A pilot study of the efficacy and safety of dupilumab versus placebo in patients with Netherton syndrome

Phase 1

• Conditions: etherton syndrome; MedDRA version: 20.0Level: PTClassification code 10062909Term: Netherton's syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

• Registration Number: EUCTR2019-001220-35-FR
• Lead Sponsor: CHU de Toulouse

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2019-04-23
• Last Update Posted: 3 June 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

- Adult patients (=18 years) affiliated to a social insurance protection regimen.
-Clinical diagnosis of NS 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.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 24
F.1.3 Elderly (>=65 years)
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

-Hypersensitivity to dupilumab or its excipients.
-Modification of the usual treatment within 2 weeks before inclusion.
-Treatment with topical calcineurin inhibitors 1 week before inclusion.
-Treatment with oral immunosuppressant , oral retinoids or phototherapy within 4 weeks before inclusion.
-Treatment with immunomodulating biologics 16 weeks before inclusion.
-Treatment with another investigational drug within 8 weeks before inclusion.
-Treatment with a systemic antibiotic within 1 week 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 ability to evaluate treatment effect.
-Known or suspected history of immunosuppression, including history of invasive opportunistic infections
-Current infections including infection with helminthes.
-Pregnant or breastfeeding women, or women planning to become pregnant or breastfeed during the study.
-Mental or physical incapacity to fill in the questionnaires.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified