- ICH GCP
- Registre américain des essais cliniques
- Essai clinique NCT00694785
A Study of the Pharmacokinetics, Pharmacodynamics, and Safety of ARC1779 Injection in Patients With Von Willebrand Disease Type 2B
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Description détaillée
Type d'étude
Inscription (Anticipé)
Phase
- Phase 2
Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
Accepte les volontaires sains
Sexes éligibles pour l'étude
La description
Inclusion Criteria:
- Male or female patients;
- ≥ 16 to ≤ 75 years of age;
- Diagnosis of VWD-2B according to national expert guidelines for the USA [1] and Europe [2] based on medical history and findings from a matrix of laboratory assays which may include: platelet count, concentration of VWF antigen (VWF:Ag), VWF ristocetin cofactor activity (VWF:RCo), Factor VIII (FVIII) activity, ristocetin-induced platelet aggregation (RIPA), platelet function analyzer (PFA-100®) closure time, bleeding time (BT), VWF multimer test, VWF: platelet-binding (VWF:PB) activity, etc.)
- Thrombocytopenia (defined as a platelet count < 100 per nL on at least 2 occasions within the month preceding enrollment;
- Female patients of reproductive age must be enrolled within 1 to 7 days of the cessation of preceding menses;
- Female patients must be non-pregnant and willing to use effective, redundant methods of contraception (i.e., for both self and male partner) throughout the study and for at least 30 days after discontinuation of study drug treatment;
- Male patients must agree to use a medically acceptable contraceptive (abstinence or use of a condom with spermicide) throughout the study and for at least 30 days after discontinuation of study drug treatment;
- All patients must be capable of understanding and complying with the protocol and must have signed the informed consent document.
Exclusion Criteria:
- Patients with a possible co-existing or alternative hematologic diagnosis which can account for the laboratory findings of thrombocytopenia, etc.;
- Any significant medical co-morbidity which would pose an increased risk of bleeding (e.g., recent trauma or surgery, a history of gastrointestinal ulcers, etc.) or thrombosis (e.g., history of recurrent deep vein thrombosis (DVT).
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: Randomisé
- Modèle interventionnel: Affectation à un seul groupe
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
---|---|
Expérimental: Group NT3
Patients will receive a total dose of approximately 1.7 mg/kg of ARC1779 over 72 hours to achieve a target plasma concentration of 3 mcg/mL
|
Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.7 mg/kg given over the remaining 72 hours at a rate of 0.0004 mg/kg/min. Group "NT3" Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.3 mg/kg given over the next 72 hours. Group "T3" Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.8 mg/kg given over the remaining 72 hours at a rate of 0.0009 mg/kg/min. Group "NT6" Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.0 mg/kg given over the remaining 72 hours. Group "T6" |
Expérimental: Group T3
Patients will receive a total dose of approximately 1.4 mg/kg of ARC1779 over 72 hours to achieve a target plasma concentration of 3 mcg/mL.
The infusion of ARC1779 will be tapered by 50% from 48 hours to 60 hours and again by 50% from 60 hours to 72 hours.
|
Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.7 mg/kg given over the remaining 72 hours at a rate of 0.0004 mg/kg/min. Group "NT3" Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.3 mg/kg given over the next 72 hours. Group "T3" Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.8 mg/kg given over the remaining 72 hours at a rate of 0.0009 mg/kg/min. Group "NT6" Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.0 mg/kg given over the remaining 72 hours. Group "T6" |
Expérimental: Group NT6
Patients will receive a total dose of approximately 3.9 mg/kg of ARC1779 over 72 hours to achieve a target plasma concentration of 6 mcg/mL.
|
Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.7 mg/kg given over the remaining 72 hours at a rate of 0.0004 mg/kg/min. Group "NT3" Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.3 mg/kg given over the next 72 hours. Group "T3" Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.8 mg/kg given over the remaining 72 hours at a rate of 0.0009 mg/kg/min. Group "NT6" Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.0 mg/kg given over the remaining 72 hours. Group "T6" |
Expérimental: Group T6
Patients will receive a total dose of approximately 3.1 mg/kg of ARC1779 over 72 hours to achieve a target plasma concentration of 6 mcg/mL.
The infusion of ARC1779 will be tapered by 50% from 48 hours to 60 hours and again by 50% from 60 hours to 72 hours.
|
Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.7 mg/kg given over the remaining 72 hours at a rate of 0.0004 mg/kg/min. Group "NT3" Initial stepwise infusion of 0.14 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 1.3 mg/kg given over the next 72 hours. Group "T3" Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.8 mg/kg given over the remaining 72 hours at a rate of 0.0009 mg/kg/min. Group "NT6" Initial stepwise infusion of 0.28 mg/kg given over 60 minutes and subsequent continuous infusion of an additional 3.0 mg/kg given over the remaining 72 hours. Group "T6" |
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Délai |
---|---|
To evaluate the effect of ARC1779 Injection on platelet counts in vWD-2B patients who have thrombocytopenia at baseline.
Délai: 10 days
|
10 days
|
Mesures de résultats secondaires
Mesure des résultats |
Délai |
---|---|
To assess the concentration-response relationships among ARC1779 pharmacokinetic (PK) and pharmacodynamic (PD) parameters
Délai: 10 days
|
10 days
|
Collaborateurs et enquêteurs
Parrainer
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude
Achèvement primaire (Anticipé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Estimation)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Estimation)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
- Processus pathologiques
- Maladies hématologiques
- Troubles de la coagulation sanguine, héréditaires
- Troubles des protéines de la coagulation
- Troubles hémorragiques
- Maladies génétiques, innées
- Troubles de la coagulation sanguine
- Troubles des plaquettes sanguines
- Maladie
- Maladies de von Willebrand
- Maladie de von Willebrand, type 2
Autres numéros d'identification d'étude
- ARC1779-010
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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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
-
TakedaComplétéMaladie de von Willebrand (MVW)Canada
Essais cliniques sur ARC1779
-
Archemix Corp.Complété
-
Archemix Corp.RésiliéPurpura thrombotique thrombotique | Microangiopathie thrombotiqueÉtats-Unis, Italie, L'Autriche, Canada, Royaume-Uni
-
Archemix Corp.ComplétéPurpura thrombotique thrombocytopénique | Maladie de von Willebrand Type-2bL'Autriche
-
Archemix Corp.St George's, University of LondonRésiliéSténose carotidienne | Embolie intracrânienne | Thromboembolie cérébraleÉtats-Unis, Royaume-Uni