A Phase 3 interventional study of Recombinant Factor VIIa BI (rFVIIa BI) and Recombinant Factor VIIa BI (rFVIIa BI) in Hemophilia A and Hemophilia B, sponsored by Baxalta now part of Shire. Completed at 16 sites in 9 countries. Open to male participants aged 12 Years to 65 Years. Per ClinicalTrials.gov, last updated 2021-05-11.
Sponsored by Baxalta now part of Shire · Phase 3, Interventional, and Treatment
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.
866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.
This study's enrollment of 40 is above the median of 28 across 512 interventional studies indexed under Hemophilia A.
Browse Hemophilia A studies →Baxalta now part of Shire is the lead sponsor of 110 studies on the registry; none are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 16 (89%) have results posted.
Counted across the registry records on this site, refreshed daily.
Main Inclusion Criteria:
Main Exclusion Criteria:
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.
Biological: Recombinant Factor VIIa BI (rFVIIa BI)
Biological: Recombinant Factor VIIa BI (rFVIIa BI)
Administered approximately every 3 hours as an intravenous bolus injection on-demand
Administered as a single intravenous bolus injection on-demand
Percentage of Bleeding Episode With "Treatment Success"
No additional hemostatic product required within 12 hours of first dose other than the prescribed dosing regimen.
Time frame: within 12 hours of first dose
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.
Time frame: 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).
Time frame: 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).
Time frame: 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 \[to rFVIIa BI\]).
Time frame: 6 months (throughout study period)
Percentage of Participants With Inhibitor Development to FVII
Development of rFVII inhibitors or FVIIa binding antibodies during the study.
Time frame: 6 months (throughout study period)
Enrollment was conduced at 16 clinical sites from the following countries: Japan, Taiwan, Poland, Romania, Russian Federation, Serbia, Spain, Ukraine and the United States.
| Milestone | Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI | Arm 2: 1 x 270 Micrograms/kg rFVIIa BI |
|---|---|---|
| Started | 18 | 20 |
| Completed | 17 | 18 |
| Not completed | 1 | 2 |
| Withdrew: Lost to follow-up | 1 | 1 |
| Withdrew: Withdrawal by subject | 0 | 1 |
No additional hemostatic product required within 12 hours of first dose other than the prescribed dosing regimen.
| percent of bleeding episodes | Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI | Arm 2: 1 x 270 Micrograms/kg rFVIIa BI |
|---|---|---|
| Percentage of Bleeding Episode With "Treatment Success" | 96.19 (93.31 to 97.86) | 79.30 (73.92 to 83.81) |
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.
| percent of bleeding episodes | Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI | Arm 2: 1 x 270 Micrograms/kg rFVIIa BI |
|---|---|---|
| Successful | 87.89 (83.62 to 91.16) | 79.30 (73.92 to 83.81) |
| Excellent | 34.60 (29.35 to 40.26) | 36.72 (31.05 to 42.78) |
| Good | 53.29 (47.53 to 58.96) | 42.58 (36.67 to 48.70) |
| Moderate | 9.69 (6.79 to 13.65) | 18.36 (14.10 to 23.56) |
| No Assessment Available | 0 (0 to 0) | 0.39 (0.07 to 2.18) |
| None | 2.42 (1.18 to 4.91) | 1.95 (0.84 to 4.49) |
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).
| percent of bleeding episodes | Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI | Arm 2: 1 x 270 Micrograms/kg rFVIIa BI |
|---|---|---|
| Percentage of Clinical Responders (Sustained Bleeding Control) for All Acute Bleeding Episodes | 93.43 (89.96 to 95.75) | 76.17 (70.59 to 80.98) |
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).
| percent of participants with AEs | Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI | Arm 2: 1 x 270 Micrograms/kg rFVIIa BI |
|---|---|---|
| SAE-Moderate-Unrelated | 5.6 | 5.0 |
| SAE-Severe-Unrelated | 11.1 | 0 |
| SAE-Severe-Related | 0 | 5.0 |
| nsAE-Mild-Unrelated | 22.2 | 30.0 |
| nsAE-Moderate-Unrelated | 0 | 15.0 |
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 \[to rFVIIa BI\]).
| percent of AEs | Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI | Arm 2: 1 x 270 Micrograms/kg rFVIIa BI |
|---|---|---|
| SAE-Moderate-Unrelated | 6.7 | 6.3 |
| SAE-Severe-Unrelated | 20.0 | 0 |
| SAE-Severe-Related | 0 | 12.5 |
| nsAE-Mild-Unrelated | 73.3 | 62.5 |
| nsAE-Moderate-Unrelated | 0 | 18.8 |
Development of rFVII inhibitors or FVIIa binding antibodies during the study.
| percent of participants | Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI | Arm 2: 1 x 270 Micrograms/kg rFVIIa BI |
|---|---|---|
| Percentage of Participants With Inhibitor Development to FVII | 0 | 0 |
Collected over 6 months (throughout study period). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI | — | 2/18 (11.1%) | 4/18 (22.2%) |
| Arm 2: 1 x 270 Micrograms/kg rFVIIa BI | — | 2/20 (10%) | 2/20 (10%) |
| Event | Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI | Arm 2: 1 x 270 Micrograms/kg rFVIIa BI |
|---|---|---|
| Muscle HaemorrhageMusculoskeletal and connective tissue disorders | 0/18 | 2/20 |
| Craniocerebral injuryInjury, poisoning and procedural complications | 1/18 | 0/20 |
| Joint injuryInjury, poisoning and procedural complications | 1/18 | 0/20 |
| Head InjuryInjury, poisoning and procedural complications | 1/18 | 0/20 |
| Limb InjuryInjury, poisoning and procedural complications | 1/18 | 0/20 |
| Drug IneffectiveGeneral disorders | 0/18 | 1/20 |
| Event | Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI | Arm 2: 1 x 270 Micrograms/kg rFVIIa BI |
|---|---|---|
| InfluenzaInfections and infestations | 0/18 | 2/20 |
| LacerationInjury, poisoning and procedural complications | 1/18 | 0/20 |
| Hepatic Enzyme IncreasedInvestigations | 1/18 | 0/20 |
| Nasal CongestionRespiratory, thoracic and mediastinal disorders | 1/18 | 0/20 |
| Oropharyngeal PainRespiratory, thoracic and mediastinal disorders | 1/18 | 0/20 |
| Drug HypersensitivityImmune system disorders | 1/18 | 0/20 |
| HeadacheNervous system disorders | 1/18 | 0/20 |
| Sinus HeadacheNervous system disorders | 1/18 | 0/20 |
| PyrexiaGeneral disorders | 1/18 | 0/20 |
| NasopharyngitisInfections and infestations | 1/18 | 0/20 |
| Age, Continuous(Years) | Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI | Arm 2: 1 x 270 Micrograms/kg rFVIIa BI | Total |
|---|---|---|---|
| Median | 28 (12 to 53) | 28 (12 to 54) | 28 (12 to 54) |
| Sex: Female, Male(Participants) | Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI | Arm 2: 1 x 270 Micrograms/kg rFVIIa BI | Total |
|---|---|---|---|
| Female | 0 | 0 | 0 |
| Male | 18 | 20 | 38 |
This study is completed, as verified in Apr 2021. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Baxalta now part of Shire