Blue vs Red Light During Levulan Based Photodynamic Therapy in Patients With Basal Cell Nevus Syndrome

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

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.

Study Overview

• Status: Completed
• Conditions: Basal Cell Nevus Syndrome
• Intervention / Treatment: Drug: Levulan; Other: Red Light PDT; Other: Blue Light PDT

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.

• Study Type: Interventional
• Enrollment (Actual): 3
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Red Light PDT and Blue Light PDT; The tumor clearance with one side treated with Levulan and Red light PDT, and the contralateral side treated with Blue light PDT.

Drug: Levulan; Levulan application followed by Red or Blue light PDT; Other Names: ALA; Levulan™; Levulan® Kerastick® for Topical Solution; 5-aminolevulinic acid; aminolevulinic acid HCL; Other: Red Light PDT; Aktilite™ (red lamp) after Levulan application on lesions; Other Names: PDT; Aktilite™; Red Lamp; Red Light; Other: Blue Light PDT; Blu-U® (blue lamp) after Levulan application on lesions; Other Names: PDT; Blue Light; Blu-U; Blue Lamp

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Maximum value reported during the illumination period
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.	6 months

Collaborators and Investigators

• Sponsor: Edward Maytin, MD, PhD
• Collaborators: DUSA Pharmaceuticals, Inc.
• Investigators: Principal Investigator: Edward V. Maytin, MD, PhD, The Cleveland Clinic

Study record dates

Study Major Dates

• Study Start (Actual): February 1, 2016
• Primary Completion (Actual): September 1, 2016
• Study Completion (Actual): December 1, 2016

Study Registration Dates

• First Submitted: June 2, 2014
• First Submitted That Met QC Criteria: June 4, 2014
• First Posted (Estimate): June 6, 2014

Study Record Updates

• Last Update Posted (Actual): July 30, 2020
• Last Update Submitted That Met QC Criteria: July 15, 2020
• Last Verified: July 1, 2020

More Information

Terms related to this study

• Keywords: Basal Cell Nevus Syndrome; Gorlin's Syndrome
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms by Histologic Type; Neoplasms; Carcinoma; Neoplasms, Glandular and Epithelial; Disease; Cysts; Congenital Abnormalities; Genetic Diseases, Inborn; Musculoskeletal Diseases; Stomatognathic Diseases; Bone Diseases; Neoplastic Syndromes, Hereditary; Nevi and Melanomas; Jaw Diseases; Abnormalities, Multiple; Bone Diseases, Developmental; Odontogenic Cysts; Jaw Cysts; Bone Cysts; Carcinoma, Basal Cell; Neoplasms, Basal Cell; Syndrome; Nevus; Basal Cell Nevus Syndrome; Nevus, Pigmented; Photosensitizing Agents; Dermatologic Agents; Aminolevulinic Acid
• Other Study ID Numbers: CASE 2614

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided