Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-01-31

Study Completion Date

2015-03-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Congenital Bleeding Disorder Congenital FXIII Deficiency

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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rFXIII 35 IU/kg

Group Type EXPERIMENTAL

catridecacog

Intervention Type DRUG

Intravenous injection of a single dose of recombinant factor XIII, 35 IU/kg body weight every 4th week

Interventions

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catridecacog

Intravenous injection of a single dose of recombinant factor XIII, 35 IU/kg body weight every 4th week

Intervention Type DRUG

Other Intervention Names

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recombinant factor XIII

Eligibility Criteria

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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

Minimum Eligible Age

1 Year

Maximum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No