Clinical Trial Results:
A Prospective, Multicenter, Open Enrollment Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Summary
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EudraCT number |
2014-003764-20 |
Trial protocol |
Outside EU/EEA |
Global end of trial date |
22 Aug 2011
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Results information
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Results version number |
v1(current) |
This version publication date |
13 Jul 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 |
BI71023_3002
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT00945906 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
CSL Behring LLC
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Sponsor organisation address |
1020 First Avenue, King of Prussia, United States, 19406-0901
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Public contact |
Clinical Trial Disclosure Manager, CSL Behring, clinicaltrials@cslbehring.com
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Scientific contact |
Clinical Trial Disclosure Manager, CSL Behring, clinicaltrials@cslbehring.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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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 |
20 Dec 2011
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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 |
22 Aug 2011
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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 |
The objectives of the study are to provide FXIII Concentrate (Human) to patients in the United States until the product becomes commercially available in the United States as well as to collect additional long-term safety data in this population.
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Protection of trial subjects |
This study was carried out in accordance with the International Conference on Harmonisation (ICH)
Good Clinical Practice guidelines, and standard operating procedures for clinical research and
development at CSL Behring (CSLB).
The study protocol and all amendments were approved by the Independent Ethics Committee(s) (IECs) /
Institutional Review Board(s) (IRBs) of the participating centers.
Before undergoing screening procedures for possible enrollment into the study, subjects were informed,
in an understandable form, about the nature, scope, and possible consequences of the study. The
investigator was responsible for obtaining a subject’s written informed consent to participate in the
study.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
18 Sep 2009
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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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 |
United States: 61
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Worldwide total number of subjects |
61
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EEA total number of subjects |
0
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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) |
4
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Children (2-11 years) |
15
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Adolescents (12-17 years) |
15
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Adults (18-64 years) |
27
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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 |
Subjects who were enrolled in pharmacokinetic (PK) Study BI71023_2002 or Phase 3b Study B171023_3001 were offered enrollment in this study. Enrollment was also offered to subjects who were enrolled in a clinical study conducted under BB-IND 5986 or not currently participating in any other study. | ||||||||||||||||
Pre-assignment
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Screening details |
Eligible subjects were males or females of any age with documented congenital Factor XIII deficiency that required prophylactic treatment with a Factor XIII-containing product. Sixty-one subjects were screened, enrolled, and received at least 1 dose of Factor XIII Concentrate (Human) in the study. | ||||||||||||||||
Period 1
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Period 1 title |
Overall Study (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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Arm title
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Factor XIII | ||||||||||||||||
Arm description |
Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%. | ||||||||||||||||
Arm type |
Experimental | ||||||||||||||||
Investigational medicinal product name |
Factor XIII Concentrate (Human)
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Investigational medicinal product code |
BI71023
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Other name |
Cluvot, Fibrogammin®, Corifact®
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Pharmaceutical forms |
Powder and solvent for solution for injection/infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
Subjects who did not complete PK Study BI71023_2002 or receive at least 3 doses of the investigational medicinal product in the Phase 3b Study BI71023_3001 received Factor XIII Concentrate (Human) at a dose of 40 U/kg by intravenous infusion. Subjects who were enrolled in a clinical study conducted under BB-IND 5986 received Factor XIII Concentrate at a dose of 40 U/kg by intravenous infusion. For all other subjects and all doses after Baseline (Day 0), the dose was guided by the individual subject’s most recent (pre-infusion) trough Factor XIII activity levels, with the objective of dosing every 28 days (4 weeks) to maintain a trough Factor XIII activity level of approximately 5 to 20%.
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Baseline characteristics reporting groups
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Reporting group title |
Factor XIII
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Reporting group description |
Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%. | ||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Factor XIII
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Reporting group description |
Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%. |
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End point title |
Adverse Events [1] | ||||||||||||
End point description |
Number of subjects with any treatment-emergent adverse event (AE), treatment-related AE or serious AE (SAE). Treatment-related AEs are defined as AEs whose relationship to treatment is related, or possibly related and AEs with missing relationship.
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End point type |
Primary
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End point timeframe |
After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
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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: Statistical analyses were not performed in this study. |
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No statistical analyses for this end point |
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End point title |
Hematology and Chemistry Testing | ||||||||||
End point description |
Number of participants with treatment-emergent clinically significant hematology and/or chemistry laboratory parameter values.
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End point type |
Secondary
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End point timeframe |
After the first infusion and at the end-of-study (or withdrawal) visit.
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No statistical analyses for this end point |
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End point title |
Factor XIII Antibody Testing | ||||||||||
End point description |
Number of participants with serum Factor XIII antibodies.
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End point type |
Secondary
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End point timeframe |
Before the first infusion, then every 48 weeks, at the end-of-study (or withdrawal) visit and after a bleeding episode requiring treatment with a Factor XIII -containing product.
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No statistical analyses for this end point |
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End point title |
Factor XIII Concentration | ||||||||||||||||||||||||||||||||||||
End point description |
Trough Factor XIII concentration.
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End point type |
Secondary
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End point timeframe |
Before the first infusion, at 24 and 48 weeks after the first infusion, and at the end-of-study (or withdrawal) visit.
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No statistical analyses for this end point |
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End point title |
Number of Subjects With at Least One Bleeding Episode | ||||||||||
End point description |
Number of subjects with at least one bleeding episode at any time after the first infusion in the study, and the number of subjects with at least one bleeding episode requiring Factor XIII treatment.
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End point type |
Secondary
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End point timeframe |
After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
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No statistical analyses for this end point |
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End point title |
Total Number of Bleeding Episodes | ||||||
End point description |
Number of bleeding episodes at any time after the first infusion in the study.
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End point type |
Secondary
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End point timeframe |
After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
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No statistical analyses for this end point |
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Adverse events information
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Timeframe for reporting adverse events |
After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
12.0
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Reporting groups
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Reporting group title |
Factor XIII
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Reporting group description |
Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Frequency threshold for reporting non-serious adverse events: 4% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
None reported |