Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A
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ClinicalTrials.gov Identifier: NCT03376516 |
Recruitment Status :
Completed
First Posted : December 18, 2017
Results First Posted : December 24, 2019
Last Update Posted : January 19, 2021
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Study Type | Interventional |
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Study Design | Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment |
Condition |
Severe Hemophilia A |
Intervention |
Drug: Wilate |
Enrollment | 11 |
Recruitment Details | |
Pre-assignment Details |
Arm/Group Title | Wilate |
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A total of 11 patients were enrolled in this study. For the pharmacokinetic (PK) assessment a single dose of Wilate (50±5 IU/kg BW) was administered to 10 patients. Prophylactic treatment: Wilate (20-40 IU/kg BW) was administered every 2-3 days for 6 months. In case of unacceptably frequent spontaneous breakthrough bleeding episodes (BEs) the dose of Wilate was to be increased by approximately 5 IU/kg. The dose (and duration) of treatment for breakthrough BEs was dependent on the location and extent of bleeding and on the clinical condition of the patient; range: 10-50 IU/kg every 12-24 hours or 8-24 hours until resolved. Two patients underwent surgery treated with Wilate (SURG population). Minor surgeries received 15-30 IU/kg of Wilate every 24 hours until healing was achieved. Major surgeries were treated with 40-50 IU/kg, repeat injection every 8-24 hours until adequate wound healing, then therapy for at least another 7 days to maintain a FVIII activity of 30% to 60%. |
Period Title: Overall Study | |
Started | 11 |
Received Treatment | 10 |
SAF Population [1] | 10 |
FAS Population [2] | 10 |
PK Population [3] | 10 |
PP Population [4] | 9 |
SURG Population [5] | 2 |
Completed | 9 |
Not Completed | 2 |
Reason Not Completed | |
Not treated | 1 |
Consent withdrawn | 1 |
[1]
SAF population = study population of patients in safety analysis
[2]
FAS population = full analysis population
[3]
PK population = study population of patients who underwent pharmacokinetic analysis
[4]
PP population = per-protocol population
[5]
SURG population = study population of patients undergoing surgery treated with Wilate
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Arm/Group Title | Wilate | |
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The safety (SAF) population includes all patients who received at least one injection of Wilate during the study (n=10). | |
Overall Number of Baseline Participants | 10 | |
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The safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10).
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Age, Customized
[1] [2] Mean (Standard Deviation) Unit of measure: Years |
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1-<6 years | Number Analyzed | 5 participants |
4.0 (1.2) | ||
6-<12 years | Number Analyzed | 5 participants |
9.8 (0.8) | ||
Total | Number Analyzed | 10 participants |
6.9 (3.2) | ||
[1]
Measure Description: Age at screening (years)
[2]
Measure Analysis Population Description: The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients aged 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed.
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Sex/Gender, Customized
[1] Measure Type: Count of Participants Unit of measure: Participants |
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1-<6 years | Number Analyzed | 5 participants |
Female |
0 0.0%
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Male |
5 100.0%
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6-<12 years | Number Analyzed | 5 participants |
Female |
0 0.0%
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Male |
5 100.0%
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Total | Number Analyzed | 10 participants |
Female |
0 0.0%
|
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Male |
10 100.0%
|
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[1]
Measure Analysis Population Description: The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed.
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Race (NIH/OMB)
[1] Measure Type: Count of Participants Unit of measure: Participants |
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1-<6 years | Number Analyzed | 5 participants |
American Indian or Alaska Native |
0 0.0%
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Asian |
0 0.0%
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Native Hawaiian or Other Pacific Islander |
0 0.0%
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Black or African American |
0 0.0%
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White |
5 100.0%
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More than one race |
0 0.0%
|
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Unknown or Not Reported |
0 0.0%
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6-<12 years | Number Analyzed | 5 participants |
American Indian or Alaska Native |
0 0.0%
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Asian |
0 0.0%
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Native Hawaiian or Other Pacific Islander |
0 0.0%
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Black or African American |
0 0.0%
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White |
5 100.0%
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More than one race |
0 0.0%
|
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Unknown or Not Reported |
0 0.0%
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Total | Number Analyzed | 10 participants |
American Indian or Alaska Native |
0 0.0%
|
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Asian |
0 0.0%
|
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Native Hawaiian or Other Pacific Islander |
0 0.0%
|
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Black or African American |
0 0.0%
|
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White |
10 100.0%
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More than one race |
0 0.0%
|
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Unknown or Not Reported |
0 0.0%
|
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[1]
Measure Analysis Population Description: The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed.
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Weight
[1] Mean (Standard Deviation) Unit of measure: Kg |
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1-<6 years | Number Analyzed | 5 participants |
16.9 (3.6) | ||
6-<12 years | Number Analyzed | 5 participants |
37.4 (5.4) | ||
Total | Number Analyzed | 10 participants |
27.2 (11.6) | ||
[1]
Measure Analysis Population Description: The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed.
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Body Mass Index (BMI)
[1] Mean (Standard Deviation) Unit of measure: Kg/m^2 |
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1-<6 years | Number Analyzed | 5 participants |
15.3 (1.3) | ||
6-<12 years | Number Analyzed | 5 participants |
17.7 (2.2) | ||
Total | Number Analyzed | 10 participants |
16.5 (2.1) | ||
[1]
Measure Analysis Population Description: The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed.
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Blood groups
[1] Measure Type: Count of Participants Unit of measure: Participants |
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1-<6 years | Number Analyzed | 5 participants |
O |
2 40.0%
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A |
1 20.0%
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AB |
0 0.0%
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B |
2 40.0%
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6-<12 years | Number Analyzed | 5 participants |
O |
2 40.0%
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A |
3 60.0%
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AB |
0 0.0%
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B |
0 0.0%
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Total | Number Analyzed | 10 participants |
O |
4 40.0%
|
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A |
4 40.0%
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AB |
0 0.0%
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B |
2 20.0%
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[1]
Measure Analysis Population Description: The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed.
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Previous Factor (F)VIII treatment
[1] Measure Type: Count of Participants Unit of measure: Participants |
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1-<6 years | Number Analyzed | 5 participants |
On-demand |
0 0.0%
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Prophylaxis |
3 60.0%
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Combination |
2 40.0%
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6-<12 years | Number Analyzed | 5 participants |
On-demand |
2 40.0%
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Prophylaxis |
0 0.0%
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Combination |
3 60.0%
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Total | Number Analyzed | 10 participants |
On-demand |
2 20.0%
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Prophylaxis |
3 30.0%
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Combination |
5 50.0%
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[1]
Measure Analysis Population Description: The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed.
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Previous annualised bleeding rate (ABR)
[1] [2] Mean (Standard Deviation) Unit of measure: Bleeding events per year (ABR) |
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1-<6 years | Number Analyzed | 5 participants |
4.8 (3.0) | ||
6-<12 years | Number Analyzed | 5 participants |
13.6 (8.9) | ||
Total | Number Analyzed | 10 participants |
9.2 (7.8) | ||
[1]
Measure Description: Previous ABR is based on the bleeding rate in the 6 months prior to entry into the study.
[2]
Measure Analysis Population Description: The overall safety (SAF) population included all patients who received at least one injection of Wilate during the study (n=10). Of these, 5 patients 1-<6 years were analyzed, and 5 patients aged 6-<12 years were analyzed.
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Name/Title: | Sylvia Werner |
Organization: | Octapharma AG |
Phone: | 604-1149 ext +1 (201) |
EMail: | sylvia.werner@octapharma.com |
Responsible Party: | Octapharma |
ClinicalTrials.gov Identifier: | NCT03376516 |
Other Study ID Numbers: |
WIL-30 |
First Submitted: | December 13, 2017 |
First Posted: | December 18, 2017 |
Results First Submitted: | October 23, 2019 |
Results First Posted: | December 24, 2019 |
Last Update Posted: | January 19, 2021 |