Trial Outcomes & Findings for Metvix Photodynamic Therapy (PDT) Versus Cryotherapy in Participants With Primary Superficial Basal Cell Carcinoma (NCT NCT00469417)

Last Updated: 2024-08-19

Results Overview

Patient Complete Response (CR) was defined as 100 percentage of the lesions within the participant having negative findings for nodular basal cell carcinoma (BCC) in the histological examination.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

120 participants

Primary outcome timeframe

3 months after last Metvix PDT or Cryotherapy cycle, up to 6 months

Results posted on

2024-08-19

Participant Flow

A total of 120 participants were randomized, of which 118 were received the study treatment. The study was carried out in 13 centers in seven European countries, they were included (Sweden, Finland, United Kingdom, Austria, France, Belgium and Italy).

Participant milestones

Participant milestones
Measure	Metvix® PDT Participants with basal cell carcinoma (BCC) lesions were administered to photodynamic therapy (PDT) with Metvix® cream 160 milligrams per gram (mg/g) applied for three hours, followed by illumination using non-coherent light with a fluency of 75 Joule per centimeter square (J/cm\2) and fluency rate of 70-200 milliwatt per centimeter square (mW/cm\2) up to 13 weeks.	Cryotherapy Cryotherapy was performed with a hand-held liquid nitrogen spray, using a double freeze-thaw cycle. After an initial icefield formation with a 3 millimeter (mm) rim of clinically healthy tissue, the icefield was to be maintained for a minimum of 20 seconds. This procedure was repeated after a thaw of 2-3 times the freeze time up to 12 weeks.
Overall Study STARTED	62	58
Overall Study Treated	60	58
Overall Study COMPLETED	32	41
Overall Study NOT COMPLETED	30	17

Reasons for withdrawal

Reasons for withdrawal
Measure	Metvix® PDT Participants with basal cell carcinoma (BCC) lesions were administered to photodynamic therapy (PDT) with Metvix® cream 160 milligrams per gram (mg/g) applied for three hours, followed by illumination using non-coherent light with a fluency of 75 Joule per centimeter square (J/cm\2) and fluency rate of 70-200 milliwatt per centimeter square (mW/cm\2) up to 13 weeks.	Cryotherapy Cryotherapy was performed with a hand-held liquid nitrogen spray, using a double freeze-thaw cycle. After an initial icefield formation with a 3 millimeter (mm) rim of clinically healthy tissue, the icefield was to be maintained for a minimum of 20 seconds. This procedure was repeated after a thaw of 2-3 times the freeze time up to 12 weeks.
Overall Study Treatment failure all lesions	18	11
Overall Study Consent withdrawn	1	1
Overall Study Adverse Event	8	2
Overall Study Lost to Follow-up	1	2
Overall Study Intercurrent disease	0	1
Overall Study Randomized but not treated	2	0

Baseline Characteristics

Metvix Photodynamic Therapy (PDT) Versus Cryotherapy in Participants With Primary Superficial Basal Cell Carcinoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Metvix® PDT n=60 Participants Participants with basal cell carcinoma (BCC) lesions were administered to photodynamic therapy (PDT) with Metvix® cream 160 milligrams per gram (mg/g) applied for three hours, followed by illumination using non-coherent light with a fluency of 75 Joule per centimeter square (J/cm\2) and fluency rate of 70-200 milliwatt per centimeter square (mW/cm\2) up to 13 weeks.	Cryotherapy n=58 Participants Cryotherapy was performed with a hand-held liquid nitrogen spray, using a double freeze-thaw cycle. After an initial icefield formation with a 3 millimeter (mm) rim of clinically healthy tissue, the icefield was to be maintained for a minimum of 20 seconds. This procedure was repeated after a thaw of 2-3 times the freeze time up to 12 weeks.	Total n=118 Participants Total of all reporting groups
Age, Continuous	63 Years STANDARD_DEVIATION 16 • n=5 Participants	64 Years STANDARD_DEVIATION 13 • n=7 Participants	64 Years STANDARD_DEVIATION 15 • n=5 Participants
Sex: Female, Male Female	20 Participants n=5 Participants	28 Participants n=7 Participants	48 Participants n=5 Participants
Sex: Female, Male Male	40 Participants n=5 Participants	30 Participants n=7 Participants	70 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	60 Participants n=5 Participants	58 Participants n=7 Participants	118 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: 3 months after last Metvix PDT or Cryotherapy cycle, up to 6 months

Population: Intent-to-treat analysis set included all the participants enrolled in the study who received at least one dose of study treatment.

Patient Complete Response (CR) was defined as 100 percentage of the lesions within the participant having negative findings for nodular basal cell carcinoma (BCC) in the histological examination.

Outcome measures

Outcome measures
Measure	Metvix® PDT n=60 Participants Participants with basal cell carcinoma (BCC) lesions were administered to photodynamic therapy (PDT) with Metvix® cream 160 milligrams per gram (mg/g) applied for three hours, followed by illumination using non-coherent light with a fluency of 75 Joule per centimeter square (J/cm\2) and fluency rate of 70-200 milliwatt per centimeter square (mW/cm\2) up to 13 weeks.	Cryotherapy n=58 Participants Cryotherapy was performed with a hand-held liquid nitrogen spray, using a double freeze-thaw cycle. After an initial icefield formation with a 3 millimeter (mm) rim of clinically healthy tissue, the icefield was to be maintained for a minimum of 20 seconds. This procedure was repeated after a thaw of 2-3 times the freeze time up to 12 weeks.
Percentage of Participants With Histologically Confirmed Patient Complete Response (CR) 3 Months After Last Metvix PDT or Cryotherapy Cycle	92 percentage of participants	90 percentage of participants

SECONDARY outcome

Timeframe: 3 months after last Metvix PDT or Cryotherapy cycle, up to 6 months

Population: Intent-to-treat analysis set included all the participants enrolled in the study who received at least one dose of study treatment.

Complete response was defined as no clinically visible BCC lesions in the treatment area.

Outcome measures

Outcome measures
Measure	Metvix® PDT n=114 lesion Participants with basal cell carcinoma (BCC) lesions were administered to photodynamic therapy (PDT) with Metvix® cream 160 milligrams per gram (mg/g) applied for three hours, followed by illumination using non-coherent light with a fluency of 75 Joule per centimeter square (J/cm\2) and fluency rate of 70-200 milliwatt per centimeter square (mW/cm\2) up to 13 weeks.	Cryotherapy n=105 lesion Cryotherapy was performed with a hand-held liquid nitrogen spray, using a double freeze-thaw cycle. After an initial icefield formation with a 3 millimeter (mm) rim of clinically healthy tissue, the icefield was to be maintained for a minimum of 20 seconds. This procedure was repeated after a thaw of 2-3 times the freeze time up to 12 weeks.
Number of Lesion With Complete Response 3 Months After Last Metvix PDT or Cryotherapy Cycle	109 lesion	94 lesion

SECONDARY outcome

Timeframe: 3 months after the last metvix PDT or Cryotherapy cycle (Up to 6 months)

Cosmetic outcome was assessed by both investigator and participants in participants with 100% of lesions in complete response. Overall cosmetic outcome was assessed with regard to occurrence of the following signs or symptoms; scarring, atrophy, induration, redness or change in pigmentation. The investigator graded the cosmetic outcome as: * excellent: no scarring, atrophy or induration, and no or slight occurrence of redness or change in pigmentation compared lo adjacent skin * good: no scarring, atrophy or induration but moderate redness or change in pigmentation compared to adjacent skin * fair: slight to moderate occurrence of scarring, atrophy or induration * poor: extensive occurrence of scarring, atrophy or induration.

Outcome measures

Outcome measures
Measure	Metvix® PDT n=53 Participants Participants with basal cell carcinoma (BCC) lesions were administered to photodynamic therapy (PDT) with Metvix® cream 160 milligrams per gram (mg/g) applied for three hours, followed by illumination using non-coherent light with a fluency of 75 Joule per centimeter square (J/cm\2) and fluency rate of 70-200 milliwatt per centimeter square (mW/cm\2) up to 13 weeks.	Cryotherapy n=50 Participants Cryotherapy was performed with a hand-held liquid nitrogen spray, using a double freeze-thaw cycle. After an initial icefield formation with a 3 millimeter (mm) rim of clinically healthy tissue, the icefield was to be maintained for a minimum of 20 seconds. This procedure was repeated after a thaw of 2-3 times the freeze time up to 12 weeks.
Overall Cosmetic Outcome Assessed by Investigator 3 Months After the Last Metvix PDT or Cryotherapy Cycle Investigator: Excellent	17 Participants	2 Participants
Overall Cosmetic Outcome Assessed by Investigator 3 Months After the Last Metvix PDT or Cryotherapy Cycle Investigator: Good	30 Participants	23 Participants
Overall Cosmetic Outcome Assessed by Investigator 3 Months After the Last Metvix PDT or Cryotherapy Cycle Investigator: Fair	6 Participants	25 Participants

SECONDARY outcome

Timeframe: 3 months after the last metvix PDT or Cryotherapy cycle (Up to 6 months)

Cosmetic outcome was assessed by both investigator and participants in participants with 100% of lesions in complete response. Overall cosmetic outcome was assessed with regard to occurrence of the following signs or symptoms; scarring, atrophy, induration, redness or change in pigmentation. The participants graded the cosmetic outcome as: * excellent: no scarring, atrophy or induration, and no or slight occurrence of redness or change in pigmentation compared lo adjacent skin * good: no scarring, atrophy or induration but moderate redness or change in pigmentation compared to adjacent skin * fair: slight to moderate occurrence of scarring, atrophy or induration * poor: extensive occurrence of scarring, atrophy or induration.

Outcome measures

Outcome measures
Measure	Metvix® PDT n=46 Participants Participants with basal cell carcinoma (BCC) lesions were administered to photodynamic therapy (PDT) with Metvix® cream 160 milligrams per gram (mg/g) applied for three hours, followed by illumination using non-coherent light with a fluency of 75 Joule per centimeter square (J/cm\2) and fluency rate of 70-200 milliwatt per centimeter square (mW/cm\2) up to 13 weeks.	Cryotherapy n=44 Participants Cryotherapy was performed with a hand-held liquid nitrogen spray, using a double freeze-thaw cycle. After an initial icefield formation with a 3 millimeter (mm) rim of clinically healthy tissue, the icefield was to be maintained for a minimum of 20 seconds. This procedure was repeated after a thaw of 2-3 times the freeze time up to 12 weeks.
Overall Cosmetic Outcome Assessed by Participants 3 Months After the Last Metvix PDT or Cryotherapy Cycle Participants: Excellent	22 Participants	9 Participants
Overall Cosmetic Outcome Assessed by Participants 3 Months After the Last Metvix PDT or Cryotherapy Cycle Participants: Good	24 Participants	24 Participants
Overall Cosmetic Outcome Assessed by Participants 3 Months After the Last Metvix PDT or Cryotherapy Cycle Participants: Fair	0 Participants	11 Participants

SECONDARY outcome

Timeframe: 12, 24, 36, 48 and 60 months after last Metvix PDT cycle or Cryotherapy (Up to 5 years)

Population: Intent-to-treat analysis set included all the participants enrolled in the study who received at least one dose of study treatment. Here "overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure.

Recurrence rate in complete clearance group was analyzed.

Outcome measures

Outcome measures
Measure	Metvix® PDT n=109 lesion Participants with basal cell carcinoma (BCC) lesions were administered to photodynamic therapy (PDT) with Metvix® cream 160 milligrams per gram (mg/g) applied for three hours, followed by illumination using non-coherent light with a fluency of 75 Joule per centimeter square (J/cm\2) and fluency rate of 70-200 milliwatt per centimeter square (mW/cm\2) up to 13 weeks.	Cryotherapy n=94 lesion Cryotherapy was performed with a hand-held liquid nitrogen spray, using a double freeze-thaw cycle. After an initial icefield formation with a 3 millimeter (mm) rim of clinically healthy tissue, the icefield was to be maintained for a minimum of 20 seconds. This procedure was repeated after a thaw of 2-3 times the freeze time up to 12 weeks.
Recurrence Rate in Complete Clearance Group 36 months	23 lesion	18 lesion
Recurrence Rate in Complete Clearance Group 48 months	23 lesion	18 lesion
Recurrence Rate in Complete Clearance Group 60 months	23 lesion	19 lesion
Recurrence Rate in Complete Clearance Group 12 months	10 lesion	12 lesion
Recurrence Rate in Complete Clearance Group 24 months	18 lesion	18 lesion

SECONDARY outcome

Timeframe: 24, 36, 48, and 60 Months After the Last Metvix PDT Cycle (Up to 5 years)

Population: Intent-to-treat analysis set included all the participants enrolled in the study who received at least one dose of study treatment. Here overall "Number of participants analyzed" signifies those participants who were evaluable for this outcome measure. "Number analyzed", signifies those participants who were evaluable for this outcome measure at the specified time point.

Outcome measures

Outcome measures
Measure	Metvix® PDT n=36 Participants Participants with basal cell carcinoma (BCC) lesions were administered to photodynamic therapy (PDT) with Metvix® cream 160 milligrams per gram (mg/g) applied for three hours, followed by illumination using non-coherent light with a fluency of 75 Joule per centimeter square (J/cm\2) and fluency rate of 70-200 milliwatt per centimeter square (mW/cm\2) up to 13 weeks.	Cryotherapy n=43 Participants Cryotherapy was performed with a hand-held liquid nitrogen spray, using a double freeze-thaw cycle. After an initial icefield formation with a 3 millimeter (mm) rim of clinically healthy tissue, the icefield was to be maintained for a minimum of 20 seconds. This procedure was repeated after a thaw of 2-3 times the freeze time up to 12 weeks.
Overall Cosmetic Outcome Assessed by Investigator 24, 36, 48, and 60 Months After the Last Metvix PDT or Cryotherapy Cycle Excellent: At month 24	19 Participants	3 Participants
Overall Cosmetic Outcome Assessed by Investigator 24, 36, 48, and 60 Months After the Last Metvix PDT or Cryotherapy Cycle Good: At month 24	12 Participants	15 Participants
Overall Cosmetic Outcome Assessed by Investigator 24, 36, 48, and 60 Months After the Last Metvix PDT or Cryotherapy Cycle Fair: At month 24	3 Participants	20 Participants
Overall Cosmetic Outcome Assessed by Investigator 24, 36, 48, and 60 Months After the Last Metvix PDT or Cryotherapy Cycle Excellent: At month 36	20 Participants	5 Participants
Overall Cosmetic Outcome Assessed by Investigator 24, 36, 48, and 60 Months After the Last Metvix PDT or Cryotherapy Cycle Good: At month 36	12 Participants	22 Participants
Overall Cosmetic Outcome Assessed by Investigator 24, 36, 48, and 60 Months After the Last Metvix PDT or Cryotherapy Cycle Fair: At month 36	4 Participants	14 Participants
Overall Cosmetic Outcome Assessed by Investigator 24, 36, 48, and 60 Months After the Last Metvix PDT or Cryotherapy Cycle Poor: At month 36	0 Participants	2 Participants
Overall Cosmetic Outcome Assessed by Investigator 24, 36, 48, and 60 Months After the Last Metvix PDT or Cryotherapy Cycle Excellent: At month 48	19 Participants	5 Participants
Overall Cosmetic Outcome Assessed by Investigator 24, 36, 48, and 60 Months After the Last Metvix PDT or Cryotherapy Cycle Good: At month 48	10 Participants	21 Participants
Overall Cosmetic Outcome Assessed by Investigator 24, 36, 48, and 60 Months After the Last Metvix PDT or Cryotherapy Cycle Fair: At month 48	4 Participants	15 Participants
Overall Cosmetic Outcome Assessed by Investigator 24, 36, 48, and 60 Months After the Last Metvix PDT or Cryotherapy Cycle Poor: At month 48	0 Participants	1 Participants
Overall Cosmetic Outcome Assessed by Investigator 24, 36, 48, and 60 Months After the Last Metvix PDT or Cryotherapy Cycle Excellent: At month 60	18 Participants	6 Participants
Overall Cosmetic Outcome Assessed by Investigator 24, 36, 48, and 60 Months After the Last Metvix PDT or Cryotherapy Cycle Good: At month 60	8 Participants	22 Participants
Overall Cosmetic Outcome Assessed by Investigator 24, 36, 48, and 60 Months After the Last Metvix PDT or Cryotherapy Cycle Fair: At month 60	6 Participants	14 Participants
Overall Cosmetic Outcome Assessed by Investigator 24, 36, 48, and 60 Months After the Last Metvix PDT or Cryotherapy Cycle Poor: At month 60	0 Participants	1 Participants

Adverse Events

Metvix® PDT

Serious events: 7 serious events

Other events: 0 other events

Deaths: 4 deaths

Cryotherapy

Serious events: 2 serious events

Other events: 0 other events

Deaths: 3 deaths

Serious adverse events

Serious adverse events
Measure	Metvix® PDT n=60 participants at risk Participants with basal cell carcinoma (BCC) lesions were administered to photodynamic therapy (PDT) with Metvix® cream 160 milligrams per gram (mg/g) applied for three hours, followed by illumination using non-coherent light with a fluency of 75 Joule per centimeter square (J/cm\2) and fluency rate of 70-200 milliwatt per centimeter square (mW/cm\2) up to 13 weeks.	Cryotherapy n=58 participants at risk Cryotherapy was performed with a hand-held liquid nitrogen spray, using a double freeze-thaw cycle. After an initial icefield formation with a 3 millimeter (mm) rim of clinically healthy tissue, the icefield was to be maintained for a minimum of 20 seconds. This procedure was repeated after a thaw of 2-3 times the freeze time up to 12 weeks.
Cardiac disorders Cardiac arrest	1.7% 1/60 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.	0.00% 0/58 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Metastatic cancer	0.00% 0/60 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.	1.7% 1/58 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.
General disorders Participant deceased	1.7% 1/60 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.	0.00% 0/58 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Death due to myeloma	1.7% 1/60 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.	0.00% 0/58 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.
General disorders Death	1.7% 1/60 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.	0.00% 0/58 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.
General disorders Sudden death	1.7% 1/60 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.	0.00% 0/58 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Hepatic cancer	1.7% 1/60 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.	0.00% 0/58 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Metastatic adenocarcinoma of lung	0.00% 0/60 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.	1.7% 1/58 • Baseline up to Months 60 Safety analysis set included all the participants enrolled in the study.

Other adverse events

Adverse event data not reported

Additional Information

Clinical Operations

Galderma

Phone: 817 961 5000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place