Clinical Trial Results:
EFFICACY AND SAFETY STUDY OF vWF SD-35-DH (WILFACTIN) IN PATIENTS UNDER LONG-TERM PROPHYLAXIS
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Summary
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EudraCT number |
2005-001746-17 |
Trial protocol |
BE PL |
Global end of trial date |
17 Jul 2012
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Results information
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Results version number |
v1(current) |
This version publication date |
30 Jun 2016
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First version publication date |
04 Jul 2015
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Other versions |
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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 |
42-73-406
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
- | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
LFB Biotechnologies
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Sponsor organisation address |
3 Avenue des Tropiques, Les Ulis, COURTABOEUF, France, 91930
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Public contact |
Global Clinical Development Leader, LFB Biotechnologies, 33 169825656,
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Scientific contact |
Global Clinical Development Leader, LFB Biotechnologies, 33 169825656,
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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 |
17 Jan 2013
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
17 Jul 2012
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Global end of trial reached? |
Yes
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Global end of trial date |
17 Jul 2012
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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 the efficacy of vWF SD-35-DH (WILFACTIN) in a long-term prophylaxis treatment regimen for the prevention of haemorrhages.
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Protection of trial subjects |
Blood sampling usually done for laboratory testing presents a potential discomfort and the possible associated risks are
slight pain at the site, feeling light-headed, bruising and, exceptionally, local infection as well as bleeding from the site
of the puncture. However, all precautionary measures will be taken to minimize potential side effects.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
30 Nov 2005
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Long term follow-up planned |
Yes
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Long term follow-up rationale |
Safety, Efficacy | ||
Long term follow-up duration |
12 Months | ||
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 |
Poland: 1
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Country: Number of subjects enrolled |
Belgium: 3
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Country: Number of subjects enrolled |
Tunisia: 6
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Worldwide total number of subjects |
10
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EEA total number of subjects |
4
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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) |
3
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Adolescents (12-17 years) |
2
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Adults (18-64 years) |
5
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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 |
10 patients were included at 5 study centers in Belgium, Poland and Tunisia. | ||||||||||
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Pre-assignment
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Screening details |
- | ||||||||||
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Pre-assignment period milestones
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Number of subjects started |
10 | ||||||||||
Number of subjects completed |
8 | ||||||||||
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Pre-assignment subject non-completion reasons
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Reason: Number of subjects |
screening failure: 2 | ||||||||||
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Period 1
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Period 1 title |
Prophylaxis study follow-up (overall period)
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Is this the baseline period? |
Yes | ||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||
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Arms
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Arm title
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WILFACTIN | ||||||||||
Arm description |
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Arm type |
Experimental | ||||||||||
Investigational medicinal product name |
WILFACTIN
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Investigational medicinal product code |
vWF SD-35-DH
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Other name |
Human von willebrand factor
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Pharmaceutical forms |
Powder and solvent for solution for infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
Individualized dose for long-term prophylaxis regimen according to historical profile of recurrent bleeding.
• 60 IU/kg in subjects with a predisposition to "mucosal" bleeds:
- twice per week if VWF:RCo level at 48 hours post-infusion was > 10 IU/dL at the time of the recovery test.
- 3 times per week if VWF:RCo level at 48 hours post-infusion was < 10 IU/dL
• 40 IU/kg in subjects with a predisposition to ‘non-mucosal’ bleeds:
- twice per week if FVIII:C level at 48 hours post-infusion was >20 IU/dL at the time of the recovery test
- 3 times per week if FVIII:C level at 48 hours post-infusion was <20 IU/dL
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| Notes [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same. Justification: One subject not completed the baseline period because of a not fatal adverse event. |
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Baseline characteristics reporting groups
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Reporting group title |
Prophylaxis study follow-up
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Reporting group description |
- | |||||||||||||||||||||||||||||||||||||||
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Subject analysis sets
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Subject analysis set title |
Total Treated Set
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Subject analysis set type |
Safety analysis | |||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
Eight subjects received study drug and were included in the safety population or TTS
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Subject analysis set title |
FAS set
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Subject analysis set type |
Full analysis | |||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
Seven subjects completed the study and were included in the FAS. One subject was excluded from the efficacy analyses because he withdrew prematurely after the first administration of the study drug.
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End points reporting groups
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Reporting group title |
WILFACTIN
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Reporting group description |
- | ||
Subject analysis set title |
Total Treated Set
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Subject analysis set type |
Safety analysis | ||
Subject analysis set description |
Eight subjects received study drug and were included in the safety population or TTS
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Subject analysis set title |
FAS set
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Subject analysis set type |
Full analysis | ||
Subject analysis set description |
Seven subjects completed the study and were included in the FAS. One subject was excluded from the efficacy analyses because he withdrew prematurely after the first administration of the study drug.
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End point title |
Reduction of haemorrages [1] | ||||||||
End point description |
All breakthrough bleeding episodes requiring VWF treatment or not were analysed.
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End point type |
Primary
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End point timeframe |
ABR calculated at the end of the study compared with historical ABR the year before prophylaxis
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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: Descriptive analyse |
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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 |
All included subjects (8) received at least one infusion; all were included in the safety analysis. Subjects were followed after the first infusion (recovery test) between 0 and 54.8 months (median: 27.3 months).
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
11.1
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Reporting groups
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Reporting group title |
safety group
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Reporting group description |
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| Frequency threshold for reporting non-serious adverse events: 3% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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27 Oct 2006 |
MSI for information. To increase the number of patients |
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18 Dec 2006 |
To allow the patients at the study end to continue the study drug up to the commercialisation.
Add details regarding the bleeding episodes as inclusion criteria.
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13 Jun 2007 |
Addition of a new center Dr El Khorassani, Maroc and LFB status = LFB SA |
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26 Nov 2007 |
Prolong study recruitment period (+ 6 months), extend the number of centres (countries: Tunisia and Poland). To change the sponsor name (LFB BIOTECHNOLOGIES) . |
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03 Sep 2008 |
This amendment concerns the Study Protocol.
The main objective is the following:
-to prolong the patient recruitment period,
-to update the procedure for reporting Serious Adverse Events (SAE),
-to add an intermediary analysis in Q4 2008,
-to specify change of clinical development director,
-to specify change of clinical project manager,
-It also includes the correction of typing errors.
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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 | |||
