Basal Cell Nevus Syndrome
Conditions
Keywords
Basal Cell Nevus Syndrome, Gorlin's Syndrome
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.
Interventions
DRUGLevulan
Levulan application followed by Red or Blue light PDT
OTHERRed Light PDT
Aktilite™ (red lamp) after Levulan application on lesions
OTHERBlue Light PDT
Blu-U® (blue lamp) after Levulan application on lesions
Sponsors
DUSA Pharmaceuticals, Inc.
Edward Maytin, MD, PhD
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
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.
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Tumor Clearance Rate Following Red or Blue Light PDT | 6 months | The rate of clearance of existing BCC tumors will be assessed in patients with BCNS, using clinical and photographic measurements. The endpoint will be assessed for tumors in a Red light treatment field and a Blue light treatment field in each patient, and compared (bilateral intrapatient comparison). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pain During Illumination | Maximum value reported during the illumination period | Pain reported by the patient, using a 0-10 Visual Analog Scale (VAS), where 0 is no pain and 10 is maximum pain possible. |
| Patient Satisfaction Survey | 6 months | Overall patient satisfaction with the technique will be assessed using a simple survey: 6 = extremely satisfied; 5 = very satisfied; 4 = somewhat satisfied; 3 = somewhat dissatisfied; 2 = very dissatisfied; 1 = extremely dissatisfied. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Red Light / Blue Light Participants Subject will be receive red light PDT on one side of the body, and blue light PDT to the contralateral side after Levulan application | 3 |
| Total | 3 |
Baseline characteristics
| Characteristic | Red Light / Blue Light Participants |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 3 Participants |
| Age, Continuous | 42 years |
| Region of Enrollment United States | 3 Participants |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 3 | 0 / 3 |
| other Total, other adverse events | 0 / 3 | 0 / 3 |
| serious Total, serious adverse events | 0 / 3 | 0 / 3 |
Outcome results
Primary
Tumor Clearance Rate Following Red or Blue Light PDT
The rate of clearance of existing BCC tumors will be assessed in patients with BCNS, using clinical and photographic measurements. The endpoint will be assessed for tumors in a Red light treatment field and a Blue light treatment field in each patient, and compared (bilateral intrapatient comparison).
Time frame: 6 months
Population: Three (3) patients with BCNS were enrolled.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Red Light Photodynamic Therapy | Tumor Clearance Rate Following Red or Blue Light PDT | 87.3 Percentage of tumors cleared |
| Blue Light Photodynamic Therapy | Tumor Clearance Rate Following Red or Blue Light PDT | 99 Percentage of tumors cleared |
Secondary
Pain During Illumination
Pain reported by the patient, using a 0-10 Visual Analog Scale (VAS), where 0 is no pain and 10 is maximum pain possible.
Time frame: Maximum value reported during the illumination period
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Red Light Photodynamic Therapy | Pain During Illumination | 5.6 VAS score value | Standard Error 2 |
| Blue Light Photodynamic Therapy | Pain During Illumination | 3.7 VAS score value | Standard Error 1.7 |
Secondary
Patient Satisfaction Survey
Overall patient satisfaction with the technique will be assessed using a simple survey: 6 = extremely satisfied; 5 = very satisfied; 4 = somewhat satisfied; 3 = somewhat dissatisfied; 2 = very dissatisfied; 1 = extremely dissatisfied.
Time frame: 6 months
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Red Light Photodynamic Therapy | Patient Satisfaction Survey | 5.3 Scores on the rating scale |
| Blue Light Photodynamic Therapy | Patient Satisfaction Survey | 5.6 Scores on the rating scale |