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En pilotstudie av effektiviteten og sikkerheten til dupilumab versus placebo hos pasienter med Netherton syndrom (NS-DUPI)

15. september 2026 oppdatert av: University Hospital, Toulouse

En randomisert dobbeltblindet pilotstudie av effektiviteten og sikkerheten til dupilumab versus placebo hos pasienter med Netherton 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.

Studieoversikt

Status

Avsluttet

Forhold

Intervensjon / Behandling

Detaljert 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.

Studietype

Intervensjonell

Registrering (Faktiske)

10

Fase

  • Fase 2
  • Fase 3

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

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

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

18 år og eldre (Voksen, Eldre voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

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

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Trippel

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: 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).
Legemiddel: 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)..
Annen: Placebo Prefilled Syringe

administration of placebo corresponding to placebo arm

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
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
Tiltaksbeskrivelse
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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

University Hospital, Toulouse

Samarbeidspartnere

MedSharing

Etterforskere

  • Hovedetterforsker: Juliette MAZEREEUW-HAUTIER, Toulouse Hospital

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

23. juli 2020

Primær fullføring (Faktiske)

1. juni 2022

Studiet fullført (Faktiske)

15. november 2022

Datoer for studieregistrering

Først innsendt

13. januar 2020

Først innsendt som oppfylte QC-kriteriene

24. januar 2020

Først lagt ut (Faktiske)

28. januar 2020

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

21. september 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

15. september 2026

Sist bekreftet

1. september 2026

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • RC31/19/0045

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

NEI

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Nei

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

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