Trial Outcomes & Findings for A Sequential Treatment Regimen of Cryotherapy and Picato® for the Treatment of Actinic Keratosis on the Face and Scalp (NCT NCT01541553)

NCT ID: NCT01541553

Last Updated: 2025-03-10

Results Overview

To determine the 11-week rate of complete clearance of AKs (defined as no clinically visible AKs) in the selected treatment area using sequential cryotherapy and field treatment with PEP005 Gel compared to cryotherapy alone.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 367 participants
• Primary outcome timeframe: 11 weeks
• Results posted on: 2025-03-10

Participant Flow

A total of 367 subjects were enrolled of whom 38 were screen failures. The remaining 329 subjects were randomised at Visit 1.

Participant milestones

Measure	PEP005 Gel, 0.015% Cryotherapy followed by PEP005 Gel, 0.015% Cryotherapy of all visible AKs (4-8 lesions, "baseline AKs") in the selected treatment area, 2-4 weeks healing time followed by field treatment with PEP005 gel, 0.015% once daily for 3 consecutive days	Vehicle Gel Cryotherapy followed by vehicle gel Cryotherapy of all visible AKs (4-8 lesions, "baseline AKs") in the selected treatment area, 2-4 weeks healing time followed by field treatment with vehicle gel once daily for 3 consecutive days.
Overall Study STARTED	167	162
Overall Study COMPLETED	155	148
Overall Study NOT COMPLETED	12	14

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Sequential Treatment Regimen of Cryotherapy and Picato® for the Treatment of Actinic Keratosis on the Face and Scalp

Baseline characteristics by cohort

Measure	PEP005 Gel, 0.015% n=167 Participants Cryotherapy followed by PEP005 Gel, 0.015% Cryotherapy of all visible AKs (4-8 lesions, "baseline AKs") in the selected treatment area, 2-4 weeks healing time followed by field treatment with PEP005 gel, 0.015% once daily for 3 consecutive days	Vehicle Gel n=162 Participants Cryotherapy followed by vehicle gel Cryotherapy of all visible AKs (4-8 lesions, "baseline AKs") in the selected treatment area, 2-4 weeks healing time followed by field treatment with vehicle gel once daily for 3 consecutive days.	Total n=329 Participants Total of all reporting groups
Age, Continuous	66.7 years STANDARD_DEVIATION 9.4 • n=5 Participants	67.4 years STANDARD_DEVIATION 9.3 • n=7 Participants	67 years STANDARD_DEVIATION 9.3 • n=5 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	72 Participants n=5 Participants	61 Participants n=7 Participants	133 Participants n=5 Participants
Age, Categorical >=65 years	95 Participants n=5 Participants	101 Participants n=7 Participants	196 Participants n=5 Participants
Sex: Female, Male Female	29 Participants n=5 Participants	29 Participants n=7 Participants	58 Participants n=5 Participants
Sex: Female, Male Male	138 Participants n=5 Participants	133 Participants n=7 Participants	271 Participants n=5 Participants
Region of Enrollment United States	167 participants n=5 Participants	162 participants n=7 Participants	329 participants n=5 Participants

PRIMARY outcome

Timeframe: 11 weeks

To determine the 11-week rate of complete clearance of AKs (defined as no clinically visible AKs) in the selected treatment area using sequential cryotherapy and field treatment with PEP005 Gel compared to cryotherapy alone.

Outcome measures

Measure	PEP005 Gel, 0.015% n=167 Participants Cryotherapy followed by PEP005 Gel, 0.015% Cryotherapy of all visible AKs (4-8 lesions, "baseline AKs") in the selected treatment area, 2-4 weeks healing time followed by field treatment with PEP005 gel, 0.015% once daily for 3 consecutive days	Vehicle Gel n=162 Participants Cryotherapy followed by vehicle gel Cryotherapy of all visible AKs (4-8 lesions, "baseline AKs") in the selected treatment area, 2-4 weeks healing time followed by field treatment with vehicle gel once daily for 3 consecutive days.
Complete Clearance of AKs at Week 11	101 participants	80 participants

SECONDARY outcome

Timeframe: Baseline to week 11

Percentage change from baseline in number of AKs at Week 11

Outcome measures

Measure	PEP005 Gel, 0.015% n=167 Participants Cryotherapy followed by PEP005 Gel, 0.015% Cryotherapy of all visible AKs (4-8 lesions, "baseline AKs") in the selected treatment area, 2-4 weeks healing time followed by field treatment with PEP005 gel, 0.015% once daily for 3 consecutive days	Vehicle Gel n=162 Participants Cryotherapy followed by vehicle gel Cryotherapy of all visible AKs (4-8 lesions, "baseline AKs") in the selected treatment area, 2-4 weeks healing time followed by field treatment with vehicle gel once daily for 3 consecutive days.
Percentage Change From Baseline in Number of AKs at Week 11	81.1 percentage of change Standard Deviation 31.8	74.1 percentage of change Standard Deviation 34.4

SECONDARY outcome

Timeframe: Week 11

Partial clearance of AKs at Week 11, defined as 75% or greater reduction from baseline in the number of clinically visible AKs in the selected treatment area at Week 11

Outcome measures

Measure	PEP005 Gel, 0.015% n=167 Participants Cryotherapy followed by PEP005 Gel, 0.015% Cryotherapy of all visible AKs (4-8 lesions, "baseline AKs") in the selected treatment area, 2-4 weeks healing time followed by field treatment with PEP005 gel, 0.015% once daily for 3 consecutive days	Vehicle Gel n=162 Participants Cryotherapy followed by vehicle gel Cryotherapy of all visible AKs (4-8 lesions, "baseline AKs") in the selected treatment area, 2-4 weeks healing time followed by field treatment with vehicle gel once daily for 3 consecutive days.
Partial Clearance of AKs at Week 11	130 participants	109 participants

Adverse Events

PEP005 Gel, 0.015%

Serious events: 7 serious events

Other events: 0 other events

Deaths: 0 deaths

Vehicle Gel

Serious events: 4 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	PEP005 Gel, 0.015% n=167 participants at risk Cryotherapy followed by PEP005 Gel, 0.015% Cryotherapy of all visible AKs (4-8 lesions, "baseline AKs") in the selected treatment area, 2-4 weeks healing time followed by field treatment with PEP005 gel, 0.015% once daily for 3 consecutive days	Vehicle Gel n=162 participants at risk Cryotherapy followed by vehicle gel Cryotherapy of all visible AKs (4-8 lesions, "baseline AKs") in the selected treatment area, 2-4 weeks healing time followed by field treatment with vehicle gel once daily for 3 consecutive days.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Basal cell carcinoma	1.2% 2/167	0.62% 1/162
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Malignant melanoma	0.60% 1/167	0.62% 1/162
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Squamous cell carcinoma	1.8% 3/167	0.62% 1/162
Nervous system disorders Syncope	0.60% 1/167	0.00% 0/162
Nervous system disorders Transient ischeamic	0.00% 0/167	0.62% 1/162

Other adverse events

Adverse event data not reported

Additional Information

Clinical trial disclosure

LEO Pharma A/S

Phone: 00 45 44 94 58 88

Email: ctr.disclosure@leo-pharma.com

Results disclosure agreements

• Principal investigator is a sponsor employee The sponsor is entitled to review and comment on publications prior to submission or presentation. Upon request of the sponsor, the investigator shall delay publication or presentation to allow for protection of the sponsors inventions and other intellectual property.
• Publication restrictions are in place

Restriction type: OTHER