Phase 3
Completed 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
Source: ClinicalTrials.gov NCT01253811 ↗
Enrolled (actual)
6
Serious AEs
16.7%
Results posted
Jun 2016
Primary outcomePrimary: Number of Treatment Emergent (Serious and Non-serious) Adverse Events — 100; 2; 98 number of events
◆ Published Evidence
Emerging
11citations · ~1 / year
Recombinant factor XIII prophylaxis is safe and effective in young children with congenital factor XIII-A deficiency: international phase 3b trial results.
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.
Linked Publications
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Recombinant factor XIII prophylaxis is safe and effective in young children with congenital factor XIII-A deficiency: international phase 3b trial results.
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. |
— | — |
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) and the linked publication. 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. Informational only — not medical advice.