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

• Sponsor: Edward Maytin, MD, PhD (Other)
• Overall Status: Completed
• CT.gov ID: NCT02157623
• Collaborator: DUSA Pharmaceuticals, Inc. (Industry)
• Enrollment: 3
• Location: 1
• Arm: 1
• Actual Duration (Months): 10
• Patients Per Site Per Month: 0.3

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Basal Cell Nevus Syndrome	Drug: Levulan; Other: Red Light PDT; Other: Blue Light PDT	N/A

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 Design

• Study Type: Interventional
• Actual Enrollment: 3 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• 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.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.
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: Feb 1, 2016
• Actual Primary Completion Date: Sep 1, 2016
• Actual Study Completion Date: Dec 1, 2016

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. 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 Outcome Measures

1. 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.

2. 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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A and Older
• Sexes Eligible for Study: All
• Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Cleveland Clinic	Cleveland	Ohio	United States	44195

Sponsors and Collaborators

• Edward Maytin, MD, PhD
• DUSA Pharmaceuticals, Inc.

Investigators

• Principal Investigator: Edward V. Maytin, MD, PhD, The Cleveland Clinic

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Edward Maytin, MD, PhD, Staff, The Cleveland Clinic
• ClinicalTrials.gov Identifier: NCT02157623
• Other Study ID Numbers: CASE 2614; NCT02258243
• First Posted: Jun 6, 2014
• Last Update Posted: Jul 30, 2020
• Last Verified: Jul 1, 2020

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Keywords provided by Edward Maytin, MD, PhD, Staff, The Cleveland Clinic: Basal Cell Nevus Syndrome; Gorlin's Syndrome
• Additional relevant MeSH terms: Nevus; Nevus, Pigmented; Basal Cell Nevus Syndrome; Syndrome; Aminolevulinic Acid

Study Results

Participant Flow

Arm/Group Title	Red Light/Blue Light Participants
Arm/Group Description	Subjects will receive Red Light PDT on one side of the body, and Blue Light PDT to the contralateral side.
Period Title: Overall Study
STARTED	3
COMPLETED	3
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Red Light / Blue Light Participants
Arm/Group Description	Subject will be receive red light PDT on one side of the body, and blue light PDT to the contralateral side after Levulan application
Overall Participants	3
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	3 100%
>=65 years	0 0%
Age (years) [Mean (Full Range) ]
Mean (Full Range) [years]	42
Sex: Female, Male (Count of Participants)
Female	1 33.3%
Male	2 66.7%
Region of Enrollment (Count of Participants)
United States	3 100%

Outcome Measures

1. Primary Outcome

Title	Tumor Clearance Rate Following Red or Blue Light PDT
Description	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

Outcome Measure Data

Analysis Population Description

Three (3) patients with BCNS were enrolled.

Arm/Group Title	Red Light Photodynamic Therapy	Blue Light Photodynamic Therapy
Arm/Group Description	One side of patient received red light PDT	The other side of the patient received blue light PDT
Measure Participants	3	3
Measure Tumors	76	65
Mean (Full Range) [Percentage of tumors cleared]	87.3	99

2. Secondary Outcome

Title	Pain During Illumination
Description	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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Pain During Red Light	Pain During Blue Light
Arm/Group Description	Pain during red light illumination (on a 0-10 visual analog scale)	Pain during blue light illumination (on a 0-10 visual analog scale)
Measure Participants	3	3
Mean (Standard Error) [VAS score value]	5.6 (2.0)	3.7 (1.7)

3. Secondary Outcome

Title	Patient Satisfaction Survey
Description	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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Red Light Treatment	Blue Light Treatment
Arm/Group Description	RATING SCALE: 6 = extremely satisfied; 5 = very satisfied; 4 = somewhat satisfied; 3 = somewhat dissatisfied; 2 = very dissatisfied; 1 = extremely dissatisfied.	1 to 6 satisfaction scale
Measure Participants	3	3
Mean (Full Range) [Scores on the rating scale]	5.3	5.6

Adverse Events

Arm/Group Title	Red Light Photodynamic Therapy		Blue Light Photodynamic Therapy
Arm/Group Description	Subject will be randomized to right or left side and assigned side will be treated with red light PDT after Levulan application Levulan: Levulan application followed by Red or Blue light PDT on randomized treatment fields Red Light Photodynamic Therapy: Aktilite™ (red lamp) after Levulan application on randomized treatment field		Subject will be randomized to right or left side and assigned side will be treated with blue light PDT after Levulan application Levulan: Levulan application followed by Red or Blue light PDT on randomized treatment fields Blue Light Photodynamic Therapy: Blu-U® (blue lamp) after Levulan application on randomized treatment field
All Cause Mortality
	Red Light Photodynamic Therapy		Blue Light Photodynamic Therapy
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/3 (0%)		0/3 (0%)
Serious Adverse Events
	Red Light Photodynamic Therapy		Blue Light Photodynamic Therapy
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/3 (0%)		0/3 (0%)
Other (Not Including Serious) Adverse Events
	Red Light Photodynamic Therapy		Blue Light Photodynamic Therapy
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/3 (0%)		0/3 (0%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.