A Multi-Centre, Open-Label, Single-Arm and Multiple Dosing Trial on Efficacy and Safety of Monthly Replacement Therapy with Recombinant Factor XIII (rFXIII) in Subjects with Congenital Factor XIII Deficiency. - F13CD-1725
- Conditions
- Congenital Factor XIII DeficencyMedDRA version: 14.1Level: PTClassification code 10016083Term: Factor XIII deficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 14.1Level: PTClassification code 10061992Term: HaemophiliaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 14.1Level: HLTClassification code 10009737Term: Coagulation factor deficienciesSystem Organ Class: 100000004851Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2006-003148-51-IT
- Lead Sponsor
- OVO NORDISK
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 40
1.Informed consent obtained before any trial-related activities (Trial-related activities are any procedure that would not have been performed during normal management of the subject). 2.Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit). 3.Treatment with regular FXIII replacement therapy initiated at least 6 months prior to screening and one of the following : a documented history of ≥1 treatment-requiring bleeding episode prior to initiation of regular replacement therapy or a documented family history of FXIII congenital deficiency (only for subjects on regular replacement therapy prior to screening). 4.Documented history of ≥ 2 bleeding episodes requiring treatment with FXIII containing blood products within the last 12 months prior to screening (only for subjects receiving on-demand treatment prior to screening). 5.Subjects with age ≥ 6 years and a weight ≥20 kg. 6.If female and of child-bearing potential: negative pregnancy test at screening.
Are the trial subjects under 18? yes
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
1.Known neutralizing antibodies (inhibitors) towards FXIII. 2.Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency. 3.Documented history of two treatment-requiring bleeding episodes per year during previous regular replacement therapy with FXIII containing blood products (FFP, pd FXIII and cryoprecipitate). 4.Platelet count (thrombocytes) < 75 × 109/L. 5.Known or suspected allergy to trial product(s) or related products. 6.Previous participation in this trial. 7.Subject has received treatment with any investigational drug within 30 days of trial enrolment, except pdFXIII. 8.Planned major surgery during the trial period. Catheter, ports and dental extractions do not count as surgeries and will not exclude the subject. 9.Renal insufficiency defined as current dialysis therapy. 10.Any history of confirmed venous or arterial thrombo-embolic events. 11.Subject has received any anti-thrombotic or anti-platelet drugs within 7 days of trial enrollment. 12.Subject has medical, social or psychosocial factors expected to impact compliance or safety. 13.Any disease or condition which, judged by the Investigator, could imply a potential hazard to the subject, interfere with the trial participation or trial outcome. 14.Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation in participating in the trial. 15.Females of childbearing potential who are pregnant, breastfeeding or intend to become pregnant or are not using adequate contraceptive methods (adequate contraceptive measures as required by local law or practice) from the time of enrollment to completion of all follow-up trial visits
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method