Hemophilia APhase 3NCT01757405
Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen
A Phase 3 study testing Recombinant Factor VIIa BI (rFVIIa BI) in people with Hemophilia A and Hemophilia B, run by Baxalta now part of Shire at 16 sites in 9 countries.
Trial summary
The purpose of the study is to determine the efficacy and safety of rFVIIa BI as part of a six-month on-demand treatment regimen in hemophilia A or B subjects with inhibitors. Read more about this study
- Who is it for?
- Ages 12 to 65 years, men only
- Conditions the study is for
- Hemophilia A, Hemophilia B
- What is being tested?
- Recombinant Factor VIIa BI (rFVIIa BI)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 16 sitesin 9 countries
- Study dates
- 20 Feb 2013to 11 Nov 2014
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Main Inclusion Criteria:
- Participant is male with hemophilia A or B with inhibitors, with a high titer (≥5 Bethesda unit (BU)) or a historical high anamnestic response.
- Participant is 12 to 65 years old at the time of screening.
- Participant is currently using or has used bypassing agents for treatment of bleeding episodes.
- Participant has an annualized bleed rate of 5 or more bleeding episodes per year on average over the 2 years prior to the Screening visit.
- Participant has a Karnofsky Performance Score ≥60.
- Participant is hepatitis C virus negative (HCV-) either by antibody testing or polymerase chain reaction (PCR); or hepatitis C virus positive (HCV+) with stable hepatic disease.
- Participant is human immunodeficiency virus negative (HIV-) or HIV+ with stable disease, CD4+ count ≥200 cells/mm^3 at screening.
- Participant is willing and able to comply with the requirements of the protocol.
Main Exclusion Criteria:
- Participant is not willing to go on an on-demand treatment scheme.
- Participant is positive for a FVII inhibitor at screening.
- Participant has clinically symptomatic liver disease.
- Participant has a platelet count <100,000/µL.
- The use of α-interferon with or without ribavirin is planned for an HCV-infected participant or the use of a protease inhibitor is planned for an HIV-infected participant.
- Participants currently taking any of these medications for ≥30 days are eligible.
- Participant has a known hypersensitivity to rFVIIa, hamster or murine proteins, or Tween 80.
- Participant has a known history of being non-responsive to rFVIIa treatment of bleeding episodes.
- Participant has a prior history of thromboembolic event or diagnosis of other diseases that may increase the participant's risk of thromboembolic complications.
- Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
- Participant is a family member or employee of the investigator.
- Participant is scheduled for surgery during the study period.
Trial locations and contacts
16 sites in 9 countries.
16 sites in 9 countries
Russia3 sites
Kirov Hematology and Blood Transfusion Research Institute under the Federal Medical and Biological Agency of Russia
Kirov, 610027
Hematology Research Center under RAMS (State Institution), Department of Reconstructive Orthopedic Surgery for Hemophilia Patients
Moscow, 125167
St. Petersburg City Healthcare Institution Municipal Policlinic # 37
Saint Petersburg, 195213
Ukraine3 sites
V.K. Gusak Institute of Urgent and Reconstructive Surgery within the Ukrainian National Academy of Medical Sciences, Hematology Department
Donetsk, 83045
Kyiv City Clinical Hospital #9, City Scientific-Practical Center for Diagnostics and Treatment of Patients with Hemostatic Pathlogies
Kiev, 79044
State Institution "Institute of Blood Pathology and Transfusion Medicine within the Ukrainian National Academy of Medical Sciences", Hematology Department
Lviv
Japan2 sites
Nara Medical University Hospital
Nara, 6348522
Tokyo Medical University Hospital
Tokyo, 1600023
Poland2 sites
Kracow Medical Center, LLC
Krakow, 31-501
Institute of Haematology and Transfusion Medicine, Clinic of Haemostatic Disorders and Internal Diseases
Warsaw, 02-776
Spain2 sites
Hospital Teresa Herrera Materno Infantil del C.H.U.Carretera del Pasajes/nlaboratorio de hematología
A Coruña, 15006
University Hospital Virgen del Rocio
Seville, 41013
United States1 site
Health Point Medical Group "St Joseph's Children's Hospital"
Tampa, Florida, 33607
Romania1 site
Louis Turcanu Emergency Clinical Children´s Hospital
Timișoara, 300011
Serbia1 site
Clinic for Hematology of the Clinical Center of Serbia
Belgrade, 11000
About this study
Official title: A PHASE 3, PROSPECTIVE, OPEN-LABEL, RANDOMIZED STUDY TO EVALUATE SAFETY AND EFFICACY OF RECOMBINANT ACTIVATED FVII BI (rFVIIa BI) IN THE TREATMENT OF ACUTE BLEEDING EPISODES PER AN ON-DEMAND REGIMEN IN PATIENTS WITH HEMOPHILIA A OR B WITH INHIBITORS
The purpose of the study is to determine the efficacy and safety of rFVIIa BI as part of a six-month on-demand treatment regimen in hemophilia A or B subjects with inhibitors.
- Conditions
- Hemophilia A, Hemophilia B
- Being tested
- Recombinant Factor VIIa BI (rFVIIa BI)
- Keywords
- with Factor VIII (FVIII) or Factor IX (FIX) inhibitors
- Medical subject headings
- Hemophilia A, Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Factor VIIa BI (rFVIIa BI)Biological
Administered approximately every 3 hours as an intravenous bolus injection on-demand
Recombinant Factor VIIa BI (rFVIIa BI)Biological
Administered as a single intravenous bolus injection on-demand
| Study group | Role | What they receive |
|---|---|---|
| ≤ 3 doses of 90 µg/kg rFVIIa BI | Experimental | Not described Receives: Biological: Recombinant Factor VIIa BI (rFVIIa BI) |
| One dose of 270 µg/kg rFVIIa BI | Experimental | Not described Receives: Biological: Recombinant Factor VIIa BI (rFVIIa BI) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Bleeding Episode With "Treatment Success"
No additional hemostatic product required within 12 hours of first dose other than the prescribed dosing regimen.
Measured: within 12 hours of first dose
Secondary outcomes (5)
Treatment Response for Each Bleeding Episode
Participants rated the treatment of each bleeding episode. If treatment occurred under direct supervision of treating physician, the physician rated the response. Ratings based on a 4 point scale; EXCELLENT - full relief of pain and cessation of objective signs of bleeding (swelling, tenderness, decrease in range of motion [for muscle bleeds]) within 9 hours of treatment initiation. No additional infusion required to control bleeding, other than prescribed dosing regimen. GOOD - Substantial relief of pain and/or cessation of objective signs of bleeding within 9 hours of treatment initiation. No additional infusion required to control bleeding, other than prescribed dosing regimen. MODERATE - slight relief of pain and slight improvement of signs of bleeding within 9 hours of treatment initiation. Requires additional infusion beyond treatment regimen. NONE - No improvement or condition worsens. SUCCESSFUL = EXCELLENT or GOOD.
Measured: within 24 hours of infusion
Percentage of Clinical Responders (Sustained Bleeding Control) for All Acute Bleeding Episodes
Clinical responders defined as sustained bleeding control, (no additional hemostatic medication including rFVIIa BI required between 12 and 24 hours after first infusion of the successfully treated bleeding episode).
Measured: 24 hours post infusion
Safety and Tolerability of Treatment Regimens by Clinical Assessment of Percentage of Participants With Adverse Events (AEs)
Safety was determined by the number of AEs (both serious AEs [SAEs] and non-serious AEs [nsAE]). Tolerability was determined by the number of AEs related to rFVIIa BI (both SAEs and nsAEs) as determined by causality assessment of the AEs by the investigator. An AE was deemed Related if the investigator judged the AE to be "possibly related" or "probably related" to rFVIIa BI. The percentage of participants with AEs were presented by seriousness (SAE, nsAE), severity (Mild, Moderate or Severe) and causality (Related or Not Related to rFVIIa BI).
Measured: 6 months (throughout study period)
Safety and Tolerability of Treatment Regimens by Clinical Assessment of Adverse Events (AEs)
Safety was determined by the number of AEs (both serious AEs [SAEs] and non-serious AEs [nsAE]). Tolerability was determined by the number of AEs related to rFVIIa BI (both SAEs and nsAEs) as determined by causality assessment of the AEs by the investigator. An AE was deemed Related if the investigator judges the AE to be "possibly related" or "probably related" to rFVIIa BI. The percentage of AEs were presented by seriousness (SAE, nsAE), severity (Mild, Moderate or Severe) and causality (Related or Not Related [to rFVIIa BI]).
Measured: 6 months (throughout study period)
Percentage of Participants With Inhibitor Development to FVII
Development of rFVII inhibitors or FVIIa binding antibodies during the study.
Measured: 6 months (throughout study period)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Dec 2012 (estimated)
- Study start20 Feb 2013
- Primary completion11 Nov 2014
- Study completion11 Nov 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- 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
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Shire
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01757405
- Sponsor's protocol ID
- 021101
- Other IDs
- 2011-006294-26
- Status last verified
- Apr 2021
- First submitted
- 21 Dec 2012
- Last updated
- 11 May 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A PHASE 3, PROSPECTIVE, OPEN-LABEL, RANDOMIZED STUDY TO EVALUATE SAFETY AND EFFICACY OF RECOMBINANT ACTIVATED FVII BI (rFVIIa BI) IN THE TREATMENT OF ACUTE BLEEDING EPISODES PER AN ON-DEMAND REGIMEN IN PATIENTS WITH HEMOPHILIA A OR B WITH INHIBITORS
- ClinicalTrials.gov ID
- NCT01757405
- Acronym / sponsor ID
- 021101
- Conditions
- Hemophilia A, Hemophilia B
- Keywords
- with Factor VIII (FVIII) or Factor IX (FIX) inhibitors
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 20 Feb 2013 (actual)
- Primary completion
- 11 Nov 2014 (actual)
- Study completion
- 11 Nov 2014 (actual)
- First posted
- 28 Dec 2012 (estimated)
- Results
- Posted 25 Oct 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to determine the efficacy and safety of rFVIIa BI as part of a six-month on-demand treatment regimen in hemophilia A or B subjects with inhibitors.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ≤ 3 doses of 90 µg/kg rFVIIa BI | Experimental | Biological: Recombinant Factor VIIa BI (rFVIIa BI) |
| One dose of 270 µg/kg rFVIIa BI | Experimental | Biological: Recombinant Factor VIIa BI (rFVIIa BI) |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| Recombinant Factor VIIa BI (rFVIIa BI)Administered approximately every 3 hours as an intravenous bolus injection on-demand | Biological | ≤ 3 doses of 90 µg/kg rFVIIa BI |
| Recombinant Factor VIIa BI (rFVIIa BI)Administered as a single intravenous bolus injection on-demand | Biological | One dose of 270 µg/kg rFVIIa BI |
Primary outcome
Percentage of Bleeding Episode With "Treatment Success"
Time frame: within 12 hours of first dose
No additional hemostatic product required within 12 hours of first dose other than the prescribed dosing regimen.
5 secondary outcomes
Treatment Response for Each Bleeding Episode
Time frame: within 24 hours of infusion
Percentage of Clinical Responders (Sustained Bleeding Control) for All Acute Bleeding Episodes
Time frame: 24 hours post infusion
Safety and Tolerability of Treatment Regimens by Clinical Assessment of Percentage of Participants With Adverse Events (AEs)
Time frame: 6 months (throughout study period)
Safety and Tolerability of Treatment Regimens by Clinical Assessment of Adverse Events (AEs)
Time frame: 6 months (throughout study period)
Percentage of Participants With Inhibitor Development to FVII
Time frame: 6 months (throughout study period)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 May 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Study Director study director
Shire
16 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Recombinant Factor VIIa BI (rFVIIa BI) in people with Hemophilia A and Hemophilia B, run by Baxalta now part of Shire at 16 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in Russia, Ukraine, Japan, Poland, Spain and 4 more countries. 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?
Yes. Results are posted in the registry (first posted 25 Oct 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percentage of Bleeding Episode With "Treatment Success" (time frame: within 12 hours of first dose). 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 May 2021.