Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725

This trial is conducted in Asia, Europe and North America. The aim of the trial is to investigate the safety of monthly replacement therapy of recombinant factor XIII in patients with congenital FXIII deficiency. The trial continues until the product is commercially available, but an interim assessment will take place when all subjects have completed 52 weeks in the trial.

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Key information

Conditions

Congenital Bleeding Disorder Congenital FXIII Deficiency

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Novo Nordisk Investigational Site, Klagenfurt, Austria

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

For subjects who participated in F13CD-1725:
Previous participation (means up to and inclusive Visit 16, (End of Trial)) in F13CD-1725
For all other subjects:
Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit or documented results from previously performed genotyping)
Body weight at least 20 kg

Exclusion criteria

Known neutralizing antibodies (inhibitors) towards FXIII
Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
Platelet count (thrombocytes) of less than 50 × 109/L. For subjects who participated in F13CD-1725 platelet count from visit 15 in F13CD-1725 must be used for evaluation.
Females of childbearing potential who are pregnant, breastfeeding or are not using adequate contraceptive methods

Treatment and study plan

catridecacog

Drug

Monthly administration of recombinant factor XIII as preventative treatment of bleeding episodes. Dose: 35 IU/kg body weight intravenous (into the vein)

Other names: recombinant factor XIII

Primary outcomes

Adverse Events (AEs)(Serious and Non-serious)

Time frame: All AEs were collected and reported from screening (week 0) for a minimum period of 52 weeks or until the end of trial visit.

An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Trial AEs (serious) included any event such as death, life-threatening experience, in-subject hospitalisation, significant disability/ congential anomaly experienced from the trial product.

Secondary outcomes

Antibody and Inhibitor Development

Time frame: From week 0 to week 52

All subjects who received rFXIII were monitored for anti-rFXIII antibodies and inhibitor development. Samples passed through 2 tiers of ELISA testing: an initial screen with a specific cut-off point (including ~5% false positives) and a second confirmatory assay for samples yielding a result above the screening cut-off point. If samples were confirmed as antibody positive in the confirmation assay, an inhibitor assay was also carried out to detect functional inhibitors. Percentage of subjects with antibody and inhibitor development were reported.

Sponsors and collaborators

Lead sponsor

Novo Nordisk A/S

Industry

Registry information

Official study title

A Multi-Centre, Open-Label, Single-Arm, and Multiple Dosing Trial on Safety of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Subjects With Congenital Factor XIII Deficiency

Acronym: mentor™2

Important dates

Study start: 2009
Primary completion: 2015
Study completion: 2015
First posted: Sep 16, 2009
Registry last updated: Jan 24, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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Completed