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Completed

NCT Number: NCT02093741

ADVATE 2 mL Post-Authorization Safety Surveillance (PASS)

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital Factor VIII (FVIII) Deficiency Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

Up to 12 year

Sex eligibility

All sexes

Study type

Observational

Primary location

CHRU Pellegrin, Hématologie - CRTH, Bordeaux, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Octocog alfa (recombinant human coagulation factor VIII) [ADVATE]

Biological

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 outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

Baxalta now part of Shire

Industry

Registry information

Official study title

ADVATE 2 mL (Reconstituted in 2 mL SWFI) POST-AUTHORIZATION SAFETY SURVEILLANCE STUDY

Important dates

Study start
2013
Primary completion
2016
Study completion
2016
First posted
Mar 21, 2014
Registry last updated
Mar 17, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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