Trial Outcomes & Findings for Topical Laser-assisted Combination Chemotherapy for Basal Cell Carcinoma- a Clinical Study (NCT NCT03541252)

Results Overview

Non-blinded, clinical evaluation of local erythema, edema, flaking, crusting/scabbing, pustulation, scarring, hypo/hyperpigmentation, infection in treated areas will be performed by a physician using a FDA-approved LSR scale at Days 1, 3-5, 14, 30 and 3 months after AFL exposure. Each parameter was graded on a standardized 5-point severity scale (0-4) representing none, mild, moderate, prominent, and severe. A total composite score reflecting overall LSR severity was then calculated based on the sum of all parameters (minimum score 0- least severe; max score: 24-most severe). lower scores are better.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

20 participants

Primary outcome timeframe

Days 1, 3-5, 14, 30 and 3 months post treatment

Results posted on

2022-04-14

Participant Flow

Participant milestones

Participant milestones
Measure	Basal Cell Carcinoma Patients Patients (\>18 years) with histologically-verified superficial or nodular basal cell carcinoma (\<20 mm on the face/scalp, \<50mm on the trunk/extremities) AFL-assisted cisplatin+5-FU: Patients will receive AFL-assisted cisplatin+5-FU as a treatment for their BCC. In brief, treatment areas consisting of tumors and a 5 mm margin will undergo CO2 laser exposure followed by 60 min topical application of a marketed and commercially available IV cisplatin solution (0.1%) After removal of cisplatin, a commercially distributed 5-FU cream (5%) will be applied to the treatment area at a dose of 0.125 ml per cm2 and left under occlusion. After skin evaluations on Day 1 and 5 after treatment, the same 5-FU dose will be applied, again left under occlusion. In total, 5-FU will remain on the skin for 7 days after AFL treatment whereafter it will be washed off. An additional repeat AFL-cisplatin+5-FU treatment on Day 30 will be offered if tumors persist, based on clinical evaluation and imaging on Day 30.
Overall Study STARTED	20
Overall Study COMPLETED	19
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Basal Cell Carcinoma Patients Patients (\>18 years) with histologically-verified superficial or nodular basal cell carcinoma (\<20 mm on the face/scalp, \<50mm on the trunk/extremities) AFL-assisted cisplatin+5-FU: Patients will receive AFL-assisted cisplatin+5-FU as a treatment for their BCC. In brief, treatment areas consisting of tumors and a 5 mm margin will undergo CO2 laser exposure followed by 60 min topical application of a marketed and commercially available IV cisplatin solution (0.1%) After removal of cisplatin, a commercially distributed 5-FU cream (5%) will be applied to the treatment area at a dose of 0.125 ml per cm2 and left under occlusion. After skin evaluations on Day 1 and 5 after treatment, the same 5-FU dose will be applied, again left under occlusion. In total, 5-FU will remain on the skin for 7 days after AFL treatment whereafter it will be washed off. An additional repeat AFL-cisplatin+5-FU treatment on Day 30 will be offered if tumors persist, based on clinical evaluation and imaging on Day 30.
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Basal Cell Carcinoma Patients n=19 Participants Patients (\>18 years) with histologically-verified superficial or nodular basal cell carcinoma (\<20 mm on the face/scalp, \<50mm on the trunk/extremities) AFL-assisted cisplatin+5-FU: Patients will receive AFL-assisted cisplatin+5-FU as a treatment for their BCC. In brief, treatment areas consisting of tumors and a 5 mm margin will undergo CO2 laser exposure followed by 60 min topical application of a marketed and commercially available IV cisplatin solution (0.1%) After removal of cisplatin, a commercially distributed 5-FU cream (5%) will be applied to the treatment area at a dose of 0.125 ml per cm2 and left under occlusion. After skin evaluations on Day 1 and 5 after treatment, the same 5-FU dose will be applied, again left under occlusion. In total, 5-FU will remain on the skin for 7 days after AFL treatment whereafter it will be washed off. An additional repeat AFL-cisplatin+5-FU treatment on Day 30 will be offered if tumors persist, based on clinical evaluation and imaging on Day 30.
Age, Continuous	73 years n=19 Participants
Sex: Female, Male Female	5 Participants n=19 Participants
Sex: Female, Male Male	14 Participants n=19 Participants
Histological Tumor Subtype Nodular BCC	13 Tumors n=19 Participants
Histological Tumor Subtype Superficial BCC	6 Tumors n=19 Participants

PRIMARY outcome

Timeframe: Days 1, 3-5, 14, 30 and 3 months post treatment

Non-blinded, clinical evaluation of local erythema, edema, flaking, crusting/scabbing, pustulation, scarring, hypo/hyperpigmentation, infection in treated areas will be performed by a physician using a FDA-approved LSR scale at Days 1, 3-5, 14, 30 and 3 months after AFL exposure. Each parameter was graded on a standardized 5-point severity scale (0-4) representing none, mild, moderate, prominent, and severe. A total composite score reflecting overall LSR severity was then calculated based on the sum of all parameters (minimum score 0- least severe; max score: 24-most severe). lower scores are better.

Outcome measures

Outcome measures
Measure	Basal Cell Carcinoma Patients n=19 Participants Patients (\>18 years) with histologically-verified superficial or nodular basal cell carcinoma (\<20 mm on the face/scalp, \<50mm on the trunk/extremities) AFL-assisted cisplatin+5-FU: Patients will receive AFL-assisted cisplatin+5-FU as a treatment for their BCC. In brief, treatment areas consisting of tumors and a 5 mm margin will undergo CO2 laser exposure followed by 60 min topical application of a marketed and commercially available IV cisplatin solution (0.1%) After removal of cisplatin, a commercially distributed 5-FU cream (5%) will be applied to the treatment area at a dose of 0.125 ml per cm2 and left under occlusion. After skin evaluations on Day 1 and 5 after treatment, the same 5-FU dose will be applied, again left under occlusion. In total, 5-FU will remain on the skin for 7 days after AFL treatment whereafter it will be washed off. An additional repeat AFL-cisplatin+5-FU treatment on Day 30 will be offered if tumors persist, based on clinical evaluation and imaging on Day 30.
Change in Occurence of Local Skin Reaction (LSR) Side Effects TOTAL COMPOSITE SCORE Day 3-5 post treatment	9 score on a scale Interval 6.0 to 11.0
Change in Occurence of Local Skin Reaction (LSR) Side Effects TOTAL COMPOSITE SCORE Day 1 post treatment	7 score on a scale Interval 6.0 to 9.0
Change in Occurence of Local Skin Reaction (LSR) Side Effects TOTAL COMPOSITE SCORE Day 30 post treatment	4 score on a scale Interval 2.0 to 6.0
Change in Occurence of Local Skin Reaction (LSR) Side Effects TOTAL COMPOSITE SCORE Month 3 post treatment	2 score on a scale Interval 1.0 to 3.0

SECONDARY outcome

Timeframe: Day 30 and Month 3 post treatment

Tumor clearance (yes or no) will be evaluated clinically at Day 30 and Month 3. If residual tumor is identified at 3 months follow-up, patients will receive conventional treatment according to national guidelines.¨

Outcome measures

Outcome measures
Measure	Basal Cell Carcinoma Patients n=19 Participants Patients (\>18 years) with histologically-verified superficial or nodular basal cell carcinoma (\<20 mm on the face/scalp, \<50mm on the trunk/extremities) AFL-assisted cisplatin+5-FU: Patients will receive AFL-assisted cisplatin+5-FU as a treatment for their BCC. In brief, treatment areas consisting of tumors and a 5 mm margin will undergo CO2 laser exposure followed by 60 min topical application of a marketed and commercially available IV cisplatin solution (0.1%) After removal of cisplatin, a commercially distributed 5-FU cream (5%) will be applied to the treatment area at a dose of 0.125 ml per cm2 and left under occlusion. After skin evaluations on Day 1 and 5 after treatment, the same 5-FU dose will be applied, again left under occlusion. In total, 5-FU will remain on the skin for 7 days after AFL treatment whereafter it will be washed off. An additional repeat AFL-cisplatin+5-FU treatment on Day 30 will be offered if tumors persist, based on clinical evaluation and imaging on Day 30.
Tumor Response- Clinical Clearance Determined by Clinical Assessment by Physician Clinical Clearance Day 30	13 Participants
Tumor Response- Clinical Clearance Determined by Clinical Assessment by Physician Clinical Clearance Month 3	18 Participants

SECONDARY outcome

Timeframe: Day 30 and 3 months post treatment

Clearance (yes or no) will be evaluated using non-invasive dynamic optical coherence tomography, high-intensity focused ultrasound and reflectance confocal microscopy imaging at Day 30 and Month 3. If residual tumor is identified at 3 months follow-up, patients will receive conventional treatment according to national guidelines.

Outcome measures

Outcome measures
Measure	Basal Cell Carcinoma Patients n=19 Participants Patients (\>18 years) with histologically-verified superficial or nodular basal cell carcinoma (\<20 mm on the face/scalp, \<50mm on the trunk/extremities) AFL-assisted cisplatin+5-FU: Patients will receive AFL-assisted cisplatin+5-FU as a treatment for their BCC. In brief, treatment areas consisting of tumors and a 5 mm margin will undergo CO2 laser exposure followed by 60 min topical application of a marketed and commercially available IV cisplatin solution (0.1%) After removal of cisplatin, a commercially distributed 5-FU cream (5%) will be applied to the treatment area at a dose of 0.125 ml per cm2 and left under occlusion. After skin evaluations on Day 1 and 5 after treatment, the same 5-FU dose will be applied, again left under occlusion. In total, 5-FU will remain on the skin for 7 days after AFL treatment whereafter it will be washed off. An additional repeat AFL-cisplatin+5-FU treatment on Day 30 will be offered if tumors persist, based on clinical evaluation and imaging on Day 30.
Imaging-based Tumor Response: Complete Tumor Clearance Determined by Physician Performing Imaging Day 30	13 Participants
Imaging-based Tumor Response: Complete Tumor Clearance Determined by Physician Performing Imaging Month 3	18 Participants

SECONDARY outcome

Timeframe: 3 months post treatment

Histological verification of tumor clearance will be performed 3 months after first treatment using tissue sections from a 4 mm punch biopsy. If residual tumor is identified at 3 months follow-up, patients will receive conventional treatment according to national guidelines. NOTE: Overall Number of Participants Analyzed is not consistent with numbers provided in any of the rows in the Participant Flow module since only 18 of the 19 patients consented to undergoing histologicla verification with biopsy. Those, presented data represents patients that underwent biopsy at the 3 month mark.

Outcome measures

Outcome measures
Measure	Basal Cell Carcinoma Patients n=18 Participants Patients (\>18 years) with histologically-verified superficial or nodular basal cell carcinoma (\<20 mm on the face/scalp, \<50mm on the trunk/extremities) AFL-assisted cisplatin+5-FU: Patients will receive AFL-assisted cisplatin+5-FU as a treatment for their BCC. In brief, treatment areas consisting of tumors and a 5 mm margin will undergo CO2 laser exposure followed by 60 min topical application of a marketed and commercially available IV cisplatin solution (0.1%) After removal of cisplatin, a commercially distributed 5-FU cream (5%) will be applied to the treatment area at a dose of 0.125 ml per cm2 and left under occlusion. After skin evaluations on Day 1 and 5 after treatment, the same 5-FU dose will be applied, again left under occlusion. In total, 5-FU will remain on the skin for 7 days after AFL treatment whereafter it will be washed off. An additional repeat AFL-cisplatin+5-FU treatment on Day 30 will be offered if tumors persist, based on clinical evaluation and imaging on Day 30.
Tumor Response- Histological Tumor Clearance Determined by Pathologist	17 Participants

Adverse Events

Basal Cell Carcinoma Patients

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Emily Wenande, MD, PhD

Bispebjerg Hospital

Phone: +4540505590

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place