A Pilot Study of the Efficacy and Safety of Dupilumab Versus Placebo in Patients With Netherton Syndrome
Status:
Recruiting
Trial end date:
2023-06-01
Target enrollment:
Participant gender:
Summary
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.