Hemophilia APhase 2NCT00879541
Study of a pd vWF/FVIII, Biostate®, in Subjects With Haemophilia A
A Phase 2 study testing Biostate® [SP] and Biostate® [RP] in people with Hemophilia A, run by CSL Behring at 14 sites in 4 countries.
Trial summary
The aim of this study are to assess the efficacy of Biostate® [Study Product (SP)] in subjects with Haemophilia A compare the pharmacokinetics of Biostate® [SP] with the previously marketed product Biostate® (here referred to as Biostate® [Reference Product (RP)]). Read more about this study
- Who is it for?
- Ages 12 years and older, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Biostate® [SP] and Biostate® [RP]
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 81 participants
- Where
- 14 sitesin 4 countries
- Study dates
- Feb 2009to Oct 2010
- Phase
- Phase 2
- 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 12 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosed with Haemophilia A with ≤ 1% Factor VIII (FVIII) levels in the absence of factor replacement
- 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) within 10 years prior to Day 1 documented in the medical notes
- At least 150 days of prior exposure to a FVIII replacement product
- Written informed consent given
- Exclusion Criteria (for participation in the pharmacokinetic (PK) component):
- Active bleeding
- Body weight > 100 kg
Who cannot join
- Receipt of an infusion of any FVIII product, cryoprecipitate, whole blood, plasma, or desmopressin acetate (DDAVP) in the 4 days prior to Day 1
- Known history of FVIII inhibitors, or FVIII inhibitor level > 0.6 Bethesda Units (BU) at screening
- Receipt of aspirin or other Non-Steroidal Anti-Inflammatory Drugs (NSAIDs) within 7 days of administration of study product.
- CD4 lymphocytes < 200/µL. Subjects wo are HIV-1 positive may be considered for the study if viral load ≤ 200 particles/µL at screening and all other eligibility criteria are met.
- Impaired liver function ie. bilirubin >1.5 x upper limit of normal (ULN) and/or AST/ALT > 2.5 x ULN at screening.
- Acute or chronic medical condition, other than haemophilia A, which may, in the opinion of the Investigator, affect the conduct of the study
Show all 11Show fewer
- von Willebrand Disease (VWD) with Von Willebrand Factor:Ristocetin Cofactor (vWF:RCo) level < 50 IU/dL at screening
- Evidence or a history (within the previous 12 months) of abuse of any drug substance, licit or illicit
- Known or suspected hypersensitivity or previous evidence of severe side effects to Biostate®, FVIII concentrates or human albumin
- Participation in a clinical study or use of an investigational compound (e.g. a new chemical entity not approved for clinical use) in the 3 months preceding the first day of study drug administration, or plans to enter such a study during the study period
- Not willing and/or not able to comply with study requirements
Trial locations and contacts
14 sites in 4 countries.
14 sites in 4 countries
Poland7 sites
Study Site
Bialystok
Study Site
Gdansk
Study Site
Krakow
Study Site
Lublin
Study Site
Poznan
Study Site
Warsaw
Study Site
Wroclaw
About this study
Official title: A Phase II, Multicentre, Double-blinded, Randomised, Cross-over Study to Evaluate Efficacy, Safety and Pharmacokinetics of Biostate® in Subjects With Haemophilia A.
The aim of this study are to
- assess the efficacy of Biostate® [Study Product (SP)] in subjects with Haemophilia A
- compare the pharmacokinetics of Biostate® [SP] with the previously marketed product Biostate® (here referred to as Biostate® [Reference Product (RP)]).
Read more
This study is divided into 3 parts:
Part 1: Cross-over pharmacokinetic (PK) component. PK subjects will be randomised to determine the order in which they receive the two study products. This part of the study is double-blinded.
Part 2: Efficacy component. All subjects will receive Biostate® [SP] as required to manage their haemophilia condition for an estimated period of 6 months (or minimum of 50 exposure days) to assess efficacy and safety of the product. This part of the study is open-label.
Part 3: Repeat pharmacokinetic assessment. Subjects who participated in Part 1 (PK component) will undergo a repeat PK assessment on Day 180 following administration of Biostate® [SP].
- Conditions
- Hemophilia A
- Being tested
- Biostate® [SP], Biostate® [RP]
- Keywords
- Hemophilia A
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biostate® [SP]Biological
Also called Human Coagulation Factor VIII / von Willebrand Factor
Single bolus intravenous dose of 50 IU/kg
Biostate® [SP]Biological
Also called Human Coagulation Factor VIII / von Willebrand Factor
The dose is dependent on the reason for use and may consist of repeated bolus doses as required to manage haemophilia condition.
Biostate® [RP]Biological
Also called Biostate®, Human Coagulation Factor VIII / von Willebrand Factor
Single bolus intravenous dose of 50 IU/kg.
| Study group | Role | What they receive |
|---|---|---|
| PK Biostate® [SP] | Other | Part 1: PK subjects are randomized to receive Biostate® [SP] either on Day 1 or Day 8.
Part 3: All PK subjects receive Biostate® [SP] on Day 180. Receives: Biological: Biostate® [SP] |
| PK Biostate® [RP] | Other | Part 1: PK subjects are randomized to receive Biostate® [RP] either on Day 1 or Day 8. Receives: Biological: Biostate® [RP] |
| Efficacy | Experimental | Part 2: This arm includes all subjects during the efficacy component of the study. Receives: Biological: Biostate® [SP] |
What is being measured
The primary outcome is the main question the study is designed to answer.
Haemostatic efficacy
Measured: Monthly, until final study visit
Number of treatments/units required to resolve any bleeding event
Measured: From Day 1 until final study visit
FVIII concentrate usage (number of infusions, IU/kg per event, per month, and per year)
Measured: From Day 1 until final study visit
Assessment of blood loss during any surgical procedure
Measured: From Day 1 until final study visit
Pharmacokinetics of FVIII activity
Measured: Up to 48 hours following infusions (Part 1 and Part 3 only)
Secondary outcomes (2)
The nature, frequency and incidence of adverse events
Measured: From Day 1 until final study visit
Development of FVIII inhibitors
Measured: From Day 1 until final study visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Apr 2009 (estimated)
- Study startFeb 2009
- Primary completionOct 2010
- Study completionOct 2010
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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 81 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Parexel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00879541
- Sponsor's protocol ID
- CSLCT-BIO-07-47
- Other IDs
- 1472
- Status last verified
- Feb 2011
- First submitted
- 9 Apr 2009
- Last updated
- 11 Feb 2011
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II, Multicentre, Double-blinded, Randomised, Cross-over Study to Evaluate Efficacy, Safety and Pharmacokinetics of Biostate® in Subjects With Haemophilia A.
- ClinicalTrials.gov ID
- NCT00879541
- Acronym / sponsor ID
- CSLCT-BIO-07-47
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 81 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Collaborators
- Parexel
- Study start
- Feb 2009
- Primary completion
- Oct 2010 (actual)
- Study completion
- Oct 2010 (actual)
- First posted
- 10 Apr 2009 (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 aim of this study are to
- assess the efficacy of Biostate® [Study Product (SP)] in subjects with Haemophilia A
- compare the pharmacokinetics of Biostate® [SP] with the previously marketed product Biostate® (here referred to as Biostate® [Reference Product (RP)]).
This study is divided into 3 parts:
Part 1: Cross-over pharmacokinetic (PK) component. PK subjects will be randomised to determine the order in which they receive the two study products. This part of the study is double-blinded.
Part 2: Efficacy component. All subjects will receive Biostate® [SP] as required to manage their haemophilia condition for an estimated period of 6 months (or minimum of 50 exposure days) to assess efficacy and safety of the product. This part of the study is open-label.
Part 3: Repeat pharmacokinetic assessment. Subjects who participated in Part 1 (PK component) will undergo a repeat PK assessment on Day 180 following administration of Biostate® [SP].
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| PK Biostate® [SP] | Other | Biological: Biostate® [SP]Part 1: PK subjects are randomized to receive Biostate® [SP] either on Day 1 or Day 8. Part 3: All PK subjects receive Biostate® [SP] on Day 180. |
| PK Biostate® [RP] | Other | Biological: Biostate® [RP]Part 1: PK subjects are randomized to receive Biostate® [RP] either on Day 1 or Day 8. |
| Efficacy | Experimental | Biological: Biostate® [SP]Part 2: This arm includes all subjects during the efficacy component of the study. |
Who may qualify
- Ages
- 12 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Diagnosed with Haemophilia A with ≤ 1% Factor VIII (FVIII) levels in the absence of factor replacement
- 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) within 10 years prior to Day 1 documented in the medical notes
- At least 150 days of prior exposure to a FVIII replacement product
- Written informed consent given
- Active bleeding
Key exclusion criteria (11 listed)
- Receipt of an infusion of any FVIII product, cryoprecipitate, whole blood, plasma, or desmopressin acetate (DDAVP) in the 4 days prior to Day 1
- Known history of FVIII inhibitors, or FVIII inhibitor level > 0.6 Bethesda Units (BU) at screening
- Receipt of aspirin or other Non-Steroidal Anti-Inflammatory Drugs (NSAIDs) within 7 days of administration of study product.
- CD4 lymphocytes < 200/µL. Subjects wo are HIV-1 positive may be considered for the study if viral load ≤ 200 particles/µL at screening and all other eligibility criteria are met.
- Impaired liver function ie. bilirubin >1.5 x upper limit of normal (ULN) and/or AST/ALT > 2.5 x ULN 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 |
|---|---|---|
| Biostate® [SP]Also: Human Coagulation Factor VIII / von Willebrand FactorSingle bolus intravenous dose of 50 IU/kg | Biological | PK Biostate® [SP] |
| Biostate® [SP]Also: Human Coagulation Factor VIII / von Willebrand FactorThe dose is dependent on the reason for use and may consist of repeated bolus doses as required to manage haemophilia condition. | Biological | Efficacy |
| Biostate® [RP]Also: Biostate®, Human Coagulation Factor VIII / von Willebrand FactorSingle bolus intravenous dose of 50 IU/kg. | Biological | PK Biostate® [RP] |
Primary outcome
Haemostatic efficacy
Time frame: Monthly, until final study visit
Number of treatments/units required to resolve any bleeding event
Time frame: From Day 1 until final study visit
FVIII concentrate usage (number of infusions, IU/kg per event, per month, and per year)
Time frame: From Day 1 until final study visit
Assessment of blood loss during any surgical procedure
Time frame: From Day 1 until final study visit
Pharmacokinetics of FVIII activity
Time frame: Up to 48 hours following infusions (Part 1 and Part 3 only)
2 secondary outcomes
The nature, frequency and incidence of adverse events
Time frame: From Day 1 until final study visit
Development of FVIII inhibitors
Time frame: From Day 1 until final study 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 11 Feb 2011.
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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.
The record lists no contact. Use the official record to reach the sponsor.
14 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Biostate® [SP] and Biostate® [RP] in people with Hemophilia A, run by CSL Behring at 14 sites in 4 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 7 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 11 Feb 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in Poland, Bulgaria, Russia, North Macedonia. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Haemostatic efficacy (time frame: Monthly, until final study visit). The study lists 5 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 11 Feb 2011.