Trial Outcomes & Findings for Patient and Clinician Reported Outcomes for Tirbanibulin Effectiveness and Safety in Actinic Keratosis (NCT NCT05260073)

Last Updated: 2024-09-20

Results Overview

Skindex-16 is a validated dermatological instrument to measure dermatology-specific Health-Related Quality of Life (HRQoL). The Skindex-16 consisted of 16 items that were divided into three domains: Symptoms (four items, range 0-24), Emotions (seven items, range 0-42), and Functioning (five items, range 0-30). Participant were asked to respond on how much their skin condition bothered them in the week prior to administration of the Skindex-16. Each item was scored on a scale ranged from 0 (never bothered) to 6 (always bothered), where higher score indicated continued/more botheration. Item scores are transformed to 0 to 100 scale, and domain scores are calculated as the average of the item scores comprising the domain. Net positive changes in respective subscale scoring indicates improvement in that particular Quality of life assessment (i.e., Symptoms, Emotions, Functioning), while net negative changes in scoring indicates decrease in that particular Quality of life assessment.

Recruitment status

COMPLETED

Target enrollment

300 participants

Primary outcome timeframe

Baseline, Week 8

Results posted on

2024-09-20

Participant Flow

This study was conducted at 32 sites in the United States from 9 March 2022 to 7 March 2023.

A total of 300 participants were enrolled, out of which 290 participants who completed the week 8 study survey were included in the full analysis set.

Participant milestones

Participant milestones
Measure	Tirbanibulin Participants with actinic keratosis (AK) applied tirbanibulin ointment 1% topically once daily using 1 single-dose packet (2.5 milligrams \[mg\] in 250 mg) per application, for 5 consecutive days were observed for 24 weeks to gather participant reported outcomes (PROs) and clinical profile.
Overall Study STARTED	300
Overall Study Full Analysis Set	290
Overall Study COMPLETED	279
Overall Study NOT COMPLETED	21

Reasons for withdrawal

Reasons for withdrawal
Measure	Tirbanibulin Participants with actinic keratosis (AK) applied tirbanibulin ointment 1% topically once daily using 1 single-dose packet (2.5 milligrams \[mg\] in 250 mg) per application, for 5 consecutive days were observed for 24 weeks to gather participant reported outcomes (PROs) and clinical profile.
Overall Study Physician Decision	1
Overall Study Withdrawal by Subject	6
Overall Study Lost to Follow-up	14

Baseline Characteristics

Patient and Clinician Reported Outcomes for Tirbanibulin Effectiveness and Safety in Actinic Keratosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Tirbanibulin n=290 Participants Participants with AK applied tirbanibulin ointment 1% topically once daily using 1 single-dose packet (2.5 mg in 250 mg) per application, for 5 consecutive days were observed for 24 weeks to gather PROs and clinical profile.
Age, Continuous	66.30 years STANDARD_DEVIATION 11.38 • n=5 Participants
Sex: Female, Male Female	91 Participants n=5 Participants
Sex: Female, Male Male	199 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	21 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	269 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · White	285 Participants n=5 Participants
Race/Ethnicity, Customized Race · Black or African American	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · American Indian or Alaska Native	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · Asian	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race/Ethnicity, Customized Race · Other	2 Participants n=5 Participants
Race/Ethnicity, Customized Race · Prefer not to answer	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 8

Population: FAS included all participants who received at least one dose of tirbanibulin during the study observation period of 8-weeks related to primary endpoint, as part of usual care and had at least some data pertaining to the key variables studied at relevant timepoints, post-baseline. Here, "number analyzed" signifies participants who were evaluable at each specified category.

Outcome measures

Outcome measures
Measure	Tirbanibulin n=290 Participants Participants with AK applied tirbanibulin ointment 1% topically once daily using 1 single-dose packet (2.5 mg in 250 mg) per application, for 5 consecutive days were observed for 24 weeks to gather PROs and clinical profile.
Change From Baseline in Domain Scores Using the Patient Reported Outcome Skindex-16 Instrument at Week 8 Symptoms Domain Score: Baseline	22.30 score on a scale Standard Deviation 22.36
Change From Baseline in Domain Scores Using the Patient Reported Outcome Skindex-16 Instrument at Week 8 Symptoms Domain Score: Change at Week 8	-14.26 score on a scale Standard Deviation 27.77
Change From Baseline in Domain Scores Using the Patient Reported Outcome Skindex-16 Instrument at Week 8 Emotions Domain Score: Baseline	38.17 score on a scale Standard Deviation 27.30
Change From Baseline in Domain Scores Using the Patient Reported Outcome Skindex-16 Instrument at Week 8 Emotions Domain Score: Change at Week 8	-24.88 score on a scale Standard Deviation 32.96
Change From Baseline in Domain Scores Using the Patient Reported Outcome Skindex-16 Instrument at Week 8 Functioning Domain Score: Baseline	14.41 score on a scale Standard Deviation 20.06
Change From Baseline in Domain Scores Using the Patient Reported Outcome Skindex-16 Instrument at Week 8 Functioning Domain Score: Change at Week 8	-9.78 score on a scale Standard Deviation 23.66

SECONDARY outcome

Timeframe: At Week 8

IGA success was defined as achieving a rating of 'completely cleared' (0) or 'partially cleared' (1) in IGA of AK status at Week 8. The IGA is 5-point scale that assessed the severity of actinic keratosis, with scores ranged from 0 (completely clear) to 4 (not cleared): 0 = completely cleared (100% clearance); 1 = partially cleared (\>=75% clearance); 2 = moderately cleared (50% to 74% clearance); 3 = minimally cleared (\<50% clearance); 4 = not cleared (0% clearance), where higher score indicated higher severity.

Outcome measures

Outcome measures
Measure	Tirbanibulin n=290 Participants Participants with AK applied tirbanibulin ointment 1% topically once daily using 1 single-dose packet (2.5 mg in 250 mg) per application, for 5 consecutive days were observed for 24 weeks to gather PROs and clinical profile.
Percentage of Participants With Investigator Global Assement (IGA) Success at Week 8	73.79 percentage of participants

Adverse Events

Tirbanibulin

Serious events: 6 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Tirbanibulin n=300 participants at risk Participants with AK applied tirbanibulin ointment 1% topically once daily using 1 single-dose packet (2.5 mg in 250 mg) per application, for 5 consecutive days were observed for 24 weeks to gather PROs and clinical profile.
Injury, poisoning and procedural complications Fall	0.33% 1/300 • From start of study up to end of the study (Week 24) Safety population included all participants who received at least one dose of tirbanibulin during the study observation period of 8-weeks related to primary endpoint, as part of usual care.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Basal Cell Carcinoma	0.67% 2/300 • From start of study up to end of the study (Week 24) Safety population included all participants who received at least one dose of tirbanibulin during the study observation period of 8-weeks related to primary endpoint, as part of usual care.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Bowen's Disease	0.33% 1/300 • From start of study up to end of the study (Week 24) Safety population included all participants who received at least one dose of tirbanibulin during the study observation period of 8-weeks related to primary endpoint, as part of usual care.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Squamous Cell Carcinoma	1.0% 3/300 • From start of study up to end of the study (Week 24) Safety population included all participants who received at least one dose of tirbanibulin during the study observation period of 8-weeks related to primary endpoint, as part of usual care.
Respiratory, thoracic and mediastinal disorders Pneumothorax	0.33% 1/300 • From start of study up to end of the study (Week 24) Safety population included all participants who received at least one dose of tirbanibulin during the study observation period of 8-weeks related to primary endpoint, as part of usual care.

Other adverse events

Other adverse events
Measure	Tirbanibulin n=300 participants at risk Participants with AK applied tirbanibulin ointment 1% topically once daily using 1 single-dose packet (2.5 mg in 250 mg) per application, for 5 consecutive days were observed for 24 weeks to gather PROs and clinical profile.
Infections and infestations COVID-19	0.67% 2/300 • From start of study up to end of the study (Week 24) Safety population included all participants who received at least one dose of tirbanibulin during the study observation period of 8-weeks related to primary endpoint, as part of usual care.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Basal Cell Carcinoma	0.67% 2/300 • From start of study up to end of the study (Week 24) Safety population included all participants who received at least one dose of tirbanibulin during the study observation period of 8-weeks related to primary endpoint, as part of usual care.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Squamous Cell Carcinoma	1.3% 4/300 • From start of study up to end of the study (Week 24) Safety population included all participants who received at least one dose of tirbanibulin during the study observation period of 8-weeks related to primary endpoint, as part of usual care.
Vascular disorders Capillary fragility	0.33% 1/300 • From start of study up to end of the study (Week 24) Safety population included all participants who received at least one dose of tirbanibulin during the study observation period of 8-weeks related to primary endpoint, as part of usual care.
Skin and subcutaneous tissue disorders Angioedema	0.33% 1/300 • From start of study up to end of the study (Week 24) Safety population included all participants who received at least one dose of tirbanibulin during the study observation period of 8-weeks related to primary endpoint, as part of usual care.

Additional Information

Head of Global Clinical Development

Almirall S.A.

Phone: +34932913000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place