- ICH GCP
- Amerikanska kliniska prövningsregistret
- Klinisk prövning NCT01213446
Study of Biostate® in Children With Von Willebrand Disease
2 oktober 2017 uppdaterad av: 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.
Studieöversikt
Studietyp
Interventionell
Inskrivning (Faktisk)
17
Fas
- Fas 3
Kontakter och platser
Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.
Deltagandekriterier
Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.
Urvalskriterier
Åldrar som är berättigade till studier
Inte äldre än 12 år (Barn)
Tar emot friska volontärer
Nej
Kön som är behöriga för studier
Allt
Beskrivning
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
Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Behandling
- Tilldelning: N/A
- Interventionsmodell: Enskild gruppuppgift
- Maskning: Ingen (Open Label)
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
---|---|
Experimentell: Biostate
|
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. |
Vad mäter studien?
Primära resultatmått
Resultatmått |
Tidsram |
---|---|
Half-life of FVIII
Tidsram: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Mean residence time (MRT) of FVIII
Tidsram: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Haemostatic efficacy
Tidsram: From Day 1 until final study visit
|
From Day 1 until final study visit
|
Incremental Recovery of VWF
Tidsram: 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
|
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
|
Incremental Recovery of FVIII
Tidsram: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Half-life of VWF
Tidsram: 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
|
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
|
Area under the concentration curve (AUC) of VWF
Tidsram: 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
|
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
|
AUC of FVIII
Tidsram: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Maximum plasma concentration (Cmax) of VWF
Tidsram: 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
|
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
|
Maximum plasma concentration (Cmax) of FVIII
Tidsram: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Minimum plasma concentration (Cmin) of VWF
Tidsram: 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
|
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
|
Minimum plasma concentration (Cmin) of FVIII
Tidsram: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Time to maximum concentration (tmax) of VWF
Tidsram: 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
|
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
|
Time to maximum concentration (tmax) of FVIII
Tidsram: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Mean residence time (MRT) of VWF
Tidsram: 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
|
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
|
Clearance (CL) of VWF
Tidsram: 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
|
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
|
Clearance (CL) of FVIII
Tidsram: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Volume of distribution of steady state (Vss) of VWF
Tidsram: 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
|
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
|
Volume of distribution of steady state (Vss) of FVIII
Tidsram: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
|
Sekundära resultatmått
Resultatmått |
Tidsram |
---|---|
Frequency of adverse events (AEs) per infusion
Tidsram: 13 months
|
13 months
|
Severity of AEs per infusion
Tidsram: 13 months
|
13 months
|
Severity of AEs per subject
Tidsram: 13 months
|
13 months
|
Relatedness of AEs per infusion
Tidsram: 13 months
|
13 months
|
Relatedness of AEs per subject
Tidsram: 13 months
|
13 months
|
Development of VWF inhibitors
Tidsram: Sample taken at baseline, then every 3 months up to 12 months
|
Sample taken at baseline, then every 3 months up to 12 months
|
Development of FVIII inhibitors
Tidsram: Sample taken at baseline, then every 3 months up to 12 months
|
Sample taken at baseline, then every 3 months up to 12 months
|
Frequency of adverse events (AEs) per subject
Tidsram: 13 months
|
13 months
|
Samarbetspartners och utredare
Det är här du hittar personer och organisationer som är involverade i denna studie.
Sponsor
Samarbetspartners
Studieavstämningsdatum
Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.
Studera stora datum
Studiestart
1 augusti 2010
Primärt slutförande (Faktisk)
1 augusti 2013
Avslutad studie (Faktisk)
1 augusti 2013
Studieregistreringsdatum
Först inskickad
1 oktober 2010
Först inskickad som uppfyllde QC-kriterierna
1 oktober 2010
Första postat (Uppskatta)
4 oktober 2010
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
3 oktober 2017
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
2 oktober 2017
Senast verifierad
1 oktober 2017
Mer information
Termer relaterade till denna studie
Nyckelord
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- CSLCT-BIO-08-52
- 1494 (CSL Behring)
- 2009-017753-34 (EudraCT-nummer)
Denna information hämtades direkt från webbplatsen clinicaltrials.gov utan några ändringar. Om du har några önskemål om att ändra, ta bort eller uppdatera dina studieuppgifter, vänligen kontakta register@clinicaltrials.gov. Så snart en ändring har implementerats på clinicaltrials.gov, kommer denna att uppdateras automatiskt även på vår webbplats .
Kliniska prövningar på Von Willebrands sjukdom
-
St. James's Hospital, IrelandOkänd
-
Fondazione IRCCS Ca' Granda, Ospedale Maggiore...RekryteringLåg Von Willebrand-faktorItalien
-
Baylor College of MedicineShireAktiv, inte rekryterande
-
Baxalta now part of ShireAvslutadVon Willebrands sjukdomFörenta staterna, Tyskland, Storbritannien, Italien, Österrike, Kanada
-
University Hospital, CaenRekryteringVon Willebrands sjukdom, typ 2BFrankrike
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Fondazione Angelo Bianchi BonomiSintesi Research SrlAktiv, inte rekryterandeTyp 3 Von Willebrands sjukdomFinland, Frankrike, Tyskland, Ungern, Iran, Islamiska republiken, Italien, Nederländerna, Spanien, Sverige, Storbritannien
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Archemix Corp.Indragen
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OctapharmaRekryteringVWD - Von Willebrands sjukdomFrankrike
-
TakedaTillgängligtVon Willebrands sjukdom (VWD)
-
Tirol Kiniken GmbHLFB BIOMEDICAMENTSOkänd
Kliniska prövningar på Biostate
-
CSL BehringAvslutadVon Willebrands sjukdomBulgarien, Tyskland, Polen, Ryska Federationen, Ukraina
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CSL BehringAvslutadVon Willebrands sjukdomFrankrike
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Spinal Restoration, Inc.AvslutadLändryggssmärtaFörenta staterna
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Spinal Restoration, Inc.AvslutadRyggont | Degenerativ disksjukdom | Kronisk ländryggssmärta | Intern skivavbrottFörenta staterna