Descrizione riassuntiva della sperimentazione (Fonte di dati: BASEC)
Es handelt sich um eine internationale Studie, die in mehreren Ländern durchgeführt wird. Weltweit werden ungefähr 200 Patienten an dieser Studie teilnehmen. Patienten die sich für die Teilnahme an dieser Studie eignen erhalten das Prüfpräparat Emicizumab. Das Ziel der Studie ist es die Sicherheit und Verträglichkeit von Emicizumab zu untersuchen.
Malattie studiate(Fonte di dati: BASEC)
Hämophilie A bei Patienten mit Hemmkörper (neutralisierende Antikörper) gegen Faktor VIII (FVIII)
Health conditions
(Fonte di dati: WHO)
Hemophilia A
Malattia rara
(Fonte di dati: BASEC)
No
Interventi esaminati (p. es. medicamento, terapia, campagna)
(Fonte di dati: BASEC)
Emicizumab wird durch eine Injektion unmittelbar unter die Hautoberfläche verabreicht. Patienten erhalten eine Dosis von 3 mg pro Kilogramm Körpergewicht Emicizumab pro Woche für die ersten 4 Wochen. Nach 4 Wochen wird die Dosis auf 1,5 mg pro Kilogramm Körpergewicht halbiert und dann einmal wöchentlich für den Rest der 2-jährigen Behandlungsphase verabreicht.
Interventions
(Fonte di dati: WHO)
Drug: Emicizumab
Criteri per la partecipazione alla sperimentazione
(Fonte di dati: BASEC)
- 12 Jahre alt oder älter
- Hämophilie A mit persistierenden Hemmkörpern (neutralisierende Antikörper) gegen Faktor VIII (FVIII)
- Behandlung mit Bypass-Medikamenten oder FVIII Konzentrat innerhalb der letzten 6 Monaten
Criteri di esclusione
(Fonte di dati: BASEC)
- andere Blutungsstörungen, ausser Hämophilie A
- laufende Behandlung mit Immuntoleranz-Induktionstherapie (prophylaktische Behandlung mit FVIII und/oder Bypass Medikamenten müssen vor dem Einschluss in die Studie abgesetzt werden).
Inclusion/Exclusion Criteria
(Fonte di dati: WHO)
Inclusion Criteria:
- As per investigator's judgement, a willingness and ability to comply with scheduled
visits, treatment plans, laboratory tests, and other study procedures, including the
patient-reported outcome (PRO) questionnaires and bleed diaries through the use of an
electronic device or paper
- Aged 12 years or older at the time of informed consent
- Diagnosis of congenital hemophilia A with persistent inhibitors against FVIII
- Documented treatment with bypassing agents or FVIII concentrates in the last 6 months
(on-demand or prophylaxis). Prophylaxis needs to be discontinued the latest by a day
before starting emicizumab
- Adequate hematologic, hepatic, and renal function
- For women of childbearing potential: agreement to remain abstinent (refrain from
heterosexual intercourse) or use a highly effective contraceptive method with a
failure rate of <1% per year during the treatment period and for at least five
elimination half-lives (24 weeks) after the last dose of emicizumab
Exclusion Criteria:
- Inherited or acquired bleeding disorder other than hemophilia A
- Ongoing (or plan to receive during the study) immune tolerance induction (ITI) therapy
(prophylaxis regimens with FVIII and/or bypassing agents must be discontinued prior to
enrollment). Patients receiving ITI therapy will be eligible following the completion
of a 72-hour washout period prior to the first emicizumab administration
- History of illicit drug or alcohol abuse within 12 months prior to screening, as per
the investigator's judgment
- High risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or
family history of TMA), as per the investigator's judgment
- Previous (in the past 12 months) or current treatment for thromboembolic disease (with
the exception of previous catheter-associated thrombosis for which antithrombotic
treatment is not currently ongoing) or current signs of thromboembolic disease
- Other conditions (e.g., certain autoimmune diseases) that may increase the risk of
bleeding or thrombosis
- History of clinically significant hypersensitivity reaction associated with monoclonal
antibody therapies or components of the emicizumab injection
- Known human immunodeficiency virus (HIV) infection with CD4 count <200 cells/µL within
6 months prior to screening
- Use of systemic immunomodulators (e.g., interferon or rituximab) at enrollment or
planned use during the study, with the exception of antiretroviral therapy
- Concurrent disease, treatment, or abnormality in clinical laboratory tests that could
interfere with the conduct of the study or that would, in the opinion of the
investigator or Sponsor, preclude the patient's safe participation in and completion
of the study or interpretation of the study results
- Receipt of: Emicizumab in a prior investigational study; An investigational drug to
treat or reduce the risk of hemophilic bleeds within five half-lives of last drug
administration; A non-hemophilia-related investigational drug within last 30 days or
five half-lives, whichever is shorter; or, Any concurrent investigational drug.
- Pregnancy or lactation, or intent to become pregnant during the study
- Positive serum pregnancy test result within 7 days prior to initiation of emicizumab
(females only)
-
Altre informazioni sulla sperimentazione
Data di registrazione della sperimentazione
15 giu 2017
Inserimento del primo partecipante
5 set 2017
Stato di reclutamento
Completed
Titolo scientifico
(Fonte di dati: WHO)
A Single-Arm, Multicenter Phase IIIB Clinical Trial to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
Tipo di sperimentazione
(Fonte di dati: WHO)
Interventional
Disegno della sperimentazione
(Fonte di dati: WHO)
Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
Fase
(Fonte di dati: WHO)
Phase 3
Punti finali primari
(Fonte di dati: WHO)
Incidence and severity of all adverse events (AEs) including thromboembolic events, microangiopathic hemolytic anemia or TMA (e.g. hemolytic uremic syndrome), systemic hypersensitivity, anaphylaxis, and anaphylactoid events
Punti finali secondari
(Fonte di dati: WHO)
Area under the plasma drug concentration-time curve (AUC) of emicizumab;Volume of distribution of emicizumab;Clearance of emicizumab;Ctrough of emicizumab;Incidence and clinical significance of anti-emicizumab antibodies;Participant preference for the emicizumab regimen compared with the previous regimen, as measured by the EmiPref questionnaire;EuroQoL Five-Dimension-Five Levels Questionnaire (EQ-5D-5L);Hemophilia Quality of Life Short Form (Haemo-QoL-SF) (12-17 y);Hemophilia Adult Quality of Life Questionnaire (Haem-A-QoL) (>= 18 y);Numbers of bleeds over time
Contatto per informazioni
(Fonte di dati: WHO)
Please refer to primary and secondary sponsors
Risultati della sperimentazione
(Fonte di dati: WHO)
Sintesi dei risultati
ancora nessuna informazione disponibile
Collegamento ai risultati nel registro primario
ancora nessuna informazione disponibile
Informazioni sulla disponibilità dei dati dei singoli partecipanti
ancora nessuna informazione disponibile
Siti di esecuzione della sperimentazione
Siti di esecuzione in Svizzera
(Fonte di dati: BASEC)
Berna
Paesi di esecuzione
(Fonte di dati: WHO)
Australia, Belgium, Brazil, Canada, Colombia, Denmark, Finland, Germany, Guatemala, Hungary, India, Israel, Italy, Mexico, Netherlands, Panama, Poland, Portugal, Romania, Russian Federation, Saudi Arabia, Spain, Sweden, Switzerland, United Kingdom
Contatto per maggiori informazioni sulla sperimentazione
Dati della persona di contatto in Svizzera
(Fonte di dati: BASEC)
Clinical Trials
+41617154391
switzerland.clinical-research@roche.com
Contatto per informazioni generali
(Fonte di dati: WHO)
Clinical Trials
Hoffmann-La Roche
Contatto per informazioni scientifiche
(Fonte di dati: WHO)
Clinical Trials
Hoffmann-La Roche
Autorizzazione da parte della commissione d’etica (Fonte di dati: BASEC)
Nome della commissione d’etica che rilascia l’autorizzazione (nel caso di studi multicentrici solo la commissione direttiva)
Kantonale
Ethikkommission Bern
Data di autorizzazione da parte della commissione d’etica
28.11.2017
Altri numeri di identificazione delle sperimentazioni
Numero di identificazione della sperimentazione della commissione d’etica (BASEC-ID)
(Fonte di dati: BASEC)
2017-01510
Secondary ID (Fonte di dati: WHO)
2016-004366-25
MO39129
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