Phase 3
N=6
Safety of a Single Intravenous Dose of Recombinant Factor XIII in Children With Congenital FXIII A-subunit Deficiency
Congenital Bleeding Disorder · Congenital FXIII Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT01230021 ↗Enrolled (actual)
6
Serious AEs
0.0%
Results posted
Jun 2014
Primary outcome: Primary: Area Under the Concentration vs. Time Curve (AUC) — 250.25 IU*h/mL
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- catridecacog (Drug)
- Age
- Pediatric · 1+ yrs
- Sex
- All
- Sponsor
- Novo Nordisk A/S
- Primary completion
- Jan 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Area Under the Concentration vs. Time Curve (AUC) |
250.25 | — |
| SECONDARY Area Under the Concentration vs. Time Curve (AUC0-∞) |
403.18 | — |
| SECONDARY Maximum Plasma Concentration (Cmax) for FXIII |
0.69 | — |
| SECONDARY Terminal Half-life (t½) |
378 | — |
| SECONDARY Mean Residence Time (MRT) |
729.01 | — |
| SECONDARY Total Plasma Clearance (CL) |
0.10 | — |
| SECONDARY Volume of Distribution at Steady State (Vss) |
66.07 | — |
| SECONDARY Percentage of Subjects With One or More Adverse Events (AEs) Recorded |
33.3 | — |
| SECONDARY Percentage of Subjects With One or More Serious Adverse Events (SAEs) |
— | — |
| SECONDARY Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors (Neutralising Antibodies Against Factor XIII) |
50; 50 | — |
| SECONDARY Coagulation Related Parameters - Fibrinogen |
3.26; 3.46 | — |
| SECONDARY Coagulation Related Parameters - Activated Partial Thromboplastin Time (aPTT, Seconds) |
33.8; 30.3 | — |
| SECONDARY Coagulation Related Parameters - Prothrombin Time (PT) (Seconds) |
12.6; 12.1 | — |
| SECONDARY Clot Solubility Test (Evaluated as Normal/Abnormal) |
4; 1; 1; 5; 0; 1 | — |
| SECONDARY Vital Signs - Pulse |
95.3; 113.2 | — |
| SECONDARY Vital Signs - Blood Pressure (Systolic and Diastolic) |
98.5; 107.0; 59.8; 64.0 | — |
| SECONDARY Physical Examination (Evaluated as Normal/Abnormal) |
6; 0; 5; 1 | — |
Summary
This trial is conducted in Europe and United States of America (USA). The aim of this clinical trial is to investigate the pharmacokinetics (at which rate the substance is distributed and eliminated from the body) and the safety profile of catridecacog (recombinant factor XIII (rFXIII)) in children with congenital FXIII A-subunit deficiency. Young children (1 to less than 6 years old) with congenital FXIII deficiency are evaluated.
Eligibility Criteria
Inclusion Criteria
- Signed Informed Consent by subject's parents or subject's legally acceptable representative before any trial related activities. Trial related activities are any procedures that would not have been performed during the normal management of the subject
- Age 1 to less than 6 years old at the time of enrolment
- Congenital FXIII subunit-A deficiency previously documented by genotyping or evaluated by genotyping through blood sampling at screening visit
- Body weight at least 10 kg
Exclusion Criteria
- Known antibodies to FXIII
- Hereditary or acquired coagulation disorder other than FXIII A-subunit congenital deficiency
- Platelet count (thrombocytes) of less than 50 × 10^9/L (at screening visit)
- Previous history of autoimmune disorder involving autoantibodies e.g., systemic lupus erythematosus
- Previous history of arterial or venous thromboembolic events e.g., cerebrovascular accident or deep vein thrombosis
- Known or suspected allergy to trial product or related products
- Any surgical procedure in the 30 days prior to enrolment and any planned surgery during the trial period
- Any disease or condition which, judged by the Investigator, could imply a potential hazard to the subject or interfere with the trial participation or trial outcome including renal and/or liver dysfunction
Data sourced from ClinicalTrials.gov (NCT01230021). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.