Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II

Brief Title

Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II

Official Title

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

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.
    

Study Phase

Phase 2

Study Type

Interventional

Primary Outcome

Time to disease progression

Secondary Outcome

 Tumor growth rate

Condition

NEUROFIBROMATOSIS 1

Intervention

aminolevulinic acid

Study Arms / Comparison Groups

 PDT Treatment

Description:  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).

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Status

Drug

Estimated Enrollment

30

Start Date

August 2016

Completion Date

August 2023

Primary Completion Date

August 2022

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.
      

Gender

All

Ages

14 Years - 30 Years

Accepts Healthy Volunteers

No

Contacts

Harry T Whelan, MD, 414-266-7540, [email protected]

Location Countries

United States

Location Countries

United States

NCT ID

NCT02728388

Organization ID

PRO00026795

Responsible Party

Sponsor-Investigator

Study Sponsor

Harry T Whelan, MD

Study Sponsor

Harry T Whelan, MD, Principal Investigator, Medical College of Wisconsin

Verification Date

November 2021