| Primary | Area Under the Concentration vs. Time Curve (AUC) | A measure of the exposure. Blood samples for the PK assessment were drawn pre-dose and up to 30 days after dosing. The PK of FXIII in children was assessed after a single i.v. dose of rFXIII 35 IU/kg. | The full analysis set including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Mean | Standard Deviation | IU*h/mL | | At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Area Under the Concentration vs. Time Curve (AUC0-∞) | | The full analysis set including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Mean | Standard Deviation | IU*h/mL | | From day 0 to day 30 | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Maximum Plasma Concentration (Cmax) for FXIII | Maximum plasma concentration of the drug reached. | The full analysis set including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Mean | Standard Deviation | U/mL | | At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Terminal Half-life (t½) | Time point when half of the maximum plasma concentration is reached. | The full analysis set including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Mean | Full Range | hours | | From day 0 to day 30 | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Mean Residence Time (MRT) | The mean residence time (MRT) of a drug in the body and related functions are derived for drugs which are intravenously administered. | The full analysis set including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Mean | Standard Deviation | hours | | From day 0 to day 30 | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Total Plasma Clearance (CL) | The total plasma clearance is a measure of the elimination of a drug from the body. Drugs are excreted primarily by the kidneys into the urine. Clearance is calculated as 'CL=Dose / AUC0-30 days'). | The full analysis set including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Mean | Standard Deviation | mL/h/kg | | From day 0 to day 30 | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Volume of Distribution at Steady State (Vss) | Volume of distribution at steady state (Vss) is the theoretical volume that the total amount of administered drug would have to occupy (if it were uniformly distributed), to provide the same concentration as it is in blood plasma at steady state. Steady state is achieved when all variables are constant in spite of ongoing processes. | The full analysis set including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Mean | Standard Deviation | mL/kg | | At steady state | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Percentage of Subjects With One or More Adverse Events (AEs) Recorded | | Safety analysis population including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Number | | percentage (%) of subjects | | From day 0 to day 30 | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Percentage of Subjects With One or More Serious Adverse Events (SAEs) | | Safety analysis population including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Number | | percentage of subjects | | From day 0 to day 30 | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors (Neutralising Antibodies Against Factor XIII) | | Safety analysis population including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Number | | percentage of subjects | | At screening and day 30 | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Coagulation Related Parameters - Fibrinogen | | Safety analysis population including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Mean | Standard Deviation | g/L | | Day 0 and at day 30 | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Coagulation Related Parameters - Activated Partial Thromboplastin Time (aPTT, Seconds) | | Safety analysis population including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Mean | Standard Deviation | Sec | | Day 0 and day 30 | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Coagulation Related Parameters - Prothrombin Time (PT) (Seconds) | | Safety analysis population including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Mean | Standard Deviation | Sec | | Day 0 and day 30 | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Clot Solubility Test (Evaluated as Normal/Abnormal) | Blood samples for clot solubility drawn at each visit (1 hour before and after dose administration). A clot solubility assay was used to screen for FXIII deficiency. The assay is based on the ability of urea to dissolve fibrin clots that have not undergone FXIII-induced stabilization. Normal blood clots generally remain stable for 24 hours or more, while clots in which fibrin molecules have not been cross-linked are soluble within minutes. The outcome of the test is normal (FXIII present; a clot is observed in the test tube) or abnormal (FXIII absent or very low level; no clot in test tube). | Safety analysis population including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Number | | participants | | Day 0 and day 30 | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Vital Signs - Pulse | | Safety analysis population including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Mean | Standard Deviation | beats/minute | | Day 0 and day 30 | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Vital Signs - Blood Pressure (Systolic and Diastolic) | | Safety analysis population including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Mean | Standard Deviation | mmHg | | Day 0 and day 30 | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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| Secondary | Physical Examination (Evaluated as Normal/Abnormal) | | Safety analysis population including all 6 children exposed to FXIII. All 6 children were exposed to one dose of trial product. | Posted | | Number | | participants | | From day 0 to day 30 | | | | ID | Title | Description |
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| OG000 | Recombinant Factor XIII | One single dose of 35 IU/kg rFXIII was administered as an intravenous (i.v.) injection to each child. The trial included one screening visit, one treatment visit (including pharmacokinetics (PK) assessments up to 24 hours) and 4 follow-up visits (at 7, 14, 21 and 30 days after dosing). Blood samples for PK assessments were drawn pre-dose and up to 30 days after dosing. |
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