Phase 2/3 Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome
ConditionNetherton Syndrome
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age4+
SponsorQuoin Pharmaceuticals
About this trial
This is a multicenter, open-label study to evaluate the safety, tolerability, and efficacy of 4% QRX003 lotion applied twice daily (BID) for 12 weeks to Netherton syndrome (NS) diseased skin in all affected areas of the body excluding the scalp (the Treatment Area), totaling approximately 50% Body Surface Area (BSA) or more.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Design model
Single group
Treatments tested in this trial
QRX003
DrugSerine Protease Inhibitor
Treatment groups
16 Participants
are divided into 1 treatment groupGroup A: QRX003 lotion, 4% BIDExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Proportion of subjects with 1-point reduction on IGA
The IGA (Investigator Global Assessment) is a 5-point scale used to assess the overall severity of a Subject's NS condition.
Time frame
Week 12Secondary outcomes
1
Proportion of subjects with 2-point improvement on the WI-NRS
Subjects will rate their itch intensity using the 11-point Worst-Itch-Numeric Rating Scale (WI-NRS)
Time frame
Week 122
PGI-C Score
Patient (or Caregiver) Global Impression of Change (PGI-C) is a 7-point, self-assessed scale measuring the patient's (or caregiver's) improvement of their NS condition.
Time frame
Week 123
CGI-C Score
Clinical Global Impression of Change (PGI-C) is a 7-point scale used by the clinician to assess improvement of the NS condition.
Time frame
Week 12Sponsors and contacts
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Source ClinicalTrials.gov