Factor X Deficiency
Conditions
Brief summary
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).
Interventions
BIOLOGICALFACTOR X
Sponsors
Bio Products Laboratory
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 11 Years
Healthy volunteers
No
Inclusion criteria
1. Children with hereditary severe or moderate FX deficiency (FX:C \<5 IU/dL), based on their lowest reliable FX:C recorded. 2. Children under 12 years old, whose parent/guardian has given informed consent. 3. Children with a history of severe bleeding e.g.: intracranial haemorrhage, before starting prophylactic therapy, OR a mutation in the F10 gene causing a documented severe bleeding phenotype.
Exclusion criteria
1. Children must not suffer from clinically significant liver disease, renal disease, or other coagulopathy or thrombophilia 2. Children must have no history or suspicion of inhibitors to factor X. 3. Children who have known or suspected hypersensitivity to the investigational medicinal product or its excipients. 4. Children with a history of unreliability or non-cooperation. 5. Children who are participating or have taken part in another trial within the last 30 days. 6. Children planning more than 4 weeks' continuous absence from the locality of the investigational site, between the Screening Visit and the End of Study Visit at approximately 6 months (26 weeks) post-Baseline.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Safety of FACTOR X: Number of Participants Experiencing Adverse Events | 6 months | 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. |
| Pharmacokinetics: FX:C Incremental Recovery | Baseline Visit and End of Study Visit, 30 minutes post-dose | 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. |
Countries
United Kingdom
Participant flow
Recruitment details
Two subjects completed less than 26 weeks in the study. The subjects were re-enrolled and data from their first treatment cycle was excluded from the per-protocol analysis. 9 unique subjects were enrolled event though there were 11 treatment cycles.
Participants by arm
| Arm | Count |
|---|---|
| Human Coagulation FACTOR X At the Baseline Visit, eligible children received a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children were treated with FACTOR X prophylactically for a period of 6 months (26 weeks).
A dosing regimen of 40-50 IU/kg twice a week was recommended, but was not mandatory. Each dose of FACTOR X was not to not exceed 60 IU/kg. | 9 |
| Total | 9 |
Baseline characteristics
| Characteristic | Human Coagulation FACTOR X |
|---|---|
| Age, Categorical <=18 years | 9 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Region of Enrollment United Kingdom | 9 participants |
| Sex: Female, Male Female | 5 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 3 / 9 |
| serious Total, serious adverse events | 1 / 9 |
Outcome results
Primary
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
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Overall Study | The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months | 9 Participants |
Secondary
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
Population: Plasma concentrations were obtained for FX:C for all 9 subjects at 30 minutes post dose at Visit 1 and Visit 5.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Overall Study | Pharmacokinetics: FX:C Incremental Recovery | 1.74 IU/dL |
Secondary
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
Population: The safety evaluation examined the summaries for any trends. No formal hypothesis was carried out.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Overall Study | Safety of FACTOR X: Number of Participants Experiencing Adverse Events | 8 Participants |