Actinic KeratosisPhase 1/2NCT02100813

Safety and Efficacy of Doses of LEO 43204 Once Daily for Two Consecutive Days on Full Balding Scalp in Subjects With Actinic Keratosis

A Phase 1/2 study testing LEO 43204 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.

Trial summary

To identify the Maximum Tolerated Dose levels of LEO 43204 after once daily treatment for two consecutive days and to evaluate efficacy of LEO 43204 in two doses after once daily treatment for two consecutive days compared to vehicle 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?: LEO 43204
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 220 participants
Where: 1 siteUnited States
Study dates: May 2014to Mar 2015
Phase: Phase 1/2
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

Part 1: Subjects with 5 to 20 clinically typical, visible and discrete AKs on the full balding scalp.
Part 2: Subjects with 5 to 20 clinically typical, visible and discrete AKs on the full balding scalp

Who cannot join

Location of the treatment area
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 gel on the treatment area
Lesions in the treatment areas 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

Gwinnett Clinical Research Center, Inc.
Snellville, Georgia, 30078

Other trials in United States

About this study

Official title: Safety and Efficacy of Escalating Doses of LEO 43204 Applied Once Daily for Two Consecutive Days on Full Balding Scalp in Subjects With Actinic Keratosis

Conditions: Actinic Keratosis
Being tested: LEO 43204, 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.

LEO 43204Drug
PlaceboDrug

Study groups in NCT02100813
Study group	Role	What they receive
Part 1: LEO 43204	Other	Open-label, dose escalation, 2 days treatment Receives: Drug: LEO 43204
Part 2: LEO 43204 x dose	Active comparator	X dose for 2 days treatment Receives: Drug: LEO 43204
Part 2: LEO 43204 Y dose	Active comparator	Y dose for 2 days treatment Receives: Drug: LEO 43204
Part 2: Placebo	Placebo comparator	Placebo for 2 days treatment Receives: Drug: Placebo

What is being measured

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

Part 1: Number of Participants Experiencing a Dose-limiting Toxicity (DLT)
The number participants experiencing a DLT was used to identify the maximum tolerated dose (MTD) of LEO 43204 after once daily treatment for 2 consecutive days.The MTD was defined as the highest dose level with less than 4 out of 12 participants experiencing a DLT.
A DLT was defined as:
- Erosion/ulceration Grade 4 on the Local Skin Response (LSR) scale
- Other clinically relevant signs or symptoms observed, which the International Co-ordinating Investigator judges to be counted as a DLT.
The Local Skin Responses consists of the following 6 categories: Erythema, flaking/scaling, crusting, swelling, vesiculation/pustulation, erosion/ulceration. Each individual LSR category are given a numeric grade of severity from 0-4. Grade 0 being no presence and Grade 4 being the highest grade of severity.
Measured: From Day 1 up to and including Day 8
Part 2: Percent Reduction From Baseline in Actinic Keratosis (AK) Lesion Count
Percent reduction from baseline in clinically visible actinic keratosis lesions (AKs) in the selected treatment area.
Measured: From baseline to Week 8

Secondary outcomes (2)

Part 2: Participants With Complete Clearance of AKs
Complete clearance of AKs at Week 8 was defined as a 100% reduction from baseline in AK count.
Measured: From baseline to Week 8
Part 2: Participants With Partial Clearance of AKs
Partial clearance of AKs at Week 8 was defined as at least 75% reduction from baseline in AK count.
Measured: From baseline to Week 8

Timeline

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

May 2014Mar 2015

First posted1 Apr 2014 (estimated)
Study startMay 2014
Primary completionMar 2015
Study completionMar 2015

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 1/2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 220 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02100813
Sponsor's protocol ID: LP0084-1014
Other IDs: 2014-000037-23, 2014
Status last verified: Nov 2018
First submitted: 27 Mar 2014
Last updated: 6 Mar 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
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

RecruitingPhase 3
Study of Reformulated Levulan Kerastick Plus PDT for Actinic Keratosis on Face and Scalp
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RecruitingPhase 3
Basal Cell Carcinoma Chemoprevention Trial
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Active, not recruitingPhase 4
Cryotherapy in Combination With Red Light PDT for Actinic Keratosis of Full Face
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Not yet recruiting
Therapeutic Induction of Dermal Remodeling in Veterans to Prevent Actinic Neoplasia Caused by Chronic Environmental Exposure to Ultraviolet Radiation
Nonmelanoma Skin Cancer, Actinic Keratosis

All Actinic Keratosis trials

Study at a glance

From the registry record, last updated by the sponsor on 6 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Safety and Efficacy of Escalating Doses of LEO 43204 Applied Once Daily for Two Consecutive Days on Full Balding Scalp in Subjects With Actinic Keratosis
ClinicalTrials.gov ID: NCT02100813
Acronym / sponsor ID: LP0084-1014
Conditions: Actinic Keratosis
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 220 participants (actual)
Lead sponsor: LEO PharmaIndustry (pharma, biotech, devices)
Study start: May 2014
Primary completion: Mar 2015 (actual)
Study completion: Mar 2015 (actual)
First posted: 1 Apr 2014 (estimated)
Results: Posted 18 Feb 2019

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: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 4

Arm	Type	Receives
Part 1: LEO 43204	Other	Drug: LEO 43204Open-label, dose escalation, 2 days treatment
Part 2: LEO 43204 x dose	Active comparator	Drug: LEO 43204X dose for 2 days treatment
Part 2: LEO 43204 Y dose	Active comparator	Drug: LEO 43204Y dose for 2 days treatment
Part 2: Placebo	Placebo comparator	Drug: PlaceboPlacebo for 2 days treatment

Who may qualify

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

Key inclusion criteria (2 listed)

Part 1: Subjects with 5 to 20 clinically typical, visible and discrete AKs on the full balding scalp.
Part 2: Subjects with 5 to 20 clinically typical, visible and discrete AKs on the full balding scalp

Key exclusion criteria (7 listed)

Location of the treatment area
Prior treatment with ingenol mebutate gel on the treatment area
Lesions in the treatment areas 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
LEO 43204	Drug	Part 1: LEO 43204, Part 2: LEO 43204 Y dose, Part 2: LEO 43204 x dose
Placebo	Drug	Part 2: Placebo

Primary outcome

Part 1: Number of Participants Experiencing a Dose-limiting Toxicity (DLT)
Time frame: From Day 1 up to and including Day 8
The number participants experiencing a DLT was used to identify the maximum tolerated dose (MTD) of LEO 43204 after once daily treatment for 2 consecutive days.The MTD was defined as the highest dose level with less than 4 out of 12 participants experiencing a DLT. A DLT was defined as: * Erosion/ulceration Grade 4 on the Local Skin Response (LSR) scale * Other clinically relevant signs or symptoms observed, which the International Co-ordinating Investigator judges to be counted as a DLT. The Local Skin Responses consists of the following 6 categories: Erythema, flaking/scaling, crusting, swelling, vesiculation/pustulation, erosion/ulceration. Each individual LSR category are given a numeric grade of severity from 0-4. Grade 0 being no presence and Grade 4 being the highest grade of severity.
Part 2: Percent Reduction From Baseline in Actinic Keratosis (AK) Lesion Count
Time frame: From baseline to Week 8
Percent reduction from baseline in clinically visible actinic keratosis lesions (AKs) in the selected treatment area.

2 secondary outcomes

Part 2: Participants With Complete Clearance of AKs
Time frame: From baseline to Week 8
Part 2: Participants With Partial Clearance of AKs
Time frame: From baseline to Week 8

Results and safety

Results posted to the registry on 18 Feb 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Part 1 - 0.018% Cohort	10	10
Part 1 - 0.025% Cohort	11	11
Part 1 - 0.037% Cohort	12	12
Part 1 - 0.05% Cohort	12	12
Part 1 - 0.075% Cohort	12	12
Part 2 - Vehicle	32	32
Part 2 - 0.037%	64	64
Part 2 - 0.05%	67	66

Primary outcome: Part 1: Number of Participants Experiencing a Dose-limiting Toxicity (DLT)

Time frame: From Day 1 up to and including Day 8. Count of participants. Unit: Participants

Measure	Part 1 - 0.018% Cohort n = 10	Part 1 - 0.025% Cohort n = 11	Part 1 - 0.037% Cohort n = 12	Part 1 - 0.05% Cohort n = 12	Part 1 - 0.075% Cohort n = 12
Yes	0	0	0	0	0
No	10	11	12	12	12

Primary outcome: Part 2: Percent Reduction From Baseline in Actinic Keratosis (AK) Lesion Count

Time frame: From baseline to Week 8. Mean (95% Confidence Interval). Unit: percentage of reduction

Measure	Part 2 - Vehicle n = 32	Part 2 - 0.037% n = 64	Part 2 - 0.05% n = 67
Value	12.6 (-9.5 to 30.3)	72.7 (66.9 to 77.5)	78.5 (73.7 to 82.5)

Part 2 - Vehicle vs Part 2 - 0.037%: Rate ratio: 0.31 (95% CI 0.23 to 0.42), p <0.001, Negative binomial regression
Part 2 - Vehicle vs Part 2 - 0.05%: Rate ratio: 0.25 (95% CI 0.18 to 0.33), p <0.001, Negative binomial regression
Part 2 - 0.037% vs Part 2 - 0.05%: Rate ratio: 0.79 (95% CI 0.59 to 1.04), p =0.096, Negative binomial regression

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: Part 1: From Day 1 to Week 2 Part 2: From Day 1 to Week 8. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Part 1 - 0.018% Cohort	0 of 10 (0%)	7 of 10 (70%)	Not reported
Part 1 - 0.025% Cohort	0 of 11 (0%)	7 of 11 (63.6%)	Not reported
Part 1 - 0.037% Cohort	0 of 12 (0%)	12 of 12 (100%)	Not reported
Part 1 - 0.05% Cohort	0 of 12 (0%)	10 of 12 (83.3%)	Not reported
Part 1 - 0.075% Cohort	0 of 12 (0%)	12 of 12 (100%)	Not reported
Part 2 - Vehicle	0 of 32 (0%)	4 of 32 (12.5%)	Not reported
Part 2 - 0.037%	2 of 64 (3.1%)	47 of 64 (73.4%)	Not reported
Part 2 - 0.05%	3 of 67 (4.5%)	50 of 67 (74.6%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 6 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

RecruitingPhase 3
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RecruitingPhase 3
Study of Reformulated Levulan Kerastick Plus PDT for Actinic Keratosis on Upper Extremities
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Active, not recruitingPhase 4
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Not yet recruiting
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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.

Jonathan Weiss, MD principal investigator

Gwinnett Clinical Research Center, Inc.

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

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing LEO 43204 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 2 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 6 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 18 Feb 2019). 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?

Part 1: Number of Participants Experiencing a Dose-limiting Toxicity (DLT) (time frame: From Day 1 up to and including Day 8). The study lists 2 primary outcomes in all. 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 NCT02100813.

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 6 Mar 2025; AzTrials last refreshed its copy on 8 Nov 2026.