Evaluation of Topical ATR12-351 in Adults With Netherton Syndrome
ConditionNetherton Syndrome
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorAzitra Inc.
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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
Trial design
Design model
Single group
Treatments tested in this trial
ATR12-351
DrugTopical ointment containing ATR12-351, a LEKTI-secreting strain of S. epidermidis
Treatment groups
12 Participants
are divided into 1 treatment groupGroup A: Internal controlled armExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Adverse events
Incidence, severity, seriousness and relatedness of all treatment-emergent adverse events
Time frame
84 daysSecondary outcomes
1
Investigator's Global Assessment (IGA)
Visual assessment of the overall severity of skin disease severity at each application site, using a 5-point scale (0, clear; 1, almost clear/minimal; 2, mild; 3, moderate; 4, severe). Higher scores indicate greater severity.
Time frame
42 days2
Patient's Global Assessment (PGA)
Visual assessment of the overall severity of skin disease severity at each application site, using a 5-point scale (0, clear; 1, almost clear/minimal; 2, mild; 3, moderate; 4, severe). Higher scores indicate greater severity.
Time frame
42 days3
NS-modified SCORAD
The investigator's objective assessment of the severity (peeling, scaling, lichenification, erythema, papules) and extent (surface of affected skin areas) of skin disease at each application site is combined with patient's subjective scores (pain and pruritus). Higher scores indicate greater severity with a maximum of 100 points possible.
Time frame
42 days4
Pharmacokinetics: plasma concentration
Concentration (ng/mL) of rhLEKTI in the plasma following topical application.
Time frame
42 daysSponsors and contacts
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Source ClinicalTrials.gov