The primary objective of the study is to assess the efficacy of FACTOR X in the prevention of bleeding when given as routine prophylaxis over 12 months. The secondary objectives of the study are: 1. To assess the pharmacokinetics of FACTOR X after a single dose of 50 IU/kg. 2. To assess the safety of FACTOR X when given as routine prophylaxis over 6 months (26 weeks).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
PREVENTION
Masking
NONE
Enrollment
9
Addenbrookes Hospital
Cambridge, United Kingdom
Great Ormond Street Hospital
London, United Kingdom
Sheffield Children's Hospital
Sheffield, United Kingdom
The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months
The Investigator's assessment of the efficacy of FACTOR X in reduction/prevention of bleeding when given as routine prophylaxis over 6 months. The efficacy was assessed according to tabulated criteria; Excellent, good, poor, unassessable.
Time frame: 6 months
Safety of FACTOR X: Number of Participants Experiencing Adverse Events
One of the secondary objectives was to assess the safety of FACTOR X when given as routine prophylaxis over 6 months (26 weeks). The general strategy of the safety evaluation was to examine the summaries for any trends. No formal hypothesis was carried out. The number of participants who experienced Adverse Events is provided.
Time frame: 6 months
Pharmacokinetics: FX:C Incremental Recovery
One of the secondary objectives was to assess the pharmacokinetics (FX:C incremental recovery 30 minute post-dose at the Visit 1 (Baseline) and the End of Study Visit after a single dose of 50 IU/kg). The overall mean IR calculated for both visits is presented in the outcome measure table.
Time frame: Baseline Visit and End of Study Visit, 30 minutes post-dose
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