ADYNOVATE Drug Use-Results Survey


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Verified May 2017 by Baxalta US Inc.

Sponsor:

Collaborator:

Baxalta Japan Limited

Information provided by (Responsible Party):

Baxalta US Inc.

ClinicalTrials.gov Identifier:

NCT03169972

First received: May 25, 2017

Last updated: May 25, 2017

Last verified: May 2017

The purpose of this survey is to understand the following items in the actual clinical use of ADYNOVATE in patients:

  1. Unexpected adverse drug reactions
  2. Occurrence of adverse drug reactions in the actual clinical use
  3. Factors that may affect safety and efficacy
  4. Occurrence of Factor VIII inhibitor development in patients with coagulation factor VIII deficiency (hereinafter hemophilia A)
  5. Safety and efficacy for hemophilia A patients who received routine prophylactic therapy and on-demand therapy

Hemophilia A Biological: ADYNOVATE

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: ADYNOVATE Drug Use-Results Survey

Primary Outcome Measures:

  • Participants who discontinued the use of ADYNOVATE – previously treated patients (PTPs) [ Time Frame: Throughout the study participation period: 1 year for previously treated patients (PTPs) ]

    Number of PTPs who discontinued the use of ADYNOVATE

  • Participants who discontinued the use of ADYNOVATE – previously untreated patients (PUPs) [ Time Frame: Throughout the study participation period: 2 years for previously untreated patients (PUPs) ]

    Number of PUPs who discontinued the use of ADYNOVATE

  • Participants who developed a Factor VIII (FVIII) inhibitor – previously treated patients (PTPs) [ Time Frame: Throughout the study participation period: 1 year for previously treated patients (PTPs) ]

    Number of PTPs who developed a Factor VIII (FVIII) inhibitor

  • Participants who developed a Factor VIII (FVIII) inhibitor – previously untreated patients (PUPs) [ Time Frame: Throughout the study participation period: 2 years for previously untreated patients (PUPs) ]

    Number of PUPs who developed a Factor VIII (FVIII) inhibitor

  • Annual bleed rate (ABR) – previously treated patients (PTPs) [ Time Frame: Throughout the study participation period: 1 year for previously treated patients (PTPs) ]

    Annual bleed rate (ABR) of PTPs on a prophylaxis regimen

  • Annual bleed rate (ABR) – previously untreated patients (PUPs) [ Time Frame: Throughout the study participation period: 2 years for previously untreated patients (PUPs) ]

    Annual bleed rate (ABR) of PUPs on a prophylaxis regimen

  • Doses to treat a bleed of participants on an on-demand regimen – previously treated patients (PTPs) [ Time Frame: At bleed resolution throughout the study participation period: 1 year for previously treated patients (PTPs) ]

    Number of doses to treat a bleed of PTPs on an on-demand regimen

  • Doses to treat a bleed of participants on an on-demand regimen – previously untreated patients (PUPs) [ Time Frame: At bleed resolution throughout the study participation period: 2 years for previously untreated patients (PUPs). ]

    Number of doses to treat a bleed of PUPs on an on-demand regimen

  • Hemostatic effectiveness of ADYNOVATE for participants on an on-demand regimen – previously treated patients (PTPs) [ Time Frame: At bleed resolution throughout the study participation period: 1 year for previously treated patients (PTPs) ]

    Based on a 4-point ordinal scale (poor, fair, good, excellent)

  • Hemostatic effectiveness of ADYNOVATE for participants on an on-demand regimen – previously untreated patients (PUPs) [ Time Frame: At bleed resolution throughout the study participation period: 2 years for previously untreated patients (PUPs). ]

    Based on a 4-point ordinal scale (poor, fair, good, excellent)

Secondary Outcome Measures:

Estimated Enrollment: 130
Actual Study Start Date: April 12, 2017
Estimated Study Completion Date: December 31, 2019
Estimated Primary Completion Date: December 31, 2019 (Final data collection date for primary outcome measure)
Previously treated patients (PTPs)

PTPs: patients who had 4 or more days to other Factor VIII (FVIII) products

Biological: ADYNOVATE

Antihemophilic Factor (Recombinant), PEGylated

Other Names:

  • BAX 855
  • BAX855
  • Recombinant Factor VIII (FVIII) PEGylated
Previously untreated patients (PUPs)

PUPs: patients who had 3 or less previous exposure days to other products

Biological: ADYNOVATE

Antihemophilic Factor (Recombinant), PEGylated

Other Names:

  • BAX 855
  • BAX855
  • Recombinant Factor VIII (FVIII) PEGylated

Ages Eligible for Study:   12 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Sampling Method:   Non-Probability Sample

Patients with hemophilia A (congenital blood coagulation factor VIII deficiency) who receive ADYNOVATE

Inclusion Criteria:

  • Hemophilia A patients who receive ADYNOVATE, including previously treated patients with Factor VIII deficiency (PTPs), and previously untreated patients with Factor VIII deficiency (PUPs) who are treated with ADYNOVATE.

Exclusion Criteria:

  • Patients not administered ADYNOVATE.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT03169972

Ogikubo Hospital
Suginami-ku, Tokyo, Japan, 167-0035

Baxalta US Inc.

Baxalta Japan Limited

Study Director: Morio Arai, MD Baxalta, now part of Shire

Responsible Party: Baxalta US Inc.
ClinicalTrials.gov Identifier: NCT03169972     History of Changes
Other Study ID Numbers: 261601
Study First Received: May 25, 2017
Last Updated: May 25, 2017

Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants

ClinicalTrials.gov processed this record on May 30, 2017