Trial Outcomes & Findings for Safety and Efficacy of FVIIa to Prevent Excessive Bleeding in Hemophilia Patients With Inhibitors Undergoing Major Surgery (NCT NCT05695391)
NCT ID: NCT05695391
Last Updated: 2026-07-31
Results Overview
Successfully treated major procedures defined by a good or excellent global hemostatic response, based on an aggregate score from 3 timepoints (intra-operative, 24h post-operative, 120h post-operative), each individual score depending on comparison between actual and preliminary-communicated expected blood loss and transfusions for a similar patient without bleeding disorder undergoing the same surgical procedure, but also on the use or not of IMP extra-doses or of rescue therapy.
TERMINATED
PHASE3
2 participants
From surgical wound closure until 120 hours after surgical wound closure
2026-07-31
Participant Flow
Male patients, aged ≥12 years to ≤65 years, with hemophilia A or B with positive inhibitor test BU ≥5 (current or past if not further successfully treated by ITI), or a condition precluding the use of FVIII or FIX products to treat/prevent bleeding, and scheduled to undergo an elective major surgery, were to be enrolled. Major surgeries were those that usually required ≥5 days of factor replacement in this population and typically involved entry into a body cavity or similarly complex procedures
During the screening period, the surgeons had to communicate at least 3 working days before the date of the planned surgery, its expected characteristics including blood loss and transfusion predictions for approval by an enrollment committee comprising an expert surgeon competent in the surgery in patients with hemophilia.
Participant milestones
| Measure |
Coagulation Factor VIIa (Recombinant)
Coagulation Factor VIIa (Recombinant): Eptacog Beta (activated) (EB) 200 μg/kg initial IV bolus dosing before the start of the surgery, followed for the intra-operative period and the first 72 hours post-surgical wound closure by repeated 75 μg/kg IV bolus every 2 hours ±15 minutes; minimum duration of treatment: 5 days at hospital
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|---|---|
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Overall Study
STARTED
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2
|
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Overall Study
COMPLETED
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2
|
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Overall Study
NOT COMPLETED
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0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety and Efficacy of FVIIa to Prevent Excessive Bleeding in Hemophilia Patients With Inhibitors Undergoing Major Surgery
Baseline characteristics by cohort
| Measure |
Coagulation Factor VIIa (Recombinant)
n=2 Participants
Coagulation Factor VIIa (Recombinant): Eptacog Beta (activated) (EB)
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=9 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=9 Participants
|
|
Age, Categorical
>=65 years
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0 Participants
n=9 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=9 Participants
|
|
Sex: Female, Male
Male
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2 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
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1 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
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1 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Asian
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0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=9 Participants
|
|
Race (NIH/OMB)
Black or African American
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1 Participants
n=9 Participants
|
|
Race (NIH/OMB)
White
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1 Participants
n=9 Participants
|
|
Race (NIH/OMB)
More than one race
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0 Participants
n=9 Participants
|
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Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
|
Region of Enrollment
United States
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1 participants
n=9 Participants
|
|
Region of Enrollment
South Africa
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1 participants
n=9 Participants
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PRIMARY outcome
Timeframe: From surgical wound closure until 120 hours after surgical wound closurePopulation: Full Analysis Set was planned to be used, comprised of all major procedures conducted from patients enrolled into the study who had received at least one dose of study drug with each patient having one treated major procedure. However, as only two patients were enrolled due to study termination caused by feasibility considerations, no population-based analysis was done. Only individual data were recorded.
Successfully treated major procedures defined by a good or excellent global hemostatic response, based on an aggregate score from 3 timepoints (intra-operative, 24h post-operative, 120h post-operative), each individual score depending on comparison between actual and preliminary-communicated expected blood loss and transfusions for a similar patient without bleeding disorder undergoing the same surgical procedure, but also on the use or not of IMP extra-doses or of rescue therapy.
Outcome measures
| Measure |
Coagulation Factor VIIa (Recombinant)
n=2 Participants
Coagulation Factor VIIa (Recombinant): Eptacog Beta (activated) (EB)
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|---|---|
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Eptacog Beta (Activated) (EB) Effectiveness Based on Protocol's Aggregate Score
Excellent or good rating
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1 Participants
|
|
Eptacog Beta (Activated) (EB) Effectiveness Based on Protocol's Aggregate Score
Moderate or poor rating
|
1 Participants
|
POST_HOC outcome
Timeframe: From surgical wound closure until 120 hours after surgical wound closurePopulation: Full Analysis Set was planned to be used, comprised of all major procedures conducted from patients enrolled into the study who had received at least one dose of study drug with each patient having one treated major procedure. However, as only two patients were enrolled due to study termination caused by feasibility considerations, no population-based analysis was done. Only individual data were recorded.
Hemostatic response assessed by independent Data Monitoring Committee based on actual bleeding data and surgery types
Outcome measures
| Measure |
Coagulation Factor VIIa (Recombinant)
n=2 Participants
Coagulation Factor VIIa (Recombinant): Eptacog Beta (activated) (EB)
|
|---|---|
|
Eptacog Beta (Activated) (EB) Effectiveness Based on Data Monitoring Committee Review
Excellent or good response
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2 Participants
|
|
Eptacog Beta (Activated) (EB) Effectiveness Based on Data Monitoring Committee Review
Moderate or poor response
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0 Participants
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Adverse Events
Coagulation Factor VIIa (Recombinant)
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Coagulation Factor VIIa (Recombinant)
n=2 participants at risk
Coagulation Factor VIIa (Recombinant): Eptacog Beta (activated) (EB)
|
|---|---|
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Cardiac disorders
Sinus tachycardia
|
50.0%
1/2 • Number of events 1 • Adverse Events (AEs) were to be collected from the time of signing of the informed consent/assent until resolution or until the follow-up call which was 30 ± 2 days after the last dose of EB, or until early termination, whichever came first. As only 2 patients were effectively enrolled in the study, any AE would have a frequency of at least 50%, thus effectively exceeding the frequency threshold above which other adverse events are reported, therefore all AEs are reported.
An AE was defined as any untoward medical occurrence in a patient to whom a medicinal product is administered, regardless of the cause. Unfavorable and unintended signs including clinically significant abnormal laboratory findings, but also symptoms, diseases, whether new or exacerbated, temporally associated with the use of the study treatment, were regarded as AEs, whether or not related to the study drug, and looked for at each visit. Bleeding from the surgical incision was not regarded as AE
|
|
Infections and infestations
Oral herpes
|
50.0%
1/2 • Number of events 1 • Adverse Events (AEs) were to be collected from the time of signing of the informed consent/assent until resolution or until the follow-up call which was 30 ± 2 days after the last dose of EB, or until early termination, whichever came first. As only 2 patients were effectively enrolled in the study, any AE would have a frequency of at least 50%, thus effectively exceeding the frequency threshold above which other adverse events are reported, therefore all AEs are reported.
An AE was defined as any untoward medical occurrence in a patient to whom a medicinal product is administered, regardless of the cause. Unfavorable and unintended signs including clinically significant abnormal laboratory findings, but also symptoms, diseases, whether new or exacerbated, temporally associated with the use of the study treatment, were regarded as AEs, whether or not related to the study drug, and looked for at each visit. Bleeding from the surgical incision was not regarded as AE
|
|
Injury, poisoning and procedural complications
Incision site pain
|
50.0%
1/2 • Number of events 1 • Adverse Events (AEs) were to be collected from the time of signing of the informed consent/assent until resolution or until the follow-up call which was 30 ± 2 days after the last dose of EB, or until early termination, whichever came first. As only 2 patients were effectively enrolled in the study, any AE would have a frequency of at least 50%, thus effectively exceeding the frequency threshold above which other adverse events are reported, therefore all AEs are reported.
An AE was defined as any untoward medical occurrence in a patient to whom a medicinal product is administered, regardless of the cause. Unfavorable and unintended signs including clinically significant abnormal laboratory findings, but also symptoms, diseases, whether new or exacerbated, temporally associated with the use of the study treatment, were regarded as AEs, whether or not related to the study drug, and looked for at each visit. Bleeding from the surgical incision was not regarded as AE
|
|
Injury, poisoning and procedural complications
Post procedural inflammation
|
50.0%
1/2 • Number of events 1 • Adverse Events (AEs) were to be collected from the time of signing of the informed consent/assent until resolution or until the follow-up call which was 30 ± 2 days after the last dose of EB, or until early termination, whichever came first. As only 2 patients were effectively enrolled in the study, any AE would have a frequency of at least 50%, thus effectively exceeding the frequency threshold above which other adverse events are reported, therefore all AEs are reported.
An AE was defined as any untoward medical occurrence in a patient to whom a medicinal product is administered, regardless of the cause. Unfavorable and unintended signs including clinically significant abnormal laboratory findings, but also symptoms, diseases, whether new or exacerbated, temporally associated with the use of the study treatment, were regarded as AEs, whether or not related to the study drug, and looked for at each visit. Bleeding from the surgical incision was not regarded as AE
|
|
Injury, poisoning and procedural complications
Procedural pain
|
50.0%
1/2 • Number of events 1 • Adverse Events (AEs) were to be collected from the time of signing of the informed consent/assent until resolution or until the follow-up call which was 30 ± 2 days after the last dose of EB, or until early termination, whichever came first. As only 2 patients were effectively enrolled in the study, any AE would have a frequency of at least 50%, thus effectively exceeding the frequency threshold above which other adverse events are reported, therefore all AEs are reported.
An AE was defined as any untoward medical occurrence in a patient to whom a medicinal product is administered, regardless of the cause. Unfavorable and unintended signs including clinically significant abnormal laboratory findings, but also symptoms, diseases, whether new or exacerbated, temporally associated with the use of the study treatment, were regarded as AEs, whether or not related to the study drug, and looked for at each visit. Bleeding from the surgical incision was not regarded as AE
|
|
Injury, poisoning and procedural complications
Unintentional medical device removal
|
50.0%
1/2 • Number of events 1 • Adverse Events (AEs) were to be collected from the time of signing of the informed consent/assent until resolution or until the follow-up call which was 30 ± 2 days after the last dose of EB, or until early termination, whichever came first. As only 2 patients were effectively enrolled in the study, any AE would have a frequency of at least 50%, thus effectively exceeding the frequency threshold above which other adverse events are reported, therefore all AEs are reported.
An AE was defined as any untoward medical occurrence in a patient to whom a medicinal product is administered, regardless of the cause. Unfavorable and unintended signs including clinically significant abnormal laboratory findings, but also symptoms, diseases, whether new or exacerbated, temporally associated with the use of the study treatment, were regarded as AEs, whether or not related to the study drug, and looked for at each visit. Bleeding from the surgical incision was not regarded as AE
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
50.0%
1/2 • Number of events 1 • Adverse Events (AEs) were to be collected from the time of signing of the informed consent/assent until resolution or until the follow-up call which was 30 ± 2 days after the last dose of EB, or until early termination, whichever came first. As only 2 patients were effectively enrolled in the study, any AE would have a frequency of at least 50%, thus effectively exceeding the frequency threshold above which other adverse events are reported, therefore all AEs are reported.
An AE was defined as any untoward medical occurrence in a patient to whom a medicinal product is administered, regardless of the cause. Unfavorable and unintended signs including clinically significant abnormal laboratory findings, but also symptoms, diseases, whether new or exacerbated, temporally associated with the use of the study treatment, were regarded as AEs, whether or not related to the study drug, and looked for at each visit. Bleeding from the surgical incision was not regarded as AE
|
|
Psychiatric disorders
Anxiety
|
50.0%
1/2 • Number of events 1 • Adverse Events (AEs) were to be collected from the time of signing of the informed consent/assent until resolution or until the follow-up call which was 30 ± 2 days after the last dose of EB, or until early termination, whichever came first. As only 2 patients were effectively enrolled in the study, any AE would have a frequency of at least 50%, thus effectively exceeding the frequency threshold above which other adverse events are reported, therefore all AEs are reported.
An AE was defined as any untoward medical occurrence in a patient to whom a medicinal product is administered, regardless of the cause. Unfavorable and unintended signs including clinically significant abnormal laboratory findings, but also symptoms, diseases, whether new or exacerbated, temporally associated with the use of the study treatment, were regarded as AEs, whether or not related to the study drug, and looked for at each visit. Bleeding from the surgical incision was not regarded as AE
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60