Hemophilia APhase 3NCT01445197
Study of Biostate for Treatment of Children With Hemophilia A Complicated by Antibody Development
A Phase 3 study testing Biostate in people with Hemophilia A, run by CSL Behring at 7 sites in 5 countries.
Trial summary
This is a clinical study to investigate how well Biostate works in treatment of male patients below the age of 12 years who have a clotting factor deficiency that is aggravated by the development of antibodies. Read more about this study
- Who is it for?
- Ages 28 days to 11 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Biostate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 7 sitesin 5 countries
- Study dates
- Dec 2012to Dec 2013
- 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 28 days to 11 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Male subjects diagnosed with haemophilia A (≤ 2% FVIII level in the absence of factor replacement, according to their medical history).
- Age 28 days to <12 years.
- Subject is eligible for immune tolerance induction (ITI) therapy
Who cannot join
- The subject has received ITI previously.
- Subjects with a historical peak inhibitor titre of ≥ 200 BU/mL.
- Concomitant treatment with drugs with immunosuppressive side effects (eg, systemic corticosteroids), azathioprine, cyclophosphamide, high dose immunoglobulin or the use of a protein A column or plasmapheresis and interferons.
- High risk of cardiovascular, cerebrovascular, or other thromboembolic events (excluding catheter thrombosis) as judged by the investigator.
- Subjects who are human immunodeficiency virus (HIV)-1 or HIV-2 positive (as reported in the medical records or determined at screening).
Trial locations and contacts
7 sites in 5 countries.
7 sites in 5 countries
About this study
Official title: A Multicentre, Interventional, Non-randomized, Open-label, Single-group Phase III Study to Evaluate Plasma-Derived Antihaemophilic Factor/Von Willebrand Factor Concentrate (Biostate®) for Immune Tolerance Induction in Male Paediatric Subjects With Haemophilia A (≤ 2%) Who Have Developed High-titre Antibodies to Factor VIII (Factor VIII Inhibitors)
This is a clinical study to investigate how well Biostate works in treatment of male patients below the age of 12 years who have a clotting factor deficiency that is aggravated by the development of antibodies. The antibodies are directed against the clotting factor that is given for replacement therapy and usually make therapy unsuccessful. The treatment used in this study is called immune tolerance therapy.
- Conditions
- Hemophilia A
- Being tested
- Biostate
- 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
200 IU/kg administered daily
| 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.
Response to immune tolerance induction (ITI) treatment
Number of subjects who achieve complete, partial, and no response (ITI failure) to treatment.
Measured: 30 months
Secondary outcomes (8)
FVIII inhibitor titre
Measured: Up to 65 months
Time to complete response (success)
Measured: Up to 65 months
Time to inhibitor titer <0.6 BU/mL for the first time
Measured: Up to 65 months
Thromboembolic complications
Number of patients with clinical symptoms or increased markers of coagulation activation
Measured: Up to 65 months
Frequency of bleeding events
Measured: Up to 65 months
Number of bleeding events per patient
Measured: Up to 65 months
Severity of bleeding events per patient
Measured: Up to 65 months
Catheter-related complications
Number of line infections
Measured: Up to 65 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2011 (estimated)
- Study startDec 2012
- Primary completionDec 2013
- Study completionDec 2013
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
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Carmen Escuriola-Ettingshausen, Principal investigator, Haemophilia Centre Rhein Main, Frankfurt - Mörfelden
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01445197
- Sponsor's protocol ID
- CSLCT-BIO-10-67
- Other IDs
- 2010-020113-85
- Status last verified
- Oct 2017
- First submitted
- 30 Sep 2011
- Last updated
- 3 Oct 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Oct 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicentre, Interventional, Non-randomized, Open-label, Single-group Phase III Study to Evaluate Plasma-Derived Antihaemophilic Factor/Von Willebrand Factor Concentrate (Biostate®) for Immune Tolerance Induction in Male Paediatric Subjects With Haemophilia A (≤ 2%) Who Have Developed High-titre Antibodies to Factor VIII (Factor VIII Inhibitors)
- ClinicalTrials.gov ID
- NCT01445197
- Acronym / sponsor ID
- CSLCT-BIO-10-67
- Conditions
- Hemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 1 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- Dec 2012
- Primary completion
- Dec 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 3 Oct 2011 (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.
This is a clinical study to investigate how well Biostate works in treatment of male patients below the age of 12 years who have a clotting factor deficiency that is aggravated by the development of antibodies. The antibodies are directed against the clotting factor that is given for replacement therapy and usually make therapy unsuccessful. The treatment used in this study is called immune tolerance therapy.
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
- 28 days to 11 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Male subjects diagnosed with haemophilia A (≤ 2% FVIII level in the absence of factor replacement, according to their medical history).
- Age 28 days to <12 years.
- Subject is eligible for immune tolerance induction (ITI) therapy
Key exclusion criteria (5 listed)
- The subject has received ITI previously.
- Subjects with a historical peak inhibitor titre of ≥ 200 BU/mL.
- Concomitant treatment with drugs with immunosuppressive side effects (eg, systemic corticosteroids), azathioprine, cyclophosphamide, high dose immunoglobulin or the use of a protein A column or plasmapheresis and interferons.
- High risk of cardiovascular, cerebrovascular, or other thromboembolic events (excluding catheter thrombosis) as judged by the investigator.
- Subjects who are human immunodeficiency virus (HIV)-1 or HIV-2 positive (as reported in the medical records or determined 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 |
|---|---|---|
| Biostate200 IU/kg administered daily | Biological | Biostate |
Primary outcome
Response to immune tolerance induction (ITI) treatment
Time frame: 30 months
Number of subjects who achieve complete, partial, and no response (ITI failure) to treatment.
8 secondary outcomes
FVIII inhibitor titre
Time frame: Up to 65 months
Time to complete response (success)
Time frame: Up to 65 months
Time to inhibitor titer <0.6 BU/mL for the first time
Time frame: Up to 65 months
Thromboembolic complications
Time frame: Up to 65 months
Frequency of bleeding events
Time frame: Up to 65 months
Number of bleeding events per patient
Time frame: Up to 65 months
Severity of bleeding events per patient
Time frame: Up to 65 months
Catheter-related complications
Time frame: Up to 65 months
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Oct 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.
Carmen Escuriola-Ettingshausen principal investigator
Haemophilia Centre Rhein Main, Frankfurt - Mörfelden
7 sites in 5 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 7 sites in 5 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 3 inclusion and 5 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: stopped early, as of the sponsor's last update on 3 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Germany, Greece, Austria, Italy, Russia. 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 was stopped early. Results are not available in the source record.
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?
Response to immune tolerance induction (ITI) treatment (time frame: 30 months). 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 3 Oct 2017.