Post Marketing Surveillance Study for ADYNOVATE in South Korea

The main aim of this study is to check for side effects and medical problems in participants with hemophilia A who are receiving ADYNOVATE in routine clinical practice. Also, it will be checked how effective ADYNOVATE is at stopping a bleed, at preventing bleeding episodes when used routinely for prevention and at controlling hemophilia-related bleeding during surgeries.

Participants with hemophilia A who will be newly prescribed with ADYNOVATE treatment or have already been taking ADYNOVATE treatment according to the physician's decision will be included in this study.

Participants will visit the study clinic as a part of their routine clinical practice and will be contacted by phone calls/home visits by home care workers for collection of treatment related data during the 6-month observation period.

Study Overview

• Status: Completed
• Conditions: Hemophilia A
• Intervention / Treatment: Biological: ADYNOVATE

• Study Type: Observational
• Enrollment (Actual): 341

Contacts and Locations

Study Locations

• Korea, Republic of
  • Daegu, Korea, Republic of, 41404
    • Kyungpook National University Chilgok Hospital
  • Daegu, Korea, Republic of, 41944
    • Kyungpook National University Hospital
  • Daegu, Korea, Republic of, 42472
    • Daegu Catholic University Medical Center
  • Seoul, Korea, Republic of, 03722
    • Severance Hospital Yonsei University Health System
  • Seoul, Korea, Republic of, 05278
    • Kyung Hee University Hospital at Gangdong
  • Seoul, Korea, Republic of, 05538
    • Kim Hugh Chul Internal Medicine
  • Seoul, Korea, Republic of, 06641
    • Korea Hemophilia Foundation (Seoul)
  • Ulsan, Korea, Republic of, 44033
    • Ulsan University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Hemophilia A participants in South Korea who will be newly prescribed with ADYNOVATE or already been prescribed with ADYNOVATE will be included in this study.

Description

Inclusion Criteria

Participants with a diagnosis of hemophilia A who will be newly prescribed with ADYNOVATE or already have been prescribed ADYNOVATE according to the study physician's judgment shall be included in this study if:

• The participant or legally authorized representative has given written informed consent to participate in the study.
• The participant is indicated for treatment according to the ADYNOVATE South Korea prescribing information (PI).

Exclusion Criteria

Participants should be excluded from this study if:

• The participant or legally authorized representative does not wish to participate in the study.
• Any of the contraindications included in the PI for ADYNOVATE apply.
• Participant is enrolled in an interventional trial using an investigational product other than ADYNOVATE.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
All Study Participants; Participants who are newly prescribed with Adynovate and participants previously treated with Adynovate will be treated with ADYNOVATE for hemophilia A at the time of enrollment according to a regimen determined by the study site treating physician.	Biological: ADYNOVATE; Pegylated recombinant human factor VIII; Other Names: PEGylated rFVIII

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of adverse events	Serious adverse events and non-serious adverse events	Throughout the duration of patient participation of up to approximately 6 months
Frequency of adverse events	Serious adverse events and non-serious adverse events	Throughout the duration of patient participation of up to approximately 6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of inhibitor titers for FVIII antibodies	By titer levels, high and lower titer categories	Throughout the duration of patient participation of up to approximately 6 months
Number of treated bleeds	Number of treated bleeds throughout the study period	Throughout the duration of patient participation of up to approximately 6 months
ADYNOVATE units required for bleed resolution	Number of ADYNOVATE units required for bleed resolution	At bleed resolution, throughout the duration of patient participation of up to approximately 6 months
Number of ADYNOVATE infusions needed for the treatment of bleeding episodes	The number of ADYNOVATE infusions needed for each bleeding episode is determined by the participant, his/her caregiver, and/or clinician treating the participant, and is based upon the participant's response to treatment	At bleed resolution, throughout the duration of patient participation of up to approximately 6 months
Hemostatic effectiveness assessment of bleeding episodes treated with ADYNOVATE during prophylaxis treatment	Using a 4-point ordinal scale (Excellent, Good, Fair, or None)	Throughout the duration of patient participation of up to approximately 6 months
Hemostatic effectiveness of ADYNOVATE for participants on an on-demand regimen	Using a 4-point ordinal scale (Excellent, Good, Fair, or None)	At bleed resolution, throughout the duration of patient participation of up to approximately 6 months
Assessment of perioperative effectiveness of ADYNOVATE	Using a 4-point ordinal scale (Excellent, Good, Fair, or None)	Throughout the duration of patient participation of up to approximately 6 months
Clinically significant changes in laboratory results	Number of clinically significant changes in laboratory results	Throughout the duration of patient participation of up to approximately 6 months

Collaborators and Investigators

• Sponsor: Takeda
• Collaborators: Takeda Pharma Korea Co. Ltd.
• Investigators: Study Director: Study Director, Takeda

Publications and helpful links

Helpful Links

• To obtain more information on the study, click here/on this link

Study record dates

Study Major Dates

• Study Start (Actual): February 25, 2019
• Primary Completion (Actual): January 5, 2024
• Study Completion (Actual): January 5, 2024

Study Registration Dates

• First Submitted: January 29, 2019
• First Submitted That Met QC Criteria: January 29, 2019
• First Posted (Actual): January 31, 2019

Study Record Updates

• Last Update Posted (Estimated): February 26, 2024
• Last Update Submitted That Met QC Criteria: February 23, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A; Coagulants; BAX 855
• Other Study ID Numbers: 261603

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
• IPD Sharing Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No