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

Phase 2

Completed

Conditions: Alopecia Universalis (AU)
Alopecia Totalis (AT)

Interventions: Drug: Vehicle
Drug: ATI-50002

Registration Number: NCT03315689

Lead Sponsor: Aclaris Therapeutics, Inc.

Brief Summary: The primary objective is to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of ATI-50002 Topical Solution compared to vehicle in subjects with AU and AT.

Detailed Description: This study is designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ATI-50002 Topical Solution in patients with Alopecia Universalis and Alopecia Totalis. Concentrations of ATI-50002 in the blood and skin will be assessed after 28 days of treatment with ATI-50002 Topical Solution.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 11

Inclusion Criteria

Able to comprehend and willing to sign an Informed Consent Form (ICF).
Male or non-pregnant, non-nursing female ≥18 years old at the time of informed consent.
Have a clinical diagnosis of stable, clinically typical, AU or AT.
Have a duration of the current episode of AU or AT for a minimum of 6 months and a maximum of seven years.
Be willing and able to follow all study instructions and to attend all study visits.

Exclusion Criteria

Females who are nursing, pregnant, or planning to become pregnant for the duration of the study including 30 days after the last application of study medication.
Patchy alopecia areata, diffuse alopecia areata or a history of an atypical pattern of Alopecia Areata (AA) (e.g., ophiasis, sisaihpo).
Active skin disease on the scalp (such as psoriasis or seborrheic dermatitis) or a history of skin disease on the scalp that in the opinion of the investigator would interfere with the study assessments of efficacy or safety.
Active scalp trauma or other condition affecting the scalp that, in the investigator's opinion, may affect the course of AU or AT or interfere with the study conduct or evaluations.
The presence of a permanent or difficult to remove hairpiece or wig that will, in the opinion of the investigator, interfere with study assessments if not removed at each visit.

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Vehicle	Vehicle	Vehicle
Active	ATI-50002	ATI-50002 Topical Solution

Primary Outcome Measures

Name	Time	Method
Double Blind Period: ATI-50002 Levels in Scalp Biopsy (ng/g) - Pharmacodynamic (PD) Population at Day 2 and Day 29	Day 2 - Day 29	ATI-50002 levels in scalp biopsies taken at Visits 3 (Day 2) and 7 (Day 29).

Secondary Outcome Measures

Name	Time	Method
Open Label Extension: Mean Change From Baseline in the Severity of Alopecia Tool (SALT) Score at Week 52	Baseline - Week 52	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.
Open Label Extension: Mean Change in Subject's Eyebrow Assessment (SEA) at Week 52	Week 4 - Week 52	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).
Open Label Extension: Mean Change in Clinician's Eyebrow Assessment (CEA) at Week 28	Week 4 - Week 28	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).
Open Label Extension: Mean Change in Clinician's Eyebrow Assessment (CEA) at Week 40	Week 4 - Week 40	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).
Open Label Extension: Mean Change From Baseline in the Severity of Alopecia Tool (SALT) Score at Week 40	Baseline - Week 40	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.
Open Label Extension: Mean Change From Baseline in the Severity of Alopecia Tool (SALT) Score at Week 28	Baseline - Week 28	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.
Open Label Extension: Change From Baseline in Alopecia Density and Extent Score (ALODEX) at Week 28	Baseline - Week 28	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.
Open Label Extension: Change From Baseline in Alopecia Density and Extent Score (ALODEX) at Week 40	Baseline - Week 40	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.
Open Label Extension: Change From Baseline in Alopecia Density and Extent Score (ALODEX) at Week 52	Baseline - Week 52	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.
Open Label Extension: Mean Change in Subject's Eyebrow Assessment (SEA) at Week 28	Week 4 - Week 28	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).
Open Label Extension: Mean Change in Subject's Eyebrow Assessment (SEA) at Week 40	Week 4 - Week 40	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).
Open Label Extension: Mean Change in Clinician's Eyebrow Assessment (CEA) at Week 52	Week 4 - Week 52	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).
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 28	Week 28	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.
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 40	Week 40	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.
Open Label Extension: Subject Global Impression of Treatment Satisfaction (SGIS) Week 52	Week 52	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.
Open Label Extension: Severity of Alopecia Tool (SALT) Scores, Relative Percent Regrowth (%) at Week 24 From Baseline	Baseline - Week 24	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).
Open Label Extension: Severity of Alopecia Tool (SALT Scores), Relative Percent Regrowth (%) at Week 40 From Baseline	Baseline - Week 40	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).
Open Label Extension: The Severity of Alopecia Tool (SALT) Scores, Relative Percent Regrowth (%) at Week 52 From Baseline	Baseline - Week 52	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).
Open Label Extension: The Alopecia Density and Extent (ALODEX) Scores, Relative Percent Regrowth (%) at 24 Weeks	Baseline - Week 24	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).
Open Label Extension: The Alopecia Density and Extent (ALODEX) Scores, Relative Percent Regrowth (%) at 40 Weeks	Baseline - Week 40	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).
Open Label Extension: The Alopecia Density and Extent (ALODEX) Scores, Relative Percent Regrowth (%) at 52 Weeks	Baseline - Week 52	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).