Severe Hemophilia A
Conditions
Brief summary
This study will determine the efficacy of human-cl rhFVIII in previously treated patients with severe hemophilia A during prophylactic treatment, treatment of bleeding episodes and in surgical prophylaxis.
Interventions
BIOLOGICALrecombinant Factor VIII
intravenous infusion of factor FVIII every other day.
Sponsors
Octapharma
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Severe hemophilia A ((FVIII:C \<= 1%) * Male subjects \>= 12 years of age * Previously treated with FVIII concentrate, at least 50 EDs * Immunocompetent (CD4+ count \> 200/ul) * Negative for anti- HIV; if positive, viral load \< 200 particles/u; or \<400,000 copies/mL
Exclusion criteria
* Other coagulation disorder than hemophilia A * Present of past FVIII inhibitor activity (.= 0.6 BU) * Severe liver and kidney disease * Receiving of scheduled to receive immuno-modulating drugs
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy Assessment After a Total of at Least 50 EDs Per Subject at the End of the Study at 6 Months | At least 50 Exposure Days and at least 6 months | Frequency of spontaneous breakthrough bleeds/months under prophylactic treatment. |
| Efficacy of Treating Bleeding Episodes | After each bleeding episode, up to 6 month | At the end of a bleeding episode, efficacy was assessed as: * Excellent: Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single infusion * Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an infusion requiring up to 2 infusions for complete resolution * Moderate: Probable or slight beneficial effect within approximately 12 hours after the first infusion requiring more than two infusions for complete resolution * None: No improvement within 12 hours, or worsening of symptoms, requiring more than 2 infusions for complete resolution Efficacy was rated |
Countries
Austria, Bulgaria, Germany, United Kingdom
Participant flow
Recruitment details
Participants were enrolled at 11 European sites beginning June 2010 and completing in January 2012.
Pre-assignment details
36 participants were enrolled and screened. 4 were screen failures. Therefore, 32 of the 36 enrolled were exposed to investigation product.
Participants by arm
| Arm | Count |
|---|---|
| Human Cl-rhFVIII recombinant Factor VIII : intravenous infusion of factor FVIII every other day. | 32 |
| Total | 32 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Death | 1 |
| Overall Study | Screen Failures | 4 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Human Cl-rhFVIII |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 1 Participants |
| Age, Categorical Between 18 and 65 years | 31 Participants |
| Age, Continuous | 37.3 years STANDARD_DEVIATION 13.6 |
| Region of Enrollment Austria | 1 participants |
| Region of Enrollment Bulgaria | 8 participants |
| Region of Enrollment Germany | 8 participants |
| Region of Enrollment United Kingdom | 15 participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 32 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 3 / 32 |
| serious Total, serious adverse events | 2 / 32 |
Outcome results
Primary
Efficacy Assessment After a Total of at Least 50 EDs Per Subject at the End of the Study at 6 Months
Frequency of spontaneous breakthrough bleeds/months under prophylactic treatment.
Time frame: At least 50 Exposure Days and at least 6 months
Population: All subjects who started prophylactic treatment were evaluated.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Human Cl-rhFVIII | Efficacy Assessment After a Total of at Least 50 EDs Per Subject at the End of the Study at 6 Months | 0.188 Bleeds per month | Standard Deviation 0.307 |
Primary
Efficacy of Treating Bleeding Episodes
At the end of a bleeding episode, efficacy was assessed as: * Excellent: Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single infusion * Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an infusion requiring up to 2 infusions for complete resolution * Moderate: Probable or slight beneficial effect within approximately 12 hours after the first infusion requiring more than two infusions for complete resolution * None: No improvement within 12 hours, or worsening of symptoms, requiring more than 2 infusions for complete resolution Efficacy was rated
Time frame: After each bleeding episode, up to 6 month
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Human Cl-rhFVIII | Efficacy of Treating Bleeding Episodes | Excellent | 71.4 Percentage of treatments |
| Human Cl-rhFVIII | Efficacy of Treating Bleeding Episodes | Good | 28.6 Percentage of treatments |