Hemophilia A
Conditions
Keywords
hemophilia, hemophilia A, ReFacto, Xyntha, moroctocog alfa, bleeding disorder
Brief summary
The purpose of this study is to collect safety information associated with the use of Xyntha in a usual care setting. Upon meeting eligibility criteria, patients will be required to have approximately 5 study visits over a 2 year period. Procedures completed throughout the study include collection of vital signs, physical exams, and laboratory assessments. Patients will be required to complete an infusion log for each Xyntha infusion.
Detailed description
The study was terminated on May 12, 2011 due to poor enrollment prospects and the study's similarity to another ongoing trial with ReFacto AF. The termination of this study has no impact on subject safety or well being. The decision to terminate the trial was not based on any safety concerns.
Interventions
BIOLOGICALMoroctocog alfa(AF-CC)
Dosing is at the discretion of the investigator during the study
PROCEDUREBlood draw for laboratory testing
Hematology and Chemistry panels, Factor VIII inhibitor and recovery studies
Sponsors
Pfizer
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male patients 12 years of age and older. * Patients transitioned to Xyntha from other recombinant or plasma-derived FVIII replacement products. * Treatment history of 150 or greater exposure days to any FVIII products prior to Enrollment visit. * Negative inhibitor at screening or documentation of negative inhibitor titer within 6 weeks or less prior to study entry except for patients entering the study on immune tolerance induction therapy.
Exclusion criteria
* Bleeding disorder other than hemophilia A. * Inhibitor titer greater than or equal to 0.6 BU during screening except for patients on immune tolerance induction therapy. * Immunomodulatory therapy during screening period. * Known hypersensitivity to hamster protein.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Factor VIII (FVIII) Inhibitor Development | Month 24 or early withdrawal | FVIII inhibitor development was defined as an inhibitor titer of more than or equal to 0.6 Bethesda Units (BU) using the Nijmegen modification of the Bethesda assay and confirmed by the central laboratory. |
Countries
New Zealand, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Xyntha Xyntha \[moroctocog alfa albumin free cell culture (AF-CC)\] administered intravenously (IV) at a dose and frequency prescribed by the treating physician as per local standard of care for up to 2 years. | 12 |
| Total | 12 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Discontinuation of study by sponsor | 8 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Xyntha |
|---|---|
| Age Continuous | 30 Years STANDARD_DEVIATION 13.6 |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 9 / 12 |
| serious Total, serious adverse events | 2 / 12 |
Outcome results
Primary
Percentage of Participants With Factor VIII (FVIII) Inhibitor Development
FVIII inhibitor development was defined as an inhibitor titer of more than or equal to 0.6 Bethesda Units (BU) using the Nijmegen modification of the Bethesda assay and confirmed by the central laboratory.
Time frame: Month 24 or early withdrawal
Population: Safety analysis population included all enrolled participants who had taken at least 1 dose of the study medication.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Xyntha | Percentage of Participants With Factor VIII (FVIII) Inhibitor Development | 0.00 Percentage of participants |