A Phase 2/3 interventional study of Dupilumab Prefilled Syringe and Placebo Prefilled Syringe in Netherton Syndrome, sponsored by University Hospital, Toulouse. Terminated at 2 sites in France. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-21.
Sponsored by University Hospital, Toulouse · Phase 2/3, Interventional, and Treatment
To date, there are no effective therapy for the management of Netherton Syndrome (NS) Patients use emollients with a limited efficacy on scaling and no efficacy on skin inflammation and pruritus. They may also use topical corticosteroids or calcineurin inhibitors in case of eczematous lesions. The use of therapies targeting skin inflammation has been reported in a few case reports. Their efficacy is very limited and their uses are limited because of the chronicity of the disease, the impaired skin barrier function and the risk for skin infections and skin cancers. Therefore, there is a huge medical need for novel therapies in NS.The expected consequences of this study are that a 16-week course of dupilumab will be more effective than placebo for the treatment of moderate to severe NS Dupilumab could therefore improve skin condition and quality of life.
This is a proof of concept (pilot) double-blind randomized placebo-controlled study evaluating the efficacy and safety of dupilumab for the treatment of NS. Patients will be randomized in a 2:1 ratio to receive dupilumab (2 doses of 300 mg), or placebo, at baseline and then 1 dose of dupilumab 300 mg, or placebo, every 2 weeks until week 14 (total of 8 administrations).Moderate to severe NS were selected in order to be able to measure the improvement of skin condition.
21 studies on the registry are indexed under Netherton Syndrome; 6 are open to participants now.
This study's enrollment of 10 is below the median of 14 across 18 interventional studies indexed under Netherton Syndrome.
Browse Netherton Syndrome studies →University Hospital, Toulouse is the lead sponsor of 794 studies on the registry; 214 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
The patient will receive 2 doses at baseline and then 1 dose every 2 weeks (8 administrations in total) of Dupilumab 300 mg (syringe of 2 mL for subcutaneous administration).
Drug: Dupilumab Prefilled Syringe
The patient will receive 2 doses at baseline and then 1 dose every 2 weeks (8 administrations in total) of placebo (syringe of 2 mL for subcutaneous administration)..
Other: Placebo Prefilled Syringe
administration of dupilumab corresponding to dupilumab arm
administration of placebo corresponding to placebo arm
The severity of the disease of the Netherton Area Severity Assessment score (NASA).
NASA score
Time frame: Day 0 and week 16
Clinical efficacy severity of pruritus and pain
NASA score
Time frame: Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28
Presence of infections (adverse event)
Number of Bacterial or viral Skin infections
Time frame: Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28
Quantity of dermocorticosteroids used between each visit will be evaluated by questioning the patient.
number of tubes multiplied by the weight of one tube
Time frame: Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28
QOL score
QOL score
Time frame: Day 0, Week 16 and 28
Skin inflammation
number of inflammation markers on biopsies
Time frame: Day 0 and week 16
Protease activity
number of protease markers on biopsies
Time frame: Day 0 and week 16
Microbiome qualitative and quantitative analysis
number and form of bacteria
Time frame: Day 0 and week 16
Transepidermal water loss (TEWL)
Measured by a Tewameter applied on a standardized area in the anterior aspect of the forearm,
Time frame: Day 0 and Week 16
Safety of dupilumab
Blood tests performed every month until Week 16 (liver and renal tests, total blood count)
Time frame: Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28
Plan to share: No
No publications or documents are linked to this record.
This study is terminated, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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University Hospital, Toulouse