Clinical trial NCT00978380: Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725
Official 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Congenital Bleeding Disorder
Congenital FXIII Deficiency
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2009-09-16
Enrollment: 63
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Adverse Events (AEs)(Serious and Non-serious)
timeFrame: All AEs were collected and reported from screening (week 0) for a minimum period of 52 weeks or until the end of trial visit.
Secondary outcomes:
measure: Antibody and Inhibitor Development
timeFrame: From week 0 to week 52
Other outcomes:
Locations:
city: Orange
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: Washington
country: United States
facility: Novo Nordisk Investigational Site
state: District of Columbia
city: Tampa
country: United States
facility: Novo Nordisk Investigational Site
state: Florida
city: Atlanta
country: United States
facility: Novo Nordisk Investigational Site
state: Georgia
city: Boise
country: United States
facility: Novo Nordisk Investigational Site
state: Idaho
city: Detroit
country: United States
facility: Novo Nordisk Investigational Site
state: Michigan
city: Minneapolis
country: United States
facility: Novo Nordisk Investigational Site
state: Minnesota
city: Columbus
country: United States
facility: Novo Nordisk Investigational Site
state: Ohio
city: Oklahoma City
country: United States
facility: Novo Nordisk Investigational Site
state: Oklahoma
city: Hershey
country: United States
facility: Novo Nordisk Investigational Site
state: Pennsylvania
city: Richmond
country: United States
facility: Novo Nordisk Investigational Site
state: Virginia
city: Seattle
country: United States
facility: Novo Nordisk Investigational Site
state: Washington
city: Klagenfurt
country: Austria
facility: Novo Nordisk Investigational Site
city: Toronto
country: Canada
facility: Novo Nordisk Investigational Site
state: Ontario
city: Helsinki
country: Finland
facility: Novo Nordisk Investigational Site
city: Le Kremlin Bicetre
country: France
facility: Novo Nordisk Investigational Site
city: Paris
country: France
facility: Novo Nordisk Investigational Site
city: Rouen
country: France
facility: Novo Nordisk Investigational Site
city: Valence Cedex 9
country: France
facility: Novo Nordisk Investigational Site
city: Bonn
country: Germany
facility: Novo Nordisk Investigational Site
city: Braunschweig
country: Germany
facility: Novo Nordisk Investigational Site
city: Duisburg
country: Germany
facility: Novo Nordisk Investigational Site
city: Petach Tikva
country: Israel
facility: Novo Nordisk Investigational Site
city: Vicenza
country: Italy
facility: Novo Nordisk Investigational Site
city: Hiroshima-shi, Hiroshima
country: Japan
facility: Novo Nordisk Investigational Site
city: Shinjuku-ku, Tokyo
country: Japan
facility: Novo Nordisk Investigational Site
city: Barcelona
country: Spain
facility: Novo Nordisk Investigational Site
city: Málaga
country: Spain
facility: Novo Nordisk Investigational Site
city: Tortosa
country: Spain
facility: Novo Nordisk Investigational Site
city: Zürich
country: Switzerland
facility: Novo Nordisk Investigational Site
city: Aberdeen
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: London
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: Manchester
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: Newcastle upon Tyne
country: United Kingdom
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References:
citation: Brand-Staufer B, Carcao M, Kerlin BA, Will A, Williams M, Tornoe CW, Sandberg Lundblad M, Nugent D. Pharmacokinetic characterization of recombinant factor XIII (FXIII)-A2 across age groups in patients with FXIII A-subunit congenital deficiency. Haemophilia. 2015 May;21(3):380-385. doi: 10.1111/hae.12616. Epub 2015 Jan 21.
citation: Kerlin B, Brand B, Inbal A, Halimeh S, Nugent D, Lundblad M, Tehranchi R. Pharmacokinetics of recombinant factor XIII at steady state in patients with congenital factor XIII A-subunit deficiency. J Thromb Haemost. 2014 Dec;12(12):2038-43. doi: 10.1111/jth.12739. Epub 2014 Oct 25.