Trial Outcomes & Findings for A Study of ATI-50002 Topical Solution for the Treatment of Vitiligo (NCT NCT03468855)

Last Updated: 2020-11-30

Results Overview

Mean change from baseline in facial depigmentation in quantified Area of Interest (AOI) using Canfield-2-D photographic image analysis from baseline (Visit 2) compared to Week 24. Facial depigmentation is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

34 participants

Primary outcome timeframe

Baseline to 24 Weeks

Results posted on

2020-11-30

Participant Flow

Participant milestones

Participant milestones
Measure	ATI-50002 0.46% Topical Solution ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Overall Study STARTED	34
Overall Study COMPLETED	23
Overall Study NOT COMPLETED	11

Reasons for withdrawal

Reasons for withdrawal
Measure	ATI-50002 0.46% Topical Solution ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Overall Study Adverse Event	2
Overall Study Lost to Follow-up	5
Overall Study Withdrawal by Subject	3
Overall Study Inclusion/Exclusion Enrollment Violator	1

Baseline Characteristics

A Study of ATI-50002 Topical Solution for the Treatment of Vitiligo

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	ATI-50002 0.46% Topical Solution n=34 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Age, Continuous	45.3 years STANDARD_DEVIATION 13.45 • n=93 Participants
Sex: Female, Male Female	18 Participants n=93 Participants
Sex: Female, Male Male	16 Participants n=93 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	9 Participants n=93 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	20 Participants n=93 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	5 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	3 Participants n=93 Participants
Race (NIH/OMB) White	27 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=93 Participants
Region of Enrollment United States	34 participants n=93 Participants
Fitzpatrick Skin Type I - Always Burns	0 Participants n=93 Participants
Fitzpatrick Skin Type II - Burns Easily	5 Participants n=93 Participants
Fitzpatrick Skin Type III - Burns Moderately	16 Participants n=93 Participants
Fitzpatrick Skin Type IV - Burns Minimally	9 Participants n=93 Participants
Fitzpatrick Skin Type V - Rarely Burns	3 Participants n=93 Participants
Fitzpatrick Skin Type VI - Never Burns	1 Participants n=93 Participants
Duration of Vitiligo Disease (years) (n)	13.6 years STANDARD_DEVIATION 10.47 • n=93 Participants
Duration of Current Episode (years) (n)	3.9 years STANDARD_DEVIATION 7.24 • n=93 Participants

PRIMARY outcome

Timeframe: Baseline to 24 Weeks

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=34 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in Facial Depigmentation in the AOI (Area of Interest) Based on Canfield 2-D Photographic Analysis From Baseline to Week 24	2.0 score on a scale Standard Deviation 8.41

SECONDARY outcome

Timeframe: Baseline to 48 Weeks

Population: Subjects completing 24 weeks of treatment have the option to extend treatment for an additional 24 weeks. Subjects not wishing to continue treatment can withdraw consent.

Mean change from baseline in facial depigmentation in quantified Area of Interest (AOI) using Canfield-2-D photographic image analysis from baseline (Visit 2) compared to Week 48. Facial depigmentation is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=16 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in Facial Depigmentation in the AOI (Area of Interest) Based on Canfield 2-D Photographic Analysis From Baseline Compared to Week 48	-1.4 score on a scale Standard Deviation 15.11

SECONDARY outcome

Timeframe: Baseline to 4 Weeks

Mean change from baseline in facial depigmentation in quantified Area of Interest (AOI) using Canfield-2-D photographic image analysis from baseline (Visit 2) compared to Week 4. Facial depigmentation is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=34 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in Facial Depigmentation in the AOI (Area of Interest) Based on Canfield 2-D Photographic Analysis From Baseline Compared to Week 4	0.2 score on a scale Standard Deviation 4.47

SECONDARY outcome

Timeframe: Baseline to 8 Weeks

Mean change from baseline in facial depigmentation in quantified Area of Interest (AOI) using Canfield-2-D photographic image analysis from baseline (Visit 2) compared to Week 8. Facial depigmentation is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=34 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in Facial Depigmentation in the AOI (Area of Interest) Based on Canfield 2-D Photographic Analysis From Baseline Compared to Week 8	0.5 score on a scale Standard Deviation 4.82

SECONDARY outcome

Timeframe: Baseline to 12 Weeks

Mean change from baseline in facial depigmentation in quantified Area of Interest (AOI) using Canfield-2-D photographic image analysis from baseline (Visit 2) compared to Week 12. Facial depigmentation is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=34 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in Facial Depigmentation in the AOI (Area of Interest) Based on Canfield 2-D Photographic Analysis From Baseline Compared to Week 12	0.3 score on a scale Standard Deviation 9.04

SECONDARY outcome

Timeframe: Baseline to 16 Weeks

Mean change from baseline in facial depigmentation in quantified Area of Interest (AOI) using Canfield-2-D photographic image analysis from baseline (Visit 2) compared to Week 4. Facial depigmentation is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=34 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in Facial Depigmentation in the AOI (Area of Interest) Based on Canfield 2-D Photographic Analysis From Baseline Compared to Week 16	-0.8 score on a scale Standard Deviation 8.20

SECONDARY outcome

Timeframe: Baseline to 20 Weeks

Mean change from baseline in facial depigmentation in quantified Area of Interest (AOI) using Canfield-2-D photographic image analysis from baseline (Visit 2) compared to Week 20. Facial depigmentation is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=34 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in Facial Depigmentation in the AOI (Area of Interest) Based on Canfield 2-D Photographic Analysis From Visit 2 Compared to Week 20	1.3 score on a scale Standard Deviation 9.22

SECONDARY outcome

Timeframe: Baseline to 32 Weeks

Population: Subjects completing 24 weeks of treatment have the option to extend treatment for an additional 24 weeks. Subjects not wishing to continue treatment can withdraw consent.

Mean change from baseline in facial depigmentation in quantified Area of Interest (AOI) using Canfield-2-D photographic image analysis from baseline (Visit 2) compared to Week 4. Facial depigmentation is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=16 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in Facial Depigmentation in the AOI (Area of Interest) Based on Canfield 2-D Photographic Analysis From Baseline Compared to Week 32	4.2 score on a scale Standard Deviation 9.09

SECONDARY outcome

Timeframe: Baseline to 40 Weeks

Population: Subjects completing 24 weeks of treatment have the option to extend treatment for an additional 24 weeks. Subjects not wishing to continue treatment can withdraw consent.

Mean change from baseline in facial depigmentation in quantified Area of Interest (AOI) using Canfield-2-D photographic image analysis from baseline (Visit 2) compared to Week 40. Facial depigmentation is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=16 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in Facial Depigmentation in the AOI (Area of Interest) Based on Canfield 2-D Photographic Analysis From Baseline Compared to Week 40	1.9 score on a scale Standard Deviation 12.14

SECONDARY outcome

Timeframe: Baseline to 52 Weeks

Population: Subjects completing 24 weeks of treatment have the option to extend treatment for an additional 24 weeks. Subjects not wishing to continue treatment can withdraw consent.

Mean change from baseline in facial depigmentation in quantified Area of Interest (AOI) using Canfield-2-D photographic image analysis from baseline (Visit 2) compared to Week 52. Facial depigmentation is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=16 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in Facial Depigmentation in the AOI (Area of Interest) Based on Canfield 2-D Photographic Analysis From Baseline Compared to Post-treatment Week 52	-1.4 score on a scale Standard Deviation 13.29

SECONDARY outcome

Timeframe: Baseline to 4 Weeks

The Facial Vitiligo Area Scoring Index (F-VASI) is an assessment of the subject's facial vitiligo modified from the Vitiligo Area Scoring Index for the face only at week 4. F-VASI is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=34 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in the Facial Assessment of the Vitiligo Area Scoring Index (F-VASI) Compared to Week 4	-0.023 score on a scale Standard Deviation 0.0946

SECONDARY outcome

Timeframe: Baseline to 8 Weeks

The Facial Vitiligo Area Scoring Index (F-VASI) is an assessment of the subject's facial vitiligo modified from the Vitiligo Area Scoring Index for the face only at Week 8. F-VASI is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=34 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in the Facial Assessment of the Vitiligo Area Scoring Index (F-VASI) Compared to Week 8	-0.043 score on a scale Standard Deviation 0.1454

SECONDARY outcome

Timeframe: Baseline to 12 Weeks

The Facial Vitiligo Area Scoring Index (F-VASI) is an assessment of the subject's facial vitiligo modified from the Vitiligo Area Scoring Index for the face only at week 12. F-VASI is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=34 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in the Facial Assessment of the Vitiligo Area Scoring Index (F-VASI) Compared to Week 12	-0.071 score on a scale Standard Deviation 0.2049

SECONDARY outcome

Timeframe: Week 16

The Facial Vitiligo Area Scoring Index (F-VASI) is an assessment of the subject's facial vitiligo modified from the Vitiligo Area Scoring Index for the face only at week 16. F-VASI is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=34 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in the Facial Assessment of the Vitiligo Area Scoring Index (F-VASI) Compared to Week 16	-0.065 score on a scale Standard Deviation 0.2116

SECONDARY outcome

Timeframe: Baseline to 20 Weeks

The Facial Vitiligo Area Scoring Index (F-VASI) is an assessment of the subject's facial vitiligo modified from the Vitiligo Area Scoring Index for the face only at week 20. F-VASI is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=34 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in the Facial Assessment of the Vitiligo Area Scoring Index (F-VASI) Compared to Week 20	-0.070 score on a scale Standard Deviation 0.2063

SECONDARY outcome

Timeframe: Baseline to 24 Weeks

The Facial Vitiligo Area Scoring Index (F-VASI) is an assessment of the subject's facial vitiligo modified from the Vitiligo Area Scoring Index for the face only at week 24. F-VASI is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=34 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in the Facial Assessment of the Vitiligo Area Scoring Index (F-VASI) Compared to Week 24	-0.067 score on a scale Standard Deviation 0.2411

SECONDARY outcome

Timeframe: Baseline to 32 Weeks

Population: Subjects completing 24 weeks of treatment have the option to extend treatment for an additional 24 weeks. Subjects not wishing to continue treatment can withdraw consent.

The Facial Vitiligo Area Scoring Index (F-VASI) is an assessment of the subject's facial vitiligo modified from the Vitiligo Area Scoring Index for the face only at week 32. F-VASI is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=16 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in the Facial Assessment of the Vitiligo Area Scoring Index (F-VASI) Compared to Week 32	-0.004 score on a scale Standard Deviation 0.1945

SECONDARY outcome

Timeframe: Baseline to 40 Weeks

Population: Subjects completing 24 weeks of treatment have the option to extend treatment for an additional 24 weeks. Subjects not wishing to continue treatment can withdraw consent.

The Facial Vitiligo Area Scoring Index (F-VASI) is an assessment of the subject's facial vitiligo modified from the Vitiligo Area Scoring Index for the face only at week 40. F-VASI is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=16 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in the Facial Assessment of the Vitiligo Area Scoring Index (F-VASI) Compared to Week 40	-0.076 score on a scale Standard Deviation 0.3567

SECONDARY outcome

Timeframe: Baseline to 48 Weeks

Population: Subjects completing 24 weeks of treatment have the option to extend treatment for an additional 24 weeks. Subjects not wishing to continue treatment can withdraw consent.

The Facial Vitiligo Area Scoring Index (F-VASI) is an assessment of the subject's facial vitiligo modified from the Vitiligo Area Scoring Index for the face only at week 48. F-VASI is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=16 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in the Facial Assessment of the Vitiligo Area Scoring Index (F-VASI) Compared to Week 48	-0.121 score on a scale Standard Deviation 0.4062

SECONDARY outcome

Timeframe: Baseline to 52 Weeks

Population: Subjects completing 24 weeks of treatment have the option to extend treatment for an additional 24 weeks. Subjects not wishing to continue treatment can withdraw consent.

The Facial Vitiligo Area Scoring Index (F-VASI) is an assessment of the subject's facial vitiligo modified from the Vitiligo Area Scoring Index for the face only at week 52. F-VASI is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=16 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in the Facial Assessment of the Vitiligo Area Scoring Index (F-VASI) Compared to Post-treatment Week 52	-0.158 score on a scale Standard Deviation 0.4785

SECONDARY outcome

Timeframe: Baseline to 4 Weeks

The Vitiligo Noticeability Scale (VNS) represents the subject's assessment of the noticeability of facial vitiligo during the study treatment period by means of answering the question, "Compared to before treatment, how noticeable is the vitiligo now?" Subject response options include, "1: More Noticeable", "2: As Noticeable", "3: Slightly Less Noticeable", "4: A Lot Less Noticeable", and "5: No Longer Noticeable". Treatment success will be defined as follows: VNS score of 1 or 2 = treatment not successful, VNS score of 3 = treatment partially successful, or VNS score of 4 or 5 = treatment successful. Higher scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=29 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Treatment Success Based on the Subject's Assessment of Vitiligo on the Vitiligo Noticeability Scale (VNS) at Week 4	2.1 score on a scale Standard Deviation 0.58

SECONDARY outcome

Timeframe: Baseline to 8 Weeks

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=27 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Treatment Success Based on the Subject's Assessment of Vitiligo on the Vitiligo Noticeability Scale (VNS) at Week 8	2.1 score on a scale Standard Deviation 0.58

SECONDARY outcome

Timeframe: Baseline to 12 Weeks

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=27 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Treatment Success Based on the Subject's Assessment of Vitiligo on the Vitiligo Noticeability Scale (VNS) at Week 12	2.1 score on a scale Standard Deviation 0.68

SECONDARY outcome

Timeframe: Baseline to 16 Weeks

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=25 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Treatment Success Based on the Subject's Assessment of Vitiligo on the Vitiligo Noticeability Scale (VNS) at Week 16	2.1 score on a scale Standard Deviation 0.67

SECONDARY outcome

Timeframe: Baseline to 20 Weeks

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=24 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Treatment Success Based on the Subject's Assessment of Vitiligo on the Vitiligo Noticeability Scale (VNS) at Week 20	2.3 score on a scale Standard Deviation 0.53

SECONDARY outcome

Timeframe: Baseline to 24 Weeks

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=24 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Treatment Success Based on the Subject's Assessment of Vitiligo on the Vitiligo Noticeability Scale (VNS) at Week 24	2.2 score on a scale Standard Deviation 0.66

SECONDARY outcome

Timeframe: Baseline to 32 Weeks

Population: Subjects completing 24 weeks of treatment have the option to extend treatment for an additional 24 weeks. Subjects not wishing to continue treatment can withdraw consent.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=14 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Treatment Success Based on the Subject's Assessment of Vitiligo on the Vitiligo Noticeability Scale (VNS) at Week 32	2.1 score on a scale Standard Deviation 0.73

SECONDARY outcome

Timeframe: Baseline to 40 Weeks

Population: Subjects completing 24 weeks of treatment have the option to extend treatment for an additional 24 weeks. Subjects not wishing to continue treatment can withdraw consent.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=14 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Treatment Success Based on the Subject's Assessment of Vitiligo on the Vitiligo Noticeability Scale (VNS) at Week 40	2.4 score on a scale Standard Deviation 0.74

SECONDARY outcome

Timeframe: Baseline to 48 Weeks

Population: Subjects completing 24 weeks of treatment have the option to extend treatment for an additional 24 weeks. Subjects not wishing to continue treatment can withdraw consent.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=14 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Treatment Success Based on the Subject's Assessment of Vitiligo on the Vitiligo Noticeability Scale (VNS) at Week 48	2.4 score on a scale Standard Deviation 1.01

SECONDARY outcome

Timeframe: Baseline to 24 Weeks

The Vitiligo Area Scoring Index (VASI) is an assessment of the subject's Vitiligo Area Scoring Index. VASI is measured on a scale of 0 to 100, with a score of 0 meaning no depigmentation and a score of 100 meaning total depigmentation. Lower scores represent better outcomes.

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=34 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in Total VASI at Week 24	0.340 score on a scale Standard Deviation 1.4829

SECONDARY outcome

Timeframe: Baseline to 48 Weeks

Outcome measures

Outcome measures
Measure	ATI-50002 0.46% Topical Solution n=34 Participants ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Mean Change From Baseline in Total VASI at Week 48	0.008 score on a scale Standard Deviation 1.5341

Adverse Events

ATI-50002 0.46% Topical Solution

Serious events: 2 serious events

Other events: 23 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	ATI-50002 0.46% Topical Solution n=34 participants at risk ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Cardiac disorders Acute myocardial infarction	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Gastrointestinal disorders Alcoholic pancreatitis	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).

Other adverse events

Other adverse events
Measure	ATI-50002 0.46% Topical Solution n=34 participants at risk ATI-50002 0.46% topical solution, high dose active, twice-daily, 24 weeks ATI-50002 0.46% topical solution: Topical Solution administered twice daily
Cardiac disorders Coronary artery disease	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Cardiac disorders Ventricular tachycardia	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Gastrointestinal disorders Gastrooesophageal reflux disease	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
General disorders Application site acne	5.9% 2/34 • Number of events 3 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
General disorders Application site discolouration	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
General disorders Application site dryness	17.6% 6/34 • Number of events 7 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
General disorders Application site pain	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
General disorders Application site rash	5.9% 2/34 • Number of events 2 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Infections and infestations Gastroenteritis	2.9% 1/34 • Number of events 2 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Infections and infestations Gastroenteritis viral	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Infections and infestations Influenza	5.9% 2/34 • Number of events 2 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Infections and infestations Nasopharyngitis	14.7% 5/34 • Number of events 6 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Infections and infestations Oral herpes	2.9% 1/34 • Number of events 2 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Infections and infestations Pharyngitis	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Infections and infestations Subcutaneous abscess	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Infections and infestations Upper respiratory tract infection	5.9% 2/34 • Number of events 2 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Infections and infestations Urinary tract infection	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Infections and infestations Viral infection	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Injury, poisoning and procedural complications Contusion	2.9% 1/34 • Number of events 2 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Injury, poisoning and procedural complications Meniscus injury	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Investigations Alanine aminotransferase increased	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Investigations Blood cholesterol increased	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Investigations Blood creatine phosphokinase increased	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Investigations Blood pressure systolic increased	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Investigations Blood thyroid stimulating hormone decreased	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Investigations Blood triglycerides increased	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Investigations Blood urine present	5.9% 2/34 • Number of events 2 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Investigations Eosinophil count increased	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Investigations Liver function test abnormal	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Investigations Low density lipoprotein increased	5.9% 2/34 • Number of events 4 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Investigations Protein urine present	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Investigations Red blood cell count increased	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Investigations Specific gravity urine decreased	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Investigations Transaminases increased	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Metabolism and nutrition disorders Hypokalaemia	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Metabolism and nutrition disorders Hypomagnesaemia	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Metabolism and nutrition disorders Metabolic acidosis	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Musculoskeletal and connective tissue disorders Arthralgia	5.9% 2/34 • Number of events 2 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Musculoskeletal and connective tissue disorders Back pain	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Musculoskeletal and connective tissue disorders Musculoskeletal pain	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Musculoskeletal and connective tissue disorders Spinal osteoarthritis	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Psychiatric disorders Alcohol abuse	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Psychiatric disorders Depression	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Renal and urinary disorders Acute kidney injury	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Respiratory, thoracic and mediastinal disorders Cough	5.9% 2/34 • Number of events 2 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Skin and subcutaneous tissue disorders Acne	5.9% 2/34 • Number of events 3 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Skin and subcutaneous tissue disorders Pruritus allergic	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Social circumstances Excessive exercise	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).
Vascular disorders Hypertension	2.9% 1/34 • Number of events 1 • The analysis included all treatment-emergent adverse events (TEAEs), defined as any recorded AE that occurred on or after the initiation of study medication and continue reporting until the end of the subject's last study visit (4-week post-treatment follow up visit).

Additional Information

David Gordon

Aclaris Therapeutics, Inc.

Phone: 484-540-6296

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place