Clinical Trial Results:
BAX326 (recombinant factor IX): a phase 2/3, prospective, uncontrolled, multicenter study evaluating pharmacokinetics, efficacy, safety, and immunogenicity in previously treated pediatric patients with severe (FIX level <1%) or moderately severe (FIX level 1-2%) hemophilia B
Summary
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EudraCT number |
2011-002437-19 |
Trial protocol |
GB BG |
Global end of trial date |
14 May 2013
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Results information
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Results version number |
v1(current) |
This version publication date |
18 Feb 2016
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First version publication date |
06 Aug 2015
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Other versions |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
251101
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT01488994 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
Baxalta US Inc.
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Sponsor organisation address |
One Baxter Way, Westlake Village, United States, CA 91362
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Public contact |
Clinical Trial Registries and Results Disclosure, Baxalta US Inc., ClinicalTrialsDisclosure@baxalta.com
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Scientific contact |
Clinical Trial Registries and Results Disclosure, Baxalta US Inc., ClinicalTrialsDisclosure@baxalta.com
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Sponsor organisation name |
Baxalta Innovations GmbH
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Sponsor organisation address |
Industriestrasse 67, Vienna, Austria, 1221
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Public contact |
Clinical Trial Registries and Results Disclosure, Baxalta Innovations GmbH, ClinicalTrialsDisclosure@baxalta.com
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Scientific contact |
Clinical Trial Registries and Results Disclosure, Baxalta Innovations GmbH, ClinicalTrialsDisclosure@baxalta.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
Yes
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EMA paediatric investigation plan number(s) |
EMEA-001139-PIP01-11 | ||
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
26 Aug 2013
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
14 May 2013
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
To evaluate all adverse events possibly or probably related to BAX326
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Protection of trial subjects |
This study was conducted in accordance with the clinical protocol, the International Conference on Harmonization Guideline for Good Clinical Practice E6 (ICH GCP, April 1996), Title 21 of the US Code of Federal Regulations (US CFR), the European Clinical Trial Directive (2001/20/EC and 2005/28/EC), and applicable national and local regulatory requirements.
There were 2 age cohorts: <6 years and 6 to <12 years. To reduce the burden of frequent blood sampling on the individual subject for the pharmacokinetic assessment (total of 7 post-infusion sampling time points over 72 hours), subjects within each age cohort were randomized to one of 2 blood sampling sequences of 4 post-infusion blood sampling time points each.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
20 Dec 2011
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Poland: 5
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Country: Number of subjects enrolled |
Romania: 5
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Country: Number of subjects enrolled |
Ukraine: 3
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Country: Number of subjects enrolled |
United Kingdom: 2
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Country: Number of subjects enrolled |
Russian Federation: 7
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Country: Number of subjects enrolled |
India: 1
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Worldwide total number of subjects |
23
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EEA total number of subjects |
12
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
23
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
0
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
Enrollment was conducted at 11 clinical sites in 6 countries (United Kingdom, Poland, Romania, Russian Federation, Ukraine, India). A total of 23 subjects were enrolled in the study. Of these, 11 were <6 years of age and 12 were 6 to <12 years of age. | |||||||||||||||
Pre-assignment
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Screening details |
- | |||||||||||||||
Pre-assignment period milestones
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Number of subjects started |
23 | |||||||||||||||
Number of subjects completed |
23 | |||||||||||||||
Period 1
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Period 1 title |
Overall trial (overall period)
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Is this the baseline period? |
Yes | |||||||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | |||||||||||||||
Arms
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Are arms mutually exclusive |
Yes
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Arm title
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Paediatric subjects <6 years of age | |||||||||||||||
Arm description |
Subjects in this cohort had their pharmacokinetic infusion with BAX326 in the morning and were assigned to the following 4 post-infusion blood sampling time points: 15-30 min, 7±1 hour(s), anytime during the 2nd day, anytime during the 3rd day | |||||||||||||||
Arm type |
Experimental | |||||||||||||||
Investigational medicinal product name |
BAX326 (recombinant factor IX)
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Investigational medicinal product code |
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Other name |
Rixubis
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Pharmaceutical forms |
Powder and solvent for solution for injection
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Routes of administration |
Intravenous use
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Dosage and administration details |
Subjects underwent a pharmacokinetic evaluation with BAX326 (1 infusion) which was followed by a twice weekly prophylactic treatment with BAX326. Bleeding episodes were also treated with BAX326.
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Arm title
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Paediatric subjects 6 to <12 years of age | |||||||||||||||
Arm description |
Subjects in this cohort had their pharmacokinetic infusion with BAX326 in the afternoon and were assigned to the following 4 post-infusion blood sampling time points: 15-30 min, 4±1 hour(s), anytime during the 2nd day, morning of the 4th day | |||||||||||||||
Arm type |
Experimental | |||||||||||||||
Investigational medicinal product name |
BAX326 (recombinant factor IX)
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Investigational medicinal product code |
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Other name |
Rixubis
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Pharmaceutical forms |
Powder and solvent for solution for injection
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Routes of administration |
Intravenous use
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Dosage and administration details |
Subjects underwent a pharmacokinetic evaluation with BAX326 (1 infusion) which was followed by a twice weekly prophylactic treatment with BAX326. Bleeding episodes were also treated with BAX326.
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Baseline characteristics reporting groups
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Reporting group title |
Paediatric subjects <6 years of age
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Reporting group description |
Subjects in this cohort had their pharmacokinetic infusion with BAX326 in the morning and were assigned to the following 4 post-infusion blood sampling time points: 15-30 min, 7±1 hour(s), anytime during the 2nd day, anytime during the 3rd day | ||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Paediatric subjects 6 to <12 years of age
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Reporting group description |
Subjects in this cohort had their pharmacokinetic infusion with BAX326 in the afternoon and were assigned to the following 4 post-infusion blood sampling time points: 15-30 min, 4±1 hour(s), anytime during the 2nd day, morning of the 4th day | ||||||||||||||||||||||||||||||||||||||||||||||||||||
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Subject analysis sets
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Subject analysis set title |
Full Analysis Set
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Subject analysis set type |
Full analysis | ||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
Comprised all subjects who received at least one infusion of investigational product
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Subject analysis set title |
Pharmacokinetic Full Analysis Set
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Subject analysis set type |
Sub-group analysis | ||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
Comprised all subjects who had at least one plasma factor IX activity level available during post-infusion time points
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End points reporting groups
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Reporting group title |
Paediatric subjects <6 years of age
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Reporting group description |
Subjects in this cohort had their pharmacokinetic infusion with BAX326 in the morning and were assigned to the following 4 post-infusion blood sampling time points: 15-30 min, 7±1 hour(s), anytime during the 2nd day, anytime during the 3rd day | ||
Reporting group title |
Paediatric subjects 6 to <12 years of age
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Reporting group description |
Subjects in this cohort had their pharmacokinetic infusion with BAX326 in the afternoon and were assigned to the following 4 post-infusion blood sampling time points: 15-30 min, 4±1 hour(s), anytime during the 2nd day, morning of the 4th day | ||
Subject analysis set title |
Full Analysis Set
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Subject analysis set type |
Full analysis | ||
Subject analysis set description |
Comprised all subjects who received at least one infusion of investigational product
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Subject analysis set title |
Pharmacokinetic Full Analysis Set
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Subject analysis set type |
Sub-group analysis | ||
Subject analysis set description |
Comprised all subjects who had at least one plasma factor IX activity level available during post-infusion time points
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End point title |
Adverse events (AEs) possibly or probably related to BAX326 [1] | ||||||||||||
End point description |
Probable, possible, or unknown causality assessment of an AE were to be counted as “related". AEs that occurred during or after treatment application were presented in summary tables. An overview summary table presented the number (%) of AEs, the number (%) of subjects with AEs by seriousness, severity, and relationship to the study product. Descriptive statistics were presented by age stratum.
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End point type |
Primary
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End point timeframe |
Approximately 7 months per subject
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Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Per protocol, descriptive statistics were collected for this endpoint. |
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No statistical analyses for this end point |
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End point title |
Pharmacokinetics (PK): Total Area under the plasma concentration versus time curve per dose (Total AUC/dose) | ||||||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
72 hours
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No statistical analyses for this end point |
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End point title |
PK: Mean residence time (MRT) | ||||||||||||||||
End point description |
Computed as total area under the first moment curve (total AUMC) divided by the total area under the concentration versus time curve (total AUC)
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End point type |
Secondary
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End point timeframe |
72 hours
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No statistical analyses for this end point |
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End point title |
PK: Factor IX (FIX) clearance (CL) | ||||||||||||||||
End point description |
Computed as the dose divided by total AUC
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End point type |
Secondary
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End point timeframe |
72 hours
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No statistical analyses for this end point |
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End point title |
PK: Incremental recovery (IR) | ||||||||||||||||
End point description |
Calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose
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End point type |
Secondary
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End point timeframe |
30 minutes
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Notes [2] - 1 subject <6 yrs had a biologically implausible FIX level at 15-30 min post-infusion -> was excluded |
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No statistical analyses for this end point |
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End point title |
PK: Elimination phase half-life (T 1/2) | ||||||||||||||||
End point description |
Calculated as log_e2/λ, where λ is the regression slope in the terminal phase of the least absolute deviations regression model
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End point type |
Secondary
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End point timeframe |
72 hours
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No statistical analyses for this end point |
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End point title |
PK: Volume of distribution at steady state (Vss) | ||||||||||||||||
End point description |
Computed as Clearance (CL) * Mean residence time (MRT)
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End point type |
Secondary
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End point timeframe |
72 hours
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No statistical analyses for this end point |
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End point title |
PK: Incremental recovery (IR) over time | ||||||||||||||||||||||||||||
End point description |
Calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose
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End point type |
Secondary
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End point timeframe |
Week 5, Week 13, Week 26 and study completion/termination visit (for participants receiving BAX326 beyond Week 26)
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Notes [3] - Only 10 subjects in the <6-year age group were analyzed for Week 13 and Week 26. [4] - Only 11 subjects in the 6-to-<12-year age group were analyzed for Week 13 and Week 26. [5] - Only 21 subjects were analyzed for Week 13 and Week 26. |
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No statistical analyses for this end point |
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End point title |
Haemostatic efficacy: Treatment of bleeding episodes: number of infusions per bleeding episode | ||||||||||||||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Approximately 7 months per subject
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Notes [6] - 7 subjects <6 yrs had total of 11 bleeding episodes after first BAX326 exposure which were treated [7] - 7 subjects 6 - <12 yrs had 15 bleeding episodes after first BAX326 exposure which were treated [8] - 14 subjects in FAS had total of 26 bleeding episodes after first BAX326 exposure which were treated |
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No statistical analyses for this end point |
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End point title |
Haemostatic efficacy: Treatment of bleeding episodes: overall haemostatic efficacy rating at resolution of bleed | ||||||||||||||||||||||||||||
End point description |
Rating Scale for Treatment of BEs (4-point ordinal scale):
- Excellent: Full relief of pain and cessation of objective signs of bleeding (eg, swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) after a single infusion. No additional infusion required for the control of bleeding. Administration of further infusions to maintain hemostasis did not affect this scoring.
- Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion. Possibly requires more than 1 infusion for complete resolution.
- Fair: Probable and/or slight relief of pain and slight improvement in signs of bleeding after single infusion. Required more than 1 infusion for complete resolution.
- None: No improvement or condition worsens.
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End point type |
Secondary
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End point timeframe |
Approximately 7 months per subject
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Notes [9] - 7 subjects <6 yrs had total of 11 bleeding episodes after first BAX326 exposure which were treated [10] - 7 subjects 6 - <12 yrs had 15 bleeding episodes after first BAX326 exposure which were treated [11] - 14 subjects in FAS had total of 26 bleeding episodes after first BAX326 exposure which were treated |
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No statistical analyses for this end point |
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End point title |
Haemostatic efficacy: Prophylaxis: annualized bleeding rate (ABR) | ||||||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Approximately 7 months per subject
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Notes [12] - All 11 subjects <6 yrs had at least 3 months of prophylactic treatment with BAX326 [13] - All 12 subjects 6 - <12 yrs had at least 3 months of prophylactic treatment with BAX326 [14] - All 23 subjects in the FAS had at least 3 months of prophylactic treatment with BAX326 |
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No statistical analyses for this end point |
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End point title |
Consumption of BAX326: number of infusions per month and per year (annualized) | ||||||||||||||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Approximately 7 months per subject
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No statistical analyses for this end point |
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End point title |
Consumption of BAX326: weight-adjusted consumption per month and per year (annualized) | ||||||||||||||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Approximately 7 months per subject
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No statistical analyses for this end point |
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End point title |
Consumption of BAX326: weight-adjusted consumption per event | ||||||||||||||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Approximately 7 months per subject
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Notes [15] - All 11 subjects received prophylactic infusions but only 7 received treatment for bleeding episodes [16] - All 12 subjects received prophylactic infusions but only 7 received treatment for bleeding episodes [17] - All 23 subjects received prophylactic infusions but only 14 received treatment for bleeding episodes |
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No statistical analyses for this end point |
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End point title |
Safety and Immunogenicity: Development of inhibitory antibodies to factor IX (FIX) | ||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Approximately 7 months per subject
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No statistical analyses for this end point |
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End point title |
Safety and Immunogenicity: Development of total binding antibodies to FIX | ||||||||||||
End point description |
If more than 2-dilution increase as compared to pre-study level at screening and titers verified for specificity in the confirmatory assay
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End point type |
Secondary
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End point timeframe |
Approximately 7 months per subject
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No statistical analyses for this end point |
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End point title |
Safety: Occurrence of severe allergic reactions (eg, anaphylaxis) | ||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Approximately 7 months per subject
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No statistical analyses for this end point |
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End point title |
Safety: Occurrence of thrombotic events | ||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Approximately 7 months per subject
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No statistical analyses for this end point |
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End point title |
Safety: Clinically significant (CS) changes in routine laboratory parameters (haematology and clinical chemistry), and vital signs | ||||||||||||||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Approximately 7 months per subject
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No statistical analyses for this end point |
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End point title |
Safety and Immunogenicity: Development of antibodies to Chinese hamster ovary (CHO) proteins and recombinant furin (rFurin) | ||||||||||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Approximately 7 months per subject
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No statistical analyses for this end point |
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End point title |
Health-related Quality of Life (HRQoL): Peds-QL: Change from baseline in total score | ||||||||||||||
End point description |
The Peds-QL is a generic HR QoL instrument designed specifically for a paediatric population. It captures the following domains: general health/activities, feelings/emotional, social functioning, school functioning. For this study, the Peds-QL questionnaires for subjects 2 to 7 years of age (parent-proxy versions for age groups 2-4 years and 5-7 years) and for subjects 8 to 12 years of age were used. Higher scores indicate better quality of life for all domains of the Peds-QL.
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End point type |
Secondary
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End point timeframe |
Approximately 6 months per subject
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Notes [18] - Results for 4 subjects 2-4 years, 2 subjects 5-7 years and 10 subjects 8-12 years |
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No statistical analyses for this end point |
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End point title |
HRQoL: Haemo-QoL (short version): Change from baseline in total score | ||||||||
End point description |
The Haemo-QOL instrument assesses specific aspects of dealing with haemophilia. The areas covered by this instrument are: physical health, sports/leisure, school, dealing with haemophilia, and outlook for the future. For this study, the Haemo-QoL for subjects 8-16 years of age was used. Higher scores indicate worse quality of life.
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End point type |
Secondary
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End point timeframe |
Approximately 6 months per subject
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Notes [19] - Results are only available for 10 subjects >8 years of age. |
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No statistical analyses for this end point |
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End point title |
HRQoL: Number of hospitalizations | ||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Approximately 6 months per subject
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No statistical analyses for this end point |
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End point title |
HRQoL: Length of hospitalization | ||||||||||||||||||||||||
End point description |
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End point type |
Secondary
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End point timeframe |
Approximately 6 months per subject
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|||||||||||||||||||||||||
Notes [20] - One subject in the <6-year age group underwent hospitalization by Week 13. [21] - Of 2 subjects 6 - <12 yrs who were hospitalized, 1 had data for Week 13 and the other for Week 26 |
|||||||||||||||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
HRQoL: Unscheduled visits to a doctor´s office | ||||||||||||
End point description |
|||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Approximately 6 months per subject
|
||||||||||||
|
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
HRQoL: Emergency Room (ER) visits | ||||||||||||
End point description |
|||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Approximately 6 months per subject
|
||||||||||||
|
|||||||||||||
Notes [22] - ER visits were recorded for 3 subjects in the <6-year age cohort. [23] - ER visits were recorded for 3 subjects in the 6-to-<12-year age cohort. [24] - ER visits were recorded for a total of 6 subjects. |
|||||||||||||
No statistical analyses for this end point |
|
|||||||||||||
End point title |
HRQoL: Days lost from school | ||||||||||||
End point description |
|||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Approximately 6 months per subject
|
||||||||||||
|
|||||||||||||
Notes [25] - 2 subjects in the <6-year age group missed days from school. [26] - 8 subjects in the 6-to-<12-year age group missed days from school. [27] - A total of 10 subjects missed days from school. |
|||||||||||||
No statistical analyses for this end point |
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Adverse events information
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Timeframe for reporting adverse events |
Approximately 7 months per subject
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Assessment type |
Non-systematic | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary name |
MedDRA | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
N/A
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting groups
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Paediatric subjects less than 6 years of age
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
Subjects in this cohort had their pharmacokinetic infusion with BAX326 in the morning and were assigned to the following 4 post-infusion blood sampling time points: 15-30 min, 7±1 hour(s), anytime during the 2nd day, anytime during the 3rd day | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Paediatric subjects between 6 and less than 12 years of age
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
Subjects in this cohort had their pharmacokinetic infusion with BAX326 in the afternoon and were assigned to the following 4 post-infusion blood sampling time points: 15-30 min, 4±1 hour(s), anytime during the 2nd day, anytime during the 3rd day | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Frequency threshold for reporting non-serious adverse events: 5% | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|
|||
Substantial protocol amendments (globally) |
|||
Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
|||
Were there any global interruptions to the trial? No | |||
Limitations and caveats |
|||
Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
None reported |