NCT03594227

ATI-501 Oral Suspension Compared to Placebo in Subjects With Alopecia Areata, Alopecia Universalis or Alopecia Totalis

Completed Phase 2 Results posted Last updated 16 September 2020

What this trial tests

Phase 2 trial testing ATI-501 400mg BID (Low dose) in Alopecia in 87 participants. Completed in 18 June 2019.

Timeline

11 June 2018

Primary endpoint
6 June 2019

18 June 2019

Quick facts

Lead sponsor	Aclaris Therapeutics, Inc.
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	triple
Primary purpose	other
Enrollment	87
Start date	11 June 2018
Primary completion	6 June 2019
Estimated completion	18 June 2019
Sites	25 locations across United States

Drugs / interventions tested

• ATI-501 400mg BID (Low dose) — full drug profile →
• ATI-501 600mg BID (Mid dose) — full drug profile →
• ATI-501 800mg BID (High dose) — full drug profile →
• Placebo

Conditions studied

• Alopecia — all drugs for Alopecia →

Sponsor

Aclaris Therapeutics, Inc. — full company profile →

Who can join

18 and older, any sex, with Alopecia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Percent Change From Baseline in the Severity of Alopecia Tool (SALT) Score at Week 24 Primary · 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. 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 by adding the percentage hair loss in the various areas (i.e. top, back, each side) of the scalp. The primary efficacy variable was the percent change from baseline in SALT score at Week 24. This was calculated as the mean of the changes from Baseline (Visit 2) SALT score to Week

Group	Value	95% CI
ATI-501 400mg BID Dosing	-25.6	± 39.75
ATI-501 600mg BID Dosing	-30.4	± 41.23
ATI-501 800mg BID Dosing	-25.9	± 29.86
Placebo	-6.3	± 23.36

Percent Change From Baseline in the Alopecia Density and Extent Score (ALODEX) at Week 24 Secondary · 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 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 negative change in the ALODEX score over time represents

Group	Value	95% CI
ATI-501 400mg BID Dosing	-26.88	± 5.165
ATI-501 600mg BID Dosing	-32.24	± 5.165
ATI-501 800mg BID Dosing	-26.87	± 5.281
Placebo	-7.70	± 5.683

Mean Change From Baseline in the Severity of Alopecia Tool (SALT) Score at Week 24 Secondary · 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. 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 by adding the percentage hair loss in the various areas (i.e. top, back, each side) of the scalp.

Group	Value	95% CI
ATI-501 400mg BID	-14.6	± 23.67
ATI-501 600mg BID	-18.6	± 27.29
ATI-501 800mg BID	-20.9	± 26.50
Placebo	-3.3	± 8.21

Mean Change From Baseline in the Alopecia Density and Extent (ALODEX) Score at Week 24 Secondary · 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 negative change in the ALODEX score over time represents hair regrowth.

Group	Value	95% CI
ATI-501 400mg BID	-17.6	± 26.93
ATI-501 600mg BID	-22.2	± 28.99
ATI-501 800mg BID	-20.7	± 25.04
Placebo	-3.2	± 8.42

Count of Subjects Achieving at Least a 50% Reduction in Alopecia Density and Extent (ALODEX) Score at Week 24 Secondary · 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 negative change in the ALODEX score over time represents hair regrowth.

Group	Value	95% CI
ATI-501 400mg BID	6
ATI-501 600mg BID	7
ATI-501 800mg BID	5
Placebo	1

Count of Subjects in Each Treatment Arm Achieving at Least a 50% Reduction in Severity of Alopecia Tool (SALT) Score at Week 24 Compared to Baseline Secondary · 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. 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 by adding the percentage hair loss in the various areas (i.e. top, back, each side) of the scalp.

Group	Value	95% CI
ATI-501 400mg BID	6
ATI-501 600mg BID	7
ATI-501 800mg BID	5
Placebo	1

Change From Baseline in Alopecia Facial Hair Appearance Assessment (AFHA)-Clinician Reported Outcome, Left Eyebrow Secondary · Baseline-Week 24

The Alopecia Facial Hair Appearance Assessment (AFHA)-Clinician Reported Outcome is the investigator's assessment of the subject's facial hair (eyebrow(s), eyelashes,and if male, beard) at a particular point in time. The investigator was instructed to NOT refer to any other assessments to assist with these assessments. The investigator or designee assessed the affected facial hair areas (eyebrows, eyelashes and if male, beard) using a 5 point scale for each target area \[1-Full (Target Area Hair); 2-Most (Target Area Hair); 3-Some (Target Area Hair); 4-A Little (Target Area Hair); 5-No (Target

Group	Value	95% CI
ATI-501 400mg BID	0
ATI-501 600mg BID	1
ATI-501 800mg BID	1
Placebo	0
ATI-501 400mg BID	1
ATI-501 600mg BID	0
ATI-501 800mg BID	1
Placebo	1
ATI-501 400mg BID	0
ATI-501 600mg BID	2
ATI-501 800mg BID	2
Placebo	1
ATI-501 400mg BID	4
ATI-501 600mg BID	5
ATI-501 800mg BID	6
Placebo	3

Change From Baseline in Alopecia Facial Hair Appearance Assessment (AFHA)-Clinician Reported Outcome, Right Eyebrow Secondary · Baseline-Week 24

The Alopecia Facial Hair Appearance Assessment (AFHA)-Clinician Reported Outcome is the investigator's assessment of the subject's facial hair (eyebrow(s), eyelashes,and if male, beard) at a particular point in time. The investigator was instructed to NOT refer to any other assessments to assist with these assessments. The investigator or designee assessed the affected facial hair areas (eyebrows, eyelashes and if male, beard) using a 5 point scale for each target area \[1-Full (Target Area Hair); 2-Most (Target Area Hair); 3-Some (Target Area Hair); 4-A Little (Target Area Hair); 5-No (Target

Group	Value	95% CI
ATI-501 400mg BID	0
ATI-501 600mg BID	1
ATI-501 800mg BID	1
Placebo	0
ATI-501 400mg BID	1
ATI-501 600mg BID	0
ATI-501 800mg BID	1
Placebo	1
ATI-501 400mg BID	0
ATI-501 600mg BID	0
ATI-501 800mg BID	3
Placebo	1
ATI-501 400mg BID	3
ATI-501 600mg BID	6
ATI-501 800mg BID	4
Placebo	4

Change From Baseline in Alopecia Facial Hair Appearance Assessment (AFHA)-Clinician Reported Outcome, Left Eyelash Secondary · Baseline-Week 24

The Alopecia Facial Hair Appearance Assessment (AFHA)-Clinician Reported Outcome is the investigator's assessment of the subject's facial hair (eyebrow(s), eyelashes,and if male, beard) at a particular point in time. The investigator was instructed to NOT refer to any other assessments to assist with these assessments. The investigator or designee assessed the affected facial hair areas (eyebrows, eyelashes and if male, beard) using a 5 point scale for each target area \[1-Full (Target Area Hair); 2-Most (Target Area Hair); 3-Some (Target Area Hair); 4-A Little (Target Area Hair); 5-No (Target

Group	Value	95% CI
ATI-501 400mg BID	1
ATI-501 600mg BID	1
ATI-501 800mg BID	0
Placebo	0
ATI-501 400mg BID	1
ATI-501 600mg BID	0
ATI-501 800mg BID	2
Placebo	1
ATI-501 400mg BID	1
ATI-501 600mg BID	1
ATI-501 800mg BID	3
Placebo	0
ATI-501 400mg BID	3
ATI-501 600mg BID	3
ATI-501 800mg BID	3
Placebo	1

Change From Baseline in Alopecia Facial Hair Appearance Assessment (AFHA)-Clinician Reported Outcome, Right Eyelash Secondary · Baseline-Week 24

The Alopecia Facial Hair Appearance Assessment (AFHA)-Clinician Reported Outcome is the investigator's assessment of the subject's facial hair (eyebrow(s), eyelashes,and if male, beard) at a particular point in time. The investigator was instructed to NOT refer to any other assessments to assist with these assessments. The investigator or designee assessed the affected facial hair areas (eyebrows, eyelashes and if male, beard) using a 5 point scale for each target area \[1-Full (Target Area Hair); 2-Most (Target Area Hair); 3-Some (Target Area Hair); 4-A Little (Target Area Hair); 5-No (Target

Group	Value	95% CI
ATI-501 400mg BID	1
ATI-501 600mg BID	1
ATI-501 800mg BID	0
Placebo	0
ATI-501 400mg BID	1
ATI-501 600mg BID	0
ATI-501 800mg BID	1
Placebo	1
ATI-501 400mg BID	1
ATI-501 600mg BID	1
ATI-501 800mg BID	3
Placebo	0
ATI-501 400mg BID	3
ATI-501 600mg BID	4
ATI-501 800mg BID	4
Placebo	1

Change From Baseline in Alopecia Facial Hair Appearance Assessment (AFHA)-Clinician Reported Outcome, Beard, Male Subjects Secondary · Baseline-Week 24

The Alopecia Facial Hair Appearance Assessment (AFHA)-Clinician Reported Outcome is the investigator's assessment of the subject's facial hair (eyebrow(s), eyelashes,and if male, beard) at a particular point in time. The investigator was instructed to NOT refer to any other assessments to assist with these assessments. The investigator or designee assessed the affected facial hair areas (eyebrows, eyelashes and if male, beard) using a 5 point scale for each target area \[1-Full (Target Area Hair); 2-Most (Target Area Hair); 3-Some (Target Area Hair); 4-A Little (Target Area Hair); 5-No (Target

Group	Value	95% CI
ATI-501 400mg BID	0
ATI-501 600mg BID	0
ATI-501 800mg BID	0
Placebo	0
ATI-501 400mg BID	0
ATI-501 600mg BID	1
ATI-501 800mg BID	3
Placebo	0
ATI-501 400mg BID	0
ATI-501 600mg BID	0
ATI-501 800mg BID	3
Placebo	0
ATI-501 400mg BID	2
ATI-501 600mg BID	1
ATI-501 800mg BID	0
Placebo	2

Change From Baseline in Alopecia Facial Hair Appearance Assessment (AFHA)-Patient Reported Outcome, Left Eyebrow Secondary · Baseline-Week 24

The Alopecia Facial Hair Appearance Assessment (AFHA)-Patient Reported Outcome is the patient's assessment of their facial hair (eyebrow(s), eyelashes, and if male, beard) at a particular point in time. The patient assessed the affected facial hair areas (eyebrows, eyelashes and if male, beard) using a 5 point scale for each target area \[1-Full (Target Area Hair); 2-Most (Target Area Hair); 3-Some (Target Area Hair); 4-A Little (Target Area Hair); 5-No (Target Area Hair)\]. Change status is categorized as 'Better by x points' when the score decreases from baseline by x points, and as 'Worse b

Group	Value	95% CI
ATI-501 400mg BID	0
ATI-501 600mg BID	1
ATI-501 800mg BID	0
Placebo	0
ATI-501 400mg BID	1
ATI-501 600mg BID	0
ATI-501 800mg BID	2
Placebo	0
ATI-501 400mg BID	4
ATI-501 600mg BID	0
ATI-501 800mg BID	0
Placebo	1
ATI-501 400mg BID	1
ATI-501 600mg BID	5
ATI-501 800mg BID	8
Placebo	3

Adverse events — posted to ClinicalTrials.gov

Time frame: Double blind period = 24 weeks; Reporting of non-serious AEs started with each subject's first study medication application and continued until the 30 days after the subject's last study visit. Reporting for SAEs started when the subject signed the informed consent document and continued until 30 days after the subject's last visit.. Reporting threshold: 3%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

ATI-501 400mg BID Dosing

Serious: 0/23 (0%)

Deaths: 0/23

ATI-501 600mg BID Dosing

Serious: 0/23 (0%)

Deaths: 0/23

ATI-501 800mg BID Dosing

Serious: 0/22 (0%)

Deaths: 0/22

Placebo

Serious: 0/19 (0%)

Deaths: 0/19

Other adverse events (22 terms — click to expand)

Reaction	System	ATI-501 400mg BID Dosing	ATI-501 600mg BID Dosing	ATI-501 800mg BID Dosing	Placebo
Alanine aminotransferase increased	Investigations	—	—	—	—
Nausea	Gastrointestinal disorders	—	—	—	—
Abdominal pain	Gastrointestinal disorders	—	—	—	—
Constipation	Gastrointestinal disorders	—	—	—	—
Dyspepsia	Gastrointestinal disorders	—	—	—	—
Feces soft	Gastrointestinal disorders	—	—	—	—
Gastroesophageal reflux disease	Gastrointestinal disorders	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—
Fatigue	General disorders	—	—	—	—
Folliculitis	Infections and infestations	—	—	—	—
Vulvovaginal mycotic infection	Infections and infestations	—	—	—	—
Blood creatine phosphokinase increased	Investigations	—	—	—	—
Aspartate aminotransferase increased	Investigations	—	—	—	—
Dizziness	Nervous system disorders	—	—	—	—
Headache	Nervous system disorders	—	—	—	—
Paraesthesia	Nervous system disorders	—	—	—	—
Chromaturia	Renal and urinary disorders	—	—	—	—
Adnexa uteri pain	Reproductive system and breast disorders	—	—	—	—
Pruritus	Skin and subcutaneous tissue disorders	—	—	—	—
Drug eruption	Skin and subcutaneous tissue disorders	—	—	—	—
Photosensitivity reaction	Skin and subcutaneous tissue disorders	—	—	—	—
Hypertension	Vascular disorders	—	—	—	—

Data from ClinicalTrials.gov NCT03594227 adverse events section.

Sponsor's own description

This Phase 2, multicenter, randomized study will evaluate the safety, tolerability and efficacy of ATI-501 for the treatment of Alopecia Areata (AA), Alopecia Universalis (AU) or Alopecia Totalis (AT) in adult subjects.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

1. Targeting the Janus Kinase Family in Autoimmune Skin Diseases.
   Howell MD, Kuo FI, Smith PA. · · 2019 · cited 180× · PMID 31649667 · DOI 10.3389/fimmu.2019.02342
2. An overview of JAK/STAT pathways and JAK inhibition in alopecia areata.
   Lensing M, Jabbari A. · · 2022 · cited 100× · PMID 36110853 · DOI 10.3389/fimmu.2022.955035
3. Comparative efficacy and safety of JAK inhibitors in the treatment of moderate-to-severe alopecia areata: a systematic review and network meta-analysis.
   Yan T, Wang T, Tang M, Liu N. · · 2024 · cited 19× · PMID 38659584 · DOI 10.3389/fphar.2024.1372810
4. Alopecia Areata: Pathogenesis, Diagnosis, and Therapies.
   Ma T, Zhang T, Miao F, Liu J, et al · · 2025 · cited 13× · PMID 40260013 · DOI 10.1002/mco2.70182
5. The Relative Efficacy and Safety of Monotherapies for Alopecia Areata: A Network Meta-Analysis Study.
   Gupta AK, Bamimore MA, Mirmirani P, Piguet V, et al · · 2025 · cited 4× · PMID 40231941 · DOI 10.1111/jocd.70185
6. Optimization of Pyrimidine Compounds as Potent JAK1 Inhibitors and the Discovery of R507 as a Clinical Candidate.
   Chen Y, Li H, Yen R, Heckrodt TJ, et al · · 2022 · cited 2× · PMID 36385926 · DOI 10.1021/acsmedchemlett.2c00411