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A Study to Investigate Safety and Efficacy of KB111 for the Treatment of Hailey-Hailey Disease (HALITE-1)

16. juli 2026 opdateret af: Krystal Biotech, Inc.

An Open-Label Study to Investigate Safety and Preliminary Efficacy of KB111 for the Treatment of Hailey-Hailey Disease

An open-label study to evaluate the safety and efficacy of KB111 in pediatric and adult subjects with Hailey-Hailey Disease

Studieoversigt

Status

Rekruttering

Betingelser

Intervention / Behandling

Detaljeret beskrivelse

This clinical study will investigate the safety, tolerability, and preliminary efficacy of open-label KB111 in pediatric and adult subjects with Hailey-Hailey Disease (HHD). Up to 7 subjects will be administered topical KB111 to treat HHD lesions for 12 weeks. KB111 will be administered in the home setting by a trained designee. The Investigator (or designee) will contact subjects weekly (or their parent/legal guardian) to assess for any adverse events or changes in medications, treatments, or procedures. Preliminary efficacy will be assessed by investigator assessments, patient-reported impressions of severity and molecular analysis of skin biopsies. A safety follow-up visit will occur 4 weeks after treatment stops.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

7

Fase

  • Tidlig fase 1

Kontakter og lokationer

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

Studiekontakt

Studiesteder

  • Forenede Stater
    • Florida
      • Miami, Florida, Forenede Stater, 33156
        • Rekruttering
        • Pediatric Skin Research
        • Kontakt:
          • Jean Marco Varanese
          • Telefonnummer: +1 786-585-5774

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

  • Barn
  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

Inclusion Criteria:

  • The participant must have read, understood, and signed an Institutional Review Board (IRB)- approved Informed Consent Form and must be willing and able to comply with study procedures and instructions
  • Aged ≥ 12 years of age at the time of informed consent/assent
  • Documented clinical diagnosis of HHD with confirmed ATP2C1 variant by genetic testing
  • A negative pregnancy test prior to initial dose for subjects of child-bearing potential
  • If taking systemic therapies, subject must be on a stable dosing regimen 1 month prior to Screening and throughout the 12-week treatment period
  • An acceptable form of contraception must be taken from the first dose (Week 1) through 3 months after the last dose of IP for subjects of childbearing potential

Exclusion Criteria:

  • Participation in another clinical study or treatment with an investigational agent 30 days or 5 half-lives, whichever is longer, prior to initial dosing in Week 1
  • Participant who is pregnant or nursing
  • Participant who is unwilling to comply with contraception requirements per protocol (for subjects of child-bearing potential)
  • Participant who is known to be non-compliant or is unlikely to comply with the requirements of the study protocol, in the opinion of the Investigator
  • Presence of any dermatologic or systemic condition that might hinder the efficacy of the study treatment or its evaluation, could possibly interfere with the interpretation of study results, or could be judged by the Investigator to be incompatible with the study visit schedule or conduct

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: KB111
Biologisk: KB111
KB111 is a replication-defective, non-integrating herpes simplex virus (HSV-1)-based vector encoding functional, full-length human ATP2C1 protein combined with a gel for topical application.

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
To evaluate the safety and tolerability of KB111
Tidsramme: 12 weeks
Number of subjects with treatment related adverse events as assessed by CTCAE v5
12 weeks

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
To evaluate the effect of KB111 on patient-reported lesion severity throughout the treatment period.
Tidsramme: 12 weeks
Change from baseline using a patient reported 5-point multi-item scale where signs and symptoms are rated from none (0) to extreme (4)
12 weeks
To evaluate the effect of KB111 on patient-reported global impression of severity throughout the treatment period
Tidsramme: 12 weeks
Change from baseline using a patient reported 5-point multi-item scale where signs and symptoms are rated from none (0) to extreme (4)
12 weeks
To evaluate the effect of KB111 on Investigator-based assessments of lesion severity
Tidsramme: 12 weeks
Change from baseline in the Investigator's assessment using a 5-point scale where 0 is clear and 4 is severe
12 weeks
To evaluate the effect of KB111 on Investigator-based assessment of global severity
Tidsramme: 12 weeks
Change from baseline in the Investigator's assessment using a 5-point scale where 0 is clear and 4 is severe
12 weeks

Samarbejdspartnere og efterforskere

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

Sponsor

Krystal Biotech, Inc.

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)

6. maj 2026

Primær færdiggørelse (Anslået)

1. november 2026

Studieafslutning (Anslået)

1. december 2026

Datoer for studieregistrering

Først indsendt

8. juli 2026

Først indsendt, der opfyldte QC-kriterier

16. juli 2026

Først opslået (Faktiske)

21. juli 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

21. juli 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

16. juli 2026

Sidst verificeret

1. juli 2026

Mere information

Begreber relateret til denne undersøgelse

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • KB111-A

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

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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