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Fase 2/3 klinisk undersøgelse af QRX003 lotion hos patienter med Netherton syndrom

24. september 2026 opdateret af: Quoin Pharmaceuticals

Et multicenter, åbent studie med QRX003-lotion til deltagere med Netherton-syndrom

This is a multicenter, open-label study to evaluate the safety, tolerability, and efficacy of 4% QRX003 lotion applied twice daily (BID) for 12 weeks to Netherton syndrome (NS) diseased skin in all affected areas of the body excluding the scalp (the Treatment Area), totaling approximately 50% Body Surface Area (BSA) or more.

Studieoversigt

Status

Rekruttering

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

16

Fase

  • Fase 2
  • Fase 3

Kontakter og lokationer

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

Studiekontakt

Undersøgelse Kontakt Backup

Studiesteder

  • Det Forenede Kongerige
      • London, Det Forenede Kongerige, SE1 9RT
        • Rekruttering
        • Guy's Hospital, Skin Therapy Research Unit
        • Kontakt:
    • England
      • London, England, Det Forenede Kongerige, WC1N 3JH
        • Rekruttering
        • Great Ormond Street Hospital, NIHR - Clinical Research
        • Kontakt:
    • West Midlands
      • Walsall, West Midlands, Det Forenede Kongerige, WS2 9PS
        • Rekruttering
        • Walsall Manor Hospital, Research & Development
        • Kontakt:
  • Forenede Stater
    • California
      • Fremont, California, Forenede Stater, 94538
        • Rekruttering
        • Center for Dermatology Clinical Research
        • Kontakt:
    • Connecticut
      • New Haven, Connecticut, Forenede Stater, 06520
        • Ikke rekrutterer endnu
        • Yale University School of Medicine
        • Kontakt:
    • Indiana
      • Indianapolis, Indiana, Forenede Stater, 46250
        • Rekruttering
        • Dawes Fretzin Clinical Reseacrh Group
        • Kontakt:
    • Massachusetts
      • Quincy, Massachusetts, Forenede Stater, 02169
    • Texas
      • San Antonio, Texas, Forenede Stater, 78218
        • Rekruttering
        • Texas Dermatology and Laser Specialists
        • Kontakt:

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

4 år og ældre (Barn, Voksen, Ældre voksen)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

Inclusion Criteria:

  1. Participant is male or female and at least 4 years of age.
  2. Participant has provided written informed consent/assent. Based on the participant's age and local regulations , a participant under legal age of consent must provide written informed assent and be accompanied by the parent or legal guardian at the time of assent/consent signing. The parent or legal guardian must provide informed consent for the participant. If a participant reaches the age at which consent is required by local regulations during the study, the participant must provide written informed consent at that time to continue study participation.
  3. Female participants must either not have experienced menarche, be postmenopausal , or be surgically sterile , or use a highly effective method of birth control , for the duration of the study and for 3 months following EOT. Women of childbearing potential (WOCBP) must have a negative urine pregnancy test (UPT) at Visit 1/Screening and Visit 2/Baseline. Males must use a condom in addition to having their female partner use a highly effective method of birth control.
  4. Participant has a genetically confirmed diagnosis of NS prior to Visit 2/Baseline. Participants without existing results must undergo genetic testing at Visit 1/Screening, which must confirm the diagnosis of NS in order for the participants to be enrolled in the study.

Exclusion Criteria:

  1. Participant is pregnant, lactating, or is planning to become pregnant during the study.
  2. Participant has active cancer of any type excluding melanoma skin cancer outside of the Treatment Area.
  3. Participant has diabetes of any type, except non-insulin dependent diabetes mellitus that is reasonably controlled, in the opinion of the investigator.
  4. Participant has evidence of active infection during Screening, or serious infection within 30 days prior to Visit 2/Baseline.
  5. Participant has known human immunodeficiency virus, hepatitis B or C virus, or active or latent tuberculosis.
  6. Participant has ongoing or recent history of any other uncontrolled and/or clinically significant systemic disease or condition which, in the opinion of the investigator, should exclude participation in the study.
  7. Participant has used ultraviolet phototherapy within the Treatment Area within 4 weeks prior to Visit 2/Baseline.
  8. Participant has used systemic non-biologics prescription treatment for NS or that in the opinion of the investigator may affect the participant's NS (examples include, but are not limited to antibiotics, retinoids, etc.) within 4 weeks prior to Visit 2/Baseline.
  9. Participant has used systemic biologic therapy for NS or that in the opinion of the investigator may affect the participant's NS within 5 half-lives of the biologic therapy prior to Visit 2/Baseline. Published or documented half-life of the product provided by the commercial supplier or Sponsor should be used to establish this value.
  10. Participant has used topical prescription treatments, including steroids (examples include, but are not limited to keratolytics, steroids, retinoids, etc.) in the Treatment Area within 2 weeks prior to Visit 2/Baseline.
  11. Participant has used any topical bland moisturizers/emollients in the Treatment Area within 24 hours prior to Visit 2/Baseline.
  12. Participant is currently enrolled in an investigational drug, biologic, or device study.

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: N/A
  • Interventionel model: Enkelt gruppeopgave
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: QRX003 lotion, 4% BID

Subjects will apply test article twice daily (BID) for 12 weeks
Medicin: QRX003

Serine Protease Inhibitor

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Proportion of subjects with 1-point reduction on IGA
Tidsramme: Week 12

The IGA (Investigator Global Assessment) is a 5-point scale used to assess the overall severity of a Subject's NS condition.
Week 12

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Proportion of subjects with 2-point improvement on the WI-NRS
Tidsramme: Week 12

Subjects will rate their itch intensity using the 11-point Worst-Itch-Numeric Rating Scale (WI-NRS)
Week 12
PGI-C Score
Tidsramme: Week 12

Patient (or Caregiver) Global Impression of Change (PGI-C) is a 7-point, self-assessed scale measuring the patient's (or caregiver's) improvement of their NS condition.
Week 12
CGI-C Score
Tidsramme: Week 12

Clinical Global Impression of Change (PGI-C) is a 7-point scale used by the clinician to assess improvement of the NS condition.
Week 12

Samarbejdspartnere og efterforskere

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

Sponsor

Quoin Pharmaceuticals

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)

19. marts 2026

Primær færdiggørelse (Anslået)

31. januar 2027

Studieafslutning (Anslået)

31. marts 2027

Datoer for studieregistrering

Først indsendt

24. marts 2026

Først indsendt, der opfyldte QC-kriterier

15. april 2026

Først opslået (Faktiske)

20. april 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

28. september 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

24. september 2026

Sidst verificeret

1. april 2026

Mere information

Begreber relateret til denne undersøgelse

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • CL-QRX003-004

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

UBESLUTET

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

produkt fremstillet i og eksporteret fra U.S.A.

Ja

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

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