Netherton syndrome MedDRA version: 20.0 Level: PT Classification code 10062909 Term: Netherton's syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Dupixent
Product Name: DUPILUMAB
Product Code: SAR231893
Pharmaceutical Form: Suspension for injection in pre-filled syringe
Pharmaceutical form of the placebo: Suspension for injection in
Sponsors
CHU de Toulouse
Eligibility
Sex/Gender
All
Inclusion criteria
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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: to assess the effect of dupilumab versus placebo on the severity of the disease in patients with moderate to severe NS as determined by the evolution at week 16 vs. baseline using a specific disease severity score (NASA).;Secondary Objective: - To assess the effect of dupilumab versus placebo on the evolution of severity of the disease in patients with moderate to severe NS at each visit versus baseline, using a specific disease severity score and other clinical parameters. - Effect on the bacterial or viral cutaneous secondary infections - Effect on the reduction of dermocorticosteroid intake - Effect on the evolution of quality of life at week 16 and week 28 versus baseline. - Effect on the evolution of systemic Th2 sensitization (total IgE blood levels and specific IgE) at week 16 versus baseline. - Effect on the evolution of skin inflammation on skin biopsies at week 16 versus baseline. - Effect on the evolution of protease activity on skin biopsies at week 16 versus baseline. - Effect on the evolution of microbiome analysis at week 16 versus baseline. - Effect on the evolution of the TEWL (transepidermal water loss), at week 16 versus baseline. - To assess the safety during the study period. ;Primary end point(s): The severity of the disease will be evaluated by measuring the evolution at week 16 vs. baseline of the Netherton Area Severity Assessment score (NASA). ;Timepoint(s) of evaluation of this end point: Day 0, Week 2, 4, 6, 8, 10, 12, 14, 16, 28. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Clinical efficacy - Bacterial or viral skin infections - Quantity of dermocorticosteroids - QOL : will be evaluated using the DLQI (Dermatology Life Quality Index (33)) and the IQoL-32 (specific ichthyosis QOL score (34)). - Systemic Th2 sensitization - Skin inflammation - Protease activity - Microbiome qualitative and quantitative analysis - TEWL: will be assessed using a Tewameter - Safety of dupilumab;Timepoint(s) of evaluation of this end point: Day 0, Week 2, 4, 6, 8, 10, 12, 14, 16, 28. | — |
Countries
France
Contacts
Public ContactALGANS
CHU de Toulouse
Outcome results
None listed