UnknownPhase 2/3

A Pilot Study of the Efficacy and Safety of Dupilumab Versus Placebo in Patients With Netherton Syndrome

France24 participantsStarted 2020-07-23

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Adult patients affiliated to a social insurance protection regimen. * Clinical diagnosis of NS (7) (trichorrhexis invaginata, extensive scaling, skin inflammation, allergic manifestations) and absent or marked reduction of LEKTI staining. * Moderate to severe forms: NASA (Netherton Area Severity Assessment score) score ≥ 5/12 at inclusion. * Patients able to understand the study procedures including the ability to complete patient-based self-assessment questionnaires. * Patients who agree to sign the written informed consent. Exclusion Criteria: * Hypersensitivity to dupilumab or its excipients. * Modification of the usual treatment (emollients and topical corticosteroids used on a regular basis) within 2 weeks before inclusion. * Treatment with topical calcineurin inhibitors 1 week before inclusion. * Treatment with oral immunosuppressant (including cyclosporine, methotrexate, azathioprine, mycophenolate mofetil), oral retinoids (acitretin, alitretinoin, isotretinoin) or phototherapy within 4 weeks before inclusion. * Treatment with immunomodulating biologics (Tumor Necrosis Factor (TNF) inhibitor) 16 weeks before inclusion. * Treatment with another investigational drug within 8 weeks before inclusion. * Treatment with a systemic antibiotic within 2 weeks before inclusion. * Active skin infection requiring the use of a systemic therapy within 2 weeks before the inclusion. * Any other condition that according to the investigator will impair the abilit…

What they're measuring

The severity of the disease of the Netherton Area Severity Assessment score (NASA).

Timeframe: Day 0 and week 16

Trial details

NCT IDNCT04244006

SponsorUniversity Hospital, Toulouse

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2024-06-01

Contact for this trial

Nadège ALGANS

0561777204 algans.n@chu-toulouse.fr