Phase 3
N=6
Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency
Congenital Bleeding Disorder · Congenital FXIII Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT01253811 ↗Enrolled (actual)
6
Serious AEs
16.7%
Results posted
Jun 2016
Primary outcome: Primary: Number of Treatment Emergent (Serious and Non-serious) Adverse Events — 100; 2; 98 number of events
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
- Mar 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Treatment Emergent (Serious and Non-serious) Adverse Events |
100; 2; 98 | — |
| SECONDARY Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors. |
— | — |
| SECONDARY Clinical Laboratory Assessments: Biochemistry: Creatinine |
30.20; 28.00; 28.67; 37.00; 31.50; 31.00 | — |
| SECONDARY Clinical Laboratory Assessments: Biochemistry: Urea |
5.50; 4.00; 3.99; 4.50; 3.48; 4.52 | — |
| SECONDARY Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT) |
17.75; 16.20; 16.40; 12.50; 21.33; 17.00 | — |
| SECONDARY Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT) |
30.75; 38.20; 31.20; 26.25; 27.00; 26.00 | — |
| SECONDARY Clinical Laboratory Assessments: Haematology: Haemoglobin |
7.366; 7.624; 7.138; 7.262; 7.635; 7.821 | — |
| SECONDARY Clinical Laboratory Assessments: Haematology: Leucocytes |
8.367; 7.373; 5.387; 5.863; 6.643; 5.027 | — |
| SECONDARY Clinical Laboratory Assessments: Haematology: Thrombocytes |
316.3; 296.0; 277.0; 283.3; 332.3; 269.7 | — |
| SECONDARY Clinical Laboratory Assessments: Haematology: Erythrocytes |
4.863; 4.745; 4.587; 4.505; 4.628; 4.247 | — |
| SECONDARY Clinical Laboratory Assessments: Haematology: Haematocrit |
35.07; 36.05; 32.83; 34.80; 36.85; 32.83 | — |
| SECONDARY Physical Examinations |
3; 1 | — |
| SECONDARY Vital Signs: Systolic BP (Blood Pressure) |
107.0; 101.0; 99.0; 106.3; 106.0; 100.7 | — |
| SECONDARY Vital Signs: Diastolic BP (Blood Pressure) |
64.0; 62.7; 58.3; 57.5; 52.0; 65.2 | — |
| SECONDARY Vital Signs: Pulse |
113.2; 97.7; 106.2; 104.5; 109.8; 105.3 | — |
| SECONDARY Rate (Number Per Subject Year) of All Bleeding Episodes Requiring Treatment With a FXIII Containing Product Other Than Recombinant Factor XIII. |
— | — |
Summary
This trial will be conducted in Asia, Europe and the United States of America (USA).
The aim of this clinical trial is to investigate long-term safety of rFXIII when administered for prevention of bleeding episodes in children aged between 1 and 6 years with congenital FXIII A-subunit deficiency. This trial is an extension to trial F13CD-3760 (mentor™4, NCT01230021). If applicable the trial will be extended up to maximum 3 years dependent on when recombinant factor XIII will be commercially available in subject's respective country for use in children of 1-6 years of age.
Eligibility Criteria
Inclusion Criteria
- Completed participation in trial F13CD-3760 (NCT01230021)
Exclusion Criteria
- Known or suspected hypersensitivity to trial product or related products
- Known history of development of inhibitors against FXIII (factor XIII)
- Hereditary or acquired coagulation disorder other than FXIII congenital deficiency
- Platelet count (thrombocytes) less than 50X10e9 / L
- 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
- Any disease or condition which, judged by the trial physician, could imply a potential hazard to the subject, interfere with the trial participation or trial outcome including renal and/or liver dysfunction
Data sourced from ClinicalTrials.gov (NCT01253811). 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.