Try the modernized ClinicalTrials.gov beta website. Learn more about the modernization effort.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

ADYNOVATE Drug Use-Results Survey

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03169972
Recruitment Status : Recruiting
First Posted : May 30, 2017
Last Update Posted : March 3, 2022
Sponsor:
Collaborator:
Takeda
Information provided by (Responsible Party):
Takeda ( Shire )

Study Description
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
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

Study Design
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Observational
Estimated Enrollment : 145 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 : January 31, 2023
Estimated Study Completion Date : January 31, 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Groups and Cohorts
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

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


Outcome Measures
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

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


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   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.
Contacts and Locations
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

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
Layout table for location contacts
Contact: Shire Contact +1 866 842 5335 clinicaltransparency@shire.com

Locations
Show Show 52 study locations
Sponsors and Collaborators
Shire
Takeda
Investigators
Layout table for investigator information
Study Director: Study Director Shire
More Information
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
Additional Information:

Layout table for additonal information
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT03169972    
Other Study ID Numbers: 261601
First Posted: May 30, 2017    Key Record Dates
Last Update Posted: March 3, 2022
Last Verified: March 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
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.
URL: https://vivli.org/ourmember/takeda/

Layout table for additional information
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
 Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
BAX 855
Coagulants