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BAY81-8973 Pediatric Safety and Efficacy Trial

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ClinicalTrials.gov Identifier: NCT01311648
Recruitment Status : Completed
First Posted : March 9, 2011
Results First Posted : November 3, 2020
Last Update Posted : November 23, 2020
Sponsor:
Information provided by (Responsible Party):
Bayer

Tracking Information
First Submitted Date  ICMJE February 18, 2011
First Posted Date  ICMJE March 9, 2011
Results First Submitted Date  ICMJE September 1, 2020
Results First Posted Date  ICMJE November 3, 2020
Last Update Posted Date November 23, 2020
Actual Study Start Date  ICMJE June 9, 2011
Actual Primary Completion Date September 9, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 9, 2020)
  • Annualized Number of Total Bleeds Within 48 h [ Time Frame: Within 48 hours post infusion ]
    Annualized 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; Part B: 50 exposure days (approximately 8 months)] was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds, untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').
  • Annualized Number of Total Bleeds Within 48 h [ Time Frame: Within 48 hours post infusion ]
    Annualized 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; Part B: 50 exposure days (approximately 8 months)] was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds, untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').
Original Primary Outcome Measures  ICMJE
 (submitted: March 8, 2011)
  • Annualized number of bleeds within 48 hours (h) after a prophylaxis injection [ Time Frame: 6 months ]
  • Number of infusions for the treatment of a bleed [ Time Frame: 6 months ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 9, 2020)
  • Annualized Number of Total Bleeds During Prophylaxis Treatment [ Time Frame: Part A: 6 months and at least 50 exposure days; Part B: 50 exposure days (approximately 8 months) ]
    Annualized number (mean +/- standard deviation) of total bleeds that occurred during prophylaxis treatment was summarized and reported. Total bleeds: sum of spontaneous bleeds, trauma bleeds, untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').
  • Annualized Number of Total Bleeds During Prophylaxis Treatment [ Time Frame: Part A: 6 months and at least 50 exposure days; Part B: 50 exposure days (approximately 8 months) ]
    Annualized 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, untreated bleeds and 'other' bleeds ('other' bleeds were infusions with reason given as 'other').
  • Hemostatic Control During Major and Minor Surgeries [ Time Frame: Part A: 6 months and at least 50 exposure days; Part B: 50 exposure days (approximately 8 months) ]
    For 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.
  • Number of Participants With Inhibitor Development [ Time Frame: Part A: 6 months and at least 50 exposure days; Part B: 50 exposure days (approximately 8 months) ]
    Number of participants with confirmed positive FVIII inhibitor titer (≥0.6 Bethesda unit [BU]) during the study was summarized and classified as participants developing low titer inhibitor (i.e. ≥0.6 to ≤ 5.0 BU) and participants developing high titer inhibitor (i.e. > 5.0 BU).
  • Factor VIII Recovery Values [ Time Frame: Part A: 6 months and at least 50 exposure days; Part B: 50 exposure days (approximately 8 months) ]
    Incremental recovery of Factor VIII (FVIII) at 20-30 min after end of infusions was determined and mean recovery values were reported.
  • Consumption of Factor VIII in All Infusions [ Time Frame: Part A: 6 months and at least 50 exposure days; Part B: 50 exposure days (approximately 8 months) ]
    Factor VIII (FVIII) usage/consumption was summarized for all infusions. Consumption per participant's body weight per year was calculated and reported.
  • Consumption of FVIII in Infusions for Prophylaxis [ Time Frame: Part A: 6 months and at least 50 exposure days; Part B: 50 exposure days (approximately 8 months) ]
    Factor VIII (FVIII) usage/consumption was summarized for prophylaxis infusions. Consumption per participant's body weight per year was calculated and reported.
  • Consumption of FVIII in Infusions for the Treatment of Bleeds [ Time Frame: Part A: 6 months and at least 50 exposure days; Part B: 50 exposure days (approximately 8 months) ]
    Factor 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.
  • Number of Infusions Per Bleed [ Time Frame: Part A: 6 months and at least 50 exposure days; Part B: 50 exposure days (approximately 8 months) ]
    The 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.
  • Response to Treatment of Bleeds [ Time Frame: Part A: 6 months and at least 50 exposure days; Part B: 50 exposure days (approximately 8 months) ]
    Participants 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.
  • Half-life (t1/2) of BAY81-8973 in Plasma [ Time Frame: Pre-infusion and until 24 hours post infusion ]
    Half-life (t1/2) of BAY81-8973 in plasma was measured. Geometric mean and percentage geometric coefficient of variation (%CV) were reported. Occurrence of "±" in relation with coefficient of variation is auto-generated by the database.
Original Secondary Outcome Measures  ICMJE
 (submitted: March 8, 2011)
  • Incidence of inhibitory antibody [ Time Frame: 6 months ]
  • Total annualized consumption of FVIII per subject [ Time Frame: 6 months ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE BAY81-8973 Pediatric Safety and Efficacy Trial
Official Title  ICMJE 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
Brief Summary

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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Haemophilia A
Intervention  ICMJE
  • Biological: Recombinant Factor VIII (Kovaltry, BAY81-8973)
    25-50 IU/kg at least 2x/week
  • Biological: Recombinant Factor VIII (Kovaltry, BAY81-8973)
    15-50 IU/kg at least 1x/week
Study Arms  ICMJE
  • Experimental: Part A: PTPs 0-<6 years
    Previously treated patients (PTPs) aged below 6 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED)
    Intervention: Biological: Recombinant Factor VIII (Kovaltry, BAY81-8973)
  • Experimental: Part A: PTPs 6-12 years
    Previously treated patients (PTPs) aged 6 to 12 years received BAY81-8973 25-50 IU/kg at least 2x/week for 6 months and at least 50 exposure days (ED)
    Intervention: Biological: Recombinant Factor VIII (Kovaltry, BAY81-8973)
  • Experimental: Part B: PUPs/MTPs 0-<6 years
    Previously untreated patients (PUPs) or minimally treated patients (MTPs, patients who had no more than 3 exposure days [EDs] with any FVIII product) received BAY81-8973 15-50 IU/kg at least 1x/week for 50 EDs
    Intervention: Biological: Recombinant Factor VIII (Kovaltry, BAY81-8973)
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 4, 2019)
94
Original Estimated Enrollment  ICMJE
 (submitted: March 8, 2011)
51
Actual Study Completion Date  ICMJE October 27, 2020
Actual Primary Completion Date September 9, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE up to 12 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Argentina,   Bulgaria,   Canada,   Denmark,   Hungary,   Ireland,   Israel,   Italy,   Latvia,   Lithuania,   Mexico,   Norway,   Poland,   Romania,   Russian Federation,   Spain,   United States
Removed Location Countries Austria,   Greece,   Serbia,   Sweden,   United Kingdom
 
Administrative Information
NCT Number  ICMJE NCT01311648
Other Study ID Numbers  ICMJE 13400
2010-021781-29 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Bayer
Study Sponsor  ICMJE Bayer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Bayer Study Director Bayer
PRS Account Bayer
Verification Date November 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP