Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II

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

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.

Study Overview

• Status: Recruiting
• Conditions: NEUROFIBROMATOSIS 1
• Intervention / Treatment: Drug: aminolevulinic acid

Detailed 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.

• Study Type: Interventional
• Enrollment (Estimated): 30
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Donald G Basel, MD; Phone Number: 414-266-4921; Email: dbasel@mcw.edu
• Study Contact Backup: Name: Paula Engelking, BS, CRC; Phone Number: 414-266-3289; Email: pengelki@mcw.edu

Study Locations

• United States
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Recruiting
      • Donald G Basel
      • Contact: Paula Engelking; Phone Number: 4142663289; Email: pengelki@mcw.edu
      • Contact: Donald G Basel, MD; Phone Number: 4142664921; Email: dbasel@mcw.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 30 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: PDT Treatment; 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).

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to disease progression	The time it takes for 50% growth in tumor size over baseline measurements.	3 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Tumor growth rate	Measure tumor size for control and treatment tumors in order to gain insights into tumor growth rates.	3 years

Collaborators and Investigators

• Sponsor: Donald Basel
• Investigators: Study Director: Donald G Basel, MD, Medical College of Wisconsin

Study record dates

Study Major Dates

• Study Start (Actual): August 1, 2016
• Primary Completion (Estimated): December 1, 2025
• Study Completion (Estimated): December 1, 2025

Study Registration Dates

• First Submitted: March 25, 2016
• First Submitted That Met QC Criteria: March 30, 2016
• First Posted (Estimated): April 5, 2016

Study Record Updates

• Last Update Posted (Actual): May 8, 2025
• Last Update Submitted That Met QC Criteria: May 5, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Keywords: Neurofibromatosis Type 1; NF1; Neurofibroma
• Additional Relevant MeSH Terms: Nervous System Diseases; Neoplasms; Neuromuscular Diseases; Genetic Diseases, Inborn; Peripheral Nervous System Diseases; Neoplasms by Histologic Type; Neurodegenerative Diseases; Neoplasms, Nerve Tissue; Nervous System Neoplasms; Heredodegenerative Disorders, Nervous System; Nerve Sheath Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Peripheral Nervous System Neoplasms; Neurofibromatoses; Neurofibromatosis 1; Neurofibroma; Dermatologic Agents; Photosensitizing Agents; Aminolevulinic Acid
• Other Study ID Numbers: PRO00026795

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No