check_circleStudy Completed

Hemophilia A

A Post Approval Commitment study to gain more information on how safe and effective KOVALTRY is in Chinese children, adolescents /adults with severe hemophilia A

Trial purpose

The goal of this study is to gather more information on safety and efficacy of Kovaltry for the prevention and treatment of bleeds in Chinese children, adolescents/adults with severe hemophilia A. In addition, pharmacokinetic parameters of Kovaltry will be assessed in a subset of patients.

Key Participants Requirements

Sex

Male

Age

0 - 65 Years

Trial summary

Enrollment Goal
45
Trial Dates
September 2020 - March 2024
Phase
Phase 4
Could I Receive a placebo
No
Products
Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973)
Accepts Healthy Volunteer
No

Where to participate

StatusInstitutionLocation
Completed
Beijing Children's Hospital, Capital Medical UniversityBeijing, 100045, China
Completed
Children‘s Hospital of ShanxiTaiyuan, 30013, China
Completed
Jiangxi Provincial People’s HospitalNanchang, 330006, China
Withdrawn
Nanfang Hospital, Southern Medical UniversityGuangzhou, 510515, China
Completed
NJ Drum Tower Hospital, the Affil Hos of NJ Univ Med SchoolNanjing, 210008, China
Completed
Peking Union Medical College Hospital CAMSBeijing, 100730, China
Completed
The Children's Hospital Zhejiang University School of MedHangzhou, 310056, China
Completed
1st Affiliated hospital of Soochow UniversitySuzhou, 215000, China
Completed
Chengdu Women & Children's Central HospitalChengdu, 610091, China
Completed
Shijiazhuang General HospitalShijiazhuang, 50000, China
Withdrawn
Shenzhen Children's HospitalShenzhen, 518017, China
Completed
Childrens Hospital of ShanghaiShanghai, TBC, China

Primary Outcome

  • Annualized bleeding rate of all bleeding episodes during prophylaxis treatment
    Annualized bleeding rate (ABR) of all bleeding episodes during prophylaxis treatment in previously treated patients (PTPs).
    date_rangeTime Frame:
    Part A: up to 6 months
  • Annualized bleeding rate within 48 hours of previous prophylaxis infusion
    Annualized bleeding rate (ABR) of bleeding episodes within 48 hours of previous prophylaxis infusion in previously untreated/minimally treated patients (PUPs/MTPs).
    date_rangeTime Frame:
    Part B: up to 48 hours post-infusion for at least 50 exposure days

Secondary Outcome

  • Annualized bleeding rate of treated bleeding episodes
    Annualized bleeding rate (ABR) of bleeding episodes treated with BAY81-8973 during prophylaxis treatment.
    date_rangeTime Frame:
    Part A: up to 6 months; Part B: up to at least 50 exposure days
  • Annualized bleeding rate of target joint bleeding episodes
    Annualized bleeding rate (ABR) of target joint bleeding episodes during prophylaxis treatment.
    date_rangeTime Frame:
    Part A: up to 6 months; Part B: up to at least 50 exposure days
  • Annualized bleeding rate within 48 hours of previous prophylaxis infusion
    Annualized bleeding rate (ABR) of bleeding episodes within 48 hours of previous prophylaxis infusion.
    date_rangeTime Frame:
    Part A: up to 48 hours post-infusion for 6 months
  • Annualized bleeding rate of all bleeding episodes during prophylaxis treatment
    Annualized bleeding rate (ABR) of all bleeding episodes during prophylaxis treatment in previously untreated/minimally treated patients (PUPs/MTPs).
    date_rangeTime Frame:
    Part B: up to at least 50 exposure days
  • Assessment of response to treatment of bleeds
    Participants or caregivers assessment to Kovaltry treatment on ISTH 4 point response scale for response to treatment of bleed [excellent, good, moderate or poor].
    date_rangeTime Frame:
    Part A: up to 6 months; Part B: up to at least 50 exposure days
  • Number of surgeries based on physician’s assessment of adequacy of hemostasis in minor surgery
    Physician’s assessment of participant's hemostatic response to Kovaltry treatment on the ISTH 4 point response scale for adequacy of hemostasis for surgical procedures [excellent, good, moderate or poor].
    date_rangeTime Frame:
    Part A: up to 6 months; Part B: up to at least 50 exposure days
  • Number of participants without bleeding episodes
    Number of participants who report no bleeding event during prophylaxis.
    date_rangeTime Frame:
    Part A: up to 6 months; Part B: up to at least 50 exposure days
  • Number of infusions per bleeding episode
    Number of infusions of BAY81-8973 needed to achieve hemostasis for a bleeding episode.
    date_rangeTime Frame:
    Part A: up to 6 months; Part B: up to at least 50 exposure days
  • Factor VIII usage
    BAY81-8973 consumption during the study.
    date_rangeTime Frame:
    Part A: up to 6 months; Part B: up to at least 50 exposure days
  • Factor VIII inhibitor development by the Nijmegen Bethesda assay
    Number of participants with positive Factor VIII (FVIII) inhibitor test (≥0.6 Bethesda unit [BU]).
    date_rangeTime Frame:
    Part A: up to 6 months; Part B: up to at least 50 exposure days
  • In-vivo recovery
    In-vivo recovery (or incremental recovery) is defined as the increase in the circulating FVIII activity level for one unit (IU) of the FVIII product per kilogram body weight.
    date_rangeTime Frame:
    Part A: baseline, Month 2 and Month 6; Part B: baseline and end of study
  • Maximum concentration (Cmax) of Kovaltry in plasma
    Cmax: Maximum observed drug concentration following an infusion of 50 IU/kg.
    date_rangeTime Frame:
    Part A: pre-infusion and up to 30 minutes post-infusion
  • Area under the plasma concentration versus time curve (AUC) from zero to infinity after single (first) dose
    AUC: The total area under the plasma concentration versus time curve following an infusion of 50 IU/kg.
    date_rangeTime Frame:
    Part A: pre-infusion and up to 48 hours post-infusion
  • Half-life (t1/2) of Kovaltry in plasma
    t1/2: Terminal half-life is the time the plasma concentration during terminal phase is halved following an infusion of 50 IU/kg.
    date_rangeTime Frame:
    Part A: pre-infusion and up to 48 hours post-infusion
  • Treatment-emergent adverse events
    date_rangeTime Frame:
    Part A: up to 6 months; Part B: up to at least 50 exposure days

Trial design

A Post Approval Commitment study to evaluate the efficacy, safety, and pharmacokinetics of KOVALTRY in Chinese children, adolescents /adults with severe hemophilia A
Trial Type
Interventional
Intervention Type
Biological/Vaccine
Trial Purpose
Treatment
Allocation
Non-randomized
Blinding
N/A
Assignment
Single Group Assignment
Trial Arms
3