Actively Recruiting
A Randomized, Double-Blind, Vehicle-Controlled, First-in-Human Safety, Tolerability and Proof-of-Concept Study of Topical ATR12-351 in Adults With Netherton Syndrome
Led by Azitra Inc. · Updated on 2026-02-05
12
Participants Needed
2
Research Sites
8 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and treatment effects of ATR12-351, a topical ointment containing a modified live biotherapeutic product, in adults with Netherton Syndrome (NS). NS is a rare genetic disorder affecting skin, hair, and immune function, characterized by red, scaly skin, hair abnormalities, and high allergy risk. Currently, there are no FDA-approved treatments for NS, and this first-in-human study aims to address the underlying cause by replacing a deficient skin protein. The study applies ATR12-351 ointment to skin lesions on one side of the body and a placebo vehicle ointment to similar lesions on the other side twice daily for 14 days. Participants serve as their own control in this randomized, double-blind, vehicle-controlled design. The treatment period is followed by a total of 84 days of safety monitoring to assess adverse effects, drug absorption, and preliminary skin improvements. During the study, about 12 adult patients will have their skin reactions assessed through clinical evaluations and pharmacokinetic measures of drug levels in skin and plasma. Skin lesion severity and patient-reported outcomes will be recorded up to 42 days post-treatment. Safety will be closely followed for 84 days. This detailed monitoring helps researchers understand how the drug behaves in the body and its potential benefits for NS skin symptoms.
CONDITIONS
Brief Title
Evaluation of Topical ATR12-351 in Adults With Netherton Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults 18 years of age or older
- Confirmed mutation in the SPINK5 gene
- Skin involvement of 20% or more of body surface area with changes consistent with Netherton Syndrome
You will not qualify if you...
- Use of biologic therapies, antibiotics, antihistamines, corticosteroids, retinoids, DMARDs, immunosuppressive agents, PDE4 inhibitors, topical calcineurin inhibitors, or topical JAK inhibitors
- Open wounds or extensive excoriation preventing proper application
- Participation in another clinical study or expanded access program within 30 days prior to screening
- Living with an immunocompromised person during treatment and 2 weeks after
- History of ultraviolet phototherapy in planned treatment area within 4 weeks prior to baseline
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 14 days
Participants apply the topical ATR12-351 ointment to affected areas on one side of the body and a vehicle ointment on the other side to assess safety and tolerability.
Daily application with visits as scheduled during treatment
Duration - Up to 84 days after treatment
Participants are monitored for safety and any adverse events following treatment.
Periodic visits during the 84-day follow-up period
Trial Site Locations
Total: 2 locations
1
Stanford University
Palo Alto, California, United States, 94304
Actively Recruiting
2
Yale University
New Haven, Connecticut, United States, 06519
Completed
Research Team
T
Travis Whitfill
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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