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ADYNOVATE Drug Use-Results Survey

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ClinicalTrials.gov Identifier: NCT03169972
Recruitment Status : Recruiting
First Posted : May 30, 2017
Last Update Posted : July 30, 2018
Sponsor:
Collaborator:
Shire Japan KK
Information provided by (Responsible Party):
Shire

Brief Summary:

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

Condition or disease Intervention/treatment
Hemophilia A Biological: ADYNOVATE

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Study Type : Observational
Estimated Enrollment : 165 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: ADYNOVATE Drug Use-Results Survey
Actual Study Start Date : April 12, 2017
Estimated Primary Completion Date : December 31, 2019
Estimated Study Completion Date : December 31, 2019


Group/Cohort Intervention/treatment
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




Primary Outcome Measures :
  1. 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

  2. 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

  3. 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

  4. 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

  5. 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

  6. 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

  7. 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

  8. 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

  9. 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)

  10. 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 :
  1. Participants who experience shock or anaphylaxis - previously treated patients (PTPs) [ Time Frame: Throughout the study participation period: 1 year for previously treated patients (PTPs) ]
    Number of PTPs who experience shock or anaphylaxis

  2. Participants who experience shock or anaphylaxis - previously untreated patients (PUPs) [ Time Frame: Throughout the study participation period: 2 years for previously untreated patients (PUPs) ]
    Number of PUPs who experience shock or anaphylaxis



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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with hemophilia A (congenital blood coagulation factor VIII deficiency) who receive ADYNOVATE
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03169972


Contacts
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Contact: Claudia Schwarz, PhD +43 1 20100 2476592 claudia.schwarz@shire.com
Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com

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Sponsors and Collaborators
Shire
Shire Japan KK
Investigators
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Study Director: Study Director Shire

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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT03169972     History of Changes
Other Study ID Numbers: 261601
First Posted: May 30, 2017    Key Record Dates
Last Update Posted: July 30, 2018
Last Verified: July 2018

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

Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
BAX 855
Coagulants