Study of Biostate® in Children With Hemophilia A
23 augusti 2017 uppdaterad av: CSL Behring
A Phase III, Open-Label, Multicentre Study to Evaluate Efficacy, Pharmacokinetics, and Safety of Biostate® in Paediatric Subjects With Haemophilia A
The objective of this study is to assess the efficacy and safety of a Von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, and to investigate the pharmacokinetics of Biostate in children with haemophilia A.
Studieöversikt
Studietyp
Interventionell
Inskrivning (Faktisk)
35
Fas
- Fas 3
Kontakter och platser
Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.
Studieorter
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Gomel, Belarus, 246040
- Study Site
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Minsk, Belarus, 223040
- Study Site
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Tbilisi, Georgien, 0179
- Study Site
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Guatemala, Guatemala, 01010
- Study Site
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Beirut, Libanon
- Study Site
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Monterrey, Mexiko, 64000
- Study Site
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Dnipropetrovsk, Ukraina
- Study Site
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Lviv, Ukraina
- Study Site
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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
Manlig
Beskrivning
Inclusion Criteria:
- Male subjects between 0 and <12 years of age.
- Diagnosed with severe haemophilia A (FVIII:C <1%), and pre-treated for a minimum of 20 to 50 exposure days.
- Have 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 immunisation), as documented in the medical notes at enrolment.
- The subject and/or legal guardian understand(s) the nature of the study and has/have given written informed consent to participate in the study and is/are willing to comply with the protocol.
Exclusion Criteria:
- For all subjects at Day 1: Are actively bleeding.
- Have received an infusion of any FVIII product, cryoprecipitate, whole blood, plasma or desmopressin acetate in the 4 days prior to their dosing within the PK component.
- Have a known history of, or who are suspected of having FVIII inhibitors.
- Have received aspirin or other non-steroidal anti-inflammatory drugs (NSAIDs) within 7 days of administration of the IMP.
- Have an impaired liver function ie, bilirubin >1.5 x upper limit of normal (ULN) and/or aspartate/alanine aminotransferase (AST/ALT) >2.5 x ULN (referring to limits of the laboratory that performs the determination) at Screening.
- Are human immunodeficiency virus [HIV]-1/-2 antibody positive with a viral load of >200/µL.
- Suffer from an acute or chronic medical condition, other than haemophilia A, which may, in the opinion of the Investigator, affect the conduct of the study.
- Suffering from von Willebrand disease (VWD) with von Willebrand factor: ristocetin cofactor (VWF:RCo) level <50 IU/dL at Screening.
- Have a known or suspected hypersensitivity or previous evidence of severe side effects to a plasma-derived FVIII product or to human albumin.
- Have participated in a clinical study or used an investigational compound in another study (eg, a new chemical entity not registered for clinical use) in the 3 months preceding the first day of IMP administration, or are planning to enter such a study during the study period.
- 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 |
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Experimentell: Biostate
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1 dose of 50 IU FVIII/kg body weight of Biostate administered intravenously on Day 1 in the PK component, followed by the Efficacy component for continuation of Biostate therapy, as required for a minimum of 50 exposure days.
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Vad mäter studien?
Primära resultatmått
Resultatmått |
Tidsram |
|---|---|
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Subjective assessment of Haemostatic efficacy
Tidsram: Over minimum of 50 exposure days
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Over minimum of 50 exposure days
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Incremental recovery of FVIII
Tidsram: 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 FVIII
Tidsram: 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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Area under the concentration curve (AUC) of FVIII
Tidsram: 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
Tidsram: 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 at 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
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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 FVIII
Tidsram: 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 FVIII
Tidsram: 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 the maximum concentration occurs (tmax) of FVIII
Tidsram: 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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Total clearance of the drug from the body (CL=dose/AUC) of FVIII
Tidsram: 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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Number of infusions per bleeding event
Tidsram: 1 day
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1 day
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Number of infusions per month
Tidsram: 1 month
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1 month
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Number of infusions per year
Tidsram: 1 year
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1 year
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Dose (IU/kg) per bleeding event
Tidsram: 1 day
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1 day
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Dose (IU/kg) per month
Tidsram: 1 month
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1 month
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Dose (IU/kg) per year
Tidsram: 1 year
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1 year
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Sekundära resultatmått
Resultatmått |
Tidsram |
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Frequency of adverse events (AEs)
Tidsram: 6 months
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6 months
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Severity of AEs per subject
Tidsram: 6 months
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6 months
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Severity of AEs per infusion
Tidsram: 6 months
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6 months
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Relatedness of AEs per subject
Tidsram: 6 months
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6 months
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Relatedness of AEs per infusion
Tidsram: 6 months
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6 months
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Development of FVIII inhibitors
Tidsram: Samples taken at screening visit, on day 2, on months 1 and 3, and at final visit
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Samples taken at screening visit, on day 2, on months 1 and 3, and at final visit
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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 juli 2014
Avslutad studie (Faktisk)
1 juli 2014
Studieregistreringsdatum
Först inskickad
1 oktober 2010
Först inskickad som uppfyllde QC-kriterierna
26 oktober 2010
Första postat (Uppskatta)
27 oktober 2010
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
24 augusti 2017
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
23 augusti 2017
Senast verifierad
1 juli 2014
Mer information
Termer relaterade till denna studie
Nyckelord
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- CSLCT-BIO-08-53
- 1495 (Recep Tayyip Erdogan University, Department of Scientific Research Project)
- 2009-015112-18 (EudraCT-nummer)
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