Ignite Creation Date:
2024-05-06 @ 7:46 PM
Last Modification Date:
2024-10-26 @ 3:14 PM
Study NCT ID:
NCT06137157
Status:
RECRUITING
Last Update Posted:
2024-06-21
First Post:
2023-11-07
Brief Title:
Evaluation of Topical ATR12-351 in Adults With Netherton Syndrome
Sponsor:
Azitra Inc
Organization:
Azitra Inc
Study Overview
Official Title:
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
Status:
RECRUITING
Status Verified Date:
2024-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
Detailed 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 deficientdysfunctional 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
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 deficientdysfunctional 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None