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

A Randomized, Double-blind, Vehicle-controlled Multicenter Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of ATI-50002 Topical Solution Administered Twice-Daily for 28 Days in Adult Subjects With Alopecia Universalis and Alopecia Totalis With a 12-Month Long-Term Open-Label Extension

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.

Study Overview

• Status: Completed
• Conditions: Alopecia Totalis (AT); Alopecia Universalis (AU)
• Intervention / Treatment: Drug: ATI-50002; Drug: Vehicle

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.

• Study Type: Interventional
• Enrollment (Actual): 11
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • New York
    • New York, New York, United States, 10032
      • Aclaris Investigational Site
    • New York, New York, United States, 10075
      • Aclaris Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Vehicle	Drug: Vehicle; Vehicle Topical Solution
Experimental: Active; ATI-50002 Topical Solution	Drug: ATI-50002; ATI-50002 Topical Solution

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Aclaris Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): December 14, 2017
• Primary Completion (Actual): June 20, 2019
• Study Completion (Actual): June 20, 2019

Study Registration Dates

• First Submitted: October 17, 2017
• First Submitted That Met QC Criteria: October 17, 2017
• First Posted (Actual): October 20, 2017

Study Record Updates

• Last Update Posted (Actual): July 2, 2020
• Last Update Submitted That Met QC Criteria: June 18, 2020
• Last Verified: June 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Pathological Conditions, Anatomical; Hypotrichosis; Hair Diseases; Alopecia; Alopecia Areata
• Other Study ID Numbers: ATI-50002-AA-202

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No