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ADVATE 2 mL Post-Authorization Safety Surveillance (PASS)

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ClinicalTrials.gov Identifier: NCT02093741
Recruitment Status : Completed
First Posted : March 21, 2014
Last Update Posted : April 19, 2019
Sponsor:
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

Brief Summary:
This is a Post-Authorization Safety Surveillance (PASS) study designed to collect data on the safety and effectiveness of ADVATE reconstituted in 2 mL Sterile water for injection (SWFI) during routine clinical practice in children until 12 years of age. This surveillance study is a post-licensure commitment for ADVATE reconstituted in 2 mL SWFI.

Condition or disease Intervention/treatment
Hemophilia A Congenital Factor VIII (FVIII) Deficiency Biological: Octocog alfa (recombinant human coagulation factor VIII) [ADVATE]

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Study Type : Observational
Actual Enrollment : 65 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: ADVATE 2 mL (Reconstituted in 2 mL SWFI) POST-AUTHORIZATION SAFETY SURVEILLANCE STUDY
Actual Study Start Date : September 20, 2013
Actual Primary Completion Date : January 20, 2016
Actual Study Completion Date : January 20, 2016


Group/Cohort Intervention/treatment
ADVATE - 2mL Biological: Octocog alfa (recombinant human coagulation factor VIII) [ADVATE]
The investigators shall determine all treatment regimens according to product labeling information and standard practice.
Other Names:
  • Antihemophilic Factor (Recombinant)- Plasma/albumin free method (rAHF-PFM)
  • ADVATE




Primary Outcome Measures :
  1. Incidence of all local and general, hypersensitivity and infusion-related reactions, irrespective of product-related causality for the adverse events (AEs). [ Time Frame: 6 months ]

Secondary Outcome Measures :
  1. Number and type of adverse events (or adverse experiences) (AEs) considered by the investigator to be causally related to ADVATE reconstituted in 2 mL sterile water for injection (SWFI) [ Time Frame: 6 months ]
    Causally related = possibly or probably related

  2. Number of Factor VIII (FVIII) inhibitors in all participants [ Time Frame: 6 months ]
  3. Number of Factor VIII (FVIII) inhibitors in Previously Treated Patients (PTPs) (> 50 Exposure Days (EDs)) with baseline Factor VIII (FVIII) < 1% and no history of FVIII inhibitors prior to study entry [ Time Frame: 6 months ]
  4. Number of Factor VIII (FVIII) inhibitors in Previously Treated Patients (PTPs) (> 50 Exposure Days (EDs)) with baseline Factor VIII (FVIII) ≤ 2% and no history of FVIII inhibitors prior to study entry [ Time Frame: 6 months ]
  5. Subjective hemostatic effectiveness rating of excellent, good, fair, or none for each bleeding episode treated [ Time Frame: 6 months ]
  6. Number of bleeding episodes treated with 1, 2, 3, ≥ 4 infusions of ADVATE reconstituted in 2 mL sterile water for injection (SWFI) [ Time Frame: 6 months ]
  7. Total units of ADVATE reconstituted in 2 mL sterile water for injection (SWFI) administered to treat each bleeding episode [ Time Frame: 6 months ]
  8. Overall effectiveness of prophylaxis in participants who are on a prophylactic regimen [ Time Frame: 6 months ]
  9. Global assessment rating of hemostatic effectiveness of ADVATE reconstituted in 2 mL sterile water for injection (SWFI) in surgical or dental procedures [ Time Frame: 6 months ]
    Global assessment rating = excellent, good, fair, or none

  10. Change in Factor VIII (FVIII) treatment satisfaction and preference ratings from caregiver between ADVATE reconstituted in 5 mL and 2 mL sterile water for injection (SWFI) [ Time Frame: 6 months ]
  11. Change in Factor VIII (FVIII) infusion volume and time to mix and infuse FVIII treatment between ADVATE reconstituted in 5 mL and 2 mL sterile water for injection (SWFI) [ Time Frame: 6 months ]


Information from the National Library of Medicine

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Ages Eligible for Study:   up to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The study population will include 60 evaluable participants aged = 12 years with severe or moderately severe hemophilia A (Factor VIII (FVIII) = 2%) with documented prior exposure to FVIII concentrates.
Criteria

Inclusion Criteria:

  • Severe or moderately severe hemophilia A (baseline Factor VIII (FVIII) ≤ 2%)
  • ≤12 years of age
  • Participant's legally authorized representative(s) has provided written informed consent
  • Participant is prescribed ADVATE and will only receive ADVATE reconstituted in 2 mL sterile water for injection (SWFI)
  • Documented history of prior exposure to ADVATE
  • Documented evidence of negative inhibitor test result during ≤10 EDs prior to study entry

Exclusion Criteria:

  • Known hypersensitivity to the active substance or to any of the excipients
  • Known allergic reaction to mouse or hamster proteins
  • Participant has a requirement for a major surgical procedure at the time of enrollment
  • Participant has no prior exposure to a FVIII concentrate
  • Participant currently being treated with an immune tolerance induction (ITI) regimen
  • Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand disease)
  • Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device or PASS registry during the course of this study

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): NCT02093741


Locations
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France
CHRU Pellegrin, Hématologie - CRTH
BORDEAUX Cedex, France, 33076
Centre Hospitalier Générale, CTH
Chambery Cedex, France, 73011
CRTH Laboratoire d'Hématologie CHRU - Hôpital du Bocage
Dijon Cedex, France, 21034
American Memorial Hospital, Service de Pédiatrie
REIMS Cedex, France, 51092
CHU de Rennes Hôpital Pontchaillou
Rennes Cedex 09, France, 35033
Hôpital Nord, Pédiatrie
Saint Priest En Jarez, France, 42270
CHRU Purpan, CRTH - Pavillon Sénac
TOULOUSE Cedex 9, France, 31059
CHU de Nancy - Hôpital de Brabois- CRTH - Laboratoire Hémato-Hémostase
VANDOEUVRE-LES-NANCY Cedex, France, 54511
Germany
Universitätskliniken des Saarlandes, Klinik für pädiatrische Onkologie und Hämatologie
Homburg, Saarland, Germany, 66421
Klinikum Stuttgart, Olgahospital, Pädiatrie 5
Stuttgart, Germany, 70176
Hungary
Heim Pál Children's Hospital, Department of Oncology
Budapest, Hungary, 1131
Borsod-Abaúj-Zemplén County Hospital, Pediatric Dep
Miskolc, Hungary, 3629
Mohacsi Korhaz, Department of Pediatrics
Mohacs, Hungary, 7700
Josa Andras Egyetemi Oktatokorhaz, Department of Pediatrics
Nyiregyhaza, Hungary, 4400
United Kingdom
Leeds General Infirmary
Leeds, West Yorkshire, United Kingdom, LS1 3EX
Birmingham Children's Hospital NHS Trust
Birmingham, United Kingdom, B4 6NH
Great Ormond Street Hospital for Children NHS Trust
London, United Kingdom, WC1N 3JH
Sponsors and Collaborators
Baxalta now part of Shire
Investigators
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Study Director: Study Director Shire

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Responsible Party: Baxalta now part of Shire
ClinicalTrials.gov Identifier: NCT02093741     History of Changes
Other Study ID Numbers: 061101
First Posted: March 21, 2014    Key Record Dates
Last Update Posted: April 19, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Shire provides access to the de-identified individual participant data for eligible studies to aid qualified researchers in addressing legitimate scientific objectives. These IPDs will be provided 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: Data sharing requests relating to this study will be considered beginning 6 months after publication of the study results. There is no end date for eligibility to submit a data sharing request for this study.
URL: https://www.shiretrials.com/en/our-commitment-to-transparency/data-sharing-with-researchers
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
Coagulants