthood.

Therefore, the investigators wish to determine the time to disease progression (defined as 50% growth in size over baseline) for benign neurofibromas treated with PDT in patients with neurofibromatosis type 1 (NF1) in subjects aged 14-30.

The treatment will consist of choosing several neurofibromas of similar size, and applying a topical drug called Levulan, or just the topical application alone (a placebo). Within 24 hours of drug treatment, the neurofibromas will be illuminated with red light (both Levulan and placebo). The Levulan is what is known as a photosensitizer, and will be activated by the red light to potentially kill some of the tumor cells. Approximately every 6 months after, for three years, the tumors will be measured by digital photography and ultrasound to see if they are growing more slowly than the ones with the placebo application alone.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: December 2025
• Est. primary completion date: December 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 14 Years to 30 Years

Eligibility

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.

Related Conditions & MeSH terms

• Neurofibroma
• Neurofibromatoses
• Neurofibromatosis 1

Intervention

Drug:
• aminolevulinic acid
Drug: Levulan Kerastick Drug: Topical placebo Device: Omnilux Revive Procedure: Photodynamic therapy Other: Tumor growth rate measurements

Locations

Country	Name	City	State
United States	Donald G Basel	Milwaukee	Wisconsin

Sponsors (1)

Lead Sponsor	Collaborator
Donald Basel

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Time to disease progression	The time it takes for 50% growth in tumor size over baseline measurements.	3 years
Secondary	Tumor growth rate	Measure tumor size for control and treatment tumors in order to gain insights into tumor growth rates.	3 years