Hemophilia A or B With InhibitorsPhase 3NCT02764489
FEIBA Reconstitution Volume Reduction and Faster Infusion Study (FEIBA STAR)
A Phase 3 study testing FEIBA in people with Hemophilia A or B With Inhibitors, run by Baxalta now part of Shire at 3 sites in 3 countries.
Trial summary
The purpose of this study is to: 1. To evaluate the tolerability and safety of infusing reduced volume Factor Eight Inhibitor Bypassing Activity (FEIBA) at the standard infusion rate of 2 U/kg/min 2. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Hemophilia A or B With Inhibitors
- What is being tested?
- FEIBA
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 12 Feb 2019to 27 Dec 2021
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Greater than or equal to (> or =) 18 to less than or equal to (< or =) 65 years old at the time of screening.
- Hemophilia A or B of any severity, with a documented > or = 3 months history of inhibitors (> or = 0.6 Bethesda units [BU]) requiring the use of bypassing agents (FEIBA or rFVIIa) prior to screening. Inhibitor level will be tested at screening if no documented history is available.
- Hepatitis C virus (HCV) negative, either by antibody testing or polymerase chain reaction (PCR); or HCV positive with stable liver disease.
- Human immune deficiency virus (HIV) negative; or HIV positive with stable disease and CD4 count > or = 200 cells per cubic millimetre (cell/mm3) at screening.
- Adequate venous access.
- Willing and able to comply with the requirements of the protocol.
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- If a female of childbearing potential, must have a negative blood pregnancy test and agrees to employ adequate birth control measures for the duration of the study, such as: a. Abstain from sexual intercourse, b. Use a reliable method of contraception (contraception such as an intrauterine device, barrier method [e.g., diaphragm or sponge; female condom not permitted] with spermicide, oral contraceptive, injectable progesterone, sub dermal implant), and have their male partner use a condom
- If female of non-childbearing potential, confirmed at screening by fulfilling 1 of the following criteria:
- Postmenopausal, defined as amenorrhea for at least 12 months following cessation of all exogenous hormonal treatments and with follicle-stimulating hormone levels within the laboratory-defined postmenopausal range or postmenopausal with amenorrhea for at least 24 months and on hormonal replacement therapy.
- Documentation of irreversible surgical sterilization by hysterectomy, bilateral oophorectomy, bilateral tubal ligation (with no subsequent pregnancy at least 1 year from bilateral tubal ligation), or bilateral salpingectomy.
Who cannot join
- Known hypersensitivity to FEIBA or any of its components.
- Advanced liver disease (e.g., liver biopsy confirmed diagnosis of cirrhosis, portal vein hypertension, ascites, prothrombin time [PT] 5 seconds above upper limit of normal).
- Planned elective surgery during participation in this study (excluding minor procedures that will not need preventative bleeding treatments, such as exchanges of peripherally inserted central catheters).
- Platelet count less than (<) 100,000/ microliter (μL).
- Taking Emicizumab (Hemlibra) for bleed prevention.
- Clinical or laboratory evidence of disseminated intravascular coagulation based on medical history.
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- Prior history or evidence of thromboembolic event: acute myocardial infarction, deep vein thrombosis, pulmonary embolism, etc.
- Diagnosis of advanced atherosclerosis, malignancy, and/or other diseases that may increase the participant's risk of thromboembolic complications.
- Participant is taking any immunomodulating drug (e.g., corticosteroid agents at a dose equivalent to hydrocortisone greater than (>) 10 milligram per day (mg/day), or α-interferon) within 30 days prior to enrollment except anti-retroviral chemotherapy.
- Herbal supplements that contain anti-platelet activity.
- 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.
- Clinically significant medical, psychiatric or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
North Macedonia1 site
PHI Institute for Transfusion Medicine of Macedonia
Skopje, 1000
About this study
Official title: A Phase 3b/4, Prospective, Multicenter, Open-label, Randomized, Crossover Study of Tolerability and Safety of FEIBA Reconstituted in Regular or 50% Reduced Volume and of Faster Infusion Rates in Patients With Hemophilia A or B With Inhibitors
The purpose of this study is to:
- 1. To evaluate the tolerability and safety of infusing reduced volume Factor Eight Inhibitor Bypassing Activity (FEIBA) at the standard infusion rate of 2 U/kg/min
- 2. To evaluate the tolerability and safety of infusing reduced volume FEIBA at increased rates of 4 and 10 U/kg/min, in comparison to the standard rate of 2 U/kg/min at the regular volume
Read the detailed description
15 JUN 2020: The temporary enrollment stop of new patients into this study due to the COVID-19 pandemic has been lifted in one or more countries/sites, and the study is now again enrolling new patients. However, some countries/sites may still have paused the enrollment of new patients due to the pandemic.
23 APRIL 2020: Enrollment of new patients into this study has been paused due to the COVID-19 situation. The duration of this pause is dependent on the leveling and control of the COVID-19 pandemic.
- Conditions
- Hemophilia A or B With Inhibitors
- Being tested
- FEIBA
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FEIBABiological
Also called FEIBA NF., AICC, anti-inhibitor coagulant complex, Anti-inhibitor Coagulant Complex Nanofiltered (activated prothrombin complex concentrate [APCC])
Anti-inhibitor Coagulant Complex Nanofiltered (activated prothrombin complex concentrate [APCC]), FEIBA NF.
| Study group | Role | What they receive |
|---|---|---|
| Part 1:FEIBA 85±15 U/kg at Regular Volume Then 50% Reduced Volume at 2 U/kg/min Rate or Vice Versa | Experimental | Participants who were eligible were randomized to receive: 3 infusions (infusions 1, 2 and 3) of factor eight inhibitor bypassing activity (FEIBA) 85 ± 15 U/kg, reconstituted in regular volume sterile water for injection (SWFI) followed by 3 infusions (infusions 4, 5 and 6) of FEIBA 85 ± 15 U/kg reconstituted in 50% reduced volume SWFI (Sequence A) or: 3 infusions (infusions 1, 2 and 3) of FEIBA 85 ± 15 U/kg, reconstituted in 50% reduced volume SWFI, followed by 3 infusions of FEIBA 85 ± 15 U/kg, reconstituted in regular volume SWFI (Sequence B). All infusions in Part 1 were given at the standard infusion rate of 2 U/kg/min. Receives: Biological: FEIBA |
| Part 2:FEIBA 85±15U/kg 50% Reduced Volume at 4U/kg/min Rate Then at 10U/kg/min Rate | Experimental | Participants who completed Part 1, received FEIBA 85 ± 15 U/kg, reconstituted in 50% reduced volume SWFI at an increased rate of 4 U/kg/min for infusions 7, 8, and 9, followed by FEIBA 85 ± 15 U/kg, reconstituted in 50% reduced volume SWFI at an increased rate of 10 U/kg/min for infusions 10, 11, and 12. Receives: Biological: FEIBA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Any Treatment Emergent Adverse Event (TEAE)
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment.
Measured: From first dose of study drug up to 7 days after the end of infusion (up to Day 41)
Number of Participants With Any Hypersensitivity Reaction
Number of participants with AEs particular to allergic-type hypersensitivity reactions were assessed. Clinical manifestations of hypersensitivity reactions included, but was not limited to skin rash, pruritus (itching), urticaria (hives), angioedema (for example, swelling of the lips and/or tongue) and anaphylactic reaction.
Measured: From first dose of study drug up to 7 days after the end of infusion (up to Day 41)
Number of Participants With Any Thromboembolic Event
Participants with adverse events related to thromboembolic event were reported. Clinical manifestations of thromboembolic events included, but was not limited to myocardial infarction, deep vein thrombosis, pulmonary embolism, stroke and transitory ischemic attack.
Measured: From first dose of study drug up to 7 days after the end of infusion (up to Day 41)
Number of Participants With Any Infusion Site Reaction
Infusion sites were monitored for pain, tenderness, erythema, and swelling. Infusion site evaluations were made by clinical staff or by the participant or caregiver.
Measured: From first dose of study drug up to 7 days after the end of infusion (up to Day 41)
Number of Participants With AEs Leading to Study Discontinuation
Measured: From first dose of study drug up to 7 days after the end of infusion (up to Day 41)
Number of Participants With Vital Signs Considered as AEs
Number of participants with vital signs considered as AEs were assessed. Vital signs included body temperature (degree Celsius or degrees Fahrenheit [°C or °F]), respiratory rate (breaths/min), pulse rate (beats/min), and systolic and diastolic blood pressure (millimeter of mercury [mmHg]).
Measured: From first dose of study drug up to 7 days after the end of infusion (up to Day 41)
Number of Participants With Laboratory Assessments Considered as AEs
Number of participants with Laboratory Assessments considered as AEs were assessed. Laboratory assessments included hematology, clinical chemistry, coagulation testing, serological testing, pregnancy testing, cluster differentiation 4 (CD4).
Measured: From first dose of study drug up to 7 days after the end of infusion (up to Day 41)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2016 (estimated)
- Study start12 Feb 2019
- Primary completion27 Dec 2021
- Study completion27 Dec 2021
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 A or B With InhibitorsStudies 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
- Crossover
- Masking
- None (open label)
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Baxalta Innovations GmbH, now part of Shire
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Shire
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02764489
- Sponsor's protocol ID
- 091501
- Other IDs
- 2015-005781-39, 2015
- Status last verified
- Feb 2023
- First submitted
- 11 Apr 2016
- Last updated
- 14 Feb 2023
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Study at a glance
From the registry record, last updated by the sponsor on 14 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3b/4, Prospective, Multicenter, Open-label, Randomized, Crossover Study of Tolerability and Safety of FEIBA Reconstituted in Regular or 50% Reduced Volume and of Faster Infusion Rates in Patients With Hemophilia A or B With Inhibitors
- ClinicalTrials.gov ID
- NCT02764489
- Acronym / sponsor ID
- 091501
- Conditions
- Hemophilia A or B With Inhibitors
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Collaborators
- Baxalta Innovations GmbH, now part of Shire
- Study start
- 12 Feb 2019 (actual)
- Primary completion
- 27 Dec 2021 (actual)
- Study completion
- 27 Dec 2021 (actual)
- First posted
- 6 May 2016 (estimated)
- Results
- Posted 9 Feb 2023
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 this study is to:
- 1. To evaluate the tolerability and safety of infusing reduced volume Factor Eight Inhibitor Bypassing Activity (FEIBA) at the standard infusion rate of 2 U/kg/min
- 2. To evaluate the tolerability and safety of infusing reduced volume FEIBA at increased rates of 4 and 10 U/kg/min, in comparison to the standard rate of 2 U/kg/min at the regular volume
Detailed description
15 JUN 2020: The temporary enrollment stop of new patients into this study due to the COVID-19 pandemic has been lifted in one or more countries/sites, and the study is now again enrolling new patients. However, some countries/sites may still have paused the enrollment of new patients due to the pandemic.
23 APRIL 2020: Enrollment of new patients into this study has been paused due to the COVID-19 situation. The duration of this pause is dependent on the leveling and control of the COVID-19 pandemic.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Part 1:FEIBA 85±15 U/kg at Regular Volume Then 50% Reduced Volume at 2 U/kg/min Rate or Vice Versa | Experimental | Biological: FEIBAParticipants who were eligible were randomized to receive: 3 infusions (infusions 1, 2 and 3) of factor eight inhibitor bypassing activity (FEIBA) 85 ± 15 U/kg, reconstituted in regular volume sterile water for injection (SWFI) followed by 3 infusions (infusions 4, 5 and 6) of FEIBA 85 ± 15 U/kg reconstituted in 50% reduced volume SWFI (Sequence A) or: 3 infusions (infusions 1, 2 and 3) of FEIBA 85 ± 15 U/kg, reconstituted in 50% reduced volume SWFI, followed by 3 infusions of FEIBA 85 ± 15 U/kg, reconstituted in regular volume SWFI (Sequence B). All infusions in Part 1 were given at the standard infusion rate of 2 U/kg/min. |
| Part 2:FEIBA 85±15U/kg 50% Reduced Volume at 4U/kg/min Rate Then at 10U/kg/min Rate | Experimental | Biological: FEIBAParticipants who completed Part 1, received FEIBA 85 ± 15 U/kg, reconstituted in 50% reduced volume SWFI at an increased rate of 4 U/kg/min for infusions 7, 8, and 9, followed by FEIBA 85 ± 15 U/kg, reconstituted in 50% reduced volume SWFI at an increased rate of 10 U/kg/min for infusions 10, 11, and 12. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Greater than or equal to (> or =) 18 to less than or equal to (< or =) 65 years old at the time of screening.
- Hemophilia A or B of any severity, with a documented > or = 3 months history of inhibitors (> or = 0.6 Bethesda units [BU]) requiring the use of bypassing agents (FEIBA or rFVIIa) prior to screening. Inhibitor level will be tested at screening if no documented history is available.
- Hepatitis C virus (HCV) negative, either by antibody testing or polymerase chain reaction (PCR); or HCV positive with stable liver disease.
- Human immune deficiency virus (HIV) negative; or HIV positive with stable disease and CD4 count > or = 200 cells per cubic millimetre (cell/mm3) at screening.
- Adequate venous access.
Key exclusion criteria (13 listed)
- Known hypersensitivity to FEIBA or any of its components.
- Advanced liver disease (e.g., liver biopsy confirmed diagnosis of cirrhosis, portal vein hypertension, ascites, prothrombin time [PT] 5 seconds above upper limit of normal).
- Planned elective surgery during participation in this study (excluding minor procedures that will not need preventative bleeding treatments, such as exchanges of peripherally inserted central catheters).
- Platelet count less than (<) 100,000/ microliter (μL).
- Taking Emicizumab (Hemlibra) for bleed prevention.
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 |
|---|---|---|
| FEIBAAlso: FEIBA NF., AICC, anti-inhibitor coagulant complex, Anti-inhibitor Coagulant Complex Nanofiltered (activated prothrombin complex concentrate [APCC])Anti-inhibitor Coagulant Complex Nanofiltered (activated prothrombin complex concentrate [APCC]), FEIBA NF. | Biological | Part 1:FEIBA 85±15 U/kg at Regular Volume Then 50% Reduced Volume at 2 U/kg/min Rate or Vice Versa, Part 2:FEIBA 85±15U/kg 50% Reduced Volume at 4U/kg/min Rate Then at 10U/kg/min Rate |
Primary outcome
Number of Participants With Any Treatment Emergent Adverse Event (TEAE)
Time frame: From first dose of study drug up to 7 days after the end of infusion (up to Day 41)
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment.
Number of Participants With Any Hypersensitivity Reaction
Time frame: From first dose of study drug up to 7 days after the end of infusion (up to Day 41)
Number of participants with AEs particular to allergic-type hypersensitivity reactions were assessed. Clinical manifestations of hypersensitivity reactions included, but was not limited to skin rash, pruritus (itching), urticaria (hives), angioedema (for example, swelling of the lips and/or tongue) and anaphylactic reaction.
Number of Participants With Any Thromboembolic Event
Time frame: From first dose of study drug up to 7 days after the end of infusion (up to Day 41)
Participants with adverse events related to thromboembolic event were reported. Clinical manifestations of thromboembolic events included, but was not limited to myocardial infarction, deep vein thrombosis, pulmonary embolism, stroke and transitory ischemic attack.
Number of Participants With Any Infusion Site Reaction
Time frame: From first dose of study drug up to 7 days after the end of infusion (up to Day 41)
Infusion sites were monitored for pain, tenderness, erythema, and swelling. Infusion site evaluations were made by clinical staff or by the participant or caregiver.
Number of Participants With AEs Leading to Study Discontinuation
Time frame: From first dose of study drug up to 7 days after the end of infusion (up to Day 41)
Number of Participants With Vital Signs Considered as AEs
Time frame: From first dose of study drug up to 7 days after the end of infusion (up to Day 41)
Number of participants with vital signs considered as AEs were assessed. Vital signs included body temperature (degree Celsius or degrees Fahrenheit [°C or °F]), respiratory rate (breaths/min), pulse rate (beats/min), and systolic and diastolic blood pressure (millimeter of mercury [mmHg]).
Number of Participants With Laboratory Assessments Considered as AEs
Time frame: From first dose of study drug up to 7 days after the end of infusion (up to Day 41)
Number of participants with Laboratory Assessments considered as AEs were assessed. Laboratory assessments included hematology, clinical chemistry, coagulation testing, serological testing, pregnancy testing, cluster differentiation 4 (CD4).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Feb 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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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
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing FEIBA in people with Hemophilia A or B With Inhibitors, run by Baxalta now part of Shire at 3 sites in 3 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 10 inclusion and 13 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 14 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Croatia, North Macedonia, Ukraine. 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 9 Feb 2023). 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?
Number of Participants With Any Treatment Emergent Adverse Event (TEAE) (time frame: From first dose of study drug up to 7 days after the end of infusion (up to Day 41)). The study lists 7 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 14 Feb 2023.