Hemophilia APhase 3NCT01229007
Study of Biostate® in Children With Hemophilia A
A Phase 3 study testing Biostate in people with Hemophilia A, run by CSL Behring at 8 sites in 6 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 12 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Biostate
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 8 sitesin 6 countries
- Study dates
- Aug 2010to Jul 2014
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 12 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
8 sites in 6 countries.
8 sites in 6 countries
About this study
Official title: 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.
- Conditions
- Hemophilia A
- Being tested
- Biostate
- Keywords
- Hemophilia A, Children
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BiostateBiological
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.
| Study group | Role | What they receive |
|---|---|---|
| Biostate | Experimental | Not described Receives: Biological: Biostate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Subjective assessment of Haemostatic efficacy
Measured: Over minimum of 50 exposure days
Incremental recovery of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Half-life of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Area under the concentration curve (AUC) of FVIII
Measured: 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
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Volume of distribution at steady state (Vss) of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Maximum Plasma Concentration (Cmax) of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Minimum Plasma Concentration (Cmin) of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Time the maximum concentration occurs (tmax) of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Total clearance of the drug from the body (CL=dose/AUC) of FVIII
Measured: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Number of infusions per bleeding event
Measured: 1 day
Number of infusions per month
Measured: 1 month
Number of infusions per year
Measured: 1 year
Dose (IU/kg) per bleeding event
Measured: 1 day
Dose (IU/kg) per month
Measured: 1 month
Dose (IU/kg) per year
Measured: 1 year
Secondary outcomes (6)
Frequency of adverse events (AEs)
Measured: 6 months
Severity of AEs per subject
Measured: 6 months
Severity of AEs per infusion
Measured: 6 months
Relatedness of AEs per subject
Measured: 6 months
Relatedness of AEs per infusion
Measured: 6 months
Development of FVIII inhibitors
Measured: Samples taken at screening visit, on day 2, on months 1 and 3, and at final visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2010 (estimated)
- Study startAug 2010
- Primary completionJul 2014
- Study completionJul 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by CSL BehringThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Parexel
- Responsible party
- Sponsor
- Investigators
- Program Director Clinical R&D, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01229007
- Sponsor's protocol ID
- CSLCT-BIO-08-53
- Other IDs
- 2009-015112-18, 1495, 2009
- Status last verified
- Jul 2014
- First submitted
- 1 Oct 2010
- Last updated
- 24 Aug 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 24 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III, Open-Label, Multicentre Study to Evaluate Efficacy, Pharmacokinetics, and Safety of Biostate® in Paediatric Subjects With Haemophilia A
- ClinicalTrials.gov ID
- NCT01229007
- Acronym / sponsor ID
- CSLCT-BIO-08-53
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A, Children
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Collaborators
- Parexel
- Study start
- Aug 2010
- Primary completion
- Jul 2014 (actual)
- Study completion
- Jul 2014 (actual)
- First posted
- 27 Oct 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Biostate | Experimental | Biological: Biostate |
Who may qualify
- Ages
- Up to 12 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Biostate1 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. | Biological | Biostate |
Primary outcome
Subjective assessment of Haemostatic efficacy
Time frame: Over minimum of 50 exposure days
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
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
Area under the concentration curve (AUC) of FVIII
Time frame: 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
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Volume of distribution at 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
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
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
Time the maximum concentration occurs (tmax) of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Total clearance of the drug from the body (CL=dose/AUC) of FVIII
Time frame: Samples taken prior and then 0.5, 4, 8, 24, and 48 h after the infusion on Day 1
Number of infusions per bleeding event
Time frame: 1 day
Number of infusions per month
Time frame: 1 month
Number of infusions per year
Time frame: 1 year
Dose (IU/kg) per bleeding event
Time frame: 1 day
Dose (IU/kg) per month
Time frame: 1 month
Dose (IU/kg) per year
Time frame: 1 year
6 secondary outcomes
Frequency of adverse events (AEs)
Time frame: 6 months
Severity of AEs per subject
Time frame: 6 months
Severity of AEs per infusion
Time frame: 6 months
Relatedness of AEs per subject
Time frame: 6 months
Relatedness of AEs per infusion
Time frame: 6 months
Development of FVIII inhibitors
Time frame: Samples taken at screening visit, on day 2, on months 1 and 3, and at final visit
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2017.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Program Director Clinical R&D study director
CSL Behring
8 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Biostate in people with Hemophilia A, run by CSL Behring at 8 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Aug 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Belarus, Ukraine, Georgia, Guatemala, Lebanon and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Subjective assessment of Haemostatic efficacy (time frame: Over minimum of 50 exposure days). The study lists 16 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Aug 2017.