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

Sponsor
University Hospital, Toulouse
Study ID
NCT04244006
Phase
PHASE2/PHASE3
Status
Unknown

Conditions

  • Netherton Syndrome

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Dupilumab Prefilled Syringe — DRUG
    administration of dupilumab corresponding to dupilumab arm
  • Placebo Prefilled Syringe — OTHER
    administration of placebo corresponding to placebo arm

Study Details

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.

Key Dates

Start date
Jul 23, 2020
Status verified
Aug 2023
Primary completion
Jun 1, 2024
Completion
Jun 1, 2024

Study Design

Enrollment
24 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Dupilumab
    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).
  • Placebo Comparator: Placebo
    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)..

Primary Outcome Measure

The severity of the disease of the Netherton Area Severity Assessment score (NASA). [ Time Frame: Day 0 and week 16 ]

Central Contacts

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