Phase 2/3 Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome

Inclusion criteria

• Participant is male or female and at least 4 years of age.
• Participant has provided written informed consent/assent. Based on the participant's age and local regulations , a participant under legal age of consent must provide written informed assent and be accompanied by the parent or legal guardian at the time of assent/consent signing. The parent or legal guardian must provide informed consent for the participant. If a participant reaches the age at which consent is required by local regulations during the study, the participant must provide written informed consent at that time to continue study participation.
• Female participants must either not have experienced menarche, be postmenopausal , or be surgically sterile , or use a highly effective method of birth control , for the duration of the study and for 3 months following EOT. Women of childbearing potential (WOCBP) must have a negative urine pregnancy test (UPT) at Visit 1/Screening and Visit 2/Baseline. Males must use a condom in addition to having their female partner use a highly effective method of birth control.
• Participant has a genetically confirmed diagnosis of NS prior to Visit 2/Baseline. Participants without existing results must undergo genetic testing at Visit 1/Screening, which must confirm the diagnosis of NS in order for the participants to be enrolled in the study.

Exclusion criteria

• Participant is pregnant, lactating, or is planning to become pregnant during the study.
• Participant has active cancer of any type excluding melanoma skin cancer outside of the Treatment Area.
• Participant has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled, in the opinion of the investigator.
• Participant has evidence of active infection during Screening, or serious infection within 30 days prior to Visit 2/Baseline.
• Participant has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.
• Participant has ongoing or recent history of any other uncontrolled and/or clinically significant systemic disease or condition which, in the opinion of the investigator, should exclude participation in the study.
• Participant has used ultraviolet phototherapy within the Treatment Area within 4 weeks prior to Visit 2/Baseline.
• Participant has used systemic non-biologics prescription treatment for NS or that in the opinion of the investigator may affect the participant's NS (examples include, but are not limited to antibiotics, retinoids, etc.) within 4 weeks prior to Visit 2/Baseline.
• Participant has used systemic biologic therapy for NS or that in the opinion of the investigator may affect the participant's NS within 5 half-lives of the biologic therapy prior to Visit 2/Baseline. Published or documented half-life of the product provided by the commercial supplier or Sponsor should be used to establish this value.
• Participant has used topical prescription treatments, including steroids (examples include, but are not limited to keratolytics, steroids, retinoids, etc.) in the Treatment Area within 2 weeks prior to Visit 2/Baseline.
• Participant has used any topical bland moisturizers/emollients in the Treatment Area within 24 hours prior to Visit 2/Baseline.
• Participant is currently enrolled in an investigational drug, biologic, or device study.