Photodynamic Therapy Using Blue Light or Red Light in Treating Basal Cell Carcinoma in Patients With Basal Cell Nevus Syndrome

PRIMARY OBJECTIVES:

I. To determine whether cyclic photodynamic therapy (PDT) treatment is effective in curing
existing basal cell carcinoma (BCC) tumors in patients with basal cell nevus syndrome
(BCNS), and whether red light or blue light is more effective in this regard.

SECONDARY OBJECTIVES:

I. To assess the tolerability (pain during treatment) of red light versus blue light in this
patient population.

II. To assess patient satisfaction with the technique.

OUTLINE: Patients are randomized to 1 of 2 treatment arms.

ARM I: Patients receive aminolevulinic acid hydrochloride topically. Beginning 4 hours
later, patients undergo photodynamic therapy using blue light on the left side of the body
and red light on the right side of the body on days 1 and 8. Treatment repeats every 2
months for 3 courses in the absence of disease progression or unacceptable toxicity.

ARM II: Patients receive aminolevulinic acid hydrochloride topically. Beginning 4 hours
later, patients undergo photodynamic therapy using blue light on the right side of the body
and red light on the left side of the body on days 1 and 8. Treatment repeats every 2 months
for 3 courses in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up for 2 years.

Inclusion Criteria:

- A diagnosis of basal cell nevus syndrome (BCNS) as defined in the Consensus Statement
(Bree et al, American Journal of Medical Genetics [Am J Med Genet] Part A
155:2091-2097)

- Major criteria are:

- BCC prior to age 20 years, or excessive number of BCCs out of proportion to
prior sun exposure and skin type

- Keratocyst of the jaw prior to age 20

- Palmar or plantar pitting

- Lamellar calcification of the falx cerebri

- Medulloblastoma

- First degree relative with BCNS

- Minor criteria are:

- Rib anomalies, or other specific skeletal malformations including
kyphoscoliosis and short 4th metacarpals

- Macrocephaly

- Cleft/lip or palate

- Fibroma of the heart or ovary

- Ocular abnormalities

- Other rare abnormalities listed in the article by Bree et al

- For diagnosis of BCNS, the patient must have either 2 major criteria, one major
and two minor criteria, or one major criterion plus molecular confirmation of a
patched 1 (PTCH1) gene mutation

- At least two BCC tumors, preferably more; these tumors must be located in different
body regions or alternatively, located > 10 cm apart at sites that can be
reproducibly separated into red and blue illumination fields

- Female subjects must not become pregnant during the study; women of child-bearing
potential must agree to use adequate contraception (double barrier method of birth
control or abstinence) prior to study entry, and for the duration of study
participation; should a woman become pregnant or suspect that she is pregnant while
she is participating in this study, she should inform the treating physician
immediately

- Subjects must be able to understand and be willing to sign a written informed consent
document

Exclusion Criteria:

- Pregnant or nursing

- Currently participating in another clinical trial

- Using any topical treatment for their BCC tumors, unless discontinued at least one
month prior.

- Currently being treated for other cancers with medical or radiation therapy

- Patients with a known hypersensitivity to 5-aminolevulinic acid or any component of
the study material

- Patients with history of a photosensitivity disease, such as porphyria cutanea tarda

- General or specific aspects of the patient's condition render the patient
unacceptable for this treatment in the judgment of the investigator