check_circleStudy Completed
Haemophilia A
Bayer Identifier:
13400
ClinicalTrials.gov Identifier:
EudraCT Number:
EU CT Number:
Not Available
BAY81-8973 pediatric safety and efficacy trial
Trial purpose
The primary objective was to evaluate the safety and efficacy of the treatment with BAY81-8973 for prophylaxis and treatment of breakthrough bleeds in children with severe hemophilia A.
The secondary objectives were
- To assess the safety and efficacy of BAY81-8973 during surgeries.
- To assess incremental recovery of BAY81-8973.
- To assess pharmacokinetic (PK) parameters in a subset of children (Previously treated patients [PTPs] and previously untreated patients [PUPs] / minimally treated patients [MTPs] - participation in PK sampling was voluntary and required consent).
The secondary objectives were
- To assess the safety and efficacy of BAY81-8973 during surgeries.
- To assess incremental recovery of BAY81-8973.
- To assess pharmacokinetic (PK) parameters in a subset of children (Previously treated patients [PTPs] and previously untreated patients [PUPs] / minimally treated patients [MTPs] - participation in PK sampling was voluntary and required consent).
Key Participants Requirements
Sex
MaleAge
0 - 12 YearsTrial summary
Enrollment Goal
94Trial Dates
June 2011 - October 2020Phase
Phase 3Could I Receive a placebo
NoProducts
Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973)Accepts Healthy Volunteer
NoWhere to participate
Status | Institution | Location |
---|---|---|
Completed | Milano, 20122, Italy | |
Completed | Padova, 35128, Italy | |
Completed | Roma, 00165, Italy | |
Completed | Timisoara, 300011, Romania | |
Completed | Bucharest, 011026, Romania | |
Completed | Bucharest, 022328, Romania | |
Completed | Mohacs, 7700, Hungary | |
Completed | Debrecen, 4032, Hungary | |
Completed | Budapest, 1089, Hungary | |
Completed | Ramat Gan, 5262000, Israel | |
Completed | Varna, 9010, Bulgaria | |
Completed | Plovdiv, 4002, Bulgaria | |
Completed | Vilnius, 08406, Lithuania | |
Withdrawn | Beograd, 11000, Serbia | |
Withdrawn | Belgrade, 11000, Serbia | |
Completed | Wroclaw, 50-368, Poland | |
Completed | Warszawa, 00-576, Poland | |
Withdrawn | Malmö, 205 02, Sweden | |
Withdrawn | Stockholm, 171 76, Sweden | |
Completed | Riga, LV-1004, Latvia | |
Withdrawn | Kragujevac, 34000, Serbia | |
Completed | Lodz, 91-738, Poland | |
Completed | Århus N, 8200, Denmark | |
Withdrawn | East Lansing, 48823, United States | |
Completed | Columbus, 43205-2696, United States | |
Completed | Toronto, M5G 1X8, Canada | |
Completed | Edmonton, T6G 2C8, Canada | |
Completed | Crumlin, 12, Ireland | |
Completed | Cleveland, 44106, United States | |
Withdrawn | Louisville, 40202, United States | |
Completed | Cincinnati, 45229, United States | |
Withdrawn | Chattanooga, 37403, United States | |
Completed | New Orleans, 70112, United States | |
Completed | Cluj-Napoca, 400177, Romania | |
Withdrawn | Brasov, 500063, Romania | |
Completed | Hamilton, L8N 3Z5, Canada | |
Completed | Bari, 70126, Italy | |
Withdrawn | San Luis Potosí, 78216, Mexico | |
Completed | Oaxaca, 68000, Mexico | |
Completed | Guadalajara, 44280, Mexico | |
Withdrawn | Wien, 1090, Austria | |
Withdrawn | Innsbruck, 6020, Austria | |
Withdrawn | Graz, 8036, Austria | |
Completed | Madrid, 28046, Spain | |
Completed | A Coruña, 15006, Spain | |
Completed | Valencia, 46026, Spain | |
Withdrawn | Barcelona, 08035, Spain | |
Withdrawn | Buenos Aires, C1425EFD, Argentina | |
Withdrawn | Chelyabinsk, 454076, Russian Federation | |
Completed | Kirov, 610027, Russian Federation | |
Completed | Kazan, 139445, Russian Federation | |
Withdrawn | La Plata, 1900, Argentina | |
Completed | Bahía Blanca, B8001HXM, Argentina | |
Withdrawn | Linz, 4020, Austria | |
Withdrawn | Liverpool, L12 2AP, United Kingdom | |
Completed | Esplugues de LLobregat, 8950, Spain | |
Completed | Volgograd, 400138, Russian Federation | |
Withdrawn | Resistencia, 3500, Argentina | |
Withdrawn | Mendoza, M5500GEC, Argentina | |
Withdrawn | Sofia, 1527, Bulgaria | |
Completed | San Juan del Río, 76800, Mexico | |
Completed | Oslo, Norway | |
Withdrawn | Pachuca, 42070, Mexico | |
Completed | Cáceres, 10003, Spain | |
Completed | Alicante, 03010, Spain | |
Withdrawn | Greenville, 27834, United States | |
Withdrawn | Charleston, 29425, United States | |
Withdrawn | Kazan, 420138, Russia | |
Completed | Catania, 95123, Italy | |
Withdrawn | San Miguel de Tucumán, 4000, Argentina | |
Withdrawn | La Plata, 1900, Argentina | |
Withdrawn | Thessaloniki, 54642, Greece | |
Withdrawn | Goudi / Athens, 11527, Greece |
Primary Outcome
- Annualized number of total bleeds within 48 hAnnualized number (mean +/- standard deviation) of total bleeds that occurred within 48 hours after all prophylaxis infusions (Part A: 6 months and at least 50 exposure days [EDs]; Part B: at least 50 EDs or until inhibitor development) was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').date_rangeTime Frame:Within 48 hours post infusion
- Annualized number of total bleeds within 48 hAnnualized number (median [inter-quartile range (Q1-Q3)]) of total bleeds that occurred within 48 hours after all prophylaxis infusions (Part A: 6 months and at least 50 exposure days [EDs]; Part B: at least 50 EDs or until inhibitor development) was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').date_rangeTime Frame:Within 48 hours post infusion
Secondary Outcome
- Annualized number of total bleeds during prophylaxis treatmentAnnualized number (mean +/- standard deviation) of total bleeds that occurred during prophylaxis treatment was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').date_rangeTime Frame:Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
- Annualized number of total bleeds during prophylaxis treatmentAnnualized number (median [inter-quartile range (Q1-Q3)]) of total bleeds that occurred during prophylaxis treatment was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds (only treated bleeds were classified as spontaneous or trauma), untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').date_rangeTime Frame:Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
- Hemostatic control during major and minor surgeriesFor participants who underwent major or minor surgeries during the study, hemostasis during the surgeries was assessed as excellent, good, moderate or poor. Number of surgeries per assessment was summarized and reported.date_rangeTime Frame:Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
- Number of participants with inhibitor development in main studyNumber of participants with confirmed positive FVIII inhibitor titer (≥ 0.6 Bethesda unit [BU/mL]) during the main study was summarized and classified as participants developing low titer inhibitor (i.e. ≥ 0.6 to ≤ 5.0 BU/mL) and participants developing high titer inhibitor (i.e. > 5.0 BU/mL).date_rangeTime Frame:Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
- Number of participants with new inhibitor development in extension studyNumber of participants who had not developed an inhibitor during the main study but developed an inhibitor (confirmed positive FVIII inhibitor titer [≥ 0.6 BU/mL]) during the extension study was summarized and classified as participants developing low titer inhibitor (i.e. ≥ 0.6 to ≤ 5.0 BU/mL) and participants developing high titer inhibitor (i.e. > 5.0 BU/mL).date_rangeTime Frame:From start of extension study to at least 100 cumulative exposure days (EDs) (median 421 EDs; median 3.8 years)
- Factor VIII recovery valuesIncremental recovery of Factor VIII (FVIII) at 20-30 min after end of infusions was determined and mean recovery values were reported.date_rangeTime Frame:Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
- Consumption of Factor VIII in all infusionsFactor VIII (FVIII) usage/consumption was summarized for all infusions. Consumption per participant's body weight per year was calculated and reported.date_rangeTime Frame:Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
- Consumption of FVIII in infusions for prophylaxisFactor VIII (FVIII) usage/consumption was summarized for prophylaxis infusions. Consumption per participant's body weight per year was calculated and reported.date_rangeTime Frame:Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
- Consumption of FVIII in infusions for the treatment of bleedsFactor VIII (FVIII) usage/consumption was summarized for infusions used to treat breakthrough bleeds. Consumption per participant's body weight per year was calculated and reported.date_rangeTime Frame:Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
- Number of infusions per bleedThe number of infusions used to treat a bleed was defined as the first infusion to treat the bleed plus all follow-up infusions to treat the same bleed, if any. The mean value of number of infusions for each bleed was calculated and reported.date_rangeTime Frame:Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
- Response to treatment of bleedsParticipants or caregivers were asked to assess the response to treatment of bleeds as excellent, good, moderate or poor. Percentage of bleeds per assessment was summarized and reported.date_rangeTime Frame:Part A: 6 months and at least 50 exposure days (EDs) (median 73 EDs; median 6 months); Part B: at least 50 EDs or until inhibitor development (median 46 EDs; median 8 months)
- Half-life (t1/2) of BAY81-8973 in plasmaHalf-life (t1/2) of BAY81-8973 in plasma was measured. Geometric mean and percentage geometric coefficient of variation (%CV) were reported.date_rangeTime Frame:Pre-infusion and until 24 hours post infusion
Trial design
Trial Type
InterventionalIntervention Type
Biological/VaccineTrial Purpose
TreatmentAllocation
N/ABlinding
Open LabelAssignment
Single Group AssignmentTrial Arms
2Additional Information
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