Topical treatment for adults with Netherton Syndrome
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
This study is testing a new ointment for adults with Netherton Syndrome to see if it helps improve their skin condition.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Azitra Inc. Industry-sponsored |
| Locations | 2 sites (Palo Alto, California and 1 other locations) |
| Trial ID | NCT06137157 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the safety and tolerability of a topical ointment, ATR12-351, in adults diagnosed with Netherton Syndrome. The study involves applying ATR12-351 to skin lesions on one side of the body while using a vehicle control on the other side, twice daily for two weeks. The trial aims to understand how the body processes ATR12-351 and to observe any treatment benefits in approximately 12 participants. Netherton Syndrome is a rare genetic condition that severely affects the skin, hair, and immune system, and currently has no FDA-approved treatments.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 and older with a confirmed mutation in the SPINK5 gene and significant skin involvement due to Netherton Syndrome.
Not a fit: Patients currently using biologic therapies or other immunosuppressive treatments may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a novel therapeutic option for patients suffering from Netherton Syndrome.
How similar studies have performed: While this approach is novel, it is based on the underlying biology of Netherton Syndrome, and similar studies have not yet been conducted.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Exclusion Criteria: * 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
Where this trial is running
Palo Alto, California and 1 other locations
- Stanford University — Palo Alto, California, United States (Recruiting)
- Yale University — New Haven, Connecticut, United States (Completed)
Study contacts
- Study coordinator: Travis Whitfill
- Email: clinicaltrials@azitrainc.com
- Phone: 203-646-6446
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.