ATI-502 Topical Solution for the Treatment of Alopecia Areata (AA), Alopecia Universalis (AU) and Alopecia Totalis (AT)

Study Overview
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Brief Summary
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This is an open label study of ATI-502 Topical Solution for the treatment of AA, AU, and AT with the following goals: To assess the safety, tolerability, and efficacy of ATI-502 Topical Solution in subjects with AA, AU or AT following 24 weeks of treatment with ATI-501 Oral Suspension or Placebo Suspension. To assess the ability of ATI-502 Topical Solution to maintain or improve hair regrowth in subjects previously treated with ATI-501 Oral Suspension or Placebo Suspension.

Detailed Description
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This Phase 2, multicenter, open-label study will evaluate the safety, tolerability, efficacy, and durability of efficacy of ATI-502 Topical Solution, 0.46% for the treatment of alopecia areata (AA), alopecia universalis (AU) and alopecia totalis (AT) in adult subjects who completed 24 weeks of treatment with ATI-501 Oral Suspension or Placebo Suspension. Subjects who complete 24 weeks of active treatment with ATI-501 Oral Suspension or Placebo Suspension in study ATI-501-AUAT-201 will be assessed for eligibility to enter the study. At Visit 9 in study ATI-501-AUAT-201, subjects who did not experience any adverse events (AEs), serious adverse events (SAEs), or tolerability issues that met study discontinuation criteria in study ATI-501-AUAT-201 and who in the opinion of the investigator are capable of re-growing or maintaining scalp hair and meet the entry criteria are eligible to enroll in this open-label study. Enrolled subjects will apply ATI-502 Topical Solution, 0.46% BID to the entire scalp and if applicable, the eyebrow(s) and return for safety and efficacy assessments as detailed in the Schedule of Assessments (Table 3). Assessment of response to treatment will be performed at Week 4, Week 8, Week 16, Week 24, and post-treatment Week 28. Safety and tolerability will be evaluated at each study visit by assessment of adverse events, clinical laboratory tests, and vital signs, and at Week 24, and physical examination findings

Official Title
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Open-Label Study of ATI-502 Topical Solution for the Treatment of Alopecia Areata (AA), Alopecia Universalis (AU) and Alopecia Totalis (AT)

Conditions
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Alopecia Areata, Alopecia Universalis, Alopecia Totalis

Intervention / Treatment
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* Drug: ATI 502 0.46% Topical Solution


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects must meet the following criteria to be eligible for participation in the study: Subject must be able to comprehend and willing to sign the Informed Consent Form (ICF). Male or non-pregnant, non-nursing female ≥ 18 years old at the time of informed consent. Subject has completed 24 weeks of treatment and the assessments for Visit 9 in study ATI-501-AUAT-201. Subject has not experienced any AEs, SAEs or tolerability issues that met study discontinuation criteria in ATI-501-AUAT-201. Subject is capable of regrowing scalp hair or maintaining prior scalp hair regrowth from ATI-501-AUAT-201 in the opinion of the investigator. If a woman of childbearing potential (WOCBP), must have a negative urine pregnancy test at Visit 1 and agree to: use a highly effective method of birth control for the duration of the study; not be planning a pregnancy during the study duration and use contraception for 30 days after last application of study medication. (Refer to Section 8.4). Be in good general health and free of any known disease state or physical condition which, in the investigator's opinion, might impair evaluation of the subject or which might expose the subject to an unacceptable risk by study participation. Be willing to maintain the same general hair style throughout the study period. Subjects who shave their scalp must be willing to refrain from shaving their scalp for at least one week or longer prior to each study visit, as determined by the investigator based on visible scalp hair growth. Hair trimming outside the treatment areas to maintain the current hair style is permitted. Be willing and able to follow all study instructions and to attend all study visits. Sexually active male subjects must agree to use a barrier method of contraception from the first application of study medication to at least 30 days after the last application of study medication. Exclusion Criteria: Subjects are excluded from this study if any 1 or more of the following criteria is met: Any study medication discontinuation criteria are met during participation in study ATI-501-AUAT-201. Females who are nursing, pregnant, or planning to become pregnant for the duration of the study and up to 30 days after the last application of study medication. 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. Sensitivity to any of the ingredients in the study medications. Unwillingness to refrain from weaves, hair extensions, or shaving of the scalp for at least one week or longer prior to each study visit, as determined by the investigator based on visible scalp hair growth the duration of the study.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: This Phase 2, multicenter, open-label study will evaluate the safety, tolerability, efficacy, and durability of efficacy of ATI-502 Topical Solution, 0.46% for the treatment of alopecia areata (AA), alopecia universalis (AU) and alopecia totalis (AT) in adult subjects who completed 24 weeks of treatment with ATI-501 Oral Suspension or Placebo Suspension.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ATI 502 0.46% Topical Solution<br>Subjects will apply ATI-502 Topical Solution, 0.46% twice-daily for 24 weeks followed by a 4-week post-treatment follow up period. | Drug: ATI 502 0.46% Topical Solution<br>* apply ATI-502 Topical Solution, 0.46% Twice a day (BID) to the entire scalp and if applicable, the eyebrow(s)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in the Severity of Alopecia Tool (SALT) Score | 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. For the primary endpoint, the primary efficacy variable was the mean relative percent change from the original baseline visit in study ATI-501-AUAT-201 and the SALT score at Week 24 on study ATI-502-AA-203. This represents the percentage of hair regrowth. It was calculated as the mean of the changes from baseline SALT score to the SALT score at Week 24, divided by baseline SALT score and expressed as a percentage. | Baseline to Week 24 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Mean Relative Percent Change From Baseline SALT Score | Mean Relative Percent Change from Baseline (from the current study ATI-502-AA-203) in Severity of Alopecia Tool (SALT). SALT score is on a percent scale where 0% is no hair loss and 100 % is total hair loss. A lower SALT score is a better outcome. A higher relative percent change is better. | Baseline to Week 24 | 
| Proportion of Subjects Achieving a SALT50 (Defined as a ≥ 50% Improvement From Baseline in SALT) by Visit | Proportion of subjects achieving a SALT 50 (defined as a ≥ 50% improvement from baseline in SALT) by visit 6. SALT score is on a percent scale where 0% is no hair loss and 100 % is total hair loss. | Baseline to Week 24 | 
| Proportion of Subjects Achieving a SALT 75 (Defined as a ≥ 75% Improvement From Baseline in SALT) by Visit | Proportion of subjects achieving SALT 75 (defined as a ≥ 75% improvement from baseline in SALT) by visit 6 SALT score is on a percent scale where 0% is no hair loss and 100 % is total hair loss. A bigger percent change is a more positive score. | Baseline to Week 24 | 
| Durability of Response | Durability of response: Proportion of subjects achieving a SALT 50 or SALT 75 at Week 24 (in study ATI-501-AUAT-201) and maintaining that response at week 24 in ATI-502-AA-203 study. | Baseline to Week 24 | 
| Change From Baseline in the Physician Global Impression of Severity (PhGIS) | Change from Baseline in the PhGIS by visit. The PhGIS is a 5 point scale from Mild to Extremely Severe. Mild is a better outcome. | Baseline to Week 24 |