Hereditary Factor VIII Deficiency Disease With InhibitorPhase 4BPATXASNCT01800435
A Comparison Study of Bypassing Agent Therapy With and Without Tranexamic Acid in Haemophilia A Patients With Inhibitor
A Phase 4 study testing aPCC, aPCC + TXA and rFVIIa, rFVIIa + TXA in people with Hereditary Factor VIII Deficiency Disease With Inhibitor, run by Oslo University Hospital at 1 site in Norway.
Trial summary
Activated prothrombin complex concentrate (aPCC) and recombinant activated factor VII (rFVIIa) are the only two drugs that are available to treat bleeds in haemophilia A patients with high titer inhibitors. Read more about this study
- Who is it for?
- Ages 18 to 65 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Hereditary Factor VIII Deficiency Disease With Inhibitor
- What is being tested?
- aPCC, aPCC + TXA and rFVIIa, rFVIIa + TXA
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteNorway
- Study dates
- Oct 2011to Oct 2012
- Phase
- Phase 4
- 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 18 to 65 years
- Men only
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Haemophilia patients with high titer inhibitors or high-responding inhibitors, aged between 18-65 and no history of aspirin or NSAID use within the last 14 days were eligible for the study.
Who cannot join
- Patients with renal failure, liver disease, infected with immune deficiency virus (HIV), platelet count <150x109/L, acquired haemophilia, ongoing bleeding, hypersensitivity to TXA or a history of arterial or venous thrombosis were excluded from the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Whole Blood Clot Stability and Thrombin Generating Capacity Following Treatment With Bypassing Agents (BPA) With and Without and Tranexamic Acid (TXA) in Haemophilia A Patients With inhibitor-an In-vivo Prospective Crossover Study
Activated prothrombin complex concentrate (aPCC) and recombinant activated factor VII (rFVIIa) are the only two drugs that are available to treat bleeds in haemophilia A patients with high titer inhibitors. However, management of bleeds in these patients can be challenging due to variation in response and lack of standardized methods to monitor the effect. We hypothesized that significant increase in whole blood clot stability could be achieved when tranexamic acid was given concomitantly with bypassing-agents while thrombin generation remains unaffected. In this prospective crossover study the effect of aPCC and rFVIIa with and without TXA on clot stability and thrombin generation capacity (ETP) were studied, using thromboelastography (ROTEM) and thrombin generation assay (TGA), respectively. In addition, the risk of thrombosis and disseminated intravascular coagulation (DIC) was assessed.
Read the detailed description
Patients receive the first day aPCC (75IU/kg) and aPCC in addition to TXA (20mg/kg orally) the second day. After a 14 days washout period they crosse over using rFVIIa (90 µg/kg) otherwise the same experimental setup. Blood sampling is performed at baseline, 15, 30, 60, 120, 180 and 240 minutes post-treatment.
- Being tested
- aPCC, aPCC + TXA, rFVIIa, rFVIIa + TXA
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
aPCC, aPCC + TXADrug
Also called Feiba i.v, Feiba i.v + Cyklokapron
Feiba 75 IU/kg i.v Feiba 75 IU/kg i.v +Cyklokapron 20 mg/kg p.o
rFVIIa, rFVIIa + TXADrug
Also called NovoSeven 90 µg/kg i.v, NovoSeven 90 µg/kg i.v + Cyklokapron 20 mg/kg
NovoSeven 90 µg/kg i.v NovoSeven 90 µg/kg i.v + Cyklokapron 20 mg/kg
| Study group | Role | What they receive |
|---|---|---|
| aPCC, aPCC + TXA | Active comparator | aPCC 75IU/kg i.v aPCC 75IU/kg i.v +TXA 20mg/kg Receives: Drug: aPCC, aPCC + TXA; Drug: rFVIIa, rFVIIa + TXA |
| rFVIIa, rFVIIa + TXA | Active comparator | rFVIIa 90 µg/kg i.v rFVIIa 90 µg/kg i.v + TXA 20 mg/kg Receives: Drug: aPCC, aPCC + TXA; Drug: rFVIIa, rFVIIa + TXA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clot stability and thrombin generation capacity following treatment with bypassing agents with and without tranexamic acid.
MCF (maximum clot formation/mm x 100-1), AUC (area under the elasticity curve AUC, mm• 100 s-1) were used as the primary outcome measures for evaluating clot stability and (ETP) the coagulable state.
Measured: 2 years
Secondary outcomes (1)
DIC or thrombosis events associated with different treatment regimens.
DIC parameters such as aPTT, PT/INR, platelet count, fibrinogen and D-dimer with the scoring system proposed by the International Society of Thrombosis and Haemostasis were used to monitor DIC in addition to common clinical signs associated with DIC were records. Symptoms or clinical signs of thrombosis such as dyspnea, chest pain, legg swelling or discomfort/pain were recorded.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2013 (estimated)
- Study startOct 2011
- Primary completionOct 2012
- Study completionOct 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Factor VIII Deficiency Disease With InhibitorStudies of the same condition, open and under way.
- More trials run by Oslo University HospitalThe 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 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oslo University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Pål Andre Holme, Oslo University Hospital
- Investigators
- PÅL A Holme, MD PhD, Principal investigator, Oslo University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01800435
- Sponsor's protocol ID
- EudraCTnr. 2010-022668-11
- Other IDs
- 2010-022668-11
- Acronym
- BPATXAS
- Status last verified
- Feb 2013
- First submitted
- 15 Feb 2013
- Last updated
- 1 Mar 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Mar 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Whole Blood Clot Stability and Thrombin Generating Capacity Following Treatment With Bypassing Agents (BPA) With and Without and Tranexamic Acid (TXA) in Haemophilia A Patients With inhibitor-an In-vivo Prospective Crossover Study
- ClinicalTrials.gov ID
- NCT01800435
- Acronym / sponsor ID
- BPATXAS / EudraCTnr. 2010-022668-11
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Oslo University HospitalUniversity, hospital or other
- Study start
- Oct 2011
- Primary completion
- Oct 2012 (actual)
- Study completion
- Oct 2012 (actual)
- First posted
- 27 Feb 2013 (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.
Activated prothrombin complex concentrate (aPCC) and recombinant activated factor VII (rFVIIa) are the only two drugs that are available to treat bleeds in haemophilia A patients with high titer inhibitors. However, management of bleeds in these patients can be challenging due to variation in response and lack of standardized methods to monitor the effect. We hypothesized that significant increase in whole blood clot stability could be achieved when tranexamic acid was given concomitantly with bypassing-agents while thrombin generation remains unaffected. In this prospective crossover study the effect of aPCC and rFVIIa with and without TXA on clot stability and thrombin generation capacity (ETP) were studied, using thromboelastography (ROTEM) and thrombin generation assay (TGA), respectively. In addition, the risk of thrombosis and disseminated intravascular coagulation (DIC) was assessed.
Detailed description
Patients receive the first day aPCC (75IU/kg) and aPCC in addition to TXA (20mg/kg orally) the second day. After a 14 days washout period they crosse over using rFVIIa (90 µg/kg) otherwise the same experimental setup. Blood sampling is performed at baseline, 15, 30, 60, 120, 180 and 240 minutes post-treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| aPCC, aPCC + TXA | Active comparator | Drug: aPCC, aPCC + TXA, Drug: rFVIIa, rFVIIa + TXAaPCC 75IU/kg i.v aPCC 75IU/kg i.v +TXA 20mg/kg |
| rFVIIa, rFVIIa + TXA | Active comparator | Drug: aPCC, aPCC + TXA, Drug: rFVIIa, rFVIIa + TXArFVIIa 90 µg/kg i.v rFVIIa 90 µg/kg i.v + TXA 20 mg/kg |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Haemophilia patients with high titer inhibitors or high-responding inhibitors, aged between 18-65 and no history of aspirin or NSAID use within the last 14 days were eligible for the study.
Key exclusion criteria (1 listed)
- Patients with renal failure, liver disease, infected with immune deficiency virus (HIV), platelet count <150x109/L, acquired haemophilia, ongoing bleeding, hypersensitivity to TXA or a history of arterial or venous thrombosis were excluded from the study.
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 |
|---|---|---|
| aPCC, aPCC + TXAAlso: Feiba i.v, Feiba i.v + CyklokapronFeiba 75 IU/kg i.v Feiba 75 IU/kg i.v +Cyklokapron 20 mg/kg p.o | Drug | aPCC, aPCC + TXA, rFVIIa, rFVIIa + TXA |
| rFVIIa, rFVIIa + TXAAlso: NovoSeven 90 µg/kg i.v, NovoSeven 90 µg/kg i.v + Cyklokapron 20 mg/kgNovoSeven 90 µg/kg i.v NovoSeven 90 µg/kg i.v + Cyklokapron 20 mg/kg | Drug | aPCC, aPCC + TXA, rFVIIa, rFVIIa + TXA |
Primary outcome
Clot stability and thrombin generation capacity following treatment with bypassing agents with and without tranexamic acid.
Time frame: 2 years
MCF (maximum clot formation/mm x 100-1), AUC (area under the elasticity curve AUC, mm• 100 s-1) were used as the primary outcome measures for evaluating clot stability and (ETP) the coagulable state.
1 secondary outcome
DIC or thrombosis events associated with different treatment regimens.
Time frame: 2 years
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 1 Mar 2013.
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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.
PÅL A Holme, MD PhD principal investigator
Oslo University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing aPCC, aPCC + TXA and rFVIIa, rFVIIa + TXA in people with Hereditary Factor VIII Deficiency Disease With Inhibitor, run by Oslo University Hospital at 1 site in Norway.
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 1 inclusion and 1 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 1 Mar 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Norway. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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?
Clot stability and thrombin generation capacity following treatment with bypassing agents with and without tranexamic acid. (time frame: 2 years). 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 1 Mar 2013.