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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01458106
Registration number
NCT01458106
Ethics application status
Date submitted
20/10/2011
Date registered
24/10/2011
Date last updated
19/12/2020
Titles & IDs
Public title
Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Pediatric Subjects With Hemophilia A
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Scientific title
An Open-Label, Multicenter Evaluation of Safety, Pharmacokinetics, and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein, BIIB031, in the Prevention and Treatment of Bleeding Episodes in Pediatric Subjects With Hemophilia A
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Secondary ID [1]
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2011-003073-28
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Secondary ID [2]
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8HA02PED
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Universal Trial Number (UTN)
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Trial acronym
Kids ALONG
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Hemophilia A
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Condition category
Condition code
Blood
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Clotting disorders
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Human Genetics and Inherited Disorders
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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
Treatment: Drugs - BIIB031 (rFVIIIFc)
Treatment: Drugs - FVIII (PK subgroup only)
Experimental: All participants - PK subgroup: After a Washout Period of =72 hours, at the Baseline Visit (28±7 days prior to Day 1), participants receive a single IV injection of prestudy FVIII over 5 (±2) minutes at a dose of 50 IU/kg, rounded up to the nearest 250 IU increment, for a PK assessment. Following a second Washout Period of =72 hours, participants receive a single IV injection of rFVIIIFc over 5 (±2) minutes at a dose of 50 IU/kg for PK assessment. The first prophylactic dose of rFVIIIFc is administered at a starting dose of 25 IU/kg IV injection on Day 1 and 50 IU/kg on Day 4. Dose increases to a maximum of 80 IU/kg, and frequency of administration to a minimum interval of once every 2 days, are allowed as indicated.
Non-PK subgroup: On Day 1, a first prophylactic dose of rFVIIIFc of 25 IU/kg IV injection is given, followed by a dose of 50 IU/kg on Day 4. Dose increases to a maximum of 80 IU/kg, and frequency of administration to a minimum interval of once every 2 days, are allowed as indicated.
Treatment: Drugs: BIIB031 (rFVIIIFc)
Vials of rFVIIIFc were combined as needed, based on the actual labeled potency to achieve the participant's calculated dose. Partial vial use was allowed, in order to achieve the calculated dose.
Treatment: Drugs: FVIII (PK subgroup only)
Baseline prestudy FVIII dosing in participants who enter the PK subgroup. Vials of prestudy FVIII were combined as needed, based on the nominal labeled potency (e.g., 250 IU, 500 IU, and 1000 IU), to achieve the participant's calculated dose.
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Intervention code [1]
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Treatment: Drugs
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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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Occurrence 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, confirmed on 2 separate samples drawn 2 to 4 weeks apart, was considered positive. Both tests were to be performed by the central laboratory using the Nijmegen-modified Bethesda Assay. Incidences were summarized for any positive inhibitor for participants with =50 EDs to rFVIIIFc. In addition, the incidence for all participants, regardless of their EDs to rFVIIIFc, was also summarized. An exact 95% CI for the proportion of participants with a confirmed inhibitor was calculated using the Clopper-Pearson exact method for a binomial proportion.
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Timepoint [1]
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Up to Week 26 +/- 7 days, or up to 50 exposure days (EDs) if reached prior to Week 26
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Secondary outcome [1]
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Annualized Bleeding Rate
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Assessment method [1]
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Annualized bleeding rate = (number of bleeding episodes during the efficacy period / total number of days during the efficacy period)*365.25. The efficacy period begins with the first prophylactic dose of rFVIIIFc and ends with the last dose (for prophylaxis or a bleed). Surgery/rehabilitation periods and PK evaluation periods are not included in the efficacy period. A bleeding episode started from the first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed, 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 injection to treat the bleeding episode taken more than 72 hours after the preceding one was considered the first injection to treat a new bleeding episode at the same location. Any bleeding at a different location was considered a separate bleeding episode, regardless of time from last injection.
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Timepoint [1]
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Up to Week 26 +/- 7 days (efficacy period as defined in description)
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Secondary outcome [2]
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Annualized Joint Bleeding Rate (Spontaneous)
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Assessment method [2]
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Annualized bleeding rate for spontaneous joint bleed=(number of bleeding episodes meeting those criteria during the efficacy period/total number of days during the efficacy period)*365.25. The efficacy period begins with the first prophylactic dose of rFVIIIFc and ends with the last dose (for prophylaxis or a bleed). Surgery/rehabilitation periods and PK evaluation periods are not included in the efficacy period. A bleeding episode started from the first sign of a bleed and ended no more than 72 hours after the last treatment for the bleed, 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 injection to treat the bleeding episode taken more than 72 hours after the preceding one was considered the first injection to treat a new bleeding episode at the same location. Any bleeding at a different location was considered a separate bleeding episode, regardless of time from last inject
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Timepoint [2]
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Up to Week 26 +/- 7 days (efficacy period as defined in description)
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Secondary outcome [3]
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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 (provided by the caregiver) of the response to the first rFVIIIFc injection for each bleeding episode. Percentages were based on the number of first injections for which a response was provided, 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 a single injection, but possibly requiring more than one injection after 24 to 48 hours for complete resolution; moderate=probable or slight beneficial effect within approximately 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 Week 26 +/- 7 days
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Secondary outcome [4]
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Physician's Global Assessment of the Participant's Response to His rFVIIIFc Regimen
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Assessment method [4]
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Investigators assessed each participant's response to his rFVIIIFc regimen using a 4-point scale: excellent=bleeding episodes responded to = the usual number of injections or = the usual dose of rFVIIIFc or the rate of breakthrough bleeding during prophylaxis was = that usually observed; effective=most bleeding episodes responded to the same number of injections and dose, but some required more injections or higher doses, or there was a minor increase in the rate of breakthrough bleeding; partially effective=bleeding episodes most often required more injections and/or higher doses than expected, or adequate breakthrough bleeding prevention during prophylaxis required more frequent injections and/or higher doses; ineffective=routine failure to control hemostasis, or hemostatic control required additional agents. Percentages are based on the total number of responses; multiple responses per participant are counted.
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Timepoint [4]
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Up to Week 26 +/- 7 days
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Secondary outcome [5]
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Annualized rFVIIIFc Consumption Per Participant
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Assessment method [5]
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Consumption is calculated for the efficacy period. The efficacy period begins with the first prophylactic dose of rFVIIIFc and ends with the last dose (for prophylaxis or a bleed). Surgery/rehabilitation periods and PK evaluation periods are not included in the efficacy period. Annualized consumption = (total IU/kg of study treatment received during the efficacy period / total number of days during the efficacy period)*365.25. Consumption was calculated overall for all participants and for the last 3 months (91 days) on study, counted backwards from the end of the efficacy period, for participants with at least 24 weeks on study.
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Timepoint [5]
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Up to Week 26 +/- 7 days (efficacy period as defined in description)
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Secondary outcome [6]
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Number of Days From Last Treatment Injection to a Spontaneous Bleeding Episode
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Assessment method [6]
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The number of days from the last prophylaxis injection to the onset of a new spontaneous bleeding episode, analyzed across all evaluable bleeding episodes per participant and per episode, based on the efficacy period. Evaluable bleeding episodes are those for which both a date and time are available for both the onset of the bleeding episode and the previous prophylactic injection. The efficacy period begins with the first prophylactic dose of rFVIIIFc and ends with the last dose (for prophylaxis or a bleed). Surgery/rehabilitation periods and PK evaluation periods are not included in the efficacy period. For 'Per participant' values, the number of days from the last prophylactic injection to a spontaneous bleeding episode is averaged across all evaluable spontaneous bleeding episodes per participant.
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Timepoint [6]
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Up to Week 26 +/- 7 days (efficacy period as defined in description)
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Secondary outcome [7]
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Number of Injections Required for Resolution of a Bleeding Episode
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Assessment method [7]
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The number of injections required to resolve a bleeding episode per participant and per episode, based on the efficacy period. The efficacy period begins with the first prophylactic dose of rFVIIIFc and ends with the last dose (for prophylaxis or a bleed). Surgery/rehabilitation periods and PK evaluation periods are not included in the efficacy period. 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. For 'Per participant' values, the number of injections required to resolve each bleed is averaged across all bleeding episodes per participant.
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Timepoint [7]
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Up to Week 26 +/- 7 days (efficacy period as defined in description)
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Secondary outcome [8]
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Total Dose Required for Resolution of a Bleeding Episode
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Assessment method [8]
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The total dose required to resolve a bleeding episode per participant and per episode, based on the efficacy period. The efficacy period begins with the first prophylactic dose of rFVIIIFc and ends with the last dose (for prophylaxis or a bleed). Surgery/rehabilitation periods and PK evaluation periods are not included in the efficacy period. For 'Per bleeding episode' values, for each bleeding episode, the total dose is the sum of the doses (IU/kg) administered across all injections given to treat that bleeding episode. For 'Per participant' values, the total dose (IU/kg) used to resolve each bleed is averaged across all bleeding episodes per participant.
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Timepoint [8]
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Up to Week 26 +/- 7 days (efficacy period as defined in description)
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Secondary outcome [9]
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Maximum Plasma Activity (Cmax; One-stage Activated Partial Thromboplastin Time [aPTT] Clotting Assay)
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Assessment method [9]
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Maximum plasma activity during a dosing interval for participants in the PK subgroup. The values for Cmax were adjusted to the nominal dose of 50 IU/kg. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [9]
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Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [10]
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Maximum Plasma Activity (Cmax; Two-stage Chromogenic Assay)
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Assessment method [10]
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Maximum plasma activity during a dosing interval for participants in the PK subgroup. The values for Cmax were adjusted to the nominal dose of 50 IU/kg. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [10]
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Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [11]
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Elimination Half Life (t1/2; One-stage aPTT Clotting Assay)
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Assessment method [11]
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Time required for the activity of the drug to reach half of its original value for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [11]
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Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [12]
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Elimination Half Life (t1/2; Two-stage Chromogenic Assay)
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Assessment method [12]
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Time required for the activity of the drug to reach half of its original value for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [12]
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Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [13]
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Clearance (CL; One-stage aPTT Clotting Assay)
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Assessment method [13]
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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 for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [13]
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Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [14]
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Clearance (CL; Two-stage Chromogenic Assay)
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Assessment method [14]
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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 for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [14]
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Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [15]
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Volume at Steady State (Vss; One-stage aPTT Clotting Assay)
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Assessment method [15]
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Volume of distribution at steady state for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [15]
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Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [16]
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Volume at Steady State (Vss; Two-stage Chromogenic Assay)
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Assessment method [16]
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Volume of distribution at steady state for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [16]
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Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [17]
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Dose Normalized Area Under the Curve (DNAUC; One-stage aPTT Clotting Assay)
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Assessment method [17]
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Dose normalized area under the FVIII activity-time curve for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [17]
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Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [18]
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Dose Normalized Area Under the Curve (DNAUC; Two-stage Chromogenic Assay)
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Assessment method [18]
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Dose normalized area under the FVIII activity-time curve for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [18]
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Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [19]
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Mean Residence Time (MRT; One-stage aPTT Clotting Assay)
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Assessment method [19]
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The average time that a drug molecule is present in the systemic circulation for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [19]
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Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [20]
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Mean Residence Time (MRT; Two-stage Chromogenic Assay)
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Assessment method [20]
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The average time that a drug molecule is present in the systemic circulation for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [20]
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Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [21]
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Incremental Recovery (IR; One-stage aPTT Clotting Assay)
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Assessment method [21]
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The rise in FVIII activity in IU/dL per unit dose administered in IU/kg for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [21]
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Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [22]
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Incremental Recovery (IR; Two-stage Chromogenic Assay)
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Assessment method [22]
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The rise in FVIII activity in IU/dL per unit dose administered in IU/kg for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [22]
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Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [23]
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Time at Maximum Activity (Tmax; One-stage aPTT Clotting Assay)
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Assessment method [23]
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Time at which maximum activity (Cmax) is observed for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [23]
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0
Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [24]
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Time at Maximum Activity (Tmax; Two-stage Chromogenic Assay)
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Assessment method [24]
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Time at which maximum activity (Cmax) is observed for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [24]
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0
Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [25]
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0
Lambda Z (One-stage aPTT Clotting Assay)
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Assessment method [25]
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First order rate constant associated with the terminal portion of the curve (lambda z) for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [25]
0
0
Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [26]
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0
Lambda Z (Two-stage Chromogenic Assay)
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Assessment method [26]
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First order rate constant associated with the terminal portion of the curve (lambda z) for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
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Timepoint [26]
0
0
Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [27]
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0
Volume at Terminal Phase (Vz; One-stage aPTT Clotting Assay)
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Assessment method [27]
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Volume of distribution estimated from the terminal phase for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
Query!
Timepoint [27]
0
0
Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
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Secondary outcome [28]
0
0
Volume at Terminal Phase (Vz; Two-stage Chromogenic Assay)
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Assessment method [28]
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0
Volume of distribution estimated from the terminal phase for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
Query!
Timepoint [28]
0
0
Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
Query!
Secondary outcome [29]
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0
Area Under the Curve to the Last Measurable Timepoint (AUClast; One-stage aPTT Clotting Assay)
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Assessment method [29]
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0
Dose-normalized area under the FVIII activity-time curve to the last measurable timepoint for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
Query!
Timepoint [29]
0
0
Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
Query!
Secondary outcome [30]
0
0
Area Under the Curve to the Last Measurable Timepoint (AUClast; Two-stage Chromogenic Assay)
Query!
Assessment method [30]
0
0
Dose-normalized area under the FVIII activity-time curve to the last measurable timepoint for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
Query!
Timepoint [30]
0
0
Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
Query!
Secondary outcome [31]
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0
Area Under the Curve to Infinity (AUCinf; One-stage aPTT Clotting Assay)
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Assessment method [31]
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0
Dose normalized area under the FVIII activity-time curve to infinity for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
Query!
Timepoint [31]
0
0
Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
Query!
Secondary outcome [32]
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0
Area Under the Curve to Infinity (AUCinf; Two-stage Chromogenic Assay)
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Assessment method [32]
0
0
Dose normalized area under the FVIII activity-time curve to infinity for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
Query!
Timepoint [32]
0
0
Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
Query!
Secondary outcome [33]
0
0
Percentage of AUCinf Extrapolated From the Last Data Point to Infinity (%AUCext; One-stage aPTT Clotting Assay)
Query!
Assessment method [33]
0
0
Percentage of AUCinf extrapolated from the last data point to infinity for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
Query!
Timepoint [33]
0
0
Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
Query!
Secondary outcome [34]
0
0
Percentage of AUCinf Extrapolated From the Last Data Point to Infinity (%AUCext; Two-stage Chromogenic Assay)
Query!
Assessment method [34]
0
0
Percentage of AUCinf extrapolated from the last data point to infinity for participants in the PK subgroup. The 95% confidence interval on the geometric mean is based on the t-statistic back-transformed from the log scale.
Query!
Timepoint [34]
0
0
Baseline (28 ±7 days prior to Day 1) Prestudy FVIII Dosing: predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours post-dose. Day 1 (rFVIIIFc Dosing): predose; 30 ±5 min, 3 hours ±30 min, 24 ±3 hours, 48 ±4 hours, 72 ±7 hours post-dose
Query!
Eligibility
Key inclusion criteria
Key
- Severe hemophilia A defined as <1 IU/dL (<1%) endogenous FVIII
- Male <12 years of age and weight =13 kg
- History of at least 50 documented prior exposure days to FVIII
- No current, or history of, inhibitor development to FVIII
Key
Query!
Minimum age
No limit
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Query!
Maximum age
11
Years
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Query!
Sex
Males
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Other coagulation disorders in addition to Hemophilia A
- History of anaphylaxis associated with any FVIII or IV immunoglobulin administration
NOTE: Other protocol-defined Inclusion/Exclusion criteria may apply.
Query!
Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Query!
Query!
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
Query!
Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/11/2012
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Date of last participant enrolment
Anticipated
Query!
Actual
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Date of last data collection
Anticipated
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Actual
1/12/2013
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Sample size
Target
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Accrual to date
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Final
71
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Recruitment in Australia
Recruitment state(s)
QLD,VIC
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Recruitment hospital [1]
0
0
Research Site - Brisbane
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Recruitment hospital [2]
0
0
Research Site - Melbourne
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Recruitment postcode(s) [1]
0
0
- Brisbane
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Recruitment postcode(s) [2]
0
0
- Melbourne
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Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
California
Query!
Country [2]
0
0
United States of America
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State/province [2]
0
0
Colorado
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Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Illinois
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Indiana
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Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Missouri
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Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Nevada
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Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Ohio
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Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Oregon
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Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
Pennsylvania
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Country [10]
0
0
Hong Kong
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State/province [10]
0
0
Hong Kong
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Country [11]
0
0
Ireland
Query!
State/province [11]
0
0
Dublin
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Country [12]
0
0
Netherlands
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State/province [12]
0
0
Groningen
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Country [13]
0
0
Poland
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State/province [13]
0
0
Lublin
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Country [14]
0
0
South Africa
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State/province [14]
0
0
Johannesburg
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Country [15]
0
0
United Kingdom
Query!
State/province [15]
0
0
Cambridgshire
Query!
Country [16]
0
0
United Kingdom
Query!
State/province [16]
0
0
Hampshire
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Country [17]
0
0
United Kingdom
Query!
State/province [17]
0
0
Glasgow
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Country [18]
0
0
United Kingdom
Query!
State/province [18]
0
0
London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Bioverativ Therapeutics Inc.
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Address
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Country
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Other collaborator category [1]
0
0
Commercial sector/Industry
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Name [1]
0
0
Swedish Orphan Biovitrum
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Address [1]
0
0
Query!
Country [1]
0
0
Query!
Ethics approval
Ethics application status
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Summary
Brief summary
The primary objective of the study is to evaluate the safety of Recombinant Coagulation
Factor VIII Fc Fusion Protein (rFVIIIFc) in previously treated pediatric subjects with
hemophilia A. Secondary objectives of this study in this study population are as follows: to
evaluate the efficacy of rFVIIIFc for prevention and treatment of bleeding episodes; to
evaluate and assess the pharmacokinetics (PK) of rFVIIIFc; and to evaluate rFVIIIFc
consumption for prevention and treatment of bleeding episodes.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01458106
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
0
0
Medical Director
Query!
Address
0
0
Bioverativ Therapeutics Inc.
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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
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01458106
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