[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"netherton-syndrome\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:netherton-syndrome":24},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,41,75,100,126,147],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":14,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":22,"conditions":23,"keywords":25,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":30,"startDateStruct":33,"completionDateStruct":35,"leadSponsor":37,"locationsCount":40},"100614476","study-of-skin-and-gut-microbiome-in-a-skin-condition-involving-skin-barrier-impairment-and-allergic-symptoms-netherton-syndrome-100614476",false,"NCT07280091","Study of Skin and Gut Microbiome in a Skin Condition Involving Skin Barrier Impairment and Allergic Symptoms: Netherton Syndrome","DERMABIOTE","Inclusion Criteria:\n\nGroup A:\n\n* Children aged 10 years and older and adults with confirmed Netherton syndrome diagnosed at age 10 years or older (clinical and histological and\u002For molecular)\n* Patients and legal guardians informed about the study and not opposed to participation in the study\n\nGroup B:\n\n* Children aged 10 years and older and adults with no skin barrier impairment, dermatosis, inflammatory disease, or autoimmune disease.\n* Subjects and legal guardians informed about the study and who do not object to participation in the study.\n\nExclusion Criteria:\n\n* Refusal by parents\u002Fguardians, children, adolescents, or adults.\n* General or local antibiotic therapy within the month preceding the consultation.","ALL","10 Years",{"count":19,"type":20},30,"ESTIMATED","OBSERVATIONAL","It is proposed to conduct an exploratory study to analyze the skin, intestinal, and salivary microbiome, as well as the skin mycobiome and virome, of patients (adolescents and young adults) with Netherton syndrome, a condition characterized by an impaired skin barrier that most likely promotes the development of allergic manifestations.\n\nThe study will be conducted on patients with Netherton syndrome and control subjects in order to investigate possible correlation factors between the three microbiomes and identify which ones.",[24],"Netherton Syndrome",[26,27],"Netherton syndrome","skin microbiome","RECRUITING","2026-06-29",{"date":31,"type":32},"2026-06-30","ACTUAL",{"date":34,"type":32},"2026-05-26",{"date":36,"type":20},"2028-05",{"name":38,"class":39},"Assistance Publique - Hôpitaux de Paris","OTHER",1,{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":46,"acronym":4,"eligibilityCriteria":47,"healthyVolunteers":48,"sex":16,"minAge":49,"maxAge":50,"enrollmentInfo":51,"targetDuration":4,"studyType":53,"phases":54,"briefSummary":56,"conditions":57,"keywords":58,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":64,"lastUpdatePostDateStruct":65,"startDateStruct":67,"completionDateStruct":69,"leadSponsor":71,"locationsCount":74},"100557545","phase-1-a-study-of-the-safety-tolerability-pharmacokinetics-and-immunogenicity-of-bcx17725-100557545","NCT06539507","A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725","A Phase 1\u002F1b Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of Single and Multiple Ascending Doses of BCX17725 in Healthy Participants and Multiple Doses of BCX17725 in Participants With Netherton Syndrome","Key Inclusion Criteria:\n\n* Male or non-pregnant, non-lactating female aged 18 to 55 years, inclusive (Parts 1 and 2), 18 to 65 years, inclusive (Part 3), or 12 to 65 years, inclusive (Part 4)\n* Confirmed diagnosis of Netherton syndrome (Parts 3 and 4)\n* IGA score of ≥ 3 (Parts 3 and 4) and IASI score of ≥ 16 (Part 4)\n* BMI between 18 and 30 kg\u002Fm\\^2, inclusive (Parts 1 and 2)\n* Estimated glomerular filtration rate (eGFR) of ≥ 90 mL\u002Fmin\u002F1.73 m\\^2 (Parts 1 and 2) or ≥ 60 mL\u002Fmin\u002F1.73 m\\^2 (Part 3)\n* Agree to follow the protocol contraception requirements from screening until 90 days after the last dose of study drug\n* In the opinion of the investigator, expected to adequately comply with all required study procedures and restrictions for the duration of the study",true,"12 Years","65 Years",{"count":52,"type":20},78,"INTERVENTIONAL",[55],"PHASE1","This is a first-in-human, Phase 1\u002F1b, 4-part study that includes the evaluation of safety, tolerability, pharmacokinetics (PK), and immunogenicity of BCX17725 when administered via single and multiple doses in healthy adult participants (Parts 1 and 2), and multiple doses in adult participants with Netherton syndrome (Part 3). In Part 4, the effectiveness, safety, and tolerability of BCX17725 when administered via multiple IV and\u002For SC doses through 12 weeks will be evaluated in adult and adolescent participants with Netherton syndrome.",[24],[59,60,61,62,63,26],"First-in-human","Single ascending dose","Multiple ascending dose","Pharmacokinetics","Placebo control","2026-06-15",{"date":66,"type":32},"2026-06-16",{"date":68,"type":32},"2024-09-26",{"date":70,"type":20},"2026-12",{"name":72,"class":73},"BioCryst Pharmaceuticals","INDUSTRY",12,{"id":76,"slug":77,"hasResults":11,"nctId":78,"briefTitle":79,"officialTitle":80,"acronym":4,"eligibilityCriteria":81,"healthyVolunteers":11,"sex":16,"minAge":82,"maxAge":4,"enrollmentInfo":83,"targetDuration":4,"studyType":53,"phases":85,"briefSummary":88,"conditions":89,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":90,"lastUpdatePostDateStruct":91,"startDateStruct":93,"completionDateStruct":95,"leadSponsor":97,"locationsCount":99},"100634353","phase-2-phase-23-clinical-study-of-qrx003-lotion-in-subjects-with-netherton-syndrome-100634353","NCT07538583","Phase 2\u002F3 Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome","A Multicenter, Open-label Study of QRX003 Lotion in Participants With Netherton Syndrome","Inclusion Criteria:\n\n1. Participant is male or female and at least 4 years of age.\n2. Participant has provided written informed consent\u002Fassent. 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\u002Fconsent 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.\n3. 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\u002FScreening and Visit 2\u002FBaseline. Males must use a condom in addition to having their female partner use a highly effective method of birth control.\n4. Participant has a genetically confirmed diagnosis of NS prior to Visit 2\u002FBaseline. Participants without existing results must undergo genetic testing at Visit 1\u002FScreening, which must confirm the diagnosis of NS in order for the participants to be enrolled in the study.\n\nExclusion Criteria:\n\n1. Participant is pregnant, lactating, or is planning to become pregnant during the study.\n2. Participant has active cancer of any type excluding melanoma skin cancer outside of the Treatment Area.\n3. Participant has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled, in the opinion of the investigator.\n4. Participant has evidence of active infection during Screening, or serious infection within 30 days prior to Visit 2\u002FBaseline.\n5. Participant has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.\n6. Participant has ongoing or recent history of any other uncontrolled and\u002For clinically significant systemic disease or condition which, in the opinion of the investigator, should exclude participation in the study.\n7. Participant has used ultraviolet phototherapy within the Treatment Area within 4 weeks prior to Visit 2\u002FBaseline.\n8. 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\u002FBaseline.\n9. 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\u002FBaseline. Published or documented half-life of the product provided by the commercial supplier or Sponsor should be used to establish this value.\n10. 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\u002FBaseline.\n11. Participant has used any topical bland moisturizers\u002Femollients in the Treatment Area within 24 hours prior to Visit 2\u002FBaseline.\n12. Participant is currently enrolled in an investigational drug, biologic, or device study.","4 Years",{"count":84,"type":20},16,[86,87],"PHASE2","PHASE3","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.",[24],"2026-04-20",{"date":92,"type":32},"2026-04-21",{"date":94,"type":32},"2026-03-19",{"date":96,"type":20},"2027-03-31",{"name":98,"class":73},"Quoin Pharmaceuticals",5,{"id":101,"slug":102,"hasResults":11,"nctId":103,"briefTitle":104,"officialTitle":105,"acronym":4,"eligibilityCriteria":106,"healthyVolunteers":11,"sex":16,"minAge":107,"maxAge":4,"enrollmentInfo":108,"targetDuration":4,"studyType":53,"phases":109,"briefSummary":110,"conditions":111,"keywords":112,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":116,"lastUpdatePostDateStruct":117,"startDateStruct":119,"completionDateStruct":121,"leadSponsor":123,"locationsCount":125},"100526623","phase-1-evaluation-of-topical-atr12-351-in-adults-with-netherton-syndrome-100526623","NCT06137157","Evaluation of Topical ATR12-351 in Adults With Netherton Syndrome","A Randomized, Double-Blind, Vehicle-Controlled, First-in-Human Safety, Tolerability and Proof-of-Concept Study of Topical ATR12-351 in Adults With Netherton Syndrome","Inclusion Criteria:\n\n* Adults ≥18 years of age\n* Confirmed mutation of the serine protease inhibitor of Kazal type 5 (SPINK5) gene\n* Involvement of ≥20% of body surface area with skin changes consistent with Netherton syndrome\n\nExclusion Criteria:\n\n* 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\n* Open wounds or extensive areas of excoriation precluding identification of appropriate application sites in the Investigator's judgment\n* Concurrent involvement in any other clinical study\u002Fexpanded access program with an investigational drug or device or has participated in a clinical study within 30 days prior to the screening visit\n* Residing with an immunocompromised person in the same dwelling from the baseline visit through 2 weeks after the treatment period\n* History of ultraviolet phototherapy within the planned treatment area 4 weeks prior to baseline","18 Years",{"count":74,"type":20},[55],"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.",[24],[113,26,114,115],"Ichthyosis","Skin disease","ichthyosis linearis circumflexa","2026-02-04",{"date":118,"type":32},"2026-02-05",{"date":120,"type":32},"2024-06-19",{"date":122,"type":20},"2026-08-01",{"name":124,"class":73},"Azitra Inc.",2,{"id":127,"slug":128,"hasResults":11,"nctId":129,"briefTitle":130,"officialTitle":131,"acronym":4,"eligibilityCriteria":132,"healthyVolunteers":11,"sex":16,"minAge":133,"maxAge":4,"enrollmentInfo":134,"targetDuration":4,"studyType":53,"phases":136,"briefSummary":137,"conditions":138,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":139,"lastUpdatePostDateStruct":140,"startDateStruct":142,"completionDateStruct":144,"leadSponsor":146,"locationsCount":40},"100589365","phase-2-clinical-study-of-qrx003-lotion-in-subjects-with-netherton-syndrome-100589365","NCT06953466","Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome","A Single Center, Open Label, Expanded Access Study of QRX003 Lotion in Subjects With Netherton Syndrome","Inclusion Criteria:\n\n1. Subject is a male or non-pregnant female at least 14 years of age.\n2. Females must be post-menopausal , surgically sterile , or use an effective method of birth control , for the duration of the study and for 3 months following completion of treatment. Women of childbearing potential (WOCBP) must have a negative urine pregnancy test (UPT) at Visit 1\u002FScreening and Visit 2\u002FBaseline.\n3. Subject has a clinical diagnosis of NS and agrees to genetic testing at Visit 1\u002FScreening for confirmation of NS diagnosis if the subject does not have test results confirming a SPINK5 mutation.\n4. Subject is in good general health and free of any disease state or physical condition that might impair evaluation of NS or exposes the subject to an unacceptable risk by study participation.\n5. Subject is on a stable treatment regimen including topical therapy for NS prior to baseline that is expected to remain stable for the duration of the study\n\nExclusion Criteria:\n\n1. Subject is pregnant, lactating, or is planning to become pregnant during the study.\n2. Subject has any skin pathology in the Treatment Area or condition that, could interfere with the evaluation of the test article or requires use of interfering topical, systemic, or surgical therapy.\n3. Subject has active cancer of any type excluding non-melanoma skin cancer outside of the Treatment Area.\n4. Subject has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled.\n5. Subject has evidence of active infection during screening, or serious infection within 30 days prior to Visit 2\u002FBaseline.\n6. Subject has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.\n7. Subject has used ultraviolet phototherapy within the Treatment Area within 10 weeks prior to Visit 2\u002FBaseline.\n8. Subject has used topical prescription treatment in the Treatment Area within 10 weeks prior to Visit 2\u002FBaseline.\n9. Subject has used any topical steroid prescription treatments in the Treatment Area within 10 weeks prior to Visit 2\u002FBaseline.\n10. Subject is currently enrolled in an investigational drug, biologic, or device study.\n11. Subject has used an investigational drug, biologic, or device treatment within 30 days prior to Visit 2\u002FBaseline.","14 Years",{"count":135,"type":20},8,[86,87],"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:\n\n1. Does QRX003 impact the clinical presentation of NS in adults and minors by improving the clinical symptoms (diseased skin area, itch, and discomfort; based on clinical scoring, subject self-assessment, and other criteria)?\n2. What medical problems do participants have when taking QRX003?\n3. What percent of subjects will require rescue therapy?\n\nParticipants will:\n\nTake 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.",[24],"2025-07-16",{"date":141,"type":32},"2025-07-20",{"date":143,"type":20},"2025-07",{"date":145,"type":20},"2026-07-30",{"name":98,"class":73},{"id":148,"slug":149,"hasResults":11,"nctId":150,"briefTitle":151,"officialTitle":152,"acronym":4,"eligibilityCriteria":153,"healthyVolunteers":11,"sex":16,"minAge":133,"maxAge":4,"enrollmentInfo":154,"targetDuration":4,"studyType":53,"phases":156,"briefSummary":157,"conditions":158,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":159,"lastUpdatePostDateStruct":160,"startDateStruct":162,"completionDateStruct":164,"leadSponsor":166,"locationsCount":167},"100499884","phase-2-open-label-safety-and-efficacy-study-of-qrx003-lotion-in-subjects-with-netherton-syndrome-100499884","NCT05789056","Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome","A Multicenter, Open Label Study of QRX003 Lotion in Subjects With Netherton Syndrome","Inclusion Criteria:\n\n* Subject is a male or non-pregnant female at least 14 years of age.\n* Females must be post-menopausal , surgically sterile , or use an effective method of birth control , for the duration of the study and for 3 months following completion of treatment. Women of childbearing potential (WOCBP) must have a negative urine pregnancy test (UPT) at Visit 1\u002FScreening and Visit 2\u002FBaseline.\n* Subject has a clinical diagnosis of NS and agrees to genetic testing at Visit 1\u002FScreening for confirmation of NS diagnosis if the subject does not have test results confirming a SPINK5 mutation.\n* Subject has NS lesions in the Treatment Area (i.e., arms or lower legs).\n* Subject is in good general health and free of any disease state or physical condition that might impair evaluation of NS or exposes the subject to an unacceptable risk by study participation.\n* Subject is on a stable treatment regimen including systemic therapy for NS prior to baseline that is expected to remain stable for the duration of the study\n\nExclusion Criteria:\n\n* Subject is pregnant, lactating, or is planning to become pregnant during the study.\n* Subject has any skin pathology in the Treatment Area or condition that, could interfere with the evaluation of the test article or requires use of interfering topical, systemic, or surgical therapy.\n* Subject has active cancer of any type excluding non-melanoma skin cancer outside of the Treatment Area.\n* Subject has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled.\n* Subject has evidence of active infection during screening, or serious infection within 30 days prior to Visit 2\u002FBaseline.\n* Subject has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.\n* Subject has used ultraviolet phototherapy within the Treatment Area within 4 weeks prior to Visit 2\u002FBaseline.\n* Subject has used topical prescription treatment in the Treatment Area within 2 weeks prior to Visit 2\u002FBaseline.\n* Subject has used any topical bland moisturizers\u002Femollients in the Treatment Area within 24 hours prior to Visit 2\u002FBaseline.\n* Subject is currently enrolled in an investigational drug, biologic, or device study.\n* Subject has used an investigational drug, biologic, or device treatment within 30 days prior to Visit 2\u002FBaseline.",{"count":155,"type":20},20,[86,87],"To assess the safety, tolerability, and efficacy of QRX003 lotion (4%) when added to standard of care treatment regimen, including systemic therapy in subjects with Netherton syndrome",[24],"2025-03-28",{"date":161,"type":32},"2025-04-02",{"date":163,"type":32},"2023-03-14",{"date":165,"type":20},"2025-12-30",{"name":98,"class":73},4]