Evaluation of Topical ATR12-351 in Adults With Netherton Syndrome

NCT06137157 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: NTH201
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

Brief Summary

The objectives of this clinical trial are to evaluate the safety and tolerability of topically applied ATR12-351, to understand what the body does to ATR12-351, and to observe treatment benefits of the drug in approximately 12 adult patients with Netherton Syndrome (NS). ATR12-351 will be applied to skin lesions on one side of the body, while the vehicle control will be applied to similar lesion on the other side of the body twice daily for 2 weeks.

Conditions

Netherton Syndrome

Keywords

Ichthyosis; Netherton syndrome; Skin disease; ichthyosis linearis circumflexa

Outcome Measures

Primary Outcomes (1)

• Adverse events

  Incidence, severity, seriousness and relatedness of all treatment-emergent adverse events

  84 days

Secondary Outcomes (5)

• Investigator's Global Assessment (IGA)

  42 days

• Patient's Global Assessment (PGA)

  42 days

• NS-modified SCORAD

  42 days

• Pharmacokinetics: plasma concentration

  42 days

• Pharmacokinetics: skin concentration (Cmax skin)

  42 days

Study Arms (1)

Internal controlled arm

EXPERIMENTAL

ATR12-351 on left side of body, vehicle on right side of body in one group; vehicle on left side of body, ATR12-351 on right side of body.

Drug: ATR12-351

Interventions

ATR12-351 DRUG

Topical ointment containing ATR12-351, a LEKTI-secreting strain of S. epidermidis

Also known as: ATR-12

Internal controlled arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adults ≥18 years of age
• Confirmed mutation of the serine protease inhibitor of Kazal type 5 (SPINK5) gene
• Involvement of ≥20% of body surface area with skin changes consistent with Netherton syndrome

You may not qualify if:

• Use of biologic therapies, antibiotics, antihistamines, corticosteroids, retinoids, disease-modifying antirheumatic drugs (DMARDs), immunosuppressive agents, phosphodiesterase-4 (PDE4) inhibitors, topical calcineurin inhibitors, or topical Janus kinase (JAK) inhibitors
• Open wounds or extensive areas of excoriation precluding identification of appropriate application sites in the Investigator's judgment
• Concurrent involvement in any other clinical study/expanded access program with an investigational drug or device or has participated in a clinical study within 30 days prior to the screening visit
• Residing with an immunocompromised person in the same dwelling from the baseline visit through 2 weeks after the treatment period
• History of ultraviolet phototherapy within the planned treatment area 4 weeks prior to baseline

Sponsors & Collaborators

• Azitra Inc.: lead

Study Sites (2)

Stanford University

Palo Alto, California, 94304, United States

RECRUITING

Yale University

New Haven, Connecticut, 06519, United States

COMPLETED

MeSH Terms

Conditions

Netherton Syndrome; Ichthyosis; Skin Diseases

Condition Hierarchy (Ancestors)

Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ichthyosiform Erythroderma, Congenital; Skin Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Infant, Newborn, Diseases; Keratosis; Skin and Connective Tissue Diseases

Study Officials

• Mary Spellman, MD

  Azitra Inc.

  STUDY DIRECTOR

Central Study Contacts

Travis Whitfill

CONTACT

203-646-6446; clinicaltrials@azitrainc.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Masking Details: Patients, investigators, and assessors will be masked as to whether patients received ATR12-351 on the right or left side of the body.
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Intrapatient, randomized controlled study: ATR12-351 will be applied to skin lesions on one side of the body, while the vehicle control will be applied to similar lesion on the other side of the body twice daily for 2 weeks. Each patient is randomized to receive ATR12-351 on either the left or right side.

Study Record Dates

• First Submitted: November 7, 2023
• First Posted: November 18, 2023
• Study Start: June 19, 2024
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): August 1, 2026
• Last Updated: February 5, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)