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En pilotundersøgelse af effektiviteten og sikkerheden af ​​Dupilumab versus placebo hos patienter med Nethertons syndrom (NS-DUPI)

15. september 2026 opdateret af: University Hospital, Toulouse

En randomiseret dobbeltblindet pilotundersøgelse af effektiviteten og sikkerheden af ​​Dupilumab versus placebo hos patienter med Nethertons syndrom

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.

Studieoversigt

Status

Afsluttet

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

This is a proof of concept (pilot) double-blind randomized placebo-controlled study evaluating the efficacy and safety of dupilumab for the treatment of NS. Patients will be randomized in a 2:1 ratio to receive dupilumab (2 doses of 300 mg), or placebo, at baseline and then 1 dose of dupilumab 300 mg, or placebo, every 2 weeks until week 14 (total of 8 administrations).Moderate to severe NS were selected in order to be able to measure the improvement of skin condition.

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

10

Fase

  • Fase 2
  • Fase 3

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

  • Frankrig
      • Paris, Frankrig, 75015
        • Dermatologie Necker
      • Toulouse, Frankrig
        • Dermatology

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

18 år og ældre (Voksen, Ældre voksen)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

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 ability to evaluate treatment effect.
  • Known or suspected history of immunosuppression, including history of invasive opportunistic infections (e.g., tuberculosis, histoplasmosis, listeriosis, coccidioidomycosis, pneumocystis, aspergillosis).
  • Current infections including infection with helminthes.
  • Pregnant or breastfeeding women, or women planning to become pregnant or breastfeed during the study. Women of childbearing age, potentially sexually active, and unwilling to use adequate birth control methods.
  • Mental or physical incapacity to fill in the questionnaires.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Tredobbelt

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: 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).
Medicin: Dupilumab Prefilled Syringe

administration of dupilumab corresponding to dupilumab arm
Placebo komparator: 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)..
Andet: Placebo Prefilled Syringe

administration of placebo corresponding to placebo arm

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
The severity of the disease of the Netherton Area Severity Assessment score (NASA).
Tidsramme: Day 0 and week 16

NASA score
Day 0 and week 16

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Clinical efficacy severity of pruritus and pain
Tidsramme: Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28

NASA score
Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28
Presence of infections (adverse event)
Tidsramme: Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28

Number of Bacterial or viral Skin infections
Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28
Quantity of dermocorticosteroids used between each visit will be evaluated by questioning the patient.
Tidsramme: Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28

number of tubes multiplied by the weight of one tube
Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28
QOL score
Tidsramme: Day 0, Week 16 and 28

QOL score
Day 0, Week 16 and 28
Skin inflammation
Tidsramme: Day 0 and week 16

number of inflammation markers on biopsies
Day 0 and week 16
Protease activity
Tidsramme: Day 0 and week 16

number of protease markers on biopsies
Day 0 and week 16
Microbiome qualitative and quantitative analysis
Tidsramme: Day 0 and week 16

number and form of bacteria
Day 0 and week 16
Transepidermal water loss (TEWL)
Tidsramme: Day 0 and Week 16

Measured by a Tewameter applied on a standardized area in the anterior aspect of the forearm,
Day 0 and Week 16
Safety of dupilumab
Tidsramme: Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28

Blood tests performed every month until Week 16 (liver and renal tests, total blood count)
Day 0, week 2, week 4, week 6, week 8, week 10, week 12, week 14, week 16, week 28

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

University Hospital, Toulouse

Samarbejdspartnere

MedSharing

Efterforskere

  • Ledende efterforsker: Juliette MAZEREEUW-HAUTIER, Toulouse Hospital

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

23. juli 2020

Primær færdiggørelse (Faktiske)

1. juni 2022

Studieafslutning (Faktiske)

15. november 2022

Datoer for studieregistrering

Først indsendt

13. januar 2020

Først indsendt, der opfyldte QC-kriterier

24. januar 2020

Først opslået (Faktiske)

28. januar 2020

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

21. september 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

15. september 2026

Sidst verificeret

1. september 2026

Mere information

Begreber relateret til denne undersøgelse

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • RC31/19/0045

Plan for individuelle deltagerdata (IPD)

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