Trial Outcomes & Findings for Safety and Pharmacokinetic Study of ATI-50002 in Subjects With Alopecia Universalis (AU) and Alopecia Totalis (AT) (NCT NCT03315689)

NCT ID: NCT03315689

Last Updated: 2020-07-02

Results Overview

ATI-50002 levels in scalp biopsies taken at Visits 3 (Day 2) and 7 (Day 29).

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 11 participants
• Primary outcome timeframe: Day 2 - Day 29
• Results posted on: 2020-07-02

Participant Flow

Participant milestones

Measure	Vehicle Topical Solution Vehicle Topical Solution applied twice daily.	ATI-50002 0.46% Topical Solution ATI-50002 0.46% Topical Solution applied twice daily.
Double-blind Period = 28 Days STARTED	4	7
Double-blind Period = 28 Days COMPLETED	3	7
Double-blind Period = 28 Days NOT COMPLETED	1	0
Open Label Extension = 52 Weeks STARTED	0	10
Open Label Extension = 52 Weeks COMPLETED	0	7
Open Label Extension = 52 Weeks NOT COMPLETED	0	3

Reasons for withdrawal

Measure	Vehicle Topical Solution Vehicle Topical Solution applied twice daily.	ATI-50002 0.46% Topical Solution ATI-50002 0.46% Topical Solution applied twice daily.
Double-blind Period = 28 Days Withdrawal by Subject	1	0
Open Label Extension = 52 Weeks Withdrawal by Subject	0	2
Open Label Extension = 52 Weeks Lost to Follow-up	0	1

Baseline Characteristics

Safety and Pharmacokinetic Study of ATI-50002 in Subjects With Alopecia Universalis (AU) and Alopecia Totalis (AT)

Baseline characteristics by cohort

Measure	Vehicle Topical Solution n=4 Participants Vehicle Topical Solution applied twice daily.	ATI-50002 0.46% Topical Solution n=7 Participants ATI-50002 0.46% Topical Solution applied twice daily.	Total n=11 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	3 Participants n=5 Participants	5 Participants n=7 Participants	8 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	7 Participants n=7 Participants	10 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	2 Participants n=5 Participants	4 Participants n=7 Participants	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	3 Participants n=7 Participants	4 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	3 Participants n=7 Participants	4 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants	4 Participants n=7 Participants	7 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	4 participants n=5 Participants	7 participants n=7 Participants	11 participants n=5 Participants
Fitzpatrick Skin Type I - Always Burns	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Fitzpatrick Skin Type II - Burns Easily	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Fitzpatrick Skin Type III - Burns Moderately	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Fitzpatrick Skin Type IV - Burns Minimally	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Fitzpatrick Skin Type V - Rarely Burns	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Fitzpatrick Skin Type VI - Never Burns	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Current Clinical Type of Alopecia Totalis	1 Participants n=5 Participants	3 Participants n=7 Participants	4 Participants n=5 Participants
Current Clinical Type of Alopecia Universalis	3 Participants n=5 Participants	4 Participants n=7 Participants	7 Participants n=5 Participants
Time Since Onset of Current Episode of AU or AT	145.8 Weeks STANDARD_DEVIATION 128.58 • n=5 Participants	218.3 Weeks STANDARD_DEVIATION 122.10 • n=7 Participants	191.9 Weeks STANDARD_DEVIATION 123.47 • n=5 Participants

PRIMARY outcome

Timeframe: Day 2 - Day 29

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

ATI-50002 levels in scalp biopsies taken at Visits 3 (Day 2) and 7 (Day 29).

Outcome measures

Measure	Vehicle n=3 Participants Vehicle Vehicle: Vehicle Topical Solution	Active n=6 Participants ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Double Blind Period: ATI-50002 Levels in Scalp Biopsy (ng/g) - Pharmacodynamic (PD) Population at Day 2 and Day 29 V7 - Day 29	NA ng/g Standard Deviation NA Of the 3 participants with available samples for analysis, all results were below quantifiable limits where the lower limit of quantification was 100 ng/g.	5710.0 ng/g Standard Deviation 3647.87
Double Blind Period: ATI-50002 Levels in Scalp Biopsy (ng/g) - Pharmacodynamic (PD) Population at Day 2 and Day 29 V3 - Day 2	166.0 ng/g Standard Deviation 83.44	579.7 ng/g Standard Deviation 322.36

SECONDARY outcome

Timeframe: Baseline - Week 52

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Severity of Alopecia Tool (SALT) score is a physician administered scale measuring the amount of scalp without any terminal hair. Possible scores range from 0 (no scalp hair loss) to 100 (complete scalp hair loss). A negative change in the SALT score over time represents hair regrowth.

Outcome measures

Measure	Vehicle n=4 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Mean Change From Baseline in the Severity of Alopecia Tool (SALT) Score at Week 52	-15.0 score on a scale Standard Deviation 16.21	—

SECONDARY outcome

Timeframe: Baseline - Week 40

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Severity of Alopecia Tool (SALT) score is a physician administered scale measuring the amount of scalp without any terminal hair. Possible scores range from 0 (no scalp hair loss) to 100 (complete scalp hair loss). A negative change in the SALT score over time represents hair regrowth.

Outcome measures

Measure	Vehicle n=4 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Mean Change From Baseline in the Severity of Alopecia Tool (SALT) Score at Week 40	-8.8 score on a scale Standard Deviation 12.84	—

SECONDARY outcome

Timeframe: Baseline - Week 28

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Severity of Alopecia Tool (SALT) score is a physician administered scale measuring the amount of scalp without any terminal hair. Possible scores range from 0 (no scalp hair loss) to 100 (complete scalp hair loss). A negative change in the SALT score over time represents hair regrowth.

Outcome measures

Measure	Vehicle n=8 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Mean Change From Baseline in the Severity of Alopecia Tool (SALT) Score at Week 28	-6.1 score on a scale Standard Deviation 11.91	—

SECONDARY outcome

Timeframe: Baseline - Week 28

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Alopecia Density and Extent (ALODEX) score is a measurement of the amount of scalp with terminal hair loss assessed by the investigator. ALODEX breaks the scalp up into a grid of 1% scalp surface areas and assigns density rating in each area on a 10 point scale of hair loss (0= no hair loss to 10 = complete baldness). Summation of scores from each 1% scalp surface area provides an overall score which may range from 0 (no scalp hair loss) to 100 (complete baldness). A negative change in the ALODEX score over time represents hair regrowth.

Outcome measures

Measure	Vehicle n=8 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Change From Baseline in Alopecia Density and Extent Score (ALODEX) at Week 28	-7.1 score on a scale Standard Deviation 12.71	—

SECONDARY outcome

Timeframe: Baseline - Week 40

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Alopecia Density and Extent (ALODEX) score is a measurement of the amount of scalp with terminal hair loss assessed by the investigator. ALODEX breaks the scalp up into a grid of 1% scalp surface areas and assigns density rating in each area on a 10 point scale of hair loss (0= no hair loss to 10 = complete baldness). Summation of scores from each 1% scalp surface area provides an overall score which may range from 0 (no scalp hair loss) to 100 (complete baldness). A negative change in the ALODEX score over time represents hair regrowth.

Outcome measures

Measure	Vehicle n=4 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Change From Baseline in Alopecia Density and Extent Score (ALODEX) at Week 40	-9.5 score on a scale Standard Deviation 10.66	—

SECONDARY outcome

Timeframe: Baseline - Week 52

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Alopecia Density and Extent (ALODEX) score is a measurement of the amount of scalp with terminal hair loss assessed by the investigator. ALODEX breaks the scalp up into a grid of 1% scalp surface areas and assigns density rating in each area on a 10 point scale of hair loss (0= no hair loss to 10 = complete baldness). Summation of scores from each 1% scalp surface area provides an overall score which may range from 0 (no scalp hair loss) to 100 (complete baldness). A negative change in the ALODEX score over time represents hair regrowth.

Outcome measures

Measure	Vehicle n=4 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Change From Baseline in Alopecia Density and Extent Score (ALODEX) at Week 52	-12.0 score on a scale Standard Deviation 11.58	—

SECONDARY outcome

Timeframe: Week 4 - Week 28

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Subject Eyebrow Assessment (SEA) is the subject's assessment of the appearance of eyebrow hair present on the affected eyebrow(s) at a particular point in time. The SEA is a five-point VRS ranging from "0 - No eyebrow hair", "1 - A little eyebrow hair", "2 - Some eyebrow hair", "3 - Most eyebrow hair", and "4 - Full eyebrow hair" with a recall period of "right now". A positive change over time represents eyebrow regrowth (better outcome).

Outcome measures

Measure	Vehicle n=8 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Mean Change in Subject's Eyebrow Assessment (SEA) at Week 28	0.4 score on a scale Standard Deviation 0.90	—

SECONDARY outcome

Timeframe: Week 4 - Week 40

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Subject Eyebrow Assessment (SEA) is the subject's assessment of the appearance of eyebrow hair present on the affected eyebrow(s) at a particular point in time. The SEA is a five-point VRS ranging from "0 - No eyebrow hair", "1 - A little eyebrow hair", "2 - Some eyebrow hair", "3 - Most eyebrow hair", and "4 - Full eyebrow hair" with a recall period of "right now". A positive change over time represents eyebrow regrowth (better outcome).

Outcome measures

Measure	Vehicle n=4 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Mean Change in Subject's Eyebrow Assessment (SEA) at Week 40	0.5 score on a scale Standard Deviation 1.58	—

SECONDARY outcome

Timeframe: Week 4 - Week 52

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Subject Eyebrow Assessment (SEA) is the subject's assessment of the appearance of eyebrow hair present on the affected eyebrow(s) at a particular point in time. The SEA is a five-point VRS ranging from "0 - No eyebrow hair", "1 - A little eyebrow hair", "2 - Some eyebrow hair", "3 - Most eyebrow hair", and "4 - Full eyebrow hair" with a recall period of "right now". A positive change over time represents eyebrow regrowth (better outcome).

Outcome measures

Measure	Vehicle n=4 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Mean Change in Subject's Eyebrow Assessment (SEA) at Week 52	0.6 score on a scale Standard Deviation 1.38	—

SECONDARY outcome

Timeframe: Week 4 - Week 28

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Clinician's Eyebrow Assessment (CEA) is the investigator's assessment of the appearance of eyebrow hair present on the affected eyebrow(s) at a particular point in time. The CEA is a five-point VRS ranging from "0 - No eyebrow hair", "1 - A little eyebrow hair", "2 - Some eyebrow hair", "3 - Most eyebrow hair", and "4 - Full eyebrow hair" with a recall period of "right now". A positive change over time represents eyebrow regrowth (better outcome).

Outcome measures

Measure	Vehicle n=8 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Mean Change in Clinician's Eyebrow Assessment (CEA) at Week 28	0.1 score on a scale Standard Deviation 1.16	—

SECONDARY outcome

Timeframe: Week 4 - Week 40

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Clinician's Eyebrow Assessment (CEA) is the investigator's assessment of the appearance of eyebrow hair present on the affected eyebrow(s) at a particular point in time. The CEA is a five-point VRS ranging from "0 - No eyebrow hair", "1 - A little eyebrow hair", "2 - Some eyebrow hair", "3 - Most eyebrow hair", and "4 - Full eyebrow hair" with a recall period of "right now". A positive change over time represents eyebrow regrowth (better outcome).

Outcome measures

Measure	Vehicle n=4 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Mean Change in Clinician's Eyebrow Assessment (CEA) at Week 40	0.6 score on a scale Standard Deviation 1.11	—

SECONDARY outcome

Timeframe: Week 4 - Week 52

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Clinician's Eyebrow Assessment (CEA) is the investigator's assessment of the appearance of eyebrow hair present on the affected eyebrow(s) at a particular point in time. The CEA is a five-point VRS ranging from "0 - No eyebrow hair", "1 - A little eyebrow hair", "2 - Some eyebrow hair", "3 - Most eyebrow hair", and "4 - Full eyebrow hair" with a recall period of "right now". A positive change over time represents eyebrow regrowth (better outcome).

Outcome measures

Measure	Vehicle n=4 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Mean Change in Clinician's Eyebrow Assessment (CEA) at Week 52	0.8 score on a scale Standard Deviation 1.19	—

SECONDARY outcome

Timeframe: Week 28

Population: All patients who have a valid assessment at the timepoint being reported are included.

The Subject Global Impression of Treatment Satisfaction (SGIS) is a 7-point descriptive scale with a recall period of "right now". The SGIS was completed by subjects during the Open Label period of the study. Scale response options ranged from "1: Extremely Satisfied", "2: Moderately Satisfied", "3: A little Satisfied", "4: Neither Satisfied or Dissatisfied", "5: A little Dissatisfied", "6: Moderately Dissatisfied", or "7: Extremely Dissatisfied" and are used to capture how satisfied or dissatisfied subjects are with the study medication treatment received for their alopecia areata.

Outcome measures

Measure	Vehicle n=8 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 28 1: Extremely Satisfied	0 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 28 2: Moderately Satisfied	2 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 28 3: A little Satisfied	1 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 28 4: Neither Satisfied or Dissatisfied	1 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 28 5: A little Dissatisfied	0 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 28 6: Moderately Dissatisfied	1 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 28 7: Extremely Dissatisfied	3 Participants	—

SECONDARY outcome

Timeframe: Week 40

Population: All patients who have a valid assessment at the timepoint being reported are included.

The Subject Global Impression of Treatment Satisfaction (SGIS) is a 7-point descriptive scale with a recall period of "right now". The SGIS was completed by subjects during the Open Label period of the study. Scale response options ranged from "1: Extremely Satisfied", "2: Moderately Satisfied", "3: A little Satisfied", "4: Neither Satisfied or Dissatisfied", "5: A little Dissatisfied", "6: Moderately Dissatisfied", or "7: Extremely Dissatisfied" and are used to capture how satisfied or dissatisfied subjects are with the study medication treatment received for their alopecia areata.

Outcome measures

Measure	Vehicle n=4 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 40 1: Extremely Satisfied	0 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 40 2: Moderately Satisfied	2 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 40 3: A little Satisfied	0 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 40 4: Neither Satisfied or Dissatisfied	1 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 40 5: A little Dissatisfied	0 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 40 6: Moderately Dissatisfied	0 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 40 7: Extremely Dissatisfied	1 Participants	—

SECONDARY outcome

Timeframe: Week 52

Population: All patients who have a valid assessment at the timepoint being reported are included.

The Subject Global Impression of Treatment Satisfaction (SGIS) is a 7-point descriptive scale with a recall period of "right now". The SGIS was completed by subjects during the Open Label period of the study. Scale response options ranged from "1: Extremely Satisfied", "2: Moderately Satisfied", "3: A little Satisfied", "4: Neither Satisfied or Dissatisfied", "5: A little Dissatisfied", "6: Moderately Dissatisfied", or "7: Extremely Dissatisfied" and are used to capture how satisfied or dissatisfied subjects are with the study medication treatment received for their alopecia areata.

Outcome measures

Measure	Vehicle n=4 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 52 1: Extremely Satisfied	0 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 52 2: Moderately Satisfied	2 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 52 3: A little Satisfied	0 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 52 4: Neither Satisfied or Dissatisfied	1 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 52 5: A little Dissatisfied	0 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 52 6: Moderately Dissatisfied	0 Participants	—
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 52 7: Extremely Dissatisfied	1 Participants	—

SECONDARY outcome

Timeframe: Baseline - Week 24

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Severity of Alopecia Tool (SALT) score is a physician administered scale measuring the amount of scalp without any terminal hair assessed by the investigator. A positive percent change over time represents hair regrowth (better outcome).

Outcome measures

Measure	Vehicle n=8 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Severity of Alopecia Tool (SALT) Scores, Relative Percent Regrowth (%) at Week 24 From Baseline	6.3 percent change Standard Deviation 12.14	—

SECONDARY outcome

Timeframe: Baseline - Week 40

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Severity of Alopecia Tool (SALT) score is a physician administered scale measuring the amount of scalp without any terminal hair assessed by the investigator. A positive percent change over time represents hair regrowth (better outcome).

Outcome measures

Measure	Vehicle n=4 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: Severity of Alopecia Tool (SALT Scores), Relative Percent Regrowth (%) at Week 40 From Baseline	9.0 percent change Standard Deviation 13.01	—

SECONDARY outcome

Timeframe: Baseline - Week 52

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Severity of Alopecia Tool (SALT) score is a physician administered scale measuring the amount of scalp without any terminal hair assessed by the investigator. A positive percent change over time represents hair regrowth (better outcome).

Outcome measures

Measure	Vehicle n=4 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: The Severity of Alopecia Tool (SALT) Scores, Relative Percent Regrowth (%) at Week 52 From Baseline	15.5 percent change Standard Deviation 16.77	—

SECONDARY outcome

Timeframe: Baseline - Week 24

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Alopecia Density and Extent (ALODEX) score is a measurement of the amount of scalp with terminal hair loss assessed by the investigator. ALODEX breaks the scalp up into a grid of 1% scalp surface areas and assigns density rating in each area on a 10 point scale of hair loss (0= no hair loss to 10 = complete baldness). Summation of scores from each 1% scalp surface area provides an overall score which may range from 0 (no scalp hair loss) to 100 (complete baldness). A positive percent change over time represents hair regrowth (better outcome).

Outcome measures

Measure	Vehicle n=8 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: The Alopecia Density and Extent (ALODEX) Scores, Relative Percent Regrowth (%) at 24 Weeks	7.2 percent change Standard Deviation 12.88	—

SECONDARY outcome

Timeframe: Baseline - Week 40

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Alopecia Density and Extent (ALODEX) score is a measurement of the amount of scalp with terminal hair loss assessed by the investigator at Baseline, Week 4, Week 8, Week 12, Week 16, Week 20, and Week 24. ALODEX breaks the scalp up into a grid of 1% scalp surface areas and assigns density rating in each area on a 10 point scale of hair loss (0= no hair loss to 10 = complete baldness). Summation of scores from each 1% scalp surface area provides an overall score which may range from 0 (no scalp hair loss) to 100 (complete baldness). A positive percent change over time represents hair regrowth (better outcome).

Outcome measures

Measure	Vehicle n=4 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: The Alopecia Density and Extent (ALODEX) Scores, Relative Percent Regrowth (%) at 40 Weeks	9.6 percent change Standard Deviation 10.81	—

SECONDARY outcome

Timeframe: Baseline - Week 52

Population: All randomized patients who have a valid assessment at baseline and the subsequent timepoint being reported are included.

The Alopecia Density and Extent (ALODEX) score is a measurement of the amount of scalp with terminal hair loss assessed by the investigator. ALODEX breaks the scalp up into a grid of 1% scalp surface areas and assigns density rating in each area on a 10 point scale of hair loss (0= no hair loss to 10 = complete baldness). Summation of scores from each 1% scalp surface area provides an overall score which may range from 0 (no scalp hair loss) to 100 (complete baldness). A positive percent change over time represents hair regrowth (better outcome).

Outcome measures

Measure	Vehicle n=4 Participants Vehicle Vehicle: Vehicle Topical Solution	Active ATI-50002 Topical Solution ATI-50002: ATI-50002 Topical Solution
Open Label Extension: The Alopecia Density and Extent (ALODEX) Scores, Relative Percent Regrowth (%) at 52 Weeks	12.2 percent change Standard Deviation 11.74	—

Adverse Events

Vehicle Topical Solution

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

ATI-50002 0.46% Topical Solution

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

ATI-50002 0.46% Topical Solution OLE

Serious events: 1 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Measure	Vehicle Topical Solution n=4 participants at risk Vehicle Topical Solution applied twice daily during the double blind period of the study.	ATI-50002 0.46% Topical Solution n=7 participants at risk ATI-50002 0.46% Topical Solution applied twice daily during the double blind period of the study	ATI-50002 0.46% Topical Solution OLE n=10 participants at risk ATI-50002 0.46% Topical Solution applied twice daily during the open label extension period of the study
Psychiatric disorders Depression	0.00% 0/4 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	0.00% 0/7 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	10.0% 1/10 • Number of events 1 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.

Other adverse events

Measure	Vehicle Topical Solution n=4 participants at risk Vehicle Topical Solution applied twice daily during the double blind period of the study.	ATI-50002 0.46% Topical Solution n=7 participants at risk ATI-50002 0.46% Topical Solution applied twice daily during the double blind period of the study	ATI-50002 0.46% Topical Solution OLE n=10 participants at risk ATI-50002 0.46% Topical Solution applied twice daily during the open label extension period of the study
General disorders Application site erosion	0.00% 0/4 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	0.00% 0/7 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	10.0% 1/10 • Number of events 1 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.
General disorders Peripheral swelling	0.00% 0/4 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	0.00% 0/7 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	10.0% 1/10 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.
Infections and infestations Ear infection	0.00% 0/4 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	0.00% 0/7 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	10.0% 1/10 • Number of events 1 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.
Infections and infestations Oral herpes	0.00% 0/4 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	0.00% 0/7 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	10.0% 1/10 • Number of events 1 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.
Metabolism and nutrition disorders Dehydration	0.00% 0/4 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	0.00% 0/7 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	10.0% 1/10 • Number of events 1 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.
Metabolism and nutrition disorders Glucose tolerance impaired	0.00% 0/4 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	0.00% 0/7 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	10.0% 1/10 • Number of events 1 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.
Metabolism and nutrition disorders Vitamin D deficiency	0.00% 0/4 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	0.00% 0/7 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	10.0% 1/10 • Number of events 1 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.
Musculoskeletal and connective tissue disorders Rotator cuff syndrome	0.00% 0/4 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	0.00% 0/7 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	10.0% 1/10 • Number of events 1 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.
Nervous system disorders Migraine	25.0% 1/4 • Number of events 1 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	0.00% 0/7 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	0.00% 0/10 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.
Vascular disorders Hypertensive urgency	0.00% 0/4 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	14.3% 1/7 • Number of events 1 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.	0.00% 0/10 • Double blind period = 28 days; Open label extension period = 12 months; Reporting of non-serious AEs started with each subject's first study medication application and continued until the end of the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until the end of the subject's last visit.

Additional Information

Marco Cardillo, Clinical Trial Manager

Aclaris Therapeutics, Inc.

Phone: 484-540-6299

Email: mcardillo@aclaristx.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place