Netherton Syndrome clinical trials
The registry lists 22 studies for Netherton Syndrome across more than 10 countries. 5 are recruiting or about to start. 20 of them are mainly about Netherton Syndrome (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-06.
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Status
Who runs them
Quoin Pharmaceuticals (4) · Daiichi Sankyo (3) · Boehringer Ingelheim (2) · Assistance Publique - Hôpitaux de Paris (2) · Northwestern University (2) · Great Ormond Street Hospital for Children NHS Foundation Trust (1) · University Hospital, Toulouse (1) · Institut National de la Santé Et de la Recherche Médicale, France (1)
What is being tested here
Drugs under test in Netherton Syndrome studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| QRX003 | 3 | 4 | 4 |
| Eflumenibep alfa | 0 | 0 | 3 |
| Secukinumab | 0 | 0 | 2 |
| Dupilumab | 0 | 1 | 2 |
| Adalimumab | 0 | 0 | 1 |
| Pimecrolimus | 0 | 0 | 1 |
| Spesolimab | 0 | 1 | 1 |
| Ustekinumab | 0 | 0 | 1 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 4, phase 3 first
- Dec 2026Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome · Phase 3 · Quoin Pharmaceuticals
- Dec 2026Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome · Phase 3 · Quoin Pharmaceuticals
- Jan 2027Phase 2/3 Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome · Phase 3 · Quoin Pharmaceuticals
- Dec 2026A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725 · BioCryst Pharmaceuticals
Who these studies take
Of the 4 Netherton Syndrome studies recruiting now, 4 (100%) set no upper age limit and 0 (0%) stop at 75 or younger. 4 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 2 (50%) | 2 (50%) |
| 18 | 4 (100%) | 4 (100%) |
| 30 | 4 (100%) | 4 (100%) |
| 40 | 4 (100%) | 4 (100%) |
| 50 | 4 (100%) | 4 (100%) |
| 65 | 4 (100%) | 4 (100%) |
| 75 | 4 (100%) | 4 (100%) |
| 85 | 4 (100%) | 4 (100%) |
Across all 22 studies, recruiting or not: 36% stop at 75 or younger and 36% at 65; 68% take children; 0% are for women only and 0% for men only; 32% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 22 of 22. search them.
- Phase 2/3 Clinical Study of QRX003 Lotion in Subjects With Netherton SyndromeRecruitingNCT07538583Phase 2/32 countries
- Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton SyndromeRecruitingNCT05789056Phase 2/31 country
- Clinical Study of QRX003 Lotion in Subjects With Netherton SyndromeRecruitingNCT06953466Phase 2/31 country
- Study of Skin and Gut Microbiome in a Skin Condition Involving Skin Barrier Impairment and Allergic Symptoms: Netherton SyndromeRecruitingNCT072800911 country
- Defining the Skin and Blood Biomarkers of IchthyosisEnrolling by invitationNCT034178561 country
- A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725Active, not recruitingNCT06539507Phase 15 countries
- A Pilot Study of the Efficacy and Safety of Dupilumab Versus Placebo in Patients With Netherton SyndromeTerminatedNCT04244006Phase 2/31 country
- Evaluation of Topical ATR12-351 in Adults With Netherton SyndromeCompletedNCT06137157Phase 11 country
- Natural History of Netherton SyndromeTerminatedNCT059026632 countries
- A Study to Explore Safety, Pharmacokinetics, and Early Clinical Signal of Efficacy of DS-2325a in Patients With Netherton SyndromeTerminatedNCT05979831Phase 1/21 country
- Clinical Trial Using Humira in Netherton SyndromeCompletedNCT02113904Phase 21 country
- Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton SyndromeActive, not recruitingNCT05521438Phase 2/31 country
- A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton SyndromeTerminatedNCT05856526Phase 2/314 countries
- A Study to Evaluate Topically Applied SXR1096 Cream in Patients With Netherton SyndromeCompletedNCT05211830Phase 1/24 countries
- Natural History and Biological Study of Netherton SyndromeCompletedNCT02081313Not applicable1 country
- A Study to Assess the Safety and Pharmacokinetics of Multiple Ascending Subcutaneous Doses of DS-2325a in Healthy SubjectsCompletedNCT05583669Phase 11 country
- A Study to Assess the Safety and Pharmacokinetics of Single Ascending Subcutaneous and Intravenous Doses of DS-2325a in Healthy SubjectsCompletedNCT05388903Phase 11 country
- The Efficacy and Safety of Secukinumab in Patients With IchthyosesCompletedNCT03041038Phase 21 country
- Scientific Substantiation and Assessment of the Effectiveness of Pathogenetic Methods of Therapy for Congenital Ichthyosis in ChildrenStatus unknownNCT04996485Phase 41 country
- A Study Evaluating the Safety and Efficacy of Topical BPR277 for the Treatment of Atopic Dermatitis and Netherton SyndromeCompletedNCT01428297Phase 12 countries
- Safety Study of Elidel (Pimecrolimus) 1% Cream to Treat Netherton SyndromeCompletedNCT00208026Phase 1/21 country
- Gene Therapy for Netherton SyndromeStatus unknownNCT01545323Phase 11 country