Actinic KeratosisPhase 2NCT01998984

Efficacy and Safety of Ingenol Mebutate Gel 0.06% When Applied Once Daily for 2, 3 or 4 Consecutive Days to a Treatment Area of Approximately 250 cm2 on Trunk and Extremities in Subjects With Actinic Keratosis

A Phase 2 study testing ingenol mebutate in people with Actinic Keratosis, run by LEO Pharma at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This is a randomised, double-blind, parallel groups, vehicle controlled, 8-week phase 2 trial. The objective is to evaluate efficacy of ingenol mebutate gel 0.06 % after once daily treatment for 2, 3 or 4 consecutive days compared to vehicle gel Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Actinic Keratosis
What is being tested?: ingenol mebutate
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 266 participants
Where: 1 siteUnited States
Study dates: Jan 2014to Jul 2014
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Subjects with 5 to 20 clinically typical, visible and discrete AKs within a contiguous area of approximately 250 cm² sun-damaged skin on either trunk (except chest), or extremities

Who cannot join

Location of the treatment area (trunk (except chest) or extremities)
within 5 cm of an incompletely healed wound,
within 5 cm of a suspected basal cell carcinoma (BCC) or squamous cell carcinoma (SCC)
Prior treatment with ingenol mebutate within the selected treatment area
Lesions in the treatment area that have:
atypical clinical appearance (e.g., hypertrophic,hyperkeratotic or cutaneous horns) and/or,
recalcitrant disease (e.g., did not respond to cryotherapy on two previous occasions)

Trial locations and contacts

1 site in 1 country.

United States1 site

Long Island Skin Cancer and Dermatologic Surgery
Smithtown, New York

Other trials in United States

About this study

Conditions: Actinic Keratosis
Being tested: ingenol mebutate, Placebo
Medical subject headings: Keratosis, Actinic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

ingenol mebutateDrug
PlaceboOther

Study groups in NCT01998984
Study group	Role	What they receive
2 days placebo and 2 days drug	Experimental	Placebo and drug Receives: Drug: ingenol mebutate; Other: Placebo
1 day placebo and 3 days drug	Experimental	Placebo and drug Receives: Drug: ingenol mebutate; Other: Placebo
4 days placebo	Placebo comparator	Placebo Receives: Other: Placebo
4 days drug	Experimental	Drug Receives: Drug: ingenol mebutate

What is being measured

The primary outcome is the main question the study is designed to answer.

Percentage of Participants With Complete Clearance of Actinic Keratosis Lesions (AKs)
Complete clearance of AKs at Week 8 was defined as a 100% reduction from baseline in number of AKs.
The table presents the mean across 1000 multiple imputations. Missing values for AK count were imputed sequentially from a negative binomial regression model with treatment group, AK counts at the previous visit, and analysis site as covariates and log baseline AK count as offset.
Measured: At Week 8

Secondary outcomes (2)

Percentage of Reduction in Actinic Keratosis (AK) Lesion Count From Baseline (Day 1) (Multiple Imputation)
The number of clinically visible AK lesions identified in the treatment area was to be recorded at Visit 1(≤14 days prior to Day 1).
The analysis was based on 1000 imputations of actinic keratosis lesion count at Week 8 using a negative binomial regression model with factors treatment and analysis site and with log of baseline actinic keratosis lesion count as offset. The table shows the adjusted percentage reduction from baseline.
Values for the Ingenol 4 days arm were calculated separately based on observed cases. On the basis of the data monitoring committee's recommendation the 4-day active treatment group was closed, and this arm was excluded from statistical models and comparisons in the secondary efficacy analyses.
Measured: At Week 8
Percentage of Participants With Partial Clearance of AKs
Partial clearance of AKs at Week 8, defined as at least 75% reduction from baseline in number of AKs, was analysed in the same way as the primary response criterion.
The percent reduction at Week 8 from baseline was analyzed using a negative binomial regression for the AK count at Week 8 with treatment group and pooled sites as factors and baseline count as offset variable (using multiple imputations to account for missing values).
The table presents the mean across 1000 multiple imputations. Missing values for AK count were imputed sequentially from a negative binomial regression model with treatment group, AK counts at the previous visit, and analysis site as covariates and log baseline AK count as offset.
Measured: At Week 8

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jan 2014Jul 2014

First posted3 Dec 2013 (estimated)
Study startJan 2014
Primary completionJul 2014
Study completionJul 2014

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Actinic KeratosisStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 266 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01998984
Sponsor's protocol ID: LP0105-1020
Status last verified: Nov 2018
First submitted: 21 Nov 2013
Last updated: 10 Mar 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingPhase 1
A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725
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RecruitingPhase 3
Study of Reformulated Levulan Kerastick Plus PDT for Actinic Keratosis on Upper Extremities
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Study of Reformulated Levulan Kerastick Plus PDT for Actinic Keratosis on Face and Scalp
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Active, not recruitingPhase 3
A Pilot Clinical Trial Comparing Topical Fluorouracil to Fluorouracil Plus Calcipotriene Field Treatments in Patients With Multiple Actinic Keratoses
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RecruitingPhase 3
Basal Cell Carcinoma Chemoprevention Trial
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All Actinic Keratosis trials

Study at a glance

From the registry record, last updated by the sponsor on 10 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Efficacy and Safety of Ingenol Mebutate Gel 0.06% When Applied Once Daily for 2, 3 or 4 Consecutive Days to a Treatment Area of Approximately 250 cm2 on Trunk and Extremities in Subjects With Actinic Keratosis
ClinicalTrials.gov ID: NCT01998984
Acronym / sponsor ID: LP0105-1020
Conditions: Actinic Keratosis
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 266 participants (actual)
Lead sponsor: LEO PharmaIndustry (pharma, biotech, devices)
Study start: Jan 2014
Primary completion: Jul 2014 (actual)
Study completion: Jul 2014 (actual)
First posted: 3 Dec 2013 (estimated)
Results: Posted 7 Dec 2018

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 4

Arm	Type	Receives
2 days placebo and 2 days drug	Experimental	Drug: ingenol mebutate, Other: PlaceboPlacebo and drug
1 day placebo and 3 days drug	Experimental	Drug: ingenol mebutate, Other: PlaceboPlacebo and drug
4 days placebo	Placebo comparator	Other: PlaceboPlacebo
4 days drug	Experimental	Drug: ingenol mebutateDrug

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (1 listed)

Subjects with 5 to 20 clinically typical, visible and discrete AKs within a contiguous area of approximately 250 cm² sun-damaged skin on either trunk (except chest), or extremities

Key exclusion criteria (7 listed)

Location of the treatment area (trunk (except chest) or extremities)
Prior treatment with ingenol mebutate within the selected treatment area
Lesions in the treatment area that have:

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
ingenol mebutate	Drug	1 day placebo and 3 days drug, 2 days placebo and 2 days drug, 4 days drug
Placebo	Other	1 day placebo and 3 days drug, 2 days placebo and 2 days drug, 4 days placebo

Primary outcome

Percentage of Participants With Complete Clearance of Actinic Keratosis Lesions (AKs)
Time frame: At Week 8
Complete clearance of AKs at Week 8 was defined as a 100% reduction from baseline in number of AKs. The table presents the mean across 1000 multiple imputations. Missing values for AK count were imputed sequentially from a negative binomial regression model with treatment group, AK counts at the previous visit, and analysis site as covariates and log baseline AK count as offset.

2 secondary outcomes

Percentage of Reduction in Actinic Keratosis (AK) Lesion Count From Baseline (Day 1) (Multiple Imputation)
Time frame: At Week 8
Percentage of Participants With Partial Clearance of AKs
Time frame: At Week 8

Results and safety

Results posted to the registry on 7 Dec 2018, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Ingenol 2 Days	55	55
Ingenol 3 Days	59	58
Ingenol 4 Days	49	48
Vehicle	61	58

Primary outcome: Percentage of Participants With Complete Clearance of Actinic Keratosis Lesions (AKs)

Time frame: At Week 8. Number. Unit: percentage of participants

Measure	Ingenol 2 Days n = 55	Ingenol 3 Days n = 59	Ingenol 4 Days n = 49	Vehicle n = 61
Value	12.7	5.1	26.8	0.0

Ingenol 3 Days vs Vehicle: Relative risk: 2.97 (95% CI 0.60 to 14.74), p = 0.18, Cochran-Mantel-Haenszel
Ingenol 2 Days vs Vehicle: Relative risk: 3.51 (95% CI 1.00 to 12.41), p = 0.051, Cochran-Mantel-Haenszel
Ingenol 2 Days vs Ingenol 3 Days: Relative risk: 0.47 (95% CI 0.13 to 1.68), p = 0.25, Mantel Haenszel

Population analysed: The analysis was based on the Full Analysis Set, which was defined as all randomized participants.

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: From Day 1 to Week 8 (Day 56). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Ingenol 2 Days	3 of 55 (5.5%)	48 of 55 (87.3%)	0 of 55
Ingenol 3 Days	5 of 59 (8.5%)	55 of 59 (93.2%)	0 of 59
Ingenol 4 Days	4 of 49 (8.2%)	47 of 49 (95.9%)	0 of 49
Vehicle	0 of 61 (0%)	7 of 61 (11.5%)	0 of 61

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Clinical Trials at LEO PharmaLink provided by the sponsor.
Individual participant dataSharing planned: No

Related trials for Actinic Keratosis

Active, not recruitingPhase 1
A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725
Netherton Syndrome
RecruitingPhase 3
Study of Reformulated Levulan Kerastick Plus PDT for Actinic Keratosis on Upper Extremities
Actinic Keratosis
RecruitingPhase 3
Study of Reformulated Levulan Kerastick Plus PDT for Actinic Keratosis on Face and Scalp
Actinic Keratosis
Active, not recruitingPhase 3
A Pilot Clinical Trial Comparing Topical Fluorouracil to Fluorouracil Plus Calcipotriene Field Treatments in Patients With Multiple Actinic Keratoses
Multiple Actinic Keratoses
RecruitingPhase 3
Basal Cell Carcinoma Chemoprevention Trial
Basal Cell Carcinoma

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Daniel M Siegel, MD, MS principal investigator

Lond Island Skin Cancer and Dermatologic Surgery

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ingenol mebutate in people with Actinic Keratosis, run by LEO Pharma at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 1 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 10 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 7 Dec 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of Participants With Complete Clearance of Actinic Keratosis Lesions (AKs) (time frame: At Week 8). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01998984.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Mar 2025.