A Study to Evaluate Topically Applied SXR1096 Cream in Patients With Netherton Syndrome

Inclusion Criteria

• •1. Male or female patients aged 18 to 65 years at the screening visit and also adolescents (12-17 years) only after initial cohort of 5 adult patients have been treated for at least three days.
• •2. Patients/legal guardian endpoint must be willing to provide written informed consent.
• •3. Clinical diagnosis of NS including at least 3 out of the 4 following clinical criteria;
• •1. Neonatal erythroderma
• •2. Bamboo hair and/or alopecia
• •3. Chronic atopy specified as food allergy, asthma, rhino conjunctivitis and/or eczema for at least 2 years
• •4. Ichthyosis linearis circumflexa
• •4. Patients must be willing and able to understand and can comply with study requirements, apply the medication as instructed and be able to complete the study.
• •5. Absent LEKTI on immunohistochemistry of skin biopsy and/or confirmed mutation in SPINK5 gene
• •6. NS involvement ≥ 20% of Body Surface Area (BSA) required at both the screening and baseline visits.
• •7. Investigator Global Assessment (IGA) of two areas to be treated, score ≥3, i.e. moderate or severe for each area required. Each target area approx. 9% of BSA. i.e. equal to one arm.
• •8. Female of childbearing potential must either commit true abstinence when this is in line with the preferred and usual lifestyle or use an adequate and approved method of contraception throughout the study and for 4 weeks after the last study drug application. This criterion also applies to a prepubertal female subject who begins menses during the study.
• •Adequate and approved methods of contraception applicable for the subject and/or her partner are defined below:
• •Progestogen-only oral hormonal contraception
• •Combination of male or female condom with cap, diaphragm, or sponge with spermicide (double barrier methods)
• •Combined (estrogen- and progestogen-containing) oral, intravaginal, or transdermal hormonal contraception
• •Injectable or implanted hormonal contraception
• •Intrauterine devices or intrauterine hormone-releasing system
• •Bilateral tubal ligation or tube insert (such as the Essure system) at least 3 months before the study
• •Vasectomy of partner at least 3 months before the study
• •Female subjects of non-childbearing potential must meet one of the following criteria:
• •Absence of menstrual bleeding for 1 year prior to screening without any other medical reason
• •Documented hysterectomy, bilateral salpingectomy, or bilateral oophorectomy at least 3 months before screening