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- Registre américain des essais cliniques
- Essai clinique NCT01213446
Study of Biostate® in Children With Von Willebrand Disease
2 octobre 2017 mis à jour par: CSL Behring
A Phase III Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy, and Safety of Biostate® in Paediatric Subjects With Von Willebrand Disease
This is an open-label study to investigate the pharmacokinetics (PK), efficacy, and safety of a von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, in children with Von Willebrand disease (VWD) in whom treatment with a VWF product is required for prophylactic therapy, haemostatic control during surgery, or control of a non-surgical, spontaneous, or traumatic bleeding event.
Aperçu de l'étude
Statut
Complété
Les conditions
Intervention / Traitement
Type d'étude
Interventionnel
Inscription (Réel)
17
Phase
- Phase 3
Contacts et emplacements
Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.
Lieux d'étude
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Bremen, Allemagne, 28177
- Study Site
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Homel, Biélorussie, 246040
- Study Site
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Minsk, Biélorussie, 223040
- Study Site
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CP
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Guatemala, CP, Guatemala, 01010
- Study Site
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Tbilisi, Géorgie, 0179
- Study Site
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Beirut, Liban
- Study Site
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Lviv, Ukraine
- Study Site
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Critères de participation
Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.
Critère d'éligibilité
Âges éligibles pour étudier
Pas plus vieux que 12 ans (Enfant)
Accepte les volontaires sains
Non
Sexes éligibles pour l'étude
Tout
La description
Inclusion Criteria:
- Male and female subjects between 0 and <12 years of age
- Diagnosed with VWD Type 1, 2A, or 3
- Desmopressin acetate (DDAVP) treatment is ineffective, contraindicated, or not available for subject
- von Willebrand factor: ristocetin cofactor (VWF:RCo) is <20% at screening or the subject has a history of VWF:RCo <10%
- Evidence of vaccination against hepatitis A and B or presence of antibodies against hepatitis A and B due to either a previous infection or prior immunization
- Written informed consent given
Exclusion Criteria:
- Active bleeding immediately prior to initial PK period
- Received treatment with DDAVP or a VWF concentrate product for their VWD in the 5 days prior to their first study treatment
- Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of commencing the PK period.
- Known history or suspicion of having VWF or FVIII inhibitors
- Acute or chronic medical condition, other than VWD, which may affect the conduct of the study
- Known or suspected hypersensitivity or previous evidence of severe side effects to other FVIII/VWF concentrates
- Participation in a clinical study or use of an investigational compound in another study in the 3 months preceding study start
- Unwillingness and/or inability to comply with the study requirements
Plan d'étude
Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: N / A
- Modèle interventionnel: Affectation à un seul groupe
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
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Expérimental: Biostate
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PK component: Single bolus infusion of 80 IU VWF:RCo/kg administered intravenously on Day 1, and approximately Day 180 in Type 3 VWD subjects only. Efficacy component: Repeated bolus doses over 12 months as required to manage VWD condition. |
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Délai |
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Half-life of FVIII
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Mean residence time (MRT) of FVIII
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Haemostatic efficacy
Délai: From Day 1 until final study visit
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From Day 1 until final study visit
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Incremental Recovery of VWF
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Incremental Recovery of FVIII
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Half-life of VWF
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Area under the concentration curve (AUC) of VWF
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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AUC of FVIII
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Maximum plasma concentration (Cmax) of VWF
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Maximum plasma concentration (Cmax) of FVIII
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Minimum plasma concentration (Cmin) of VWF
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Minimum plasma concentration (Cmin) of FVIII
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Time to maximum concentration (tmax) of VWF
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Time to maximum concentration (tmax) of FVIII
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Mean residence time (MRT) of VWF
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Clearance (CL) of VWF
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Clearance (CL) of FVIII
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Volume of distribution of steady state (Vss) of VWF
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 and 6 months after initial dose
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Volume of distribution of steady state (Vss) of FVIII
Délai: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
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Mesures de résultats secondaires
Mesure des résultats |
Délai |
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Frequency of adverse events (AEs) per infusion
Délai: 13 months
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13 months
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Severity of AEs per infusion
Délai: 13 months
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13 months
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Severity of AEs per subject
Délai: 13 months
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13 months
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Relatedness of AEs per infusion
Délai: 13 months
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13 months
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Relatedness of AEs per subject
Délai: 13 months
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13 months
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Development of VWF inhibitors
Délai: Sample taken at baseline, then every 3 months up to 12 months
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Sample taken at baseline, then every 3 months up to 12 months
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Development of FVIII inhibitors
Délai: Sample taken at baseline, then every 3 months up to 12 months
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Sample taken at baseline, then every 3 months up to 12 months
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Frequency of adverse events (AEs) per subject
Délai: 13 months
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13 months
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Collaborateurs et enquêteurs
C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.
Parrainer
Collaborateurs
Dates d'enregistrement des études
Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.
Dates principales de l'étude
Début de l'étude
1 août 2010
Achèvement primaire (Réel)
1 août 2013
Achèvement de l'étude (Réel)
1 août 2013
Dates d'inscription aux études
Première soumission
1 octobre 2010
Première soumission répondant aux critères de contrôle qualité
1 octobre 2010
Première publication (Estimation)
4 octobre 2010
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
3 octobre 2017
Dernière mise à jour soumise répondant aux critères de contrôle qualité
2 octobre 2017
Dernière vérification
1 octobre 2017
Plus d'information
Termes liés à cette étude
Mots clés
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- CSLCT-BIO-08-52
- 1494 (CSL Behring)
- 2009-017753-34 (Numéro EudraCT)
Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .
Essais cliniques sur Maladie de von Willebrand
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St. James's Hospital, IrelandInconnue
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Baxalta now part of ShireComplétéMaladie de von WillebrandÉtats-Unis, Allemagne, Royaume-Uni, Italie, L'Autriche, Canada
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University Hospital, CaenRecrutementMaladie de von Willebrand, type 2BFrance
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Archemix Corp.Retiré
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Fondazione Angelo Bianchi BonomiSintesi Research SrlActif, ne recrute pasMaladie de von Willebrand de type 3Finlande, France, Allemagne, Hongrie, Iran (République islamique d, Italie, Pays-Bas, Espagne, Suède, Royaume-Uni
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Archemix Corp.ComplétéPurpura thrombotique thrombocytopénique | Maladie de von Willebrand Type-2bL'Autriche
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OctapharmaRecrutementVWD - Maladie de von WillebrandFrance
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TakedaDisponibleMaladie de von Willebrand (MVW)
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Tirol Kiniken GmbHLFB BIOMEDICAMENTSInconnueMaladie de von Willebrand acquiseL'Autriche
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National Center for Research Resources (NCRR)University of North CarolinaComplétéLa maladie de von Willebrand
Essais cliniques sur Biostate
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Spinal Restoration, Inc.Complété
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Spinal Restoration, Inc.RésiliéMal au dos | Discopathie dégénérative | Lombalgie chronique | Interruption du disque interneÉtats-Unis