check_circleStudy Completed
Hemophilia A
Bayer Identifier:
19855
ClinicalTrials.gov Identifier:
EudraCT Number:
EU CT Number:
Not Available
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
MaleAge
0 - 65 YearsTrial summary
Enrollment Goal
45Trial Dates
September 2020 - March 2024Phase
Phase 4Could I Receive a placebo
NoProducts
Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973)Accepts Healthy Volunteer
NoWhere to participate
Status | Institution | Location |
---|---|---|
Completed | Beijing Children's Hospital, Capital Medical University | Beijing, 100045, China |
Completed | Children‘s Hospital of Shanxi | Taiyuan, 30013, China |
Completed | Jiangxi Provincial People’s Hospital | Nanchang, 330006, China |
Withdrawn | Nanfang Hospital, Southern Medical University | Guangzhou, 510515, China |
Completed | NJ Drum Tower Hospital, the Affil Hos of NJ Univ Med School | Nanjing, 210008, China |
Completed | Peking Union Medical College Hospital CAMS | Beijing, 100730, China |
Completed | The Children's Hospital Zhejiang University School of Med | Hangzhou, 310056, China |
Completed | 1st Affiliated hospital of Soochow University | Suzhou, 215000, China |
Completed | Chengdu Women & Children's Central Hospital | Chengdu, 610091, China |
Completed | Shijiazhuang General Hospital | Shijiazhuang, 50000, China |
Withdrawn | Shenzhen Children's Hospital | Shenzhen, 518017, China |
Completed | Childrens Hospital of Shanghai | Shanghai, TBC, China |
Primary Outcome
- Annualized bleeding rate of all bleeding episodes during prophylaxis treatmentAnnualized 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 infusionAnnualized 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 episodesAnnualized 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 episodesAnnualized 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 infusionAnnualized 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 treatmentAnnualized 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 bleedsParticipants 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 surgeryPhysician’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 episodesNumber 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 episodeNumber 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 usageBAY81-8973 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 assayNumber 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 recoveryIn-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 plasmaCmax: 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) doseAUC: 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 plasmat1/2: 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 eventsdate_rangeTime Frame:Part A: up to 6 months; Part B: up to at least 50 exposure days
Trial design
Trial Type
InterventionalIntervention Type
Biological/VaccineTrial Purpose
TreatmentAllocation
Non-randomizedBlinding
N/AAssignment
Single Group AssignmentTrial Arms
3