Trial Condition(s):
Haemophilia A
BAY81-8973 pediatric safety and efficacy trial
Bayer Identifier:
13400
ClinicalTrials.gov Identifier:
EudraCT Number:
EU CT Number:
Not Available
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).
Inclusion Criteria
- Male - PTPs (previously treated patients): aged <= 12 years - PUPs (previously untreated patients) / MTPs (minimally treated patients): aged < 6 years - Severe hemophilia A defined as < 1% FVIII concentration (FVIII:C) - PTPs: >= 50 exposure days (EDs) with any FVIII concentrate, no current evidence of inhibitory antibody, and no history of FVIII inhibitor formation - PUPs: no prior exposure to any FVIII product - MTPs: having no more than 3 EDs with any FVIII product, no current evidence of inhibitory antibody and no history of FVIII inhibitor formation
Exclusion Criteria
- With another bleeding disorder that is different from Hemophilia A - With thrombocytopenia (platelet count < 100 000/mm^3) - Creatinine > 2x upper limit of normal or Aspartate aminotransferase (AST)/Alanine aminotransferase (ALT) > 5x upper limit of normal - Without a negative inhibitor testing at screening (except for PUPs) - Receiving chemotherapy, immune modulatory drugs, has received another investigational FVIII product within the last month, or received another experimental drug within the last 3 months - Requires any pre-medication to tolerate FVIII treatment - Known hypersensitivity to active substance, mouse, or hamster protein
Trial Summary
Enrollment Goal
Trial Dates
Phase
Could I receive a placebo?
Products
Accepts Healthy Volunteers
Where to Participate
| Locations | |
|---|---|
Locations Investigative Site Milano, Italy, 20122 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Padova, Italy, 35128 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Roma, Italy, 00165 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Timisoara, Romania, 300011 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Bucharest, Romania, 011026 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Bucharest, Romania, 022328 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Mohacs, Hungary, 7700 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Debrecen, Hungary, 4032 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Budapest, Hungary, 1089 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Ramat Gan, Israel, 5262000 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Varna, Bulgaria, 9010 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Plovdiv, Bulgaria, 4002 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Vilnius, Lithuania, 08406 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Wroclaw, Poland, 50-368 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Warszawa, Poland, 00-576 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Riga, Latvia, LV-1004 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Lodz, Poland, 91-738 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Århus N, Denmark, 8200 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Columbus, United States, 43205-2696 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Toronto, Canada, M5G 1X8 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Edmonton, Canada, T6G 2C8 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Crumlin, Ireland, 12 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Cleveland, United States, 44106 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Cincinnati, United States, 45229 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site New Orleans, United States, 70112 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Cluj-Napoca, Romania, 400177 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Hamilton, Canada, L8N 3Z5 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Bari, Italy, 70126 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Oaxaca, Mexico, 68000 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Guadalajara, Mexico, 44280 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Madrid, Spain, 28046 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site A Coruña, Spain, 15006 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Valencia, Spain, 46026 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Kirov, Russia, 610027 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Kazan, Russia, 139445 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Bahía Blanca, Argentina, B8001HXM | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Esplugues de LLobregat, Spain, 8950 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Volgograd, Russia, 400138 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site San Juan del Río, Mexico, 76800 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Oslo, Norway | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Cáceres, Spain, 10003 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Alicante, Spain, 03010 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Locations Investigative Site Catania, Italy, 95123 | Contact Us: E-mail: [email protected] Phone: (+)1-888-84 22937 |
Trial Design
A multicenter Phase III uncontrolled open-label trial to evaluate safety and efficacy of BAY81-8973 in children with severe hemophilia A under prophylaxis therapy
Trial Type:
Interventional
Intervention Type:
Biological/Vaccine
Trial Purpose:
Treatment
Allocation:
N/A
Blinding:
Open Label
Assignment:
Single Group Assignment
Trial Arms:
2
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').Timeframe: 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').Timeframe: 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').Timeframe: 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').Timeframe: 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.Timeframe: 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).Timeframe: 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).Timeframe: 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.Timeframe: 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.Timeframe: 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.Timeframe: 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.Timeframe: 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.Timeframe: 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.Timeframe: 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.Timeframe: Pre-infusion and until 24 hours post infusion
Additional Information
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