Evaluation of Topical ATR12-351 in Adults With Netherton Syndrome

Status Recruiting

Clinical Trial 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.

Clinical Trial Description

Netherton syndrome (NS) is a rare but severe autosomal recessive disease that affects the skin, hair, and immune system. NS presents at birth with red skin covered by fine scales, a specific hair shaft abnormality ("bamboo hair"), atopic lesions, and elevated systemic immunoglobulin E (IgE) levels. Patients often develop allergies and asthma, and the unfortunate prognosis for infants with NS is poor, with 10% mortality in the first 6 months of life and recurrent infections. There are no FDA-approved treatment options for NS. NS is caused by mutations in in the SPINK5 gene encoding the serine protease inhibitor lymphoepithelial Kazal-type related inhibitor (LEKTI). Absence of LEKTI causes unregulated activity of proteases in the kallikrein subfamily, causing excessive desquamation of skin. ATR12-351 is a topical ointment containing a lyophilized version of a live biotherapeutic product (LBP), Staphylococcus epidermidis (SE) strain designated SE351. This strain has been modified to be auxotrophic and to express recombinant human LEKTI protein (rhLEKTI). ATR12-351 is intended to address the underlying cause of NS by replacing deficient/dysfunctional LEKTI. The treatment consists of applying ATR12-351 to affected areas, where rhLEKTI produced by ATR12-351 will counter the dysregulated skin serine protease activity observed in NS patients. This first-in-human study is a randomized, double-blind, vehicle-controlled clinical study to assess the safety, tolerability, and pharmacokinetics (PK) of topical ATR12-351 in approximately 12 adult Netherton syndrome patients who will serve as their own control. This study will include a 14-day treatment period to investigate the safety and tolerability of ATR12-351 applied to the skin and will be followed a total of 84 days for safety. The results of this first-in-human clinical study will establish safety and tolerability as well as initial efficacy of ATR12-351 application in Netherton syndrome patients. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT06137157
Study type	Interventional
Source	Azitra Inc.
Contact	Travis Whitfill
Phone	203-646-6446
Email	clinicaltrials@azitrainc.com
Status	Recruiting
Phase	Phase 1
Start date	June 19, 2024
Completion date	August 1, 2025

View Details

See also
Status	Clinical Trial	Phase
Recruiting	`NCT05979831` - A Study to Explore Safety, Pharmacokinetics, and Early Clinical Signal of Efficacy of DS-2325a in Patients With Netherton Syndrome	Phase 1/Phase 2
Completed	`NCT00208026` - Safety Study of Elidel (Pimecrolimus) 1% Cream to Treat Netherton Syndrome	Phase 1/Phase 2
Completed	`NCT05583669` - A Study to Assess the Safety and Pharmacokinetics of Multiple Ascending Subcutaneous Doses of DS-2325a in Healthy Subjects	Phase 1
Completed	`NCT01428297` - A Study Evaluating the Safety and Efficacy of Topical BPR277 for the Treatment of Atopic Dermatitis and Netherton Syndrome	Phase 1
Recruiting	`NCT05856526` - A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome	Phase 2/Phase 3
Recruiting	`NCT05789056` - Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome	Phase 2/Phase 3
Recruiting	`NCT05521438` - Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome	Phase 2/Phase 3
Completed	`NCT05388903` - A Study to Assess the Safety and Pharmacokinetics of Single Ascending Subcutaneous and Intravenous Doses of DS-2325a in Healthy Subjects	Phase 1
Not yet recruiting	`NCT05902663` - Natural History of Netherton Syndrome
Recruiting	`NCT04244006` - A Pilot Study of the Efficacy and Safety of Dupilumab Versus Placebo in Patients With Netherton Syndrome	Phase 2/Phase 3
Recruiting	`NCT03417856` - Defining the Skin and Blood Biomarkers of Ichthyosis
Completed	`NCT02113904` - Clinical Trial Using Humira in Netherton Syndrome	Phase 2
Recruiting	`NCT05211830` - A Study to Evaluate Topically Applied SXR1096 Cream in Patients With Netherton Syndrome	Phase 1/Phase 2
Completed	`NCT03041038` - The Efficacy and Safety of Secukinumab in Patients With Ichthyoses	Phase 2
Recruiting	`NCT01545323` - Gene Therapy for Netherton Syndrome	Phase 1
Recruiting	`NCT02081313` - Natural History and Biological Study of Netherton Syndrome	N/A

Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.