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Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02728388
Recruitment Status : Recruiting
First Posted : April 5, 2016
Last Update Posted : January 4, 2023
Information provided by (Responsible Party):
Harry T Whelan, MD, Medical College of Wisconsin

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.

Condition or disease Intervention/treatment Phase
NEUROFIBROMATOSIS 1 Drug: aminolevulinic acid Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Topical Photodynamic Therapy (PDT) With Levulan® Kerastick® for Benign Dermal Neurofibromas Phase II
Study Start Date : August 2016
Estimated Primary Completion Date : December 2025
Estimated Study Completion Date : December 2025

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 Name: Photodynamic therapy

Primary Outcome Measures :
  1. Time to disease progression [ Time Frame: 3 years ]
    The time it takes for 50% growth in tumor size over baseline measurements.

Secondary Outcome Measures :
  1. Tumor growth rate [ Time Frame: 3 years ]
    Measure tumor size for control and treatment tumors in order to gain insights into tumor growth rates.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   14 Years to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02728388

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Contact: Harry T Whelan, MD 414-266-7540
Contact: Marsha Malloy, RN, BSN, MBA 414-955-0704

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United States, Wisconsin
The Medical College of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Harry T Whelan, MD    414-266-7540   
Sponsors and Collaborators
Harry T Whelan, MD
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Principal Investigator: Harry T Whelan, MD Medical College of Wisconsin
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Responsible Party: Harry T Whelan, MD, Bleser Professor of Neurology, Medical College of Wisconsin Identifier: NCT02728388    
Other Study ID Numbers: PRO00026795
First Posted: April 5, 2016    Key Record Dates
Last Update Posted: January 4, 2023
Last Verified: January 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Harry T Whelan, MD, Medical College of Wisconsin:
Neurofibromatosis Type 1
Additional relevant MeSH terms:
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Neurofibromatosis 1
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Neuromuscular Diseases
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Aminolevulinic Acid
Photosensitizing Agents
Dermatologic Agents