Clinical trial NCT02157623: Blue vs Red Light During Levulan Based Photodynamic Therapy in Patients With Basal Cell Nevus Syndrome
Official title: Pilot Trial Comparing Two Different Wavelengths of Light (Blue Versus Red) During Levulan™-Based Photodynamic Therapy of Basal Cell Carcinoma in Patients With Basal Cell Nevus Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Basal Cell Nevus Syndrome
Keywords: Basal Cell Nevus Syndrome
Gorlin's Syndrome
Lead sponsor: The Cleveland Clinic
Sponsor class: OTHER
Collaborators: name: DUSA Pharmaceuticals, Inc.
First posted: 2014-06-06
Enrollment: 3
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: All patients received a different treatment to each half of their body. First Levulan with red light PDT (to tumors on one side of the body), and then Levulan with blue light PDT (to tumors on the other side of the body), at six treatment visits.
Primary purpose: TREATMENT

Brief summary: The investigators will be testing whether aminolevulinate-based (Levulan™) Photodynamic Therapy (PDT) shows effectiveness in the treatment and prevention of cutaneous basal cell carcinoma (BCC) in Basal Cell Nevus Syndrome (BCNS) patients. Levulan™ PDT is an FDA-approved method widely used currently for squamous precancers of the skin. The investigators hypothesize that PDT will provide exceptional benefit in the BCNS population because PDT is nonmutagenic, nonscarring, and can be safely repeated many times. Additionally, the study will investigate whether there are any differences in tumor clearance between the Blu-U® (blue lamp) and Aktilite™(red lamp) therapies.

Detailed description: This is a pilot intra-patient comparative study to evaluate the ability of cyclic Photodynamic Therapy (PDT) using Red (635 nm) or Blue (400 nm) light with 5-Aminolevulinic acid, to eliminate BCC skin cancers that occur in patients with Basal Cell Nevus Syndrome (Gorlin-Goltz Syndrome).

Patients will receive 3 cycles of Red light and Blue light PDT treatments, for a total of 6 treatments, over a 4 month period. Cycles will be spaced 2 months apart. Each cycle consists of a double course of PDT treatment with treatments spaced one week apart. Every subject will be treated with two light sources, blue and red, according to randomized assignments made to left side or right side of the body. There will be a final assessment visit at month 6.

Eligibility criteria: Inclusion Criteria:

* Men, women, and children of any age or ethnic group who meet eligibility
* Patients under 18 years of age must be accompanied and co-consented, by a parent or legal guardian.
* Patients must have a diagnosis of Basal Cell Nevus Syndrome (BCNS)
* 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 PTCH1 gene mutation
* Major criteria are:

  * (1) BCC prior to age 20 years, or excessive number of BCCs out of proportion to prior sun exposure and skin type
  * (2) keratocyst of the jaw prior to age 20
  * (3) palmar or plantar pitting
  * (4) lamellar calcification of the falx cerebri
  * (5) medulloblastoma
  * (6) first degree relative with BCNS
* Minor criteria are:

  * (1) rib anomalies, or other specific skeletal malformations including kyphoscoliosis and short 4th metacarpals
  * (2) macrocephaly
  * (3) cleft/lip or palate;
  * (4) fibroma of the heart or ovary
  * (5) ocular abnormalities
  * (6) other rare abnormalities
* At least two BCC tumors, preferably more, located in different body regions or located greater than 10 cm apart in locations that can be reproducibly separated into red and blue illumination fields
* Female subjects are not pregnant or nursing or planning to become pregnant during the study
* Subjects must have the ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria:

* Pregnant, planning on getting pregnant or nursing
* Currently participating in another clinical trial
* Using any topical treatment for their BCC tumors, unless discontinued at least 1 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 a history of a photosensitivity disease, including porphyria cutanea tarda

Primary outcomes:
measure: Tumor Clearance Rate Following Red or Blue Light PDT
timeFrame: 6 months
Secondary outcomes:
measure: Pain During Illumination
timeFrame: Maximum value reported during the illumination period
measure: Patient Satisfaction Survey
timeFrame: 6 months
Other outcomes:
Locations:
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
Study officials:
affiliation: The Cleveland Clinic
name: Edward V. Maytin, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: