Clinical trial NCT00571974: Treatment of Oral Premalignant Lesions With 5-ALA PDT
Official title: A Combined Phase I/II Single Site Study to Determine the Safety and Efficacy of Photodynamic Therapy (PDT) Utilizing 5-aminolevulinic Acid (5-ALA) and PDT in the Treatment of Premalignant Oral and/or Oropharynx Lesions.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Leukoplakia
Erythroplakia
Lead sponsor: University of Arkansas
Sponsor class: OTHER
First posted: 2007-12-12
Enrollment: 29
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: Oral leukoplakia within the mouth is a visible white patch which can develop into cancer if not treated. There is no good treatment for these lesions, apart from surgery which is associated with significant side effects and physical deformation of the treated area.

The investigators hypothesized that photodynamic therapy can be used safely and effectively to induce significant regression of oral leukoplakia.

Detailed description: This is a combined Phase I/II non-randomized prospective study designed to determine the safety and assess the clinical efficacy of PDT in the treatment of oral leukeplekia with 5-ALA and 585-nm PDL with 1.5 ms pulse time. In the first part of the study we determined the maximum tolerated dose (MTD) of the PDL radiant exposure in combination with 5-ALA. In the second phase of the study, this dose is used to treat subjects at the MTD in order to determine the efficacy of the treatment by documenting the regression of the treated lesions.

Eligibility criteria: Inclusion Criteria:

* At least one grossly visible premalignant lesion (i.e. leukoplakia or erythroplakia) in the oral cavity or oropharynx, with a confirmed diagnosis of leukoplakia with or without dysplasia,measuring ≥ 10 mm in diameter.
* Informed of alternative treatment methods including watchful waiting, laser ablation, or surgical resection.
* Eligible for long-term follow-up for at least one year and be able to tolerate biopsies.
* Subject has signed an informed consent.
* Subject is between the ages of 18 - 80 years of age.
* Male or Female
* Zubrod performance status of 0 or 1 at screening. See Appendix A

Exclusion Criteria:

* Known sensitivity to porphyrins or photoactive medications - See Appendix B
* Invasive carcinoma of the lesion as demonstrated by biopsy.
* Subjects with inherited or acquired blood clotting defects
* Women who are breast feeding, have a positive (+) urine pregnancy test, or refuse to use 2 effective means of contraception during drug exposure and up to 48 hours after.
* Subjects with porphyria
* Life expectancy less than 12 months
* Inability or unwillingness of subject to give written informed consent

Primary outcomes:
measure: Maximum Tolerated Dose
timeFrame: Day 2
measure: The Objective Response Rate is the Number of Participants With Significant Response (SR), Partial Response (PR) or No Response (NR).
timeFrame: Day 90
Secondary outcomes:
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences
state: Arkansas
Study officials:
affiliation: University of Arkansas
name: Gal Shafirstein, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Shafirstein G, Friedman A, Siegel E, Moreno M, Baumler W, Fan CY, Morehead K, Vural E, Stack BC Jr, Suen JY. Using 5-aminolevulinic acid and pulsed dye laser for photodynamic treatment of oral leukoplakia. Arch Otolaryngol Head Neck Surg. 2011 Nov;137(11):1117-23. doi: 10.1001/archoto.2011.178.