Trial Outcomes & Findings for A Multi-center Study to Evaluate the Efficacy and Safety of PEP005 (Ingenol Mebutate) Gel, When Used to Treat Actinic Keratoses on the Head (Face or Scalp) (NCT NCT00916006)

NCT ID: NCT00916006

Last Updated: 2015-04-02

Results Overview

Complete clearance rate of actinic keratosis (AK) lesions, defined as the proportion of patients with no clinically visible AK lesions in the selected treatment area.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 269 participants
• Primary outcome timeframe: 57 days
• Results posted on: 2015-04-02

Participant Flow

Study PEP005-016 was conducted at 21 study centers in the United States and Australia. A total of 269 patients were randomized. Patient screening was started on 01 June 2009. The first patient was randomized on 05 June 2009 and the last patient completed the Day 57 visit on 10 September 2009

Participant milestones

Measure	PEP005 (Ingenol Mebutate) Gel, 0.015% PEP005 (ingenol mebutate) Gel 0.05% once daily for 3 consecutive days	Vehicle Vehicle gel once daily for 3 consecutive days
Overall Study STARTED	135	134
Overall Study COMPLETED	132	127
Overall Study NOT COMPLETED	3	7

Reasons for withdrawal

Measure	PEP005 (Ingenol Mebutate) Gel, 0.015% PEP005 (ingenol mebutate) Gel 0.05% once daily for 3 consecutive days	Vehicle Vehicle gel once daily for 3 consecutive days
Overall Study Adverse Event	1	1
Overall Study Withdrawal by Subject	2	5
Overall Study Protocol Violation	0	1

Baseline Characteristics

A Multi-center Study to Evaluate the Efficacy and Safety of PEP005 (Ingenol Mebutate) Gel, When Used to Treat Actinic Keratoses on the Head (Face or Scalp)

Baseline characteristics by cohort

Measure	PEP005 (Ingenol Mebutate) Gel, 0.015% n=135 Participants PEP005 (ingenol mebutate) Gel 0.05% once daily for 3 consecutive days	Vehicle n=134 Participants Vehicle gel once daily for 3 consecutive days	Total n=269 Participants Total of all reporting groups
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	71 Participants n=5 Participants	77 Participants n=7 Participants	148 Participants n=5 Participants
Age, Categorical >=65 years	64 Participants n=5 Participants	57 Participants n=7 Participants	121 Participants n=5 Participants
Sex: Female, Male Female	19 Participants n=5 Participants	14 Participants n=7 Participants	33 Participants n=5 Participants
Sex: Female, Male Male	116 Participants n=5 Participants	120 Participants n=7 Participants	236 Participants n=5 Participants
Region of Enrollment United States	125 participants n=5 Participants	126 participants n=7 Participants	251 participants n=5 Participants
Region of Enrollment Australia	10 participants n=5 Participants	8 participants n=7 Participants	18 participants n=5 Participants

PRIMARY outcome

Timeframe: 57 days

Population: Intention to treat population

Complete clearance rate of actinic keratosis (AK) lesions, defined as the proportion of patients with no clinically visible AK lesions in the selected treatment area.

Outcome measures

Measure	PEP005 (Ingenol Mebutate) Gel n=135 Participants PEP005 (ingenol mebutate) Gel 0.05% once daily for 3 consecutive days	Vehicle Gel n=134 Participants Vehicle Gel once daily for 3 consecutive days
Patients With Complete Clearance of Actinic Keratosis (AK) Lesions.	50 participants	3 participants

SECONDARY outcome

Timeframe: baseline and 57 days

Population: Intention to treat population

Patients with partial clearance defined as ≥ 75% reduction in the number of actinic keratosis (AK) lesions identified at baseline in the treatment area

Outcome measures

Measure	PEP005 (Ingenol Mebutate) Gel n=135 Participants PEP005 (ingenol mebutate) Gel 0.05% once daily for 3 consecutive days	Vehicle Gel n=134 Participants Vehicle Gel once daily for 3 consecutive days
Patients With Partial Clearance of Actinic Keratosis (AK)	81 participants	9 participants

Adverse Events

PEP005 (Ingenol Mebutate) Gel, 0.015%

Serious events: 2 serious events

Other events: 39 other events

Deaths: 0 deaths

Vehicle

Serious events: 2 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Measure	PEP005 (Ingenol Mebutate) Gel, 0.015% n=132 participants at risk PEP005 (ingenol mebutate) Gel 0.05% once daily for 3 consecutive days	Vehicle n=135 participants at risk Vehicle gel once daily for 3 consecutive days
Infections and infestations Campylobacter infection	0.76% 1/132 • Number of events 1 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.	0.00% 0/135 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.
Gastrointestinal disorders Small Intestinal Obstruction	0.76% 1/132 • Number of events 1 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.	0.00% 0/135 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.
Musculoskeletal and connective tissue disorders Meniscus Lesion	0.76% 1/132 • Number of events 1 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.	0.00% 0/135 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.
Injury, poisoning and procedural complications Injury	0.76% 1/132 • Number of events 1 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.	0.00% 0/135 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.
Respiratory, thoracic and mediastinal disorders Hypoxia	0.76% 1/132 • Number of events 1 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.	0.00% 0/135 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.
Vascular disorders Vascular pseudoaneurysm	0.00% 0/132 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.	0.74% 1/135 • Number of events 1 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.00% 0/132 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.	0.74% 1/135 • Number of events 1 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.

Other adverse events

Measure	PEP005 (Ingenol Mebutate) Gel, 0.015% n=132 participants at risk PEP005 (ingenol mebutate) Gel 0.05% once daily for 3 consecutive days	Vehicle n=135 participants at risk Vehicle gel once daily for 3 consecutive days
Skin and subcutaneous tissue disorders APPLICATION SITE PAIN	18.9% 25/132 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.	0.00% 0/135 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.
Skin and subcutaneous tissue disorders APPLICATION SITE PRURITUS	10.6% 14/132 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.	2.2% 3/135 • 57 days For the safety population, the total sample size was 132 patients who received PEP005 Gel (2 patients never received study medication and one patient actually received vehicle gel) and 135 patients who received vehicle gel.

Additional Information

Dr. Torsten Skov

LEO Pharma

Phone: +4520736294

Email: tosdk@leo-pharma.com

Results disclosure agreements

• Principal investigator is a sponsor employee For US sites: The institution and investigator agree not to publish the results of this study without prior written consent of the Sponsor. As used herein, the term "publish" shall include oral presentations,written abstracts, written manuscripts, etc.
• Publication restrictions are in place

Restriction type: OTHER