Clinical Trial Results:
Efficacy and safety study of vWF SD-35-DH (WILFACTIN) in children under 6 years of age
Summary
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EudraCT number |
2004-005051-34 |
Trial protocol |
BE |
Global end of trial date |
05 Aug 2014
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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 |
22 Feb 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 |
42-73-305
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
- | ||
WHO universal trial number (UTN) |
- | ||
Other trial identifiers |
WIL1-0305: WIL1-0305 | ||
Sponsors
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Sponsor organisation name |
LFB Biotechnologies
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Sponsor organisation address |
3 Avenue des Tropiques , COURTABOEUF, France, 91930
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Public contact |
Françoise BRIDEY, LFB Biotechnologies, 33 169827010,
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Scientific contact |
Françoise BRIDEY, LFB Biotechnologies, 33 169827010,
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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? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
04 Dec 2014
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
05 Aug 2014
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Global end of trial reached? |
Yes
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Global end of trial date |
05 Aug 2014
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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 |
Main objective : to evaluate, in children presenting with inherited von Willebrand factor (VWF) deficiencies, the biological and clinical efficacy of WILFACTIN for the treatment of bleeding episodes and for the prevention of haemorrhages during surgery or invasive procedures when desmopressin is ineffective or contraindicated.
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Protection of trial subjects |
Blood sampling usually done for laboratory testing presents a potential discomfort and the 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 minimizes potential side effects in children.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
01 Feb 2006
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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 |
18 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: 3
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Country: Number of subjects enrolled |
Belgium: 1
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Country: Number of subjects enrolled |
Greece: 1
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Country: Number of subjects enrolled |
Tunisia: 4
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Worldwide total number of subjects |
9
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EEA total number of subjects |
5
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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) |
2
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Children (2-11 years) |
7
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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 |
9 patients were included at 5 study centers in Belgium, Greece, Poland and Tunisia. | ||||||
Pre-assignment
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Screening details |
- | ||||||
Pre-assignment period milestones
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Number of subjects started |
10 [1] | ||||||
Number of subjects completed |
9 | ||||||
Pre-assignment subject non-completion reasons
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Reason: Number of subjects |
selection criteria missing: 1 | ||||||
Notes [1] - The number of subjects reported to have started the pre-assignment period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same. Justification: 1 subject withdrawn after the pre-assigment period (1 selection criteria missing). |
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Period 1
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Period 1 title |
Inclusion visit
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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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Wilfactin treatment | ||||||
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 |
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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 |
No administration at inclusion.
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Period 2
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Period 2 title |
Recovery study period
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Is this the baseline period? |
No | ||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||
Arms
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Arm title
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Wilfactin treatment | ||||||
Arm description |
- | ||||||
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 |
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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 |
100 IU/kg by intravenous route
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Period 3
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Period 3 title |
Efficacy period
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Is this the baseline period? |
No | ||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||
Arms
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Arm title
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Efficacy | ||||||
Arm description |
- | ||||||
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 |
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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 |
For surgical procedures and treatment of bleeding episodes
- when the recovery of von Willebrand factor is known: individualized dosing to provide plasma level of 100 % (VWF:RCo) ,
- when recovery information of von Willebrand factor was not available: 60-100 IU/kg
The treatment duration depends on the clinical status of the subject and the baseline blood levels of VWF and factor VIII.
For long-term prophylaxis, 50 IU/kg once per week to 30 IU/kg every other day to minimize spontaneous bleeding episodes.
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Baseline characteristics reporting groups
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Reporting group title |
Inclusion visit
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Reporting group description |
- | ||||||||||||||||||||||||||||||||||||
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Subject analysis sets
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Subject analysis set title |
TTS
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Subject analysis set type |
Full analysis | ||||||||||||||||||||||||||||||||||||
Subject analysis set description |
Total Treated Set
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End points reporting groups
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Reporting group title |
Wilfactin treatment
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Reporting group description |
- | ||
Reporting group title |
Wilfactin treatment
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Reporting group description |
- | ||
Reporting group title |
Efficacy
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Reporting group description |
- | ||
Subject analysis set title |
TTS
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Subject analysis set type |
Full analysis | ||
Subject analysis set description |
Total Treated Set
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End point title |
Percentage of Excellent/Good response [1] | ||||||||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Hemostasis in bleeding episodes and surgical/invasive procedures was evaluated by the investigator at the end of the bleeding episode or course of treatment
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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 analysis |
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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 |
throughout the study
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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 |
Total Treated Set
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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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08 Jun 2007 |
To prolong the study recuitment period.
To add a recovery study to be performed after 6 months for children with type 3 VWD.
To extand the number of centres including the participation of other countries. |
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12 Nov 2007 |
To change the name of the sponsor (LFB BIOTECHNOLOGIES ).
To prolong the patient recruitment period (+ 6 month) and to add vWF inhibitor information in accordance with the guidelines on clinical investigation of human plasma derived von Willebrand factor products. |
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12 Nov 2007 |
To allow the patient to continue the IMP after 18 months follow-up under the same conditions if the product is registered but not yet commercialized.
To specify the 2nd recovery study in the information patient sheet related to amendment n°4.
To change the clinical project manager and extend the number of center and countries.
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05 Sep 2008 |
To prolong the patient recruitment period; To update the procedure for SAE reporting; To add an interim analysis in Q2 2008.
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15 Mar 2011 |
To prolong the recruitment period; To transfer storageand distribution to a sub-contractor; To clarify criteria for recovery study.
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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 |