Clinical Trial: Photodynamic Therapy (PDT) for Benign Dermal Neurofibromas (NF1)

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Photodynamic Therapy for Benign Dermal Neurofibromas Using Levulan Kerastick For Topical Solution, Plus Illumination With Red Light

Brief Summary:

GENERAL OBJECTIVE The general objective is to assess the safety and efficacy of photodynamic therapy (PDT) in the treatment of neurofibromatosis 1 (NF1) tumors in the skin.

SPECIFIC OBJECTIVE This is a light dose escalation pilot study to determine the safety and efficacy of PDT using 5-aminolevulinic acid (ALA) and 630 nm light in the treatment of benign dermal neurofibromas.

Specifically, the primary goal of the current study is to determine the maximum tolerable light doses that can be administered to subjects undergoing topical photoillumination photodynamic therapy with standard application of Levulan Kerastick (ALA) for Topical Solution.

Detailed Summary:

STUDY DESIGN This protocol is a Phase I light dose escalation pilot study to determine the safety and, secondarily, the efficacy of PDT using Levulan and 630 nm light in the treatment of benign dermal neurofibromas. This protocol represents the first two parts of a planned three part study including both pediatric and adult subjects. Part 1 will consist of studying the penetration and uptake of the PS in neurofibromas that are scheduled for excision. These tumors will be excised for therapeutic reasons unrelated to this study, and so this study will place no further burden on the subject other than a 3-24 hr incubation of the Levulan on the tumor prior to excision. The primary hypothesis to be tested is whether Levulan will accumulate, and be converted to PpIX, by the tumor tissue more than by the surrounding normal tissue. Secondary hypotheses are that tumors incubated with Levulan will show greater fluorescence than untreated tumors and tumors incubated with vehicle only (placebo application).

As the Institutional Review Boards involved generally desire pilot data on adult populations first, we will with then proceed with the adult clinical trial portion of this protocol as part 2. Part 2 will use the optimum incubation time, if one has been identified in part 1, and add a dose escalation study of the amount of red light used to activate the Levulan. Part 3, with pediatric subjects, will commence at a future date, pending review of the initial adult study results.

Sponsor: Harry T Whelan, MD

Current Primary Outcome:

• Part 1: Photosensitizer uptake [ Time Frame: 24 hours ]
  Measurement of the ratio of the mean fluorescence intensity in excised, sectioned tumors, to the fluorescence in immediately adjacent tissue, as determined by fluorescence microscopy
• Part 2: Maximum tolerated dose (MTD) [ Time Frame: 48 hours ]
  Maximum tolerated dose (MTD) as determined by dose limiting toxicity.

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Part 1: Optimal Occlusion time [ Time Frame: 24 hours ]
  An optimal occlusion time may be apparent from the results of the three time points. If no optimal occlusion time is seen, any occlusion time from 3-24 hours may be chosen for part 2. This secondary outcome measure is not critical to continuing the study, but may be useful in guiding treatment protocols
• Part 2: Efficacy - lesion size [ Time Frame: 1 year ]
  Decrease in lesion size as measured by ruler (directly or using a digital photograph). The area of the lesion will be estimated by the nurse or dermatologist by measuring the radius if circular, or by length and width if not. Irregular areas can be measured using the application of digital imaging programs such as ImageJ to the digital photograph.
• Part 2: Cosmetic Improvement [ Time Frame: 1 year ]
  Potential cosmetic improvement using subject satisfaction scale.
• Part 2: Pain Reduction [ Time Frame: 1 year ]
  Potential pain reduction, as measured by standard visual analog 1-10 scale.

Original Secondary Outcome: Same as current

Information By: Medical College of Wisconsin

Dates:
Date Received: September 5, 2012
Date Started: November 2011
Date Completion: November 2017
Last Updated: October 13, 2016
Last Verified: October 2016