Hemophilia APhase 4NCT00284193
Combination Therapy of Low Doses of rFVIIa and FEIBA for Severe Hemophilia A Patients With an Inhibitor to Factor VIII
A Phase 4 study testing rFVIIa-FEIBA therapy for hemophilia A inhibitors and FEIBA- Activated Prothrombin Complexes in people with Hemophilia A, run by Sheba Medical Center.
Trial summary
Patients with severe hemophilia and inhibitors can be treated effectively by Activated Prothrombin Complex Concentrates (APCC, eg. FEIBA) or High dose recombinant factor VIIa (rFVIIa). Read more about this study
- Who is it for?
- Ages 16 to 65 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- rFVIIa-FEIBA therapy for hemophilia A inhibitors and FEIBA- Activated Prothrombin Complexes
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- No sites listed yet
- Study dates
- Jan 2005to Nov 2009
- 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 16 to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Hemophilia patients with inhibitors
- Patients signing informed consent
Who cannot join
- Patients under 16 or above 65
- Patients with allergic reaction or adverse events in previous use the concentrates
- Patients with high risk of thrombosis
About this study
Patients with severe hemophilia and inhibitors can be treated effectively by Activated Prothrombin Complex Concentrates (APCC, eg. FEIBA) or High dose recombinant factor VIIa (rFVIIa). Rarely, such patients develop refractoriness to these products for whom therapy with sequential FEIBA and rFVIIa has been recently suggested.
The impetus for the present report was a hemophilia A patient with high titer inhibitor (1300BU) who had life threatening hematuria that was resistant to repeated doses of 400µg/kg rFVIIa up to a cumulative dose of 1200 µg/kg given over 6-9 hours.
Thrombin generation (TG) tested in vitro was consistent with resistance to high concentrations of rFVIIa but yielded good response to combinations of low doses of rFVIIa+FEIBA. In a desperate attempt to control the bleeding, concomitant therapy of 25 U/kg FEIBA and 40µg/kg rFVIIa was infused and resulted in arrest of bleeding within minutes. Over a span of about one year the patient has been successfully treated by this combination for more than 200 bleeding episodes in muscles and joints.
Read the detailed description
Inhibitor patients with HR inhibitors were eligible for study enrollment. After consent blood was drawn and ex- vivo spiked with rFVIIa/FEIBA and combinations, assayed by thrombin generation tests.
The combination yielding sufficient hemostasis was depicted for patients' therapy of future bleeding episodes.
Following actual therapy hemostasis and safety parameters were monitored.
- Conditions
- Hemophilia A
- Being tested
- rFVIIa-FEIBA therapy for hemophilia A inhibitors, FEIBA- Activated Prothrombin Complexes
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rFVIIa-FEIBA therapy for hemophilia A inhibitorsDrug
Also called NOVOSEVEN, APCC
DOses tailored per ex vivo spiking thrombin generation
FEIBA- Activated Prothrombin ComplexesDrug
| Study group | Role | What they receive |
|---|---|---|
| feiba-VIIa, hemophilia A-inhibitor therapy | Experimental | COMBINED PATIENT- TAILORED THERAPY WITH CONCOMITANT ADMINISTRATION OF BOTH DRUGS , FOLLOWING EX VIVO THROMBIN GENERATION PREDICTING ASSAYS Receives: Drug: rFVIIa-FEIBA therapy for hemophilia A inhibitors; Drug: FEIBA- Activated Prothrombin Complexes |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hemostasis achieved post therapy
Following acute bleeding therapy hemostasis was defined as good, partial or non-satisfactory
Measured: 6-24 hours
Safety
Following therapy presence of any adverse events, especially thromboembolic complications was assessed
Measured: 0-24 HOURS
Secondary outcomes (1)
Time to Hemostasis
Following therapy patients documented time to "GOOD" response
Measured: 0-24 HOURS
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2006 (estimated)
- Study startJan 2005
- Primary completionNov 2008
- Study completionNov 2009
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 Sheba Medical CenterThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sheba Medical Center
- Sponsor type
- Other government
- Responsible party
- Principal investigator, Prof. Uriel Martinowitz, Sheba Medical Center
- Investigators
- Uri Martinowitz, MD, Principal investigator, Sheba Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00284193
- Sponsor's protocol ID
- SHEBA-05-3768-UM-CTIL
- Status last verified
- Jul 2012
- First submitted
- 17 Jan 2006
- Last updated
- 27 Jul 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Combination Therapy of Low Doses of rFVIIa and FEIBA for Severe Hemophilia A Patients With an Inhibitor to Factor VIII
- ClinicalTrials.gov ID
- NCT00284193
- Acronym / sponsor ID
- SHEBA-05-3768-UM-CTIL
- Conditions
- Hemophilia A
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Sheba Medical CenterOther government
- Study start
- Jan 2005
- Primary completion
- Nov 2008 (actual)
- Study completion
- Nov 2009 (actual)
- First posted
- 31 Jan 2006 (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.
Patients with severe hemophilia and inhibitors can be treated effectively by Activated Prothrombin Complex Concentrates (APCC, eg. FEIBA) or High dose recombinant factor VIIa (rFVIIa). Rarely, such patients develop refractoriness to these products for whom therapy with sequential FEIBA and rFVIIa has been recently suggested.
The impetus for the present report was a hemophilia A patient with high titer inhibitor (1300BU) who had life threatening hematuria that was resistant to repeated doses of 400µg/kg rFVIIa up to a cumulative dose of 1200 µg/kg given over 6-9 hours.
Thrombin generation (TG) tested in vitro was consistent with resistance to high concentrations of rFVIIa but yielded good response to combinations of low doses of rFVIIa+FEIBA. In a desperate attempt to control the bleeding, concomitant therapy of 25 U/kg FEIBA and 40µg/kg rFVIIa was infused and resulted in arrest of bleeding within minutes. Over a span of about one year the patient has been successfully treated by this combination for more than 200 bleeding episodes in muscles and joints.
Detailed description
Inhibitor patients with HR inhibitors were eligible for study enrollment. After consent blood was drawn and ex- vivo spiked with rFVIIa/FEIBA and combinations, assayed by thrombin generation tests.
The combination yielding sufficient hemostasis was depicted for patients' therapy of future bleeding episodes.
Following actual therapy hemostasis and safety parameters were monitored.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| feiba-VIIa, hemophilia A-inhibitor therapy | Experimental | Drug: rFVIIa-FEIBA therapy for hemophilia A inhibitors, Drug: FEIBA- Activated Prothrombin ComplexesCOMBINED PATIENT- TAILORED THERAPY WITH CONCOMITANT ADMINISTRATION OF BOTH DRUGS , FOLLOWING EX VIVO THROMBIN GENERATION PREDICTING ASSAYS |
Who may qualify
- Ages
- 16 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Hemophilia patients with inhibitors
- Patients signing informed consent
Key exclusion criteria (3 listed)
- Patients under 16 or above 65
- Patients with allergic reaction or adverse events in previous use the concentrates
- Patients with high risk of thrombosis
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 |
|---|---|---|
| rFVIIa-FEIBA therapy for hemophilia A inhibitorsAlso: NOVOSEVEN, APCCDOses tailored per ex vivo spiking thrombin generation | Drug | feiba-VIIa, hemophilia A-inhibitor therapy |
| FEIBA- Activated Prothrombin Complexes | Drug | feiba-VIIa, hemophilia A-inhibitor therapy |
Primary outcome
Hemostasis achieved post therapy
Time frame: 6-24 hours
Following acute bleeding therapy hemostasis was defined as good, partial or non-satisfactory
Safety
Time frame: 0-24 HOURS
Following therapy presence of any adverse events, especially thromboembolic complications was assessed
1 secondary outcome
Time to Hemostasis
Time frame: 0-24 HOURS
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 27 Jul 2012.
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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.
Uri Martinowitz, MD principal investigator
Sheba Medical Center
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing rFVIIa-FEIBA therapy for hemophilia A inhibitors and FEIBA- Activated Prothrombin Complexes in people with Hemophilia A, run by Sheba Medical Center.
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 2 inclusion and 3 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 27 Jul 2012. Status can change between updates, so confirm with the study contact before making plans.
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?
Hemostasis achieved post therapy (time frame: 6-24 hours). The study lists 2 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 27 Jul 2012.