Study of Biostate® in Children With Von Willebrand Disease
2 oktober 2017 bijgewerkt door: 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.
Studie Overzicht
Studietype
Ingrijpend
Inschrijving (Werkelijk)
17
Fase
- Fase 3
Contacten en locaties
In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.
Studie Locaties
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Bremen, Duitsland, 28177
- Study Site
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Tbilisi, Georgië, 0179
- Study Site
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CP
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Guatemala, CP, Guatemala, 01010
- Study Site
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Beirut, Libanon
- Study Site
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Lviv, Oekraïne
- Study Site
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Homel, Wit-Rusland, 246040
- Study Site
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Minsk, Wit-Rusland, 223040
- Study Site
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Deelname Criteria
Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
Niet ouder dan 12 jaar (Kind)
Accepteert gezonde vrijwilligers
Nee
Geslachten die in aanmerking komen voor studie
Allemaal
Beschrijving
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
Studie plan
Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: NVT
- Interventioneel model: Opdracht voor een enkele groep
- Masker: Geen (open label)
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
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Experimenteel: 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. |
Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Tijdsspanne |
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Half-life of FVIII
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 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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Secundaire uitkomstmaten
Uitkomstmaat |
Tijdsspanne |
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Frequency of adverse events (AEs) per infusion
Tijdsspanne: 13 months
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13 months
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Severity of AEs per infusion
Tijdsspanne: 13 months
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13 months
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Severity of AEs per subject
Tijdsspanne: 13 months
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13 months
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Relatedness of AEs per infusion
Tijdsspanne: 13 months
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13 months
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Relatedness of AEs per subject
Tijdsspanne: 13 months
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13 months
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Development of VWF inhibitors
Tijdsspanne: 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
Tijdsspanne: 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
Tijdsspanne: 13 months
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13 months
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Medewerkers en onderzoekers
Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.
Sponsor
Medewerkers
Studie record data
Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.
Bestudeer belangrijke data
Studie start
1 augustus 2010
Primaire voltooiing (Werkelijk)
1 augustus 2013
Studie voltooiing (Werkelijk)
1 augustus 2013
Studieregistratiedata
Eerst ingediend
1 oktober 2010
Eerst ingediend dat voldeed aan de QC-criteria
1 oktober 2010
Eerst geplaatst (Schatting)
4 oktober 2010
Updates van studierecords
Laatste update geplaatst (Werkelijk)
3 oktober 2017
Laatste update ingediend die voldeed aan QC-criteria
2 oktober 2017
Laatst geverifieerd
1 oktober 2017
Meer informatie
Termen gerelateerd aan deze studie
Trefwoorden
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- CSLCT-BIO-08-52
- 1494 (CSL Behring)
- 2009-017753-34 (EudraCT-nummer)
Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .