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 Deficiency; MedDRA version: 9.1Level: LLTClassification code 10010432Term: Congenital deficiency of other clotting factors; MedDRA version: 9.1Level: LLTClassification code 10061992Term: Haemophilia; MedDRA version: 9.1Level: HLTClassification code 10009735Term: Coagulation disorders congenital

• Registration Number: EUCTR2006-003148-51-DE
• Lead Sponsor: ovo Nordisk A/S

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2008-06-12
• Last Update Posted: 27 January 2014

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 45

Inclusion Criteria

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. For subjects on regular replacement therapy prior to screening the following applies: 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
4. For subjects receiving on-demand treatment prior to screening the following applies: Documented history of = 2 bleeding episodes requiring treatment with FXIII containing blood products within the last 12 months prior to screening
5. Subjects with age = 6 years and a weight =20 kg. Before enrolling subjects = 6 to < 12 years of age in the EU countries, 7 subjects have to be exposed for 12 weeks (3 exposures) to trial product with a safe safety profile. For DE only: Subjects with age = 12 years.
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:
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

Exclusion Criteria

1. Known neutralizing antibodies (inhibitors) towards FXIII.
2. Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency.
3. Documented history of = 2 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 × 10^9/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, , if there is any risk of pregnancy in the opinion of the investigator.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified