Trial Outcomes & Findings for PDT With Metvix 160 mg/g Cream Versus PDT With Placebo Cream in Participants With Primary Nodular Basal Call Carcinoma (NCT NCT00472043)

NCT ID: NCT00472043

Last Updated: 2024-08-06

Results Overview

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. Histological examination included evaluation of all the microscopical slides from the excised tissue for presence of malignant basal cells. Complete response was defined as complete disappearance of lesion. Percentage of participants with histologically confirmed complete response were reported.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 66 participants
• Primary outcome timeframe: up to 6 months
• Results posted on: 2024-08-06

Participant Flow

This study was conducted in Australia between 1 October 2000 to 30 September 2002.

A total of 66 participants were included in this study.

Participant milestones

Measure	Metvix® Cream 160 Milligram Per Gram Methyl aminolevulinate hydrochloride 160 milligram (mg)/gram (g) cream were received by participants with primary nodular basal cell carcinoma. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.	Placebo Participants with primary nodular basal cell carcinoma received Metvix® matching placebo cream. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.
Overall Study STARTED	33	33
Overall Study COMPLETED	31	33
Overall Study NOT COMPLETED	2	0

Reasons for withdrawal

Measure	Metvix® Cream 160 Milligram Per Gram Methyl aminolevulinate hydrochloride 160 milligram (mg)/gram (g) cream were received by participants with primary nodular basal cell carcinoma. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.	Placebo Participants with primary nodular basal cell carcinoma received Metvix® matching placebo cream. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.
Overall Study Withdrawal by Subject	1	0
Overall Study Death	1	0

Baseline Characteristics

PDT With Metvix 160 mg/g Cream Versus PDT With Placebo Cream in Participants With Primary Nodular Basal Call Carcinoma

Baseline characteristics by cohort

Measure	Metvix® Cream 160 Milligram Per Gram n=33 Participants Methyl aminolevulinate hydrochloride 160 milligram (mg)/gram (g) cream were received by participants with primary nodular basal cell carcinoma. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.	Placebo n=33 Participants Participants with primary nodular basal cell carcinoma received Metvix® matching placebo cream. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.	Total n=66 Participants Total of all reporting groups
Age, Continuous	70 years STANDARD_DEVIATION 10 • n=5 Participants	66 years STANDARD_DEVIATION 11 • n=7 Participants	68 years STANDARD_DEVIATION 11 • n=5 Participants
Sex: Female, Male Female	11 Participants n=5 Participants	6 Participants n=7 Participants	17 Participants n=5 Participants
Sex: Female, Male Male	22 Participants n=5 Participants	27 Participants n=7 Participants	49 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	33 Participants n=5 Participants	33 Participants n=7 Participants	66 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: up to 6 months

Population: Intent to treat (ITT) population that included all treated participants.

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. Histological examination included evaluation of all the microscopical slides from the excised tissue for presence of malignant basal cells. Complete response was defined as complete disappearance of lesion. Percentage of participants with histologically confirmed complete response were reported.

Outcome measures

Measure	Metvix® Cream 160 Milligram Per Gram n=33 Participants Methyl aminolevulinate hydrochloride 160 milligram (mg)/gram (g) cream were received by participants with primary nodular basal cell carcinoma. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.	Placebo n=33 Participants Participants with primary nodular basal cell carcinoma received Metvix® matching placebo cream. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.
Percentage of Participants With Histologically Confirmed Complete Response (CR)	67 percentage of participants Interval 48.0 to 82.0	18 percentage of participants Interval 7.0 to 36.0

SECONDARY outcome

Timeframe: Up to 3 months

Population: ITT Population that included all treated participants

Histological response weight means no signs of malignant basal cells in all microscopical slides containing excised tissue. The histologically confirmed participant weighted response, weighted by percentage of lesions per participant are reported in this outcome measure. Number of lesions per participant with-in treatment group were calculated in following way: ni = number of lesions within 1 participant, ci= number of lesions in complete response within 1 participant, xi= 100%. ci/ni= response rate within one participant, Nt= number of participant within one treatment, nt=number of lesions with-in one treatment, wi=ni/nt= weight for one participant with Nt Σ wi (i=1) = 1 for each treatment.

Outcome measures

Measure	Metvix® Cream 160 Milligram Per Gram n=34 Lesions Methyl aminolevulinate hydrochloride 160 milligram (mg)/gram (g) cream were received by participants with primary nodular basal cell carcinoma. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.	Placebo n=36 Lesions Participants with primary nodular basal cell carcinoma received Metvix® matching placebo cream. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.
Percentage of Lesions Per Participant: Histologically Confirmed Participant Weighted Response	67.6 Percentage of lesions per participant Standard Deviation 8.3	19.4 Percentage of lesions per participant Standard Deviation 6.7

SECONDARY outcome

Timeframe: Up to 3 months

Population: ITT population included all treated participants

Histological examination included from the excised tissue were examined for presence of malignant basal cells, where complete response (CR) was no signs of malignant basal cells and non-CR was evidence of malignant basal cells

Outcome measures

Measure	Metvix® Cream 160 Milligram Per Gram n=34 Lesions Methyl aminolevulinate hydrochloride 160 milligram (mg)/gram (g) cream were received by participants with primary nodular basal cell carcinoma. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.	Placebo n=36 Lesions Participants with primary nodular basal cell carcinoma received Metvix® matching placebo cream. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.
Histological Lesion Response Lesions in CR	23 Lesions	7 Lesions
Histological Lesion Response Lesions in non-CR	8 Lesions	29 Lesions
Histological Lesion Response Missing	3 Lesions	0 Lesions

SECONDARY outcome

Timeframe: Up to 9 months

Population: ITT population that includes all treated participants

A CR to treatment was documented clinically by visual evaluation and palpation. The on-site investigator evaluated the lesion response by comparing with the lesion size before treatment using the following definitions: CR - complete disappearance of a lesion. Partial response (PR) -the longest diameter of the lesion is reduced by 50% or more. No response (NR) - the longest diameter of the lesion is less than 50% reduced. Progression - the longest diameter is increased by 20% or more.

Outcome measures

Measure	Metvix® Cream 160 Milligram Per Gram n=33 Participants Methyl aminolevulinate hydrochloride 160 milligram (mg)/gram (g) cream were received by participants with primary nodular basal cell carcinoma. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.	Placebo n=33 Participants Participants with primary nodular basal cell carcinoma received Metvix® matching placebo cream. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.
Percentage of Participants With Clinically Confirmed Participant Complete Response (CR)	82 percentage of participants	24 percentage of participants

SECONDARY outcome

Timeframe: Up to 3 months

Population: ITT population that includes all treated participants

Cosmetic outcome for those lesions with complete histological response was assessed by both the investigator and by the participant. Cosmetic outcome was assessed with regards to occurrence of the following signs or symptoms like scarring, atrophy, induration, redness, and change in pigmentation. The cosmetic outcome was graded as excellent: no scarring, atrophy or induration, and no or slight occurrence of redness or change in pigmentation compared to 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

Measure	Metvix® Cream 160 Milligram Per Gram n=23 Lesions Methyl aminolevulinate hydrochloride 160 milligram (mg)/gram (g) cream were received by participants with primary nodular basal cell carcinoma. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.	Placebo n=7 Lesions Participants with primary nodular basal cell carcinoma received Metvix® matching placebo cream. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.
Cosmetic Outcomes for Lesions Assessed by Investigator Investigator: Excellent	13 Lesions	2 Lesions
Cosmetic Outcomes for Lesions Assessed by Investigator Investigator: Good	8 Lesions	3 Lesions
Cosmetic Outcomes for Lesions Assessed by Investigator Investigator: Fair	1 Lesions	1 Lesions
Cosmetic Outcomes for Lesions Assessed by Investigator Investigator: Not Applicable	1 Lesions	1 Lesions

SECONDARY outcome

Timeframe: Up to 6 months

Population: The analysis was performed on safety set population defined as all participants who received study drug and had at least one post-baseline safety assessment

An AE was any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which does not necessarily had a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory value), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. A SAE was any untoward medical occurrence that at any dose: results in death, was life-threatening, required hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect.

Outcome measures

Measure	Metvix® Cream 160 Milligram Per Gram n=33 Participants Methyl aminolevulinate hydrochloride 160 milligram (mg)/gram (g) cream were received by participants with primary nodular basal cell carcinoma. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.	Placebo n=33 Participants Participants with primary nodular basal cell carcinoma received Metvix® matching placebo cream. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.
Number of Participants With Serious Adverse Events (SAEs) and AEs Leading to Discontinuation Serious adverse events	1 Participants	2 Participants
Number of Participants With Serious Adverse Events (SAEs) and AEs Leading to Discontinuation Adverse events leading to discontinuation	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Up to 3 months

Population: ITT population that includes all treated participants

Cosmetic outcome for those lesions with complete histological response was assessed by both the investigator and by the participant. Cosmetic outcome was assessed with regards to occurrence of the following signs or symptoms like scarring, atrophy, induration, redness, and change in pigmentation. The cosmetic outcome was graded as excellent: no scarring, atrophy or induration, and no or slight occurrence of redness or change in pigmentation compared to 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

Measure	Metvix® Cream 160 Milligram Per Gram n=23 Lesions Methyl aminolevulinate hydrochloride 160 milligram (mg)/gram (g) cream were received by participants with primary nodular basal cell carcinoma. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.	Placebo n=7 Lesions Participants with primary nodular basal cell carcinoma received Metvix® matching placebo cream. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.
Cosmetic Outcomes for Lesions Assessed by Participants Participants: Excellent	14 Lesions	5 Lesions
Cosmetic Outcomes for Lesions Assessed by Participants Participants: Good	8 Lesions	1 Lesions
Cosmetic Outcomes for Lesions Assessed by Participants Participants: Fair	0 Lesions	0 Lesions
Cosmetic Outcomes for Lesions Assessed by Participants Participants: Not Applicable	1 Lesions	1 Lesions

Adverse Events

Metvix® Cream 160 Milligram Per Gram

Serious events: 1 serious events

Other events: 0 other events

Deaths: 1 deaths

Placebo

Serious events: 2 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Metvix® Cream 160 Milligram Per Gram n=33 participants at risk Methyl aminolevulinate hydrochloride 160 milligram (mg)/gram (g) cream were received by participants with primary nodular basal cell carcinoma. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.	Placebo n=33 participants at risk Participants with primary nodular basal cell carcinoma received Metvix® matching placebo cream. A thick layer of study cream was applied directly on the lesion and on 5 mm of the surrounding tissue. An approximately 1 mm thick layer of cream was applied to cover the lesion completely. The study cream was applied for at least 3 hours followed by illumination using non-coherent red (570-670 nm) light at a fluence of 50- 75 J/cm\^2.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Carcinoma Bile Duct	3.0% 1/33 • Number of events 1 • Up to 6 months Analysis was performed on safety population: randomized participants who applied the study medication at least once.	0.00% 0/33 • Up to 6 months Analysis was performed on safety population: randomized participants who applied the study medication at least once.
Respiratory, thoracic and mediastinal disorders Pulmonary Oedema	0.00% 0/33 • Up to 6 months Analysis was performed on safety population: randomized participants who applied the study medication at least once.	3.0% 1/33 • Number of events 1 • Up to 6 months Analysis was performed on safety population: randomized participants who applied the study medication at least once.
Cardiac disorders Myocardial Infarction	0.00% 0/33 • Up to 6 months Analysis was performed on safety population: randomized participants who applied the study medication at least once.	3.0% 1/33 • Number of events 1 • Up to 6 months Analysis was performed on safety population: randomized participants who applied the study medication at least once.

Other adverse events

Adverse event data not reported

Additional Information

Clinical Operations

Galderma

Phone: 817 961 5000

Email: Clinical.Studies@galderma.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place