Eine Multizentrische, Offene Studie zur Beurteilung der Sicherheit, Wirksamkeit, Pharmakokinetik und Pharmakodynamik von Emicizumab bei patienten mit leichter oder mittelschwerer Hämophilie A ohne FVIII-Hemmkörper

A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors

  • Hämophilie
  • Hämophilie A
Bitte beachten Sie, dass der Rekrutierungsstatus der Studie an Ihrem Standort vom Gesamtstatus der Studie abweichen kann, da einige Studienzentren früher rekrutieren als andere.
Status der Studie:

Aktiv, keine Rekrutierung

Diese Studie läuft in
Städte
  • Bonn
  • München
Studien-ID:

NCT04158648 2023-506610-52-00 BO41423

      Studienzentren finden

      Die folgenden Informationen stammen aus der öffentlich zugänglichen Website ClinicalTrials.gov und werden für Laien bearbeitet.

      Die folgenden Informationen stammen aus der öffentlich zugänglichen Website ClinicalTrials.gov und werden nicht geändert.

      Results Disclaimer

      Studienzusammenfassung

      This is a multicenter, open-label, single-arm study designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of emicizumab in participants with mild or moderate hemophilia A without inhibitors against factor VIII (FVIII).

      Hoffmann-La Roche Sponsor
      Phase 3 Phase
      NCT04158648, BO41423, 2023-506610-52-00 Studien-ID
      Emicizumab Treatments
      Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Hemophilia A Condition
      Official Title

      A Multicenter, Open-Label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Emicizumab in Patients With Mild or Moderate Hemophilia A Without FVIII Inhibitors

      Einschlusskriterien

      All Gender
      No Healthy Volunteers
      Inclusion Criteria
      • Diagnosis of mild (FVIII level between >5% and <40%) or moderate (FVIII level between ≥1% and ≤5%) congenital Hemophilia A without FVIII inhibitors
      • Weight ≥3 kilograms (kg)
      • Need for prophylaxis based on investigator assessment
      • A negative test for inhibitor (i.e., <0.6 Bethesda Units per milliliter [BU/mL]) within 8 weeks prior to enrollment
      • No documented inhibitor (i.e., <0.6 BU/mL), FVIII half-life <6 hours, or FVIII recovery <66% in the last 5 years
      • Documentation of the details of prophylactic or episodic FVIII treatment and of number of bleeding episodes for at least the last 24 weeks prior to enrollment
      • Adequate hematologic, hepatic, and renal function
      • For women of childbearing potential: agreement to remain abstinent or use contraception (as defined in the protocol) during the treatment period and for at least 24 weeks after the final dose of study drug
      Exclusion Criteria
      • Inherited or acquired bleeding disorder other than mild or moderate congenital hemophilia A
      • History of illicit drug or alcohol abuse within 48 weeks prior to screening, in the investigator's judgment
      • Previous (within the last 12 months) or current treatment for thromboembolic disease or signs of thromboembolic disease
      • Other conditions that may currently increase the risk of bleeding or thrombosis
      • History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
      • Planned surgery during the emicizumab loading dose phase (surgeries in participants on emicizumab from Week 5 onwards are allowed)
      • Known HIV infection with CD4 counts <200 cells per microlitre (/μL)
      • Concomitant disease, condition, significant abnormality on screening evaluation or laboratory tests, or treatment that could interfere with the conduct of the study, or that would in the opinion of the investigator, pose an additional unacceptable risk in administering study drug to the participant
      • Receipt of any of the following: An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration with the exception of prior emicizumab prophylaxis; A non-hemophilia-related investigational drug within last 30 days or 5 half-lives, whichever is shorter; or Any other investigational drug currently being administered or planned to be administered
      • Inability to comply with the study protocol in the opinion of the investigator
      • Pregnant or breastfeeding, or intending to become pregnant during the study (women of childbearing potential must have a negative serum pregnancy test result within 7 days prior to initiation of study drug)

      Um die aktuellste Version dieser Seite aufzurufen gehen Sie bitte auf www.Klinische-studien-für-patienten.de

       

      Über klinische Studien

      Erfahren Sie mehr über klinische Studien und wie sie durchgeführt werden

      Erfahren Sie mehr