Study of Biostate® in Children With Von Willebrand Disease
This study is ongoing, but not recruiting participants.
Sponsor:
CSL Behring
Collaborator:
Parexel
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT01213446
First received: October 1, 2010
Last updated: May 12, 2013
Last verified: May 2013
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Von Willebrand Disease |
Biological: Biostate |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Phase III Open-label, Multi-centre Study to Assess the Pharmacokinetics, Efficacy, and Safety of Biostate® in Paediatric Subjects With Von Willebrand Disease |
Resource links provided by NLM:
Further study details as provided by CSL Behring:
Primary Outcome Measures:
- Haemostatic efficacy [ Time Frame: From Day 1 until final study visit ] [ Designated as safety issue: No ]
- Incremental Recovery of VWF [ Time Frame: 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 ] [ Designated as safety issue: No ]
- Incremental Recovery of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ] [ Designated as safety issue: No ]
- Half-life of VWF [ Time Frame: 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 ] [ Designated as safety issue: No ]
- Half-life of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ] [ Designated as safety issue: No ]
- Area under the concentration curve (AUC) of VWF [ Time Frame: 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 ] [ Designated as safety issue: No ]
- AUC of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ] [ Designated as safety issue: No ]
- Maximum plasma concentration (Cmax) of VWF [ Time Frame: 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 ] [ Designated as safety issue: No ]
- Maximum plasma concentration (Cmax) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ] [ Designated as safety issue: No ]
- Minimum plasma concentration (Cmin) of VWF [ Time Frame: 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 ] [ Designated as safety issue: No ]
- Minimum plasma concentration (Cmin) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ] [ Designated as safety issue: No ]
- Time to maximum concentration (tmax) of VWF [ Time Frame: 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 ] [ Designated as safety issue: No ]
- Time to maximum concentration (tmax) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ] [ Designated as safety issue: No ]
- Mean residence time (MRT) of VWF [ Time Frame: 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 ] [ Designated as safety issue: No ]
- Mean residence time (MRT) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ] [ Designated as safety issue: No ]
- Clearance (CL) of VWF [ Time Frame: 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 ] [ Designated as safety issue: No ]
- Clearance (CL) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ] [ Designated as safety issue: No ]
- Volume of distribution of steady state (Vss) of VWF [ Time Frame: 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 ] [ Designated as safety issue: No ]
- Volume of distribution of steady state (Vss) of FVIII [ Time Frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1 ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Frequency of adverse events (AEs) per infusion [ Time Frame: 13 months ] [ Designated as safety issue: Yes ]
- Severity of AEs per infusion [ Time Frame: 13 months ] [ Designated as safety issue: Yes ]
- Severity of AEs per subject [ Time Frame: 13 months ] [ Designated as safety issue: Yes ]
- Relatedness of AEs per infusion [ Time Frame: 13 months ] [ Designated as safety issue: Yes ]
- Relatedness of AEs per subject [ Time Frame: 13 months ] [ Designated as safety issue: Yes ]
- Development of VWF inhibitors [ Time Frame: Sample taken at baseline, then every 3 months up to 12 months ] [ Designated as safety issue: Yes ]
- Development of FVIII inhibitors [ Time Frame: Sample taken at baseline, then every 3 months up to 12 months ] [ Designated as safety issue: Yes ]
- Frequency of adverse events (AEs) per subject [ Time Frame: 13 months ] [ Designated as safety issue: Yes ]
| Estimated Enrollment: | 20 |
| Study Start Date: | August 2010 |
| Estimated Study Completion Date: | August 2013 |
| Arms | Assigned Interventions |
|---|---|
| Experimental: Biostate |
Biological: 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. |
Eligibility| Ages Eligible for Study: | up to 12 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01213446
Locations
| Belarus | |
| Study site | |
| Homel, Belarus, 246040 | |
| Study site | |
| Minsk, Belarus, 223040 | |
| Georgia | |
| Study site | |
| Tbilisi, Georgia, 0179 | |
| Germany | |
| Study site | |
| Bremen, Germany, 28177 | |
| Guatemala | |
| Study site | |
| Guatemala, CP, Guatemala, 01010 | |
| Lebanon | |
| Study site | |
| Beirut, Lebanon | |
| Ukraine | |
| Study Site | |
| Lviv, Ukraine | |
Sponsors and Collaborators
CSL Behring
Parexel
Investigators
| Study Director: | Program Director, Clinical R&D | CSL Behring |
More Information
Additional Information:
No publications provided
| Responsible Party: | CSL Behring |
| ClinicalTrials.gov Identifier: | NCT01213446 History of Changes |
| Other Study ID Numbers: | CSLCT-BIO-08-52, 2009-017753-34, 1494 |
| Study First Received: | October 1, 2010 |
| Last Updated: | May 12, 2013 |
| Health Authority: | Belarus: Ministry of Health Germany: Paul-Ehrlich-Institut Guatemala: Ministry of Public Health and Social Assistance Mexico: Ministry of Health Georgia: Ministry of Labour Health and Social Affairs of Georgia Bulgaria: Bulgarian Drug Agency Ukraine: Ministry of Health |
Keywords provided by CSL Behring:
|
Von Willebrand Disease |
Additional relevant MeSH terms:
|
Von Willebrand Diseases Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Blood Platelet Disorders Hemorrhagic Disorders Genetic Diseases, Inborn |
ClinicalTrials.gov processed this record on May 19, 2013