Severe hemophilia A
Conditions
Interventions
This is a phase 3b continuation study for safety and efficacy of PEGylated
recombinant factor VIII (PEG-rFVIII
BAX 855) administred as profylaxis of
bleedings in previously treated patients with severe hemophilia A.
The patients can continue after participation in a qualifying study (Prolongate
261201 (clos
Sponsors
Baxalta Innovations GmbH
Eligibility
Age
2 Years to 99 Years
Inclusion criteria
Inclusion criteria: SUBJECTS TRANSITIONING FROM OTHER BAX855 TRIALS: Subjects transitioning from other BAX 855 studies who meet ALL of the following criteria are eligible for this study: 1. Subject has completed a previous BAX 855 study and is willing to immediately transition into this continuation study. 2. Subject is * 75 years of age at screening of the previous BAX 855study. 3. Subject continues to have a Karnofsky (for subjects aged * 16 years) or Lansky (for subjects aged
Exclusion criteria
Exclusion criteria: SUBJECTS TRANSITIONING FROM OTHER BAX855 STUDIES: Subjects transitioning from other BAX 855 studies who meet ANY of the following criteria are not eligible for this study: 1. Subject had detectable FVIII inhibitory antibodies (* 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening. 2. Subject has developed FVIII inhibitory antibodies (* 0.6 BU using the Nijmegen modification of the Bethesda assay as determined at central laboratory in a previous BAX 855 study). 3. Subject has acquired a hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease) in a previous BAX 855 study. 4. Subject has severe chronic hepatic dysfunction (eg, * 5 times upper limit of normal alanine aminotransferase [ALT], as confirmed by central laboratory at screening). 5. Subject has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening. 6. Subject experienced a life-threatening or gastrointestinal bleeding episode within 3 months prior to study entry. 7. Subject is scheduled to use other PEGylated drugs during study participation. 8. Subject is planning to take part in any other clinical study during the course of the continuation study, with the exception of any other parallel BAX 855 study. 9. Subject has medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject safety or compliance. 10. Subject is a family member or employee of the investigator.;BAX 855 NAIVE SUBJECTS: BAX 855 naïve subjects who meet ANY of the following criteria are not eligible for this study: 1. Subject has detectable FVIII inhibitory antibodies (* 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening. 2. Subject has history of FVIII inhibitory antibodies (* 0.6 BU using the Nijmegen modification of the Bethesda assay or the Bethesda assay) at any time prior to screening. 3. Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease). 4. Subject has known hypersensitivity towards mouse or hamster proteins, PEG, or Tween 80. 5. Subject has severe chronic hepatic dysfunction (eg, * 5 times upper limit of normal ALT, as confirmed by central laboratory at screening). 6. Subject has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening. 7. Subject experienced a life-threatening or gastrointestinal bleeding episode within 3 months prior to study entry. 8. Subject has current or recent (
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety: Development of inhibitory antibodies to FVIII Efficacy: Spontaneous ABR | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary Outcome Measure(s) Efficacy 1. Total ABR (spontaneous and traumatic bleeding episodes) 2. Overall hemostatic efficacy rating of BAX 855 to treat bleeding episodes 3. Number of BAX 855 infusions to treat bleeding episodes 4. Time intervals between bleeding episodes 5. Weight-adjusted consumption of BAX 855 Safety 1. Occurrence of AEs and SAEs 2. Changes in vital signs and clinical laboratory parameters (hematology, clinical chemistry, and lipids) 3. Immunogenicity a. Binding antibodies (IgG and IgM) to FVIII, BAX 855, and PEG b. Anti-CHO antibodies Patient Reported Outcomes (PROs) Changes from baseline in the parent study, if applicable, in the following: 1. Bleed and pain severity as measured using the Haemo-SYM questionnaire 2. HRQoL as assessed using the SF-36/PedsQL questionnaire Exploratory Outcome Measure(s) 1. Patient satisfaction with treatment will be assessed using the Satisfaction Question Set 2. Patient Activity Level 3. Health resource use data (eg, physician office visits, hospitalizations, length of stay, days missed from work/school) | — |
Countries
Netherlands
Outcome results
None listed