Factor X Deficiency
Conditions
Brief summary
The main objective of the study is to assess the pharmacokinetics of FACTOR X after a single dose of 25IU/kg. The secondary objectives of the study are to assess efficacy and safety of FACTOR X in the treatment of bleeding episodes over at least 6 months.
Interventions
BIOLOGICALHuman Coagulation FACTOR X
Standard dose 25IU/kg to be administered at the Baseline Visit (1st PK assessment) and at the repeat PK assessment. Also administered to treat a bleed or to prevent a bleed.
Sponsors
Bio Products Laboratory
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Written informed consent given, or for subjects aged 12-17 years, have given written assent and whose parent/guardian has given written informed consent * At least 12 years of age at dtae of written informed consent * Have hereditary severe or moderate FX deficiency * Currently treated with Fresh Frozen Plasma FFP, Prothrombin Complex Concentrate PCC or factor IX/X concentrate * Must have a minimum of one spontaneous or menorrhagic bleed in the last 12 months which required treatment of FFP, PCC or factor IX/X concentrate. Newly diagnosed subjects who present at the hospital with a bleed may be included * Must have had at least 7 days, and ideally 10-14 days, since an infusion of either FFP, PCC or factor IX/X concentrate at Baseline Visit * Females of child bearing potential must have a negative result on a HCG based pregnancy test. If they are or become sexually active, they must practise contraception by using a method of proven reliability for the duration of the study
Exclusion criteria
* Have a history of inhibitor development to FX or a positive result at the Screening Visit * Bleeding at the appointment for the PK assessment * Subjects who have thrombocytopenia * Have clinically significant liver disease * Known to have other coagulopathy or thrombophilia * Have known or suspected hypersensitivity to the investigational medicinal product or its excipients * Have abused chemicals or drugs within the past 12 months * Have a history of unreliability or non-cooperation * Participating or have taken part in another trial within the last 30 days, with the exception of BPL FX surgery study - Protocol Ten03. In such cases, subjects should have completed their End of Study Visit either before or on the day of Screening Visit for this study * Female subjects who are pregnant or lactating * Subjects planning greater than 4 weeks absence from the locality of the Investigational site, between the screening visit and the repeat PK assessment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| FX:C Incremental Recovery | At Baseline (during first 60 minutes post-dose) and at 6 months post-Baseline (during first 60 minutes post-dose) | Incremental recovery is defined as the peak rise in plasma FX levels (IU/dL), as measured at 15, 30 and 60 minutes post-dose, divided by the dose (IU/kg). Value given is the mean of 31 results: 16 for Baseline Visit + 15 for Repeat PK assessment |
| FX:C Half-life | At Baseline and at 6 months post-Baseline | Value given is the mean of 31 results: 16 for Baseline Visit + 15 for Repeat PK assessment |
Countries
Germany, Spain, Turkey (Türkiye), United Kingdom, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Human Coagulation FACTOR X Human Coagulation FACTOR X: Standard dose 25IU/kg to be administered at the Baseline Visit (1st PK assessment) and at the repeat PK assessment. Also administered to treat a bleed or to prevent a bleed. | 16 |
| Total | 16 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Death | 1 |
Baseline characteristics
| Characteristic | Human Coagulation FACTOR X |
|---|---|
| Age, Categorical <=18 years | 6 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 10 Participants |
| Region of Enrollment Germany | 1 participants |
| Region of Enrollment Spain | 4 participants |
| Region of Enrollment Turkey | 6 participants |
| Region of Enrollment United Kingdom | 3 participants |
| Region of Enrollment United States | 2 participants |
| Sex: Female, Male Female | 10 Participants |
| Sex: Female, Male Male | 6 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 15 / 16 |
| serious Total, serious adverse events | 6 / 16 |
Outcome results
Primary
FX:C Half-life
Value given is the mean of 31 results: 16 for Baseline Visit + 15 for Repeat PK assessment
Time frame: At Baseline and at 6 months post-Baseline
| Arm | Measure | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|
| Human Coagulation FACTOR X | FX:C Half-life | 29.36 hours | Geometric Coefficient of Variation 22.89 |
Primary
FX:C Incremental Recovery
Incremental recovery is defined as the peak rise in plasma FX levels (IU/dL), as measured at 15, 30 and 60 minutes post-dose, divided by the dose (IU/kg). Value given is the mean of 31 results: 16 for Baseline Visit + 15 for Repeat PK assessment
Time frame: At Baseline (during first 60 minutes post-dose) and at 6 months post-Baseline (during first 60 minutes post-dose)
| Arm | Measure | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|
| Human Coagulation FACTOR X | FX:C Incremental Recovery | 2.07 IU/dL per IU/kg | Geometric Coefficient of Variation 21.01 |