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Clinical Trial Results:
Efficacy and safety study of vWF SD-35-DH (WILFACTIN) in children under 6 years of age

Summary
EudraCT number
 2004-005051-34
Trial protocol
 BE  
Global end of trial date
05 Aug 2014

Results information
Results version number
 v1(current)
This version publication date
30 Jun 2016
First version publication date
22 Feb 2015
Other versions

Trial information

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Trial identification
Sponsor protocol code
42-73-305
Additional study identifiers
ISRCTN number
 -
US NCT number
 -
WHO universal trial number (UTN)
 -
Other trial identifiers
 WIL1-0305: WIL1-0305
Sponsors
Sponsor organisation name
LFB Biotechnologies
Sponsor organisation address
3 Avenue des Tropiques , COURTABOEUF, France, 91930
Public contact
Françoise BRIDEY, LFB Biotechnologies, 33 169827010,
Scientific contact
Françoise BRIDEY, LFB Biotechnologies, 33 169827010,
Paediatric regulatory details
Is trial part of an agreed paediatric investigation plan (PIP)
No
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
No
Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
No
Results analysis stage
Analysis stage
Final
Date of interim/final analysis
04 Dec 2014
Is this the analysis of the primary completion data?
Yes
Primary completion date
05 Aug 2014
Global end of trial reached?
Yes
Global end of trial date
05 Aug 2014
Was the trial ended prematurely?
No
General information about the trial
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.
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.
Background therapy
 -
Evidence for comparator
 -
Actual start date of recruitment
01 Feb 2006
Long term follow-up planned
Yes
Long term follow-up rationale
 Safety, Efficacy
Long term follow-up duration
 18 Months
Independent data monitoring committee (IDMC) involvement?
No
Population of trial subjects
Number of subjects enrolled per country
Country: Number of subjects enrolled
Tunisia: 4
Country: Number of subjects enrolled
Belgium: 1
Country: Number of subjects enrolled
Greece: 1
Country: Number of subjects enrolled
Poland: 3
Worldwide total number of subjects
9
EEA total number of subjects
5
Number of subjects enrolled per age group
In utero
0
Preterm newborn - gestational age
0
Newborns (0-27 days)
0
Infants and toddlers (28 days-23 months)
2
Children (2-11 years)
7
Adolescents (12-17 years)
0
Adults (18-64 years)
0
From 65 to 84 years
0
85 years and over
0

Subject disposition

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Recruitment
Recruitment details
 9 patients were included at 5 study centers in Belgium, Greece, Poland and Tunisia.

Pre-assignment
Screening details
 -

Pre-assignment period milestones
Number of subjects started
 10 [1]
Number of subjects completed
 9

Pre-assignment subject non-completion reasons
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).
Period 1
Period 1 title
Inclusion visit
Is this the baseline period?
 Yes
Allocation method
Not applicable
Blinding used
 Not blinded

Arms
Arm title
 Wilfactin treatment
Arm description
 -
Arm type
 Experimental

Investigational medicinal product name
Wilfactin
Investigational medicinal product code
vWF SD-35-DH
Other name
Pharmaceutical forms
Powder and solvent for solution for infusion
Routes of administration
Intravenous use
Dosage and administration details
No administration at inclusion.

Number of subjects in period 1
Wilfactin treatment
Started
9
Completed
9
Period 2
Period 2 title
Recovery study period
Is this the baseline period?
 No
Allocation method
Not applicable
Blinding used
 Not blinded

Arms
Arm title
 Wilfactin treatment
Arm description
 -
Arm type
 Experimental

Investigational medicinal product name
Wilfactin
Investigational medicinal product code
vWF SD-35-DH
Other name
Pharmaceutical forms
Powder and solvent for solution for infusion
Routes of administration
Intravenous use
Dosage and administration details
100 IU/kg by intravenous route

Number of subjects in period 2
Wilfactin treatment
Started
9
Completed
9
Period 3
Period 3 title
Efficacy period
Is this the baseline period?
 No
Allocation method
Not applicable
Blinding used
 Not blinded

Arms
Arm title
 Efficacy
Arm description
 -
Arm type
 Experimental

Investigational medicinal product name
Wilfactin
Investigational medicinal product code
vWF SD-35-DH
Other name
Pharmaceutical forms
Powder and solvent for solution for infusion
Routes of administration
Intravenous use
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.

Number of subjects in period 3
Efficacy
Started
9
Completed
9

Baseline characteristics

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Baseline characteristics reporting groups
Reporting group title
Inclusion visit
Reporting group description
 -

Reporting group values
 Inclusion visit Total
Number of subjects
 9 9
Age categorical
Units: Subjects
    less than 6 years old
 9 9
Age continuous
Units: years
    median (full range (min-max))
 2 (0 to 5) -
Gender categorical
Units: Subjects
    Female
 4 4
    Male
 5 5
Subject analysis sets

Subject analysis set title
TTS
Subject analysis set type
 Full analysis
Subject analysis set description
Total Treated Set

Subject analysis sets values
 TTS
Number of subjects
9
Age categorical
Units: Subjects
    less than 6 years old
9
Age continuous
Units: years
    median (full range (min-max))
2 (0 to 5)
Gender categorical
Units: Subjects
    Female
4
    Male
5

End points

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End points reporting groups
Reporting group title
Wilfactin treatment
Reporting group description
 -
Reporting group title
Wilfactin treatment
Reporting group description
 -
Reporting group title
Efficacy
Reporting group description
 -

Subject analysis set title
TTS
Subject analysis set type
 Full analysis
Subject analysis set description
Total Treated Set

Primary: Percentage of Excellent/Good response

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End point title
 Percentage of Excellent/Good response [1]
End point description
End point type
Primary
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
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
End point values
 Efficacy
Number of subjects analysed
9
Units: Four-point scale
    Excellent
49
    Good
43
    Moderate
8
    None
0
No statistical analyses for this end point

Adverse events

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Adverse events information
Timeframe for reporting adverse events
throughout the study
Assessment type
 Systematic
Dictionary used for adverse event reporting
Dictionary name
 MedDRA
Dictionary version
11.1
Reporting groups
Reporting group title
Total Treated Set
Reporting group description
 -

Serious adverse events
 Total Treated Set
Total subjects affected by serious adverse events
     subjects affected / exposed
3 / 9 (33.33%)
     number of deaths (all causes)
0
     number of deaths resulting from adverse events
0
Respiratory, thoracic and mediastinal disorders
Lung disorder
     subjects affected / exposed
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 1
     deaths causally related to treatment / all
0 / 0
Eye disorders
Eyelid oedema
     subjects affected / exposed
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 1
     deaths causally related to treatment / all
0 / 0
General disorders and administration site conditions
Gait disturbance
     subjects affected / exposed
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 1
     deaths causally related to treatment / all
0 / 0
Infections and infestations
Pneumonia
     subjects affected / exposed
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 1
     deaths causally related to treatment / all
0 / 0
Hordeolum
     subjects affected / exposed
1 / 9 (11.11%)
     occurrences causally related to treatment / all
0 / 1
     deaths causally related to treatment / all
0 / 0
Frequency threshold for reporting non-serious adverse events: 3%
Non-serious adverse events
 Total Treated Set
Total subjects affected by non serious adverse events
     subjects affected / exposed
9 / 9 (100.00%)
Injury, poisoning and procedural complications
Face injury
     subjects affected / exposed
4 / 9 (44.44%)
     occurrences all number
6
Gingival injury
     subjects affected / exposed
2 / 9 (22.22%)
     occurrences all number
6
Limb injury
     subjects affected / exposed
3 / 9 (33.33%)
     occurrences all number
6
Traumatic haematoma
     subjects affected / exposed
4 / 9 (44.44%)
     occurrences all number
11
Mouth injury
     subjects affected / exposed
3 / 9 (33.33%)
     occurrences all number
5
Surgical and medical procedures
Hepatitis A immunisation
     subjects affected / exposed
7 / 9 (77.78%)
     occurrences all number
7
Infections and infestations
Bronchitis
     subjects affected / exposed
4 / 9 (44.44%)
     occurrences all number
9
Dental caries
     subjects affected / exposed
3 / 9 (33.33%)
     occurrences all number
5
Nasopharyngitis
     subjects affected / exposed
4 / 9 (44.44%)
     occurrences all number
13
Tonsillitis
     subjects affected / exposed
2 / 9 (22.22%)
     occurrences all number
5

More information

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Substantial protocol amendments (globally)
Were there any global substantial amendments to the protocol? Yes
Date
Amendment
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.
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.
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.
05 Sep 2008
To prolong the patient recruitment period; To update the procedure for SAE reporting; To add an interim analysis in Q2 2008.
15 Mar 2011
To prolong the recruitment period; To transfer storageand distribution to a sub-contractor; To clarify criteria for recovery study.

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

Sponsors en medewerkers

Medische omstandigheden

Geneesmiddelinterventies

3
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