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

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

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.

Study Overview

• Status: Withdrawn
• Conditions: Alopecia Areata; Alopecia Totalis; Alopecia Universalis
• Intervention / Treatment: Drug: Normal saline; Drug: BNZ-1

• Study Type: Interventional
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. 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
2. 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.
3. 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:

1. 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.
2. 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.
3. 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.
4. Any ongoing topical treatment for alopecia areata
5. History of or currently active primary or secondary immunodeficiency.
6. 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.
7. Received other investigational products or therapy in the 60 days prior to study drug administration.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

3

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Equillium

Study record dates

Study Major Dates

• Study Start (Anticipated): October 1, 2021
• Primary Completion (Anticipated): November 1, 2022
• Study Completion (Anticipated): November 1, 2022

Study Registration Dates

• First Submitted: May 10, 2018
• First Submitted That Met QC Criteria: May 10, 2018
• First Posted (Actual): May 22, 2018

Study Record Updates

• Last Update Posted (Actual): September 16, 2022
• Last Update Submitted That Met QC Criteria: September 14, 2022
• Last Verified: September 1, 2022

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: BNZ1-CT-204

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes