Phase 2 Trial of BNZ-1 in Patients With Moderate to Severe Alopecia Areata

Study Overview
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Brief Summary
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This study is a randomized, double-blind, placebo-controlled, multi-center, dose-ranging study to characterize the efficacy and safety of BNZ-1 administered by slow IV push weekly for 3 months to adults diagnosed with moderate to severe alopecia areata, defined as having a >50% loss of terminal hair on the scalp. The study has three periods: 30-Day Screening Period 3-Month Treatment Period 3-Month Follow-up Period The study will be conducted at approximately 15-20 clinical sites in the United States.

Official Title
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A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Trial of Intravenous BNZ-1 in Patients With Moderate to Severe Alopecia Areata

Conditions
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Alopecia Areata, Alopecia Totalis, Alopecia Universalis

Intervention / Treatment
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* Drug: BNZ-1
* Drug: Normal saline


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Must have a diagnosis of moderate to severe AA defined as the presence of ≥50% total terminal hair loss at baseline as measured using the SALT score for > 6 months, but <10 yrs. Includes Alopecia Totalis and Alopecia Universalis Patients may be naïve to treatment or have been treated with intralesional (IL) steroids or other treatments for AA, with a washout of at least 30 days or 5 times the elimination half-life prior to Day 1. Prior treatment with a janus kinase (JAK) inhibitor (e.g., tofacitinib, ruxolitnib) is allowed, but patients considered refractory to a JAK inhibitor are excluded from this trial. Exclusion Criteria: e subjects from this study if any of the following criteria are met: Clinically relevant hepatic, neurological, pulmonary, ophthalmological, endocrine, renal, or other major systemic disease making implementation of the protocol or interpretation of the study results difficult, or that would put the subject at risk by participating in the study in the opinion of the Investigator. Other active dermatologic conditions, including but not limited to vitiligo, atopic dermatitis, or non-scalp psoriasis are not exclusionary. Patients with active inflammatory skin disease on the scalp, including but not limited to psoriasis, seborrheic dermatitis or folliculitis, which cannot be adequately controlled prior to screening. Ongoing treatment with an immune system modulator or suppressant that cannot be discontinued prior to screening and at least 30 days or 5-times the elimination half-life prior to treatment. Any ongoing topical treatment for alopecia areata History of or currently active primary or secondary immunodeficiency. Known active bacterial, viral, fungal, mycobacterial infection, or other infection (including latent tuberculosis [TB] unless treatment is documented or atypical mycobacterial disease [but excluding fungal infection of nail beds, minor upper respiratory tract infection, and minor skin conditions]), or any major episode of infection that required hospitalization or treatment with IV antibiotics within 60 days of study drug administration or oral antibiotics within 30 days prior to study drug administration. Received other investigational products or therapy in the 60 days prior to study drug administration.

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
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: Placebo<br>Normal saline | Drug: Normal saline<br>* Dose volume consistent with weight-based dosing of BNZ-1<br>|
| Experimental: Low Dose BNZ-1<br>0.5 mg/kg QW | Drug: BNZ-1<br>* PEGylated peptide inhibitor of IL-2, IL-9, and IL-15<br>|
| Experimental: Moderate Dose BNZ-1<br>2 mg/kg QW | Drug: BNZ-1<br>* PEGylated peptide inhibitor of IL-2, IL-9, and IL-15<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline using the Severity of Alopecia Tool (SALT) score |  | 3 months | 
| Treatment-Emergent Adverse Events | Safety profile defined as incidence, severity and relationship of treatment-emergent adverse events | 3 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Alopecia Areata Investigator Global Assessment (AA-IGA) | Hair Satisfaction Scale | 3 & 6 months | 
| Patient Global Assessment | Hair satisfaction scale | 3 & 6 months | 
| Proportion of Patients with SALT50 |  | 3 months & 6 months | 
| Proportion of Patients with SALT75 |  | 3 months & 6 months | 
| Proportion of Patients with SALT90 |  | 3 months & 6 months | 
| Proportion of Patients with SALT100 (Disease-free) |  | 3 months & 6 months | 
| Change from Baseline on Alopecia Areata Symptom Impact Scale (AASIS) |  | 3 months | 
| Change from Baseline on Dermatology Life Quality Index (DLQI) |  | 3 months |