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Långsiktig säkerhet och effekt av CSL312 (Garadacimab) vid profylaktisk behandling av ärftliga angioödemsattacker

6 maj 2026 uppdaterad av: CSL Behring

En öppen studie för att utvärdera den långsiktiga säkerheten och effekten av CSL312 (Garadacimab) vid profylaktisk behandling av ärftligt angioödem

Denna fas 3b-studie kommer att utvärdera långsiktig säkerhet och effekt av CSL312 (även känd som garadacimab) när det administreras subkutant (SC)

Studieöversikt

Status

Avslutad

Betingelser

Intervention / Behandling

Studietyp

Interventionell

Inskrivning (Faktisk)

171

Fas

  • Fas 3

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studieorter

  • 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
  • Förenta staterna
    • Alabama
      • Birmingham, Alabama, Förenta staterna, 35209
        • Clinical Research Center of Alabama
    • Arizona
      • Litchfield Park, Arizona, Förenta staterna, 85340
        • Research Solutions of Arizona
      • Scottsdale, Arizona, Förenta staterna, 85251
        • Medical Research of Arizona
    • Arkansas
      • Little Rock, Arkansas, Förenta staterna, 72205
        • Little Rock Allergy & Asthma Clinic
    • California
      • Orange, California, Förenta staterna, 92868
        • Donald S. Levy M.D.
      • Santa Monica, California, Förenta staterna, 90404
        • Raffi Tachdjian MD, Inc.
      • Walnut Creek, California, Förenta staterna, 94598
        • Allergy & Asthma Clinical Research
    • Maryland
      • Chevy Chase, Maryland, Förenta staterna, 20815
        • Institute for Asthma and Allergy PC
    • Ohio
      • Cincinnati, Ohio, Förenta staterna, 45236
        • Bernstein Clinical Research Center, LLC
    • Pennsylvania
      • Hershey, Pennsylvania, Förenta staterna, 17033
        • PennState Health Milton S. Hershey Medical Center
    • Texas
      • Dallas, Texas, Förenta staterna, 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
  • Kanada
    • Alberta
      • Edmonton, Alberta, Kanada, T6G 2B7
        • University of Alberta - Research Transition Facility
    • Ontario
      • Hamilton, Ontario, Kanada, L8N 3Z5
        • McMaster University
      • Ottawa, Ontario, Kanada, K1H 1E4
        • Ottawa Allergy Research Corp
      • Toronto, Ontario, Kanada, M3B 3S6
        • Gordon Sussman Clinical Research
    • Quebec
      • Montreal, Quebec, Kanada, H2W 1R7
        • Montreal Clinical Research Institute
  • Nederländerna
      • Amsterdam, Nederländerna, 1105
        • Amsterdam UMC, location AMC
  • Nya Zeeland
    • Auckland
      • Grafton, Auckland, Nya Zeeland, 1023
        • Auckland City Hospital
  • Ryssland
      • Moscow, Ryssland, 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
  • Tjeckien
      • Brno, Tjeckien, 65691
        • University hospital St. Anna Ustav klinicke imunologie a alergologie, Fakultní nemocnice u sv. Anny v Brně
      • Prague, Tjeckien, 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
  • Ungern
      • Budapest, Ungern, 1088
        • Semmelweis Egyetem Altalanos Orvostudományi Kar Belgyógyászati és Hematológiai Klinika

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

12 år och äldre (Barn, Vuxen, Äldre vuxen)

Tar emot friska volontärer

Nej

Beskrivning

Inklusionskriterier:

  • Hanar och honor i åldern ≥ 12 år
  • Diagnostiserats med kliniskt bekräftad C1-INH HAE
  • Upplevde ≥ 3 HAE-attacker under de 3 månaderna före screening
  • Deltagit i inkörningsperioden i minst 1 månad (endast CSL312-naiva ämnen)
  • Upplevde minst ett genomsnitt av 1 HAE-attack per månad under inkörningsperioden

Exklusions kriterier:

  • Samtidig diagnos av annan form av angioödem, såsom idiopatiskt eller förvärvat angioödem eller återkommande angioödem i samband med urtikaria
  • Användning av C1-INH-produkter, androgener, antifibrinolytika eller andra småmolekylära läkemedel för rutinprofylax mot HAE-attacker minst 2 veckor före den första dagen av inkörningsperioden
  • Användning av monoklonala antikroppar såsom lanadelumab (Takhzyro®) 3 månader före den första dagen av inkörningsperioden.
  • Kvinnliga försökspersoner använder östrogeninnehållande p-piller eller hormonersättningsterapi inom 4 veckor före screening
  • Kvinnliga eller manliga försökspersoner som är fertila och sexuellt aktiva som inte använder eller inte är villiga att använda en acceptabel preventivmetod för att undvika graviditet under studien och i 30 dagar efter mottagandet av den sista dosen av CSL312
  • Gravid, ammar eller inte villig att sluta amma

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Förebyggande
  • Tilldelning: N/A
  • Interventionsmodell: Enskild gruppuppgift
  • Maskning: Ingen (Open Label)

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: CSL312
Helt humant immunglobulin G subklass 4/lambda rekombinant hämmare monoklonal antikropp administrerad subkutant
Biologisk: CSL312
Helt humant immunglobulin G subklass 4/lambda rekombinant hämmare monoklonal antikropp
Andra namn:
  • garadacimab
  • Faktor XIIa-hämmare monoklonal antikropp

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Number of Participants With Treatment-emergent Adverse Events (TEAE)
Tidsram: Approximately up to 54 months
Approximately up to 54 months
Percentage of Participants With TEAE
Tidsram: Approximately up to 54 months
The percentage of participants was rounded to one place of decimal.
Approximately up to 54 months
Number of TEAE
Tidsram: Approximately up to 54 months
Approximately up to 54 months
TEAE Rates Per Injection
Tidsram: 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
Tidsram: 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

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
The Time-normalized Number (Per Month) of Hereditary Angioedema (HAE) Attacks During the Run-in Period and Treatment Period
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: Approximately up to 54 months
Approximately up to 54 months
Percentage of Participants Experiencing SAE, Experiencing Death, Related TEAE, TEAE Leading to Study Discontinuation
Tidsram: 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
Tidsram: 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
Tidsram: 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)
Tidsram: 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
Tidsram: 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
Tidsram: Approximately up to 54 months
Approximately up to 54 months
Percentage of Participants With Laboratory Findings Reported as TEAE
Tidsram: 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
Tidsram: Approximately up to 54 months
Approximately up to 54 months
Percentage of Participants With nC1-INH Experiencing TEAE
Tidsram: Approximately up to 54 months
Approximately up to 54 months
Number of Participants With Anti-CSL312 Antibodies
Tidsram: 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
Tidsram: 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)

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

CSL Behring

Utredare

  • Studierektor: Study Director, CSL Behring

Publikationer och användbara länkar

Den som ansvarar för att lägga in information om studien tillhandahåller frivilligt dessa publikationer. Dessa kan handla om allt som har med studien att göra.

Allmänna publikationer

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

29 mars 2021

Primärt slutförande (Faktisk)

21 november 2025

Avslutad studie (Faktisk)

21 november 2025

Studieregistreringsdatum

Först inskickad

1 februari 2021

Först inskickad som uppfyllde QC-kriterierna

1 februari 2021

Första postat (Faktisk)

4 februari 2021

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

2 juni 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

6 maj 2026

Senast verifierad

1 april 2026

Mer information

Termer relaterade till denna studie

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

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

Plan för individuella deltagardata (IPD)

Planerar du att dela individuella deltagardata (IPD)?

JA

IPD-planbeskrivning

CSL kommer att överväga förfrågningar om att dela individuella patientdata (IPD) från systematiska granskningsgrupper eller bonafide forskare. För information om processen och kraven för att skicka in en frivillig begäran om datadelning för IPD, vänligen kontakta CSL på clinicaltrials@cslbehring.com.

Tillämplig landsspecifik sekretess och andra lagar och förordningar kommer att övervägas och kan förhindra delning av IPD.

Om begäran godkänns och forskaren har genomfört ett lämpligt datadelningsavtal, kommer IPD som har anonymiserats på lämpligt sätt att vara tillgänglig.

Tidsram för IPD-delning

IPD-förfrågningar kan skickas till CSL tidigast 12 månader efter publicering av resultaten av denna studie via en artikel som görs tillgänglig på en offentlig webbplats.

Kriterier för IPD Sharing Access

Begäran får endast göras av systematiska granskningsgrupper eller bonafide forskare vars föreslagna användning av IPD är icke-kommersiell till sin natur och har godkänts av en intern granskningskommitté.

En IPD-begäran kommer inte att behandlas av CSL om inte den föreslagna forskningsfrågan syftar till att besvara en viktig och okänd medicinsk vetenskaps- eller patientvårdsfråga som fastställts av CSL:s interna granskningskommitté.

Den begärande parten måste utföra ett lämpligt datadelningsavtal innan IPD kommer att göras tillgänglig.

IPD-delning som stöder informationstyp

  • STUDY_PROTOCOL
  • SAV

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Ja

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

Denna information hämtades direkt från webbplatsen clinicaltrials.gov utan några ändringar. Om du har några önskemål om att ändra, ta bort eller uppdatera dina studieuppgifter, vänligen kontakta register@clinicaltrials.gov. Så snart en ändring har implementerats på clinicaltrials.gov, kommer denna att uppdateras automatiskt även på vår webbplats .

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