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| ID | Type | Description | Link |
|---|---|---|---|
| Sponsor Funded | Other Identifier | Quoin Pharmaceuticals, Ltd. |
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The goal of this clinical trial (via an expanded access Treatment Protocol) is to learn if QRX003 (an investigational drug) applied topically to the skin (including up to the entire body [except the scalp]) works to treat the genetic disease Netherton syndrome. It will also learn about the safety of QRX003. The main questions the trial aims to answer are:
Participants will:
Take drug QRX003 twice daily (applied topically to all affected areas of the body excluding the scalp) for 3 months, visit the clinic once every 4-6 weeks for checkups and tests, and to keep a dosing diary that records the times they applied the drug.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| QRX003, 4% BID | Experimental | Subjects will apply test article twice daily (BID) for 12 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| QRX003, 4% Lotion | Drug | QRX003 Topical Lotion containing 4% active drug (serine protease inhibitor) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of subjects with 1-point reduction on IGA | Description: Proportion of subjects with 1-point reduction on the Investigator's Global Assessment (IGA) from Baseline. The IGA assesses overall severity of a subject's NS based on a 5-point scale where 0 = clear, 1 = almost clear, 2 = mild, 3 = moderate, and 4 = severe. | Up to week 16 |
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| Measure | Description | Time Frame |
|---|---|---|
| Proportion of subjects with 2-point reduction on IGA | Proportion of subjects with 2-point reduction in IGA from Baseline. | Up to week 16 |
| NS surface area change | Change from Baseline in total Body Surface Area (BSA) affected by NS in the Treatment Area |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| TI Clinical Research | Contact | 858-571-1800 | 147 | clinicalresearch@therapeuticsinc.com |
| Oleg G Khatsenko | Contact | 858-571-1800 | 166 | okhatsenko@therapeuticsinc.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Northwestern Memorial Hospital | Recruiting | Chicago | Illinois | 60611 | United States |
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| ID | Term |
|---|---|
| D056770 | Netherton Syndrome |
| ID | Term |
|---|---|
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016113 | Ichthyosiform Erythroderma, Congenital |
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Open-label
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| Up to week 16 |
| WI-NRS score change | Change from Baseline in the Worst Itch-Numeric Rating Scale (WI-NRS) score ≥4. Itch severity is graded on an 11-point scale where 0 represents "no itch" and 10 represents "worst itch imaginable". | Up to week 16 |
| Safety Assessment-AEs | Any local and systemic AEs (Adverse Events)/serious AEs | Up to week 16 |
| D007057 | Ichthyosis |
| D012868 | Skin Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D007232 | Infant, Newborn, Diseases |
| D007642 | Keratosis |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |