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Langsigtet sikkerhed og effekt af CSL312 (Garadacimab) i profylaktisk behandling af arvelige angioødemanfald

6. maj 2026 opdateret af: CSL Behring

En åben-label undersøgelse til evaluering af den langsigtede sikkerhed og effektivitet af CSL312 (Garadacimab) i profylaktisk behandling af arveligt angioødem

Dette fase 3b-studie vil evaluere langsigtet sikkerhed og effekt af CSL312 (også kendt som garadacimab), når det administreres subkutant (SC)

Studieoversigt

Status

Afsluttet

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

171

Fase

  • Fase 3

Kontakter og lokationer

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

Studiesteder

  • Australien
    • New South Wales
      • Campbelltown, New South Wales, Australien, 2560
        • Campbelltown Hospital / Western Sydney University
    • Victoria
      • Melbourne, Victoria, Australien, 3004
        • The Alfred Hospital
    • Western Australia
      • Murdoch, Western Australia, Australien, 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
  • Forenede Stater
    • Alabama
      • Birmingham, Alabama, Forenede Stater, 35209
        • Clinical Research Center of Alabama
    • Arizona
      • Litchfield Park, Arizona, Forenede Stater, 85340
        • Research Solutions of Arizona
      • Scottsdale, Arizona, Forenede Stater, 85251
        • Medical Research of Arizona
    • Arkansas
      • Little Rock, Arkansas, Forenede Stater, 72205
        • Little Rock Allergy & Asthma Clinic
    • California
      • Orange, California, Forenede Stater, 92868
        • Donald S. Levy M.D.
      • Santa Monica, California, Forenede Stater, 90404
        • Raffi Tachdjian MD, Inc.
      • Walnut Creek, California, Forenede Stater, 94598
        • Allergy & Asthma Clinical Research
    • Maryland
      • Chevy Chase, Maryland, Forenede Stater, 20815
        • Institute for Asthma and Allergy PC
    • Ohio
      • Cincinnati, Ohio, Forenede Stater, 45236
        • Bernstein Clinical Research Center, LLC
    • Pennsylvania
      • Hershey, Pennsylvania, Forenede Stater, 17033
        • PennState Health Milton S. Hershey Medical Center
    • Texas
      • Dallas, Texas, Forenede Stater, 75231
        • AARA Research Center
  • Holland
      • Amsterdam, Holland, 1105
        • Amsterdam UMC, location AMC
  • 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
  • New Zealand
    • Auckland
      • Grafton, Auckland, New Zealand, 1023
        • Auckland City Hospital
  • Rusland
      • Moscow, Rusland, 115522
        • NRC Institute of Immunology FMBA Russia
  • Spanien
      • Barcelona, Spanien, 8035
        • Hospital Universitari General de La Vall d'Hebron
      • Madrid, Spanien, 28007
        • Hospital Gregorio Marañón, Servicio de Alergia
  • Taiwan
      • Taichung, Taiwan, 407
        • Taichung Veterans General Hospital
  • Tjekkiet
      • Brno, Tjekkiet, 65691
        • University hospital St. Anna Ustav klinicke imunologie a alergologie, Fakultní nemocnice u sv. Anny v Brně
      • Prague, Tjekkiet, 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

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

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

Tager imod sunde frivillige

Ingen

Beskrivelse

Inklusionskriterier:

  • Hanner og kvinder i alderen ≥ 12 år
  • Diagnosticeret med klinisk bekræftet C1-INH HAE
  • Oplevet ≥ 3 HAE-anfald i løbet af de 3 måneder før screening
  • Deltog i indkøringsperioden i mindst 1 måned (kun CSL312-naive forsøgspersoner)
  • Oplevet mindst et gennemsnit på 1 HAE-angreb om måneden i løbet af indkøringsperioden

Ekskluderingskriterier:

  • Samtidig diagnosticering af en anden form for angioødem, såsom idiopatisk eller erhvervet angioødem eller tilbagevendende angioødem forbundet med urticaria
  • Brug af C1-INH-produkter, androgener, antifibrinolytika eller andre småmolekylære lægemidler til rutinemæssig profylakse mod HAE-anfald mindst 2 uger før den første dag i indkøringsperioden
  • Brug af monoklonale antistoffer såsom lanadelumab (Takhzyro®) 3 måneder før den første dag i indkøringsperioden.
  • Kvindelige forsøgspersoner bruger østrogenholdige orale præventionsmidler eller hormonsubstitutionsterapi inden for 4 uger før screening
  • Kvindelige eller mandlige forsøgspersoner, der er fertile og seksuelt aktive, der ikke bruger eller ikke er villige til at bruge en acceptabel præventionsmetode for at undgå graviditet under undersøgelsen og i 30 dage efter modtagelsen af ​​den sidste dosis CSL312
  • Gravid, ammende eller ikke villig til at stoppe med at amme

Studieplan

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

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Forebyggelse
  • Tildeling: N/A
  • Interventionel model: Enkelt gruppeopgave
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: CSL312
Fuldt humant immunoglobulin G subklasse 4/lambda rekombinant inhibitor monoklonalt antistof administreret subkutant
Biologisk: CSL312
Fuldt humant immunoglobulin G underklasse 4/lambda rekombinant inhibitor monoklonalt antistof
Andre navne:
  • garadacimab
  • Faktor XIIa-hæmmer monoklonalt antistof

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
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
Foranstaltningsbeskrivelse
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)

Samarbejdspartnere og efterforskere

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

Sponsor

CSL Behring

Efterforskere

  • Studieleder: Study Director, CSL Behring

Publikationer og nyttige links

Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.

Generelle publikationer

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)

29. marts 2021

Primær færdiggørelse (Faktiske)

21. november 2025

Studieafslutning (Faktiske)

21. november 2025

Datoer for studieregistrering

Først indsendt

1. februar 2021

Først indsendt, der opfyldte QC-kriterier

1. februar 2021

Først opslået (Faktiske)

4. februar 2021

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

2. juni 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

6. maj 2026

Sidst verificeret

1. april 2026

Mere information

Begreber relateret til denne undersøgelse

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

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

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-planbeskrivelse

CSL vil overveje anmodninger om at dele individuelle patientdata (IPD) fra systematiske gennemgangsgrupper eller bonafide forskere. Kontakt CSL på clinicaltrials@cslbehring.com for information om processen og kravene til indsendelse af en frivillig anmodning om datadeling for IPD.

Gældende landespecifikke privatliv og andre love og regler vil blive taget i betragtning og kan forhindre deling af IPD.

Hvis anmodningen godkendes, og forskeren har indgået en passende datadelingsaftale, vil IPD, der er blevet passende anonymiseret, være tilgængelig.

IPD-delingstidsramme

IPD-anmodninger kan indsendes til CSL tidligst 12 måneder efter offentliggørelsen af ​​resultaterne af denne undersøgelse via en artikel, der er tilgængelig på et offentligt websted.

IPD-delingsadgangskriterier

Anmodninger kan kun fremsættes af systematiske bedømmelsesgrupper eller bonafide forskere, hvis påtænkte brug af IPD er ikke-kommerciel og er godkendt af et internt bedømmelsesudvalg.

En IPD-anmodning vil ikke blive behandlet af CSL, medmindre det foreslåede forskningsspørgsmål søger at besvare et væsentligt og ukendt medicinsk videnskabs- eller patientplejespørgsmål som bestemt af CSL's interne revisionsudvalg.

Den anmodende part skal udføre en passende datadelingsaftale, før IPD bliver gjort tilgængelig.

IPD-deling Understøttende informationstype

  • STUDY_PROTOCOL
  • SAP

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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