Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II

NCT ID: NCT02728388

Last Updated: 2025-05-08

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-08-31
• Study Completion Date: 2025-12-31

Brief 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.

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.

Conditions

NEUROFIBROMATOSIS 1

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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).

Group Type: EXPERIMENTAL

aminolevulinic acid

Intervention Type: DRUG

Drug: Levulan Kerastick

Drug: Topical placebo

Device: Omnilux Revive

Procedure: Photodynamic therapy

Other: Tumor growth rate measurements

Interventions

aminolevulinic acid

Drug: Levulan Kerastick

Drug: Topical placebo

Device: Omnilux Revive

Procedure: Photodynamic therapy

Other: Tumor growth rate measurements

Intervention Type: DRUG

Other Intervention Names

Photodynamic therapy

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.

• Minimum Eligible Age: 14 Years
• Maximum Eligible Age: 30 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Donald Basel

OTHER

Sponsor Role: lead

Responsible Party

Donald Basel

Professor

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Donald G Basel, MD

Role: STUDY_DIRECTOR

Medical College of Wisconsin

Locations

Donald G Basel

Milwaukee, Wisconsin, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Donald G Basel, MD

Role: CONTACT

dbasel@mcw.edu

414-266-4921

Paula Engelking, BS, CRC

Role: CONTACT

pengelki@mcw.edu

414-266-3289

Facility Contacts

Donald G Basel, MD

Role: primary

dbasel@mcw.edu

4142664921

Paula Engelking

Role: backup

pengelki@mcw.edu

4142663289

Other Identifiers

PRO00026795

Identifier Type: -

Identifier Source: org_study_id