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Langsiktig sikkerhet og effekt av CSL312 (Garadacimab) i profylaktisk behandling av arvelige angioødemangrep

6. mai 2026 oppdatert av: CSL Behring

En åpen studie for å evaluere den langsiktige sikkerheten og effekten av CSL312 (Garadacimab) i profylaktisk behandling av arvelig angioødem

Denne fase 3b-studien vil evaluere langsiktig sikkerhet og effekt av CSL312 (også kjent som garadacimab) når det administreres subkutant (SC)

Studieoversikt

Status

Fullført

Forhold

Intervensjon / Behandling

Studietype

Intervensjonell

Registrering (Faktiske)

171

Fase

  • Fase 3

Kontakter og plasseringer

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

Studiesteder

  • Australia
    • New South Wales
      • Campbelltown, New South Wales, Australia, 2560
        • Campbelltown Hospital / Western Sydney University
    • Victoria
      • Melbourne, Victoria, Australia, 3004
        • The Alfred Hospital
    • Western Australia
      • Murdoch, Western Australia, Australia, 6150
        • Fiona Stanley Hospital, Department of Clinical Immunology
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2B7
        • University of Alberta - Research Transition Facility
    • Ontario
      • Hamilton, Ontario, Canada, L8N 3Z5
        • McMaster University
      • Ottawa, Ontario, Canada, K1H 1E4
        • Ottawa Allergy Research Corp
      • Toronto, Ontario, Canada, M3B 3S6
        • Gordon Sussman Clinical Research
    • Quebec
      • Montreal, Quebec, Canada, H2W 1R7
        • Montreal Clinical Research Institute
  • Forente stater
    • Alabama
      • Birmingham, Alabama, Forente stater, 35209
        • Clinical Research Center of Alabama
    • Arizona
      • Litchfield Park, Arizona, Forente stater, 85340
        • Research Solutions of Arizona
      • Scottsdale, Arizona, Forente stater, 85251
        • Medical Research of Arizona
    • Arkansas
      • Little Rock, Arkansas, Forente stater, 72205
        • Little Rock Allergy & Asthma Clinic
    • California
      • Orange, California, Forente stater, 92868
        • Donald S. Levy M.D.
      • Santa Monica, California, Forente stater, 90404
        • Raffi Tachdjian MD, Inc.
      • Walnut Creek, California, Forente stater, 94598
        • Allergy & Asthma Clinical Research
    • Maryland
      • Chevy Chase, Maryland, Forente stater, 20815
        • Institute for Asthma and Allergy PC
    • Ohio
      • Cincinnati, Ohio, Forente stater, 45236
        • Bernstein Clinical Research Center, LLC
    • Pennsylvania
      • Hershey, Pennsylvania, Forente stater, 17033
        • PennState Health Milton S. Hershey Medical Center
    • Texas
      • Dallas, Texas, Forente stater, 75231
        • AARA Research Center
  • Hong Kong
      • Hong Kong, Hong Kong
        • The University of Hong Kong, Queen Mary Hospital
  • Israel
      • Ashkelon, Israel, 7830604
        • Barzilai University Medical Center
  • Japan
    • Daikakuji Yaizu-shi
      • Shizuoka, Daikakuji Yaizu-shi, Japan, 425-0088
        • Koga Community Hospital
    • Edobashi, Tsu-shi
      • Mie, Edobashi, Tsu-shi, Japan, Edobashi, Tsu-shi
        • Mie University Hospital
    • Hongo Bunkyo-ku
      • Tokyo, Hongo Bunkyo-ku, Japan, 113-8431
        • Juntendo University Hospital
    • Kamoda Kawagoe-shi
      • Saitama, Kamoda Kawagoe-shi, Japan, 350-8550
        • Saitama Medical Center
    • Kawasaki-shi
      • Kanagawa, Kawasaki-shi, Japan, 216-8511
        • St. Marianna University School of Medicine Hospital
    • Kugenumaishigami, Fujisawa-shi
      • Kanagawa, Kugenumaishigami, Fujisawa-shi, Japan, 251-0025
        • Clover Hospital
    • Matsubara Soka-shi
      • Saitama, Matsubara Soka-shi, Japan, 340-0041
        • Saiyu Soka Hospital
    • Midoricho, Tachikawa-shi
      • Tokyo, Midoricho, Tachikawa-shi, Japan, 190-0014
        • National Hospital Organization Disaster Medical Center
    • Nebeshima, Saga-shi
      • Saga, Nebeshima, Saga-shi, Japan, 849-8501
        • Saga University Hospital
    • Osaka-shi
      • Miyakojima-ku, Osaka-shi, Japan, 534-0021
        • Local Incorporated Administrative Agency Osaka City Hospital Organization Osaka City General Hospital
  • Nederland
      • Amsterdam, Nederland, 1105
        • Amsterdam UMC, location AMC
  • New Zealand
    • Auckland
      • Grafton, Auckland, New Zealand, 1023
        • Auckland City Hospital
  • Russland
      • Moscow, Russland, 115522
        • NRC Institute of Immunology FMBA Russia
  • Spania
      • Barcelona, Spania, 8035
        • Hospital Universitari General de La Vall d'Hebron
      • Madrid, Spania, 28007
        • Hospital Gregorio Marañón, Servicio de Alergia
  • Taiwan
      • Taichung, Taiwan, 407
        • Taichung Veterans General Hospital
  • Tsjekkia
      • Brno, Tsjekkia, 65691
        • University hospital St. Anna Ustav klinicke imunologie a alergologie, Fakultní nemocnice u sv. Anny v Brně
      • Prague, Tsjekkia, 150 06
        • University Hospital Motol
  • Tyskland
      • Berlin, Tyskland, 10117
        • Charité - Universitätsmedizin Berlin
      • Frankfurt, Tyskland, 60590
        • Universitätsklinikum Frankfurt
      • Mainz, Tyskland, 55131
        • Johannes Gutenberg-Universität KöR, Hautklinik und Poliklinik der Universitätsmedizin, Clinical Research Center
      • Mörfelden-Walldorf, Tyskland, 64546
        • HZRM Haemophilie Zentrum Rhein Main GmbH
  • Ungarn
      • Budapest, Ungarn, 1088
        • Semmelweis Egyetem Altalanos Orvostudományi Kar Belgyógyászati és Hematológiai Klinika

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

12 år og eldre (Barn, Voksen, Eldre voksen)

Tar imot friske frivillige

Nei

Beskrivelse

Inklusjonskriterier:

  • Hanner og kvinner i alderen ≥ 12 år
  • Diagnostisert med klinisk bekreftet C1-INH HAE
  • Opplevde ≥ 3 HAE-anfall i løpet av 3 måneder før screening
  • Deltok i innkjøringsperioden i minst 1 måned (kun CSL312-naive fag)
  • Opplevde minst et gjennomsnitt på 1 HAE-angrep per måned i løpet av innkjøringsperioden

Ekskluderingskriterier:

  • Samtidig diagnose av en annen form for angioødem, slik som idiopatisk eller ervervet angioødem eller tilbakevendende angioødem assosiert med urticaria
  • Bruk av C1-INH-produkter, androgener, antifibrinolytika eller andre småmolekylære medisiner for rutineprofylakse mot HAE-anfall minst 2 uker før den første dagen i innkjøringsperioden
  • Bruk av monoklonale antistoffer som lanadelumab (Takhzyro®) 3 måneder før den første dagen i innkjøringsperioden.
  • Kvinnelige forsøkspersoner bruker østrogenholdige p-piller eller hormonerstatningsterapi innen 4 uker før screening
  • Kvinnelige eller mannlige forsøkspersoner som er fertile og seksuelt aktive som ikke bruker eller ikke er villige til å bruke en akseptabel prevensjonsmetode for å unngå graviditet under studien og i 30 dager etter mottak av siste dose av CSL312
  • Gravid, ammer eller ikke villig til å slutte å amme

Studieplan

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

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Forebygging
  • Tildeling: N/A
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: CSL312
Fullt humant immunglobulin G subklasse 4/lambda rekombinant hemmer monoklonalt antistoff administrert subkutant
Biologisk: CSL312
Fullt humant immunglobulin G underklasse 4/lambda rekombinant hemmer monoklonalt antistoff
Andre navn:
  • garadacimab
  • Faktor XIIa-hemmer monoklonalt antistoff

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Number of Participants With Treatment-emergent Adverse Events (TEAE)
Tidsramme: Approximately up to 54 months
Approximately up to 54 months
Percentage of Participants With TEAE
Tidsramme: Approximately up to 54 months
The percentage of participants was rounded to one place of decimal.
Approximately up to 54 months
Number of TEAE
Tidsramme: Approximately up to 54 months
Approximately up to 54 months
TEAE Rates Per Injection
Tidsramme: Approximately up to 54 months
The TEAE rate per injection was calculated as the number of TEAE/ total number of injections. The number of injections was defined as the total injections received by participants during the respective safety evaluation period.
Approximately up to 54 months
TEAE Rates Per Participant Year
Tidsramme: Approximately up to 54 months
The TEAE rate per participant year was calculated as the total number of TEAE/ participant years. Participant years was defined as the sum of the time (in years) that participant were exposed to study treatment during the respective safety evaluation period.
Approximately up to 54 months

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
The Time-normalized Number (Per Month) of Hereditary Angioedema (HAE) Attacks During the Run-in Period and Treatment Period
Tidsramme: Run-in Period: Up to Day 60 and Treatment Period: Approximately up to 52 months
Time-normalized number of HAE attacks per month during treatment was calculated per participant as: [number of HAE attacks / length of participant treatment in days]*30.4375.
Run-in Period: Up to Day 60 and Treatment Period: Approximately up to 52 months
The Time-normalized Number (Per Year) of HAE Attacks During Treatment Period
Tidsramme: Approximately up to 52 months
Time-normalized number of HAE attacks per year during treatment was calculated per participant as: [number of HAE attacks / length of participant treatment in days]*365.25.
Approximately up to 52 months
Percentage Reduction in the Attack Rate During the Treatment Period Compared to the Run-in Period
Tidsramme: Run-in Period: Up to 60 days and Treatment Period: Approximately up to 52 months
The percentage reduction in the time-normalized number of HAE attacks was calculated within a participant as: 100*(1- time normalized number of HAE attacks per month during Treatment Period/time-normalized number of HAE attacks per month during Run-in Period).
Run-in Period: Up to 60 days and Treatment Period: Approximately up to 52 months
Number of Participants With Percentage Reduction (of >=50%, >=70%, >=90%, and 100%) in HAE Attacks
Tidsramme: Run-in Period: Up to Day 60 and Treatment Period: Approximately up to 52 months
The number of participants who achieved a percentage reduction in HAE attacks of >=50% (also considered as responders), >=70%, >=90%, and 100% (attack free) during the Treatment Period compared with the Run-in Period. The percentage reduction in the time-normalized number of HAE attacks per month was calculated as 100*[1 - (time-normalized number of HAE attacks per month under CSL312 treatment / time-normalized number of HAE attacks per month during Run-in Period)].
Run-in Period: Up to Day 60 and Treatment Period: Approximately up to 52 months
The Time-normalized Number (Per Month) of HAE Attacks Requiring On-demand Treatment
Tidsramme: Approximately up to 52 months
Time-normalized number of HAE attacks per month requiring on demand treatment was calculated per participant as: [number of HAE attacks requiring on demand treatment during treatment period / length of participant treatment in days]*30.4375.
Approximately up to 52 months
The Time-normalized Number (Per Year) of HAE Attacks Requiring On-demand Treatment
Tidsramme: Approximately up to 52 months
Time-normalized number of HAE attacks requiring on demand treatment per year was calculated per participant as: [number of HAE attacks requiring on demand treatment during treatment period / length of participant treatment in days]*365.25.
Approximately up to 52 months
The Time-normalized Number (Per Month) of Moderate and/or Severe HAE Attacks
Tidsramme: Approximately up to 52 months
Time-normalized number of moderate or severe HAE attacks per month during treatment period was calculated per participant as: [number of moderate or severe HAE attacks / length of participant treatment in days]*30.4375.
Approximately up to 52 months
The Time-normalized Number (Per Year) of Moderate and/or Severe HAE Attacks
Tidsramme: Approximately up to 52 months
Time-normalized number of moderate or severe HAE attacks per year during treatment period was calculated per participant as: [number of moderate or severe HAE attacks /length of participant treatment in days]*365.25.
Approximately up to 52 months
Number of Participants Rating Their Response to Therapy as Good or Excellent
Tidsramme: At Months 12, 24, and 36
Number of participants rating their response to therapy as good or excellent was evaluated as per Subject's Global Assessment of Response to Therapy (SGART) questionnaire. SGART is a patient-reported outcome that represents the participant's overall response to treatment using the following ratings: (0) none: worse or no response at all, not acceptable, (1) poor: very little response, not acceptable, (2) fair: some response, acceptable but could be better, (3) good: good response, acceptable, and (4) excellent: excellent response, as good as can be imagined. Cumulative responses as "Good or Excellent" are reported for this outcome measure.
At Months 12, 24, and 36
Percentage of Participants Rating Their Response to Therapy as Good or Excellent
Tidsramme: At Months 12, 24, and 36
Percentage of participants rating their response to therapy as good or excellent was evaluated as per SGART questionnaire. SGART is a patient-reported outcome that represents the participant's overall response to treatment using the following ratings: (0) none: worse or no response at all, not acceptable, (1) poor: very little response, not acceptable, (2) fair: some response, acceptable but could be better, (3) good: good response, acceptable, and (4) excellent: excellent response, as good as can be imagined. Cumulative responses as "Good or Excellent" are reported for this outcome measure. The percentage of participants was rounded to one place of decimal.
At Months 12, 24, and 36
Number of Participants Experiencing Serious Adverse Events (SAE), Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation
Tidsramme: Approximately up to 54 months
Approximately up to 54 months
Percentage of Participants Experiencing SAE, Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation
Tidsramme: Approximately up to 54 months
The percentage of participants was rounded to one decimal place.
Approximately up to 54 months
Number of Participants Experiencing TEAE by Severity
Tidsramme: Approximately up to 54 months
Severity of AE was assessed by the investigator and categorized as mild, moderate and severe. A mild AE that is usually transient and may require only minimal treatment or therapeutic intervention. The event does not generally interfere with usual activities of daily living. A moderate AE that is usually alleviated with additional specific therapeutic intervention. The event interferes with usual activities of daily living, causing discomfort but poses no significant or permanent risk of harm to the research participant. A severe AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention.
Approximately up to 54 months
Percentage of Participants Experiencing TEAE by Severity
Tidsramme: Approximately up to 54 months
Severity of AE was assessed by the investigator and categorized as mild, moderate and severe. A mild AE that is usually transient and may require only minimal treatment or therapeutic intervention. The event does not generally interfere with usual activities of daily living. A moderate AE that is usually alleviated with additional specific therapeutic intervention. The event interferes with usual activities of daily living, causing discomfort but poses no significant or permanent risk of harm to the research participant. A severe AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention. The percentage of participants was rounded to one decimal place.
Approximately up to 54 months
Number of Participants Experiencing Adverse Events of Special Interest (AESI)
Tidsramme: Approximately up to 54 months
The AESI defined for this study were thromboembolic events, abnormal bleeding events, and severe hypersensitivity including anaphylaxis. The AESI reported have been identified by investigators.
Approximately up to 54 months
Percentage of Participants Experiencing AESI
Tidsramme: Approximately up to 54 months
The AESI defined for this study were thromboembolic events, abnormal bleeding events, and severe hypersensitivity including anaphylaxis. The AESI reported have been identified by investigators.
Approximately up to 54 months
Number of Participants With Laboratory Findings Reported as TEAE
Tidsramme: Approximately up to 54 months
Approximately up to 54 months
Percentage of Participants With Laboratory Findings Reported as TEAE
Tidsramme: Approximately up to 54 months
The percentage of participant was rounded to one place of decimal.
Approximately up to 54 months
Number of Participants With Normal C1-esterase Inhibitor (nC1-INH) Experiencing TEAE
Tidsramme: Approximately up to 54 months
Approximately up to 54 months
Percentage of Participants With nC1-INH Experiencing TEAE
Tidsramme: Approximately up to 54 months
Approximately up to 54 months
Number of Participants With Anti-CSL312 Antibodies
Tidsramme: At Day 1, Months 6, 12, 36 and 43 (end of treatment [EOT])
At Day 1, Months 6, 12, 36 and 43 (end of treatment [EOT])
Percentage of Participants With Anti-CSL312 Antibodies
Tidsramme: At Day 1, Months 6, 12, 36 and 43 (EOT)
The percentage of participants was rounded to one decimal place.
At Day 1, Months 6, 12, 36 and 43 (EOT)

Samarbeidspartnere og etterforskere

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

Sponsor

CSL Behring

Etterforskere

  • Studieleder: Study Director, CSL Behring

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Generelle publikasjoner

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)

29. mars 2021

Primær fullføring (Faktiske)

21. november 2025

Studiet fullført (Faktiske)

21. november 2025

Datoer for studieregistrering

Først innsendt

1. februar 2021

Først innsendt som oppfylte QC-kriteriene

1. februar 2021

Først lagt ut (Faktiske)

4. februar 2021

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

2. juni 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

6. mai 2026

Sist bekreftet

1. april 2026

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • CSL312_3002
  • 2020-003918-12 (EudraCT-nummer)

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

JA

IPD-planbeskrivelse

CSL vil vurdere forespørsler om å dele individuelle pasientdata (IPD) fra systematiske gjennomgangsgrupper eller godtroende forskere. For informasjon om prosessen og kravene for å sende inn en frivillig forespørsel om datadeling for IPD, vennligst kontakt CSL på clinicaltrials@cslbehring.com.

Gjeldende landsspesifikke personvern og andre lover og forskrifter vil bli vurdert og kan forhindre deling av IPD.

Hvis forespørselen godkjennes og forskeren har utført en passende datadelingsavtale, vil IPD som er riktig anonymisert være tilgjengelig.

IPD-delingstidsramme

IPD-forespørsler kan sendes til CSL tidligst 12 måneder etter publisering av resultatene av denne studien via en artikkel som er gjort tilgjengelig på et offentlig nettsted.

Tilgangskriterier for IPD-deling

Forespørsler kan kun fremsettes av systematiske vurderingsgrupper eller godtroende forskere hvis foreslåtte bruk av IPD er ikke-kommersiell og har blitt godkjent av en intern vurderingskomité.

En IPD-forespørsel vil ikke bli vurdert av CSL med mindre det foreslåtte forskningsspørsmålet søker å besvare et betydelig og ukjent medisinsk vitenskapelig eller pasientbehandlingsspørsmål som bestemt av CSLs interne vurderingskomité.

Anmodende part må utføre en passende datadelingsavtale før IPD vil bli gjort tilgjengelig.

IPD-deling Støtteinformasjonstype

  • STUDY_PROTOCOL
  • SEVJE

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

Kliniske studier på Arvelig angioødem

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