- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg NCT01213446
Study of Biostate® in Children With Von Willebrand Disease
2. oktober 2017 opdateret af: 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.
Studieoversigt
Undersøgelsestype
Interventionel
Tilmelding (Faktiske)
17
Fase
- Fase 3
Kontakter og lokationer
Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.
Studiesteder
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Tbilisi, Georgien, 0179
- Study Site
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CP
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Guatemala, CP, Guatemala, 01010
- Study Site
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Homel, Hviderusland, 246040
- Study Site
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Minsk, Hviderusland, 223040
- Study Site
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Beirut, Libanon
- Study Site
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Bremen, Tyskland, 28177
- Study Site
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Lviv, Ukraine
- Study Site
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Deltagelseskriterier
Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.
Berettigelseskriterier
Aldre berettiget til at studere
Ikke ældre end 12 år (Barn)
Tager imod sunde frivillige
Ingen
Køn, der er berettiget til at studere
Alle
Beskrivelse
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
Studieplan
Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
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Eksperimentel: 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. |
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Tidsramme |
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Half-life of FVIII
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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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Sekundære resultatmål
Resultatmål |
Tidsramme |
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Frequency of adverse events (AEs) per infusion
Tidsramme: 13 months
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13 months
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Severity of AEs per infusion
Tidsramme: 13 months
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13 months
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Severity of AEs per subject
Tidsramme: 13 months
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13 months
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Relatedness of AEs per infusion
Tidsramme: 13 months
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13 months
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Relatedness of AEs per subject
Tidsramme: 13 months
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13 months
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Development of VWF inhibitors
Tidsramme: 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
Tidsramme: 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
Tidsramme: 13 months
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13 months
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Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Sponsor
Samarbejdspartnere
Datoer for undersøgelser
Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.
Studer store datoer
Studiestart
1. august 2010
Primær færdiggørelse (Faktiske)
1. august 2013
Studieafslutning (Faktiske)
1. august 2013
Datoer for studieregistrering
Først indsendt
1. oktober 2010
Først indsendt, der opfyldte QC-kriterier
1. oktober 2010
Først opslået (Skøn)
4. oktober 2010
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
3. oktober 2017
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
2. oktober 2017
Sidst verificeret
1. oktober 2017
Mere information
Begreber relateret til denne undersøgelse
Nøgleord
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- CSLCT-BIO-08-52
- 1494 (CSL Behring)
- 2009-017753-34 (EudraCT nummer)
Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .
Kliniske forsøg med Von Willebrands sygdom
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St. James's Hospital, IrelandUkendt
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Fondazione IRCCS Ca' Granda, Ospedale Maggiore...RekrutteringLav Von Willebrand FaktorItalien
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Baylor College of MedicineShireAktiv, ikke rekrutterende
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University Hospital, CaenRekrutteringVon Willebrands sygdom, type 2BFrankrig
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Archemix Corp.Trukket tilbage
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Fondazione Angelo Bianchi BonomiSintesi Research SrlAktiv, ikke rekrutterendeType 3 Von Willebrands sygdomFinland, Frankrig, Tyskland, Ungarn, Iran, Islamisk Republik, Italien, Holland, Spanien, Sverige, Det Forenede Kongerige
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OctapharmaRekrutteringVWD - Von Willebrands sygdomFrankrig
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TakedaLedigVon Willebrands sygdom (VWD)
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Tirol Kiniken GmbHLFB BIOMEDICAMENTSUkendt
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CSL BehringAfsluttetVon Willebrands sygdomBulgarien, Tyskland, Polen, Den Russiske Føderation, Ukraine
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CSL BehringAfsluttetVon Willebrands sygdomFrankrig
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Spinal Restoration, Inc.Afsluttet
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