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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT01181128
Registration number
NCT01181128
Ethics application status
Date submitted
12/08/2010
Date registered
13/08/2010
Date last updated
8/01/2021
Titles & IDs
Public title
Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Subjects With Severe Hemophilia A
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Scientific title
A-LONG: An Open-Label, Multicenter Evaluation of the Safety, Pharmacokinetics, and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding in Previously Treated Subjects With Severe Hemophilia A
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Secondary ID [1]
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997HA301
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Severe Hemophilia A
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Condition category
Condition code
Blood
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0
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Clotting disorders
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Human Genetics and Inherited Disorders
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0
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Experimental: Individualized (Tailored) Prophylaxis - On rFVIIIFc Day 0, all participants underwent pharmacokinetic (PK) analysis with 50 IU/kg rFVIIIFc to estimate their PK parameters and guide the appropriate dose or interval of dosing. A subset of participants (Sequential PK subgroup) also had PK analyses performed with a single dose of 50 IU/kg Advate (Advate Day 0) within 8 weeks prior to rFVIIIFc Day 0. A \>= 96 hour washout was performed before the PK dose of Advate or rFVIIIFc was administered. Repeat PK profiling with a single dose of 50 IU/kg rFVIIIFc was conducted at Week 14 or after 12 to 24 weeks of prophylaxis with rFVIIIFc.
After PK assessments, all participants started twice weekly treatment with 25 IU/kg of rFVIIIFc via intravenous (IV) injection on Day 1 and 50 IU/kg on Day 4, followed by individualized dose and interval modification within the range of 25 to 65 IU/kg every 3 to 5 days, as determined by rFVIIIFc PK analysis, to maintain a trough level of 1% to 3% (or higher, as clinically indicated) FVIII activity.
Experimental: Weekly Prophylaxis - 65 IU/kg of rFVIIIFc via IV injection every 7 days
Experimental: Episodic (On-Demand) Dosing - 10 to 50 IU/kg rFVIIIFc via IV injection, as required to treat a bleeding episode
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incidence Rate of FVIII Inhibitor Development
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Assessment method [1]
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An inhibitor test result =0.6 Bethesda units (BU)/mL, identified and confirmed by re-testing of a second sample obtained within 2 to 4 weeks, was considered positive. Both tests were to be performed using the Nijmegen-modified Bethesda Assay by the central laboratory. The incidence rates along with the 95% confidence interval (CI) were summarized for all titers for subjects with 50 or more exposure days (EDs) to rFVIIIFc and a valid inhibitor test after the 50th exposure. In addition, the incidence rates for all subjects regardless of their exposure days to rFVIIIFc were also summarized. The 95% CI was calculated using Clopper-Pearson exact method.
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Timepoint [1]
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up to 52 weeks ± 2 weeks
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Primary outcome [2]
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Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
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Assessment method [2]
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AE=any untoward medical occurrence that did not necessarily have a causal relationship with treatment, and could be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of study product, whether related or not. TEAE=AE present prior to receiving the first injection of Advate or rFVIIIFc that subsequently worsened in severity or not present prior to receiving the first injection but subsequently appeared before the last visit on study. Serious AE (SAE)=AE resulting in death, immediate risk of death, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity, or a congenital anomaly/birth defect, or any other medically important event. AEs emergent between the first Advate injection and first on-study rFVIIIFc injection (Sequential PK Subgroup) or during the surgical/rehabilitation period (Surgery Subgroup) are presented separately.
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Timepoint [2]
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up to 52 weeks + 30 days ± 1 week
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Primary outcome [3]
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Number of Participants With Potentially Clinically Significant Abnormal Laboratory Values From Baseline
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Assessment method [3]
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Clinical laboratory evaluations included hematology and blood chemistry. Table does not include laboratory tests evaluated during the surgical/rehabilitation period. ULN=upper limit of normal.
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Timepoint [3]
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up to 52 weeks ± 2 weeks
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Primary outcome [4]
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Number of Participants With Clinically Relevant Abnormalities in Vital Signs or Relevant Changes From Baseline in Vital Signs
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Assessment method [4]
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Number of participants with clinically relevant abnormalities or relevant changes from baseline in temperature, pulse (beats per minute \[bpm\]), systolic blood pressure (SBP), and diastolic blood pressure (DBP) are presented. Baseline (BL) is defined as the last non-missing evaluable assessment taken prior and closest to the first rFVIIIFc dose. ? signifies increase and ? signifies decrease.
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Timepoint [4]
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0
up to 52 weeks ± 2 weeks
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Primary outcome [5]
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Annualized Bleeding Rate
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Assessment method [5]
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Annualized bleeding episodes = (number of bleeding episodes during the efficacy period / number of days during the efficacy period)\*365.25. The efficacy period in Arms 1 and 2 began with the first prophylactic dose of rFVIIIFc and ended with the last dose (regardless of reason for dosing). The efficacy period in Arm 3 began at the time of last PK rFVIIIFc sampling timepoint and ended at the date of the last study visit. Periods of PK evaluations and surgery/rehabilitation are not included in the efficacy period. A bleeding episode started from the first sign of a bleed, and ended 72 hours after the last treatment for the bleeding, within which any symptoms of bleeding at the same location, or injections less than or equal to 72 hours apart, were considered the same bleeding episode.
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Timepoint [5]
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up to 52 weeks ± 2 weeks (efficacy period as defined in description)
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Primary outcome [6]
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Comparison of Annualized Bleeding Rates: Arm 1 Versus Arm 3
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Assessment method [6]
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Estimated using the negative binomial model with treatment arm as covariate, based on whole study duration for all participants. Annualized bleeding episodes = (number of bleeding episodes / number of days in the respective period)\*365.25.The efficacy period in Arm 1 began with the first prophylactic dose of rFVIIIFc and ended with the last dose (regardless of reason for dosing). The efficacy period in Arm 3 began at the time of last PK rFVIIIFc sampling timepoint and ended at the date of the last study visit. Periods of PK evaluations and surgery/rehabilitation are not included in the efficacy period. A bleeding episode started from the first sign of a bleed, and ended 72 hours after the last treatment for the bleeding, within which any symptoms of bleeding at the same location, or injections less than or equal to 72 hours apart, were considered part of the same bleeding episode.
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Timepoint [6]
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0
up to 52 weeks ± 2 weeks (efficacy period as defined in description)
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Primary outcome [7]
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Area Under the Curve (AUC) Per Dose (One-stage Clotting Assay)
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Assessment method [7]
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Dose normalized area under the drug concentration-time curve. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
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Timepoint [7]
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0
See Measure Description for complete time frame.
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Primary outcome [8]
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Elimination Half Life (t1/2; One-stage Clotting Assay)
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Assessment method [8]
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Time required for the activity of the drug to reach half of its original value. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
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Timepoint [8]
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0
See Measure Description for complete time frame.
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Primary outcome [9]
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Clearance (CL; One-stage Clotting Assay)
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Assessment method [9]
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Rate at which the body removes the drug, measured as the volume of the plasma cleared of drug per unit time per unit weight. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
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Timepoint [9]
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0
See Measure Description for complete time frame.
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Primary outcome [10]
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Mean Residence Time (MRT; One-stage Clotting Assay)
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Assessment method [10]
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The average time that a drug molecule is present in the systemic circulation. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
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Timepoint [10]
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0
See Measure Description for complete time frame.
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Primary outcome [11]
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Incremental Recovery (One-stage Clotting Assay)
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Assessment method [11]
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The rise in FVIII activity in IU/dL per unit dose administered in IU/kg. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
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Timepoint [11]
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0
See Measure Description for complete time frame.
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Secondary outcome [1]
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Comparison of Annualized Bleeding Rates: Arm 2 Versus Arm 3
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Assessment method [1]
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0
Estimated using the negative binomial model with treatment arm as covariate, based on whole study duration for all participants. Annualized bleeding episodes = (number of bleeding episodes / number of days in the respective period)\*365.25.The efficacy period in Arm 2 began with the first prophylactic dose of rFVIIIFc and ended with the last dose (regardless of reason for dosing). The efficacy period in Arm 3 began at the time of last PK rFVIIIFc sampling timepoint and ended at the date of the last study visit. Periods of PK evaluations and surgery/rehabilitation are not included in the efficacy period. A bleeding episode started from the first sign of a bleed, and ended 72 hours after the last treatment for the bleeding, within which any symptoms of bleeding at the same location, or injections less than or equal to 72 hours apart, were considered the same bleeding episode.
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Timepoint [1]
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0
up to 52 weeks ± 2 weeks (efficacy period as defined in description)
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Secondary outcome [2]
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Annualized rFVIIIFc Consumption Per Participant
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Assessment method [2]
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Consumption is calculated for the efficacy period. The efficacy period in Arms 1 and 2 began with the first prophylactic dose of rFVIIIFc and ended with the last dose (regardless of reason for dosing). The efficacy period in Arm 3 began at the time of last PK rFVIIIFc sampling timepoint and ended at the date of the last study visit. Periods of PK evaluations and surgery/rehabilitation are not included in the efficacy period. Overall units (IU/kg) of annualized rFVIIIFc consumption = \[Total rFVIIIFc IU/kg received during the efficacy period / number of days in efficacy period\] x 365.25.
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Timepoint [2]
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0
up to 52 weeks ± 2 weeks (efficacy period as defined in description)
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Secondary outcome [3]
0
0
Participant Assessment of Response to Injections to Treat a Bleeding Episode
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Assessment method [3]
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Participant's assessment of the response to the first rFVIIIFc injection for each bleeding episode. Percentages were based on the number of bleeding episodes for which a response was provided for the first injection, using the following 4-point scale: excellent=abrupt pain relief and/or improvement in signs of bleeding within approximately 8 hours after the initial injection; good=definite pain relief and/or improvement in signs of bleeding within approximately 8 hours after an injection, but possibly requiring more than one injection after 24 to 48 hours for complete resolution; moderate=probable or slight beneficial effect within 8 hours after the initial injection and requiring more than one injection; no response=no improvement, or condition worsened, within approximately 8 hours after the initial injection.
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Timepoint [3]
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up to 52 weeks ± 2 weeks
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Secondary outcome [4]
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0
Investigator's Assessment of Participants' Bleeding Response to rFVIIIFc Injection
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Assessment method [4]
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The investigator was given the opportunity to record an assessment of a participant's response to treatment, if the participant was treated in the hospital for a major bleed, using the following 4-point scale: excellent=abrupt pain relief and/or improvement in signs of bleeding within approximately 8 hours after the initial injection; good=definite pain relief and/or improvement in signs of bleeding within approximately 8 hours after an injection, but possibly requiring more than one injection after 24 to 48 hours for complete resolution; moderate=probable or slight beneficial effect within 8 hours after the initial injection and requiring more than one injection; no response=no improvement, or condition worsened, within approximately 8 hours after the initial injection.
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Timepoint [4]
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up to 52 weeks ± 2 weeks
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Secondary outcome [5]
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Annualized Bleeding Rate by Location of Bleed (Joint, Muscle, Internal, Skin/Mucosa)
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Assessment method [5]
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0
Annualized bleeding episodes = (Number of bleeding episodes at the specified location / number of days in efficacy period) x 365.25. The efficacy period in Arms 1 and 2 began with the first prophylactic dose of rFVIIIFc and ended with the last dose (regardless of reason for dosing). The efficacy period in Arm 3 began at the time of last PK rFVIIIFc sampling timepoint and ended at the date of the last study visit. Periods of PK evaluations and surgery/rehabilitation were not included in the efficacy period. A bleeding episode started from the first sign of a bleed, and ended 72 hours after the last treatment for the bleeding, within which any symptoms of bleeding at the same location, or injections less than or equal to 72 hours apart, were considered the same bleeding episode. Any bleeding at a different location was a separate bleeding episode regardless of time from the last injection.
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Timepoint [5]
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0
up to 52 weeks ± 2 weeks (efficacy period as defined in description)
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Secondary outcome [6]
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Annualized Joint Bleeding Rate (Spontaneous and Traumatic)
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Assessment method [6]
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0
Annualized bleeding episodes = (Number of bleeding episodes of the specified type / number of days in efficacy period) x 365.25. The efficacy period in Arms 1 and 2 began with the first prophylactic dose of rFVIIIFc and ended with the last dose (regardless of reason for dosing). The efficacy period in Arm 3 began at the time of last PK rFVIIIFc sampling timepoint and ended at the date of the last study visit. Periods of PK evaluations and surgery/rehabilitation were not included in the efficacy period. A bleeding episode started from the first sign of a bleed, and ended 72 hours after the last treatment for the bleeding, within which any symptoms of bleeding at the same location, or injections less than or equal to 72 hours apart, were considered the same bleeding episode.
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Timepoint [6]
0
0
up to 52 weeks ± 2 weeks (efficacy period as defined in description)
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Secondary outcome [7]
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Number of Days From Last Treatment Injection to a New Bleeding Episode
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Assessment method [7]
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Number of days from the last injection to treat a bleeding episode to a new bleeding episode, analyzed for per evaluable bleeding episode and per participant. For "per participant" values, number of days from last injection to treat a bleed to a new bleeding episode is averaged across all evaluable bleeding episodes for each participant first, and then descriptive statistics were calculated across participants. A follow-up injection administered \>72 hours after the most recent injection given to treat a bleed was considered a new bleed at the same location and was classified as type=Unknown (not evaluable). The efficacy period in Arms 1 and 2 began with the first prophylactic dose of rFVIIIFc and ended with the last dose (regardless of reason for dosing). The efficacy period in Arm 3 began at the time of last PK rFVIIIFc sampling timepoint and ended at the date of the last study visit. Periods of PK evaluations and surgery/rehabilitation were not included in the efficacy period.
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Timepoint [7]
0
0
up to 52 weeks ± 2 weeks (efficacy period as defined in description)
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Secondary outcome [8]
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0
Number of Injections Required for Resolution of a Bleeding Episode
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Assessment method [8]
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0
A bleeding episode started from the first sign of a bleed, and ended 72 hours after the last treatment for the bleeding, within which any symptoms of bleeding at the same location, or injections less than or equal to 72 hours apart, were considered the same bleeding episode. All injections given from the initial sign of a bleed until the last date/time within the bleed window are counted. The resolution of a bleed is defined as no sign of bleeding following injection for the bleed. The efficacy period in Arms 1 and 2 began with the first prophylactic dose of rFVIIIFc and ended with the last dose (regardless of reason for dosing). The efficacy period in Arm 3 began at the time of last PK rFVIIIFc sampling timepoint and ended at the date of the last study visit. Periods of PK evaluations and surgery/rehabilitation are not included in the efficacy period.
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Timepoint [8]
0
0
up to 52 weeks ± 2 weeks (efficacy period as defined in description)
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Secondary outcome [9]
0
0
Number of Injections Required for Resolution of a Bleeding Episode by Location of Bleed
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Assessment method [9]
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0
Please see Outcome Measure 20 for a definition of the efficacy period. A bleeding episode started from the first sign of a bleed, and ended 72 hours after the last treatment for the bleeding, within which any symptoms of bleeding at the same location, or injections less than or equal to 72 hours apart, were considered the same bleeding episode. All injections given from the initial sign of a bleed until the last date/time within the bleed window were counted. The resolution of a bleed was defined as no sign of bleeding following injection for the bleed. Bleeding episodes that presented in multiple locations are included as a single event in the overall summary for the number of injections to resolve that bleeding episode but are included in summaries for each location.
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Timepoint [9]
0
0
up to 52 weeks ± 2 weeks (efficacy period as defined in description)
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Secondary outcome [10]
0
0
Total Dose Per Injection Required for Resolution of a Bleeding Episode by Location of Bleed
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Assessment method [10]
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0
For each bleeding episode at one location, the total dose is the sum of the doses (IU/kg) administered across all injections given to treat that bleeding episode. Please see Outcome Measure 20 for a definition of the efficacy period. A bleeding episode started from the first sign of a bleed, and ended 72 hours after the last treatment for the bleeding, within which any symptoms of bleeding at the same location, or injections less than or equal to 72 hours apart, were considered part of the same bleeding episode. Bleeding episodes that presented in multiple locations are included as a single event in the overall summary for dose administered to resolve that bleeding episode but are included in the individual summaries for each location.
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Timepoint [10]
0
0
up to 52 weeks ± 2 weeks (efficacy period as defined in description)
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Secondary outcome [11]
0
0
Volume at Steady State (Vss; One-stage Clotting Assay)
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Assessment method [11]
0
0
Volume of distribution at steady state. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
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Timepoint [11]
0
0
See Measure Description for complete time frame.
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Secondary outcome [12]
0
0
Volume at Steady State (Vss; Two-stage Chromogenic Assay)
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Assessment method [12]
0
0
Volume of distribution at steady state. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
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Timepoint [12]
0
0
See Measure Description for complete time frame.
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Secondary outcome [13]
0
0
Time to 1% and 3% FVIII Activity (One-stage Clotting Assay)
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Assessment method [13]
0
0
Estimated time after dose (in days) when FVIII activity has declined to approximately 1 or 3 IU/dL (1% or 3%) above baseline, respectively. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
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Timepoint [13]
0
0
See Measure Description for complete time frame.
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Secondary outcome [14]
0
0
Time to 1% and 3% FVIII Activity (Two-stage Chromogenic Assay)
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Assessment method [14]
0
0
Estimated time after dose (in days) when FVIII activity has declined to approximately 1 or 3 IU/dL (1% or 3%) above baseline, respectively. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
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Timepoint [14]
0
0
See Measure Description for complete time frame.
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Secondary outcome [15]
0
0
Time at Maximum Activity (Tmax; One-stage Clotting Assay)
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Assessment method [15]
0
0
Time at which maximum activity (Cmax) is observed. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
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Timepoint [15]
0
0
See Measure Description for complete time frame.
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Secondary outcome [16]
0
0
Time at Maximum Activity (Tmax; Two-stage Chromogenic Assay)
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Assessment method [16]
0
0
Time at which the maximum activity (Cmax) is observed. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
Query!
Timepoint [16]
0
0
See Measure Description for complete time frame.
Query!
Secondary outcome [17]
0
0
Area Under the Curve (AUC) Per Dose (Two-stage Chromogenic Assay)
Query!
Assessment method [17]
0
0
Dose normalized area under the drug concentration-time curve. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
Query!
Timepoint [17]
0
0
See Measure Description for complete time frame.
Query!
Secondary outcome [18]
0
0
Elimination Half Life (t1/2; Two-stage Chromogenic Assay)
Query!
Assessment method [18]
0
0
Time required for the activity of the drug to reach half of its original value. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
Query!
Timepoint [18]
0
0
See Measure Description for complete time frame.
Query!
Secondary outcome [19]
0
0
Clearance (CL; Two-stage Chromogenic Assay)
Query!
Assessment method [19]
0
0
Rate at which the body removes the drug, measured as the volume of the plasma cleared of drug per unit time per unit weight. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
Query!
Timepoint [19]
0
0
See Measure Description for complete time frame.
Query!
Secondary outcome [20]
0
0
Mean Residence Time (MRT; Two-stage Chromogenic Assay)
Query!
Assessment method [20]
0
0
The average time that a drug molecule is present in the systemic circulation. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
Query!
Timepoint [20]
0
0
See Measure Description for complete time frame.
Query!
Secondary outcome [21]
0
0
Incremental Recovery (Two-stage Chromogenic Assay)
Query!
Assessment method [21]
0
0
The rise in FVIII activity in IU/dL per unit dose administered in IU/kg. Sampling for Advate PK profiling was conducted, following at least 96 hours of washout, at these timepoints: preinjection and at either 30 (±3) minutes or 10 (±3) minutes, 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 48 (±2) hours (Day 2), and 72 (±2) hours (Day 3) from the start of the injection. Sampling for rFVIIIFc PK profiling was conducted at Day 0 (directly following at least 96 hours washout from Advate PK profiling or within 4 weeks of the Advate dose or other rFVIII product) and repeated 12 to 24 weeks later at the following timepoints: preinjection and at 30 (±3) minutes (or 10 (±3) minutes), 1 hour (±15 minutes), 6 (±1) hours, 24 (±2) hours (Day 1), 72 (±2) hours (Day 3), 96 (±2) hours (Day 4), and 120 (±2) hours (Day 5) from the start of the injection.
Query!
Timepoint [21]
0
0
See Measure Description for complete time frame.
Query!
Secondary outcome [22]
0
0
Hemophilia-Specific Quality of Life Index for Adults (Haem-A-QoL) Questionnaire: Change From Baseline to Week 14
Query!
Assessment method [22]
0
0
The Haem-A-QoL consists of items pertaining to 10 domains specific to living with hemophilia and was administered to adult participants (17 years and older). The 10 domains are: physical health, feeling, view of yourself, sports/leisure, school/work, dealing with hemophilia, and treatment (time frame for all 7 domains, during the last month) and future, family planning, and outlook for the future (time frame for all 3 domains, recently). Lower scores represent better QoL; therefore, a negative change from baseline represents improvement during the course of the study. Scores on a scale range between 0 and 100. Participants in Arm 1 were stratified by their prestudy regimen (either prophylaxis or on-demand).
Query!
Timepoint [22]
0
0
Baseline, Week 14
Query!
Secondary outcome [23]
0
0
Hemophilia-Specific Quality of Life Index for Adults (Haem-A-QoL) Questionnaire: Change From Baseline to Week 28
Query!
Assessment method [23]
0
0
The Haem-A-QoL consists of items pertaining to 10 domains specific to living with hemophilia and was administered to adult participants (17 years and older). The 10 domains are: physical health, feeling, view of yourself, sports/leisure, school/work, dealing with hemophilia, and treatment (time frame for all 7 domains, during the last month) and future, family planning, and outlook for the future (time frame for all 3 domains, recently). Lower scores represent better QoL; therefore, a negative change from baseline represents improvement during the course of the study. Scores on a scale range between 0 and 100. Participants in Arm 1 were stratified by their prestudy regimen (either prophylaxis or on-demand).
Query!
Timepoint [23]
0
0
Baseline, Week 28
Query!
Secondary outcome [24]
0
0
Hemophilia-Specific Quality of Life Index for Children (Haemo-QoL) Questionnaire: Change From Baseline to Week 14 and Week 28 in Haemo-QoL III Total Score
Query!
Assessment method [24]
0
0
The Haemo-QoL III, a quality of life assessment instrument for adolescents with hemophilia, was administered to participants from 13 to 16 years old. This instrument assesses domains specific to living with hemophilia and consists of 12 domains: physical health, feeling, view of yourself, family, friends, others, sports and school, treatment, perceived support, dealing with hemophilia, future, and relationships. Total HAEMO-QoL score is the sum of all raw scores for all subscales for participants for whom at least the minimum number of required questions have been answered. Total scores are presented as the Transformed Scale Score (TSS) from 0-100%, with lower scores indicating a better quality of life. A negative change indicates improvement.
Query!
Timepoint [24]
0
0
Baseline, Week 14, Week 28
Query!
Secondary outcome [25]
0
0
Investigators'/Surgeons' Assessment of Participants' Response to rFVIIIFc for Major Surgery
Query!
Assessment method [25]
0
0
Based on the first assessment of hemostasis by the surgeon/investigator 24 hours or later post-surgery. Scaled responses: Excellent = 1, Good = 2, Fair = 3, Poor/none = 4.
Query!
Timepoint [25]
0
0
up to 52 weeks
Query!
Secondary outcome [26]
0
0
Number of Injections Required to Maintain Hemostasis During Major Surgery
Query!
Assessment method [26]
0
0
The number of injections to maintain hemostasis during surgery includes all injections for surgery purposes, including from the loading dose to the end date/time of surgery.
Query!
Timepoint [26]
0
0
up to 52 weeks
Query!
Secondary outcome [27]
0
0
Dose Per Injection and Total Dose Required to Maintain Hemostasis During Major Surgery
Query!
Assessment method [27]
0
0
Mean dose per injection is the average dose for all injections (including loading dose) needed to maintain hemostasis during surgery. Total dose is the sum across all injections (including loading dose) needed to maintain hemostasis during surgery.
Query!
Timepoint [27]
0
0
up to 52 weeks ± 2 weeks
Query!
Secondary outcome [28]
0
0
Estimated Total Blood Loss During Major Surgery
Query!
Assessment method [28]
0
0
Query!
Timepoint [28]
0
0
up to 52 weeks ± 2 weeks
Query!
Secondary outcome [29]
0
0
Number of Transfusions Required Per Surgery
Query!
Assessment method [29]
0
0
Number of blood component transfusions during a single surgery.
Query!
Timepoint [29]
0
0
up to 52 weeks ± 2 weeks
Query!
Eligibility
Key inclusion criteria
* Male, =12 years of age with weight at least 40 kg
* Diagnosed with severe hemophilia A, defined as <1 IU/dL (<1%) endogenous Factor VIII)
* History of at least 150 documented prior exposure days to any Factor VIII product
* Platelet count =100,000 cells/µL
Query!
Minimum age
12
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Males
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
* History of Factor VIII inhibitors
* Kidney and liver dysfunction
* Diagnosed with other coagulation disorder(s) in addition to hemophilia A
* Prior history of hypersensitivity or anaphylaxis associated with any FVIII or IV immunoglobulin administration
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Non-randomised trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
1/11/2010
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
1/08/2012
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
165
Query!
Recruitment in Australia
Recruitment state(s)
NSW,SA,WA
Query!
Recruitment hospital [1]
0
0
Research Site - Camperdown
Query!
Recruitment hospital [2]
0
0
Research Site - Adelaide
Query!
Recruitment hospital [3]
0
0
Research Site - Perth
Query!
Recruitment postcode(s) [1]
0
0
- Camperdown
Query!
Recruitment postcode(s) [2]
0
0
- Adelaide
Query!
Recruitment postcode(s) [3]
0
0
- Perth
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Arkansas
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
California
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
District of Columbia
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Indiana
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Iowa
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Kentucky
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Louisiana
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Massachusetts
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Michigan
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
Nevada
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
New York
Query!
Country [12]
0
0
United States of America
Query!
State/province [12]
0
0
North Carolina
Query!
Country [13]
0
0
United States of America
Query!
State/province [13]
0
0
Pennsylvania
Query!
Country [14]
0
0
United States of America
Query!
State/province [14]
0
0
Texas
Query!
Country [15]
0
0
United States of America
Query!
State/province [15]
0
0
Utah
Query!
Country [16]
0
0
United States of America
Query!
State/province [16]
0
0
Washington
Query!
Country [17]
0
0
Austria
Query!
State/province [17]
0
0
Wien
Query!
Country [18]
0
0
Belgium
Query!
State/province [18]
0
0
Bruxelles
Query!
Country [19]
0
0
Brazil
Query!
State/province [19]
0
0
Campinas
Query!
Country [20]
0
0
Canada
Query!
State/province [20]
0
0
Calgary
Query!
Country [21]
0
0
Canada
Query!
State/province [21]
0
0
Toronto
Query!
Country [22]
0
0
Canada
Query!
State/province [22]
0
0
Vancouver
Query!
Country [23]
0
0
France
Query!
State/province [23]
0
0
Lyon Cedex 3
Query!
Country [24]
0
0
Germany
Query!
State/province [24]
0
0
BE
Query!
Country [25]
0
0
Germany
Query!
State/province [25]
0
0
Northwest
Query!
Country [26]
0
0
Hong Kong
Query!
State/province [26]
0
0
Hong Kong
Query!
Country [27]
0
0
India
Query!
State/province [27]
0
0
Delhi
Query!
Country [28]
0
0
India
Query!
State/province [28]
0
0
Karna
Query!
Country [29]
0
0
India
Query!
State/province [29]
0
0
Mahara
Query!
Country [30]
0
0
India
Query!
State/province [30]
0
0
Punjab
Query!
Country [31]
0
0
India
Query!
State/province [31]
0
0
Tamilnadu
Query!
Country [32]
0
0
Israel
Query!
State/province [32]
0
0
Ramat Gan
Query!
Country [33]
0
0
Italy
Query!
State/province [33]
0
0
FI
Query!
Country [34]
0
0
Italy
Query!
State/province [34]
0
0
MI
Query!
Country [35]
0
0
Italy
Query!
State/province [35]
0
0
VI
Query!
Country [36]
0
0
Japan
Query!
State/province [36]
0
0
Kashihara-shi
Query!
Country [37]
0
0
Japan
Query!
State/province [37]
0
0
Kawasaki-shi
Query!
Country [38]
0
0
Japan
Query!
State/province [38]
0
0
Kitakyushu-shi
Query!
Country [39]
0
0
Japan
Query!
State/province [39]
0
0
Nagoya-shi
Query!
Country [40]
0
0
Japan
Query!
State/province [40]
0
0
Shinjuku-ku
Query!
Country [41]
0
0
Japan
Query!
State/province [41]
0
0
Suginami-ku
Query!
Country [42]
0
0
New Zealand
Query!
State/province [42]
0
0
Auckland
Query!
Country [43]
0
0
New Zealand
Query!
State/province [43]
0
0
Christchurch
Query!
Country [44]
0
0
New Zealand
Query!
State/province [44]
0
0
Palmerston North
Query!
Country [45]
0
0
South Africa
Query!
State/province [45]
0
0
Gauteng
Query!
Country [46]
0
0
South Africa
Query!
State/province [46]
0
0
W Cape
Query!
Country [47]
0
0
Spain
Query!
State/province [47]
0
0
Barcelona
Query!
Country [48]
0
0
Spain
Query!
State/province [48]
0
0
Madrid
Query!
Country [49]
0
0
Sweden
Query!
State/province [49]
0
0
Göteborg
Query!
Country [50]
0
0
Switzerland
Query!
State/province [50]
0
0
Zurich
Query!
Country [51]
0
0
United Kingdom
Query!
State/province [51]
0
0
Basingstoke
Query!
Country [52]
0
0
United Kingdom
Query!
State/province [52]
0
0
Cambridge
Query!
Country [53]
0
0
United Kingdom
Query!
State/province [53]
0
0
Glasgow
Query!
Country [54]
0
0
United Kingdom
Query!
State/province [54]
0
0
London
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Bioverativ Therapeutics Inc.
Query!
Address
Query!
Country
Query!
Other collaborator category [1]
0
0
Commercial sector/industry
Query!
Name [1]
0
0
Swedish Orphan Biovitrum
Query!
Address [1]
0
0
Query!
Country [1]
0
0
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
The primary objectives of this study are: to evaluate the safety and tolerability of rFVIIIFc administered as a prophylaxis (Arm 1), weekly (Arm 2), on-demand (Arm 3), and surgical treatment regimen; to evaluate the efficacy of the rFVIIIFc tailored prophylaxis regimen (Arm 1); to evaluate the efficacy of rFVIIIFc administered as an on-demand (Arm 3) and surgical treatment regimen. The secondary objectives of this study are: to characterize the PK profile of rFVIIIFc and compare the PK of rFVIIIFc with the currently marketed product, Advate®; to characterize the range of dose and schedules required to adequately prevent bleeding in a prophylaxis regimen, maintain hemostasis in a surgical setting, or to treat bleeding episodes in an on-demand, weekly treatment, or prophylaxis setting.
Query!
Trial website
https://clinicaltrials.gov/study/NCT01181128
Query!
Trial related presentations / publications
Mahlangu J, Powell JS, Ragni MV, Chowdary P, Josephson NC, Pabinger I, Hanabusa H, Gupta N, Kulkarni R, Fogarty P, Perry D, Shapiro A, Pasi KJ, Apte S, Nestorov I, Jiang H, Li S, Neelakantan S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Dodd N, Nugent K, Vigliani G, Luk A, Brennan A, Pierce GF; A-LONG Investigators. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014 Jan 16;123(3):317-25. doi: 10.1182/blood-2013-10-529974. Epub 2013 Nov 13. Shapiro AD, Ragni MV, Kulkarni R, Oldenberg J, Srivastava A, Quon DV, Pasi KJ, Hanabusa H, Pabinger I, Mahlangu J, Fogarty P, Lillicrap D, Kulke S, Potts J, Neelakantan S, Nestorov I, Li S, Dumont JA, Jiang H, Brennan A, Pierce GF. Recombinant factor VIII Fc fusion protein: extended-interval dosing maintains low bleeding rates and correlates with von Willebrand factor levels. J Thromb Haemost. 2014 Nov;12(11):1788-800. doi: 10.1111/jth.12723. Epub 2014 Oct 10. Katragadda S, Neelakantan S, Diao L, Wong N. Population Pharmacokinetic Analysis of Recombinant Factor VIII Fc Fusion Protein in Subjects With Severe Hemophilia A: Expanded to Include Pediatric Subjects. J Clin Pharmacol. 2021 Jul;61(7):889-900. doi: 10.1002/jcph.1854. Epub 2021 Apr 14.
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Public notes
Query!
Contacts
Principal investigator
Name
0
0
Medical Director
Query!
Address
0
0
Bioverativ Therapeutics Inc.
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
Type
Citations or Other Details
Journal
Mahlangu J, Powell JS, Ragni MV, Chowdary P, Josep...
[
More Details
]
Journal
Shapiro AD, Ragni MV, Kulkarni R, Oldenberg J, Sri...
[
More Details
]
Results are available at
https://clinicaltrials.gov/study/NCT01181128
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