Clinical trial NCT01230021: Safety of a Single Intravenous Dose of Recombinant Factor XIII in Children With Congenital FXIII A-subunit Deficiency
Official title: A Phase 3b Trial Investigating the Pharmacokinetics and Safety Profile of a Single Intravenous Dose of rFXIII in Paediatric (1 to Less Than 6 Years Old) Subjects With Congenital FXIII A-subunit 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: 2010-10-28
Enrollment: 6
Sex: ALL
Minimum age (years): 1
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This trial is conducted in Europe and United States of America (USA). The aim of this clinical trial is to investigate the pharmacokinetics (at which rate the substance is distributed and eliminated from the body) and the safety profile of catridecacog (recombinant factor XIII (rFXIII)) in children with congenital FXIII A-subunit deficiency. Young children (1 to less than 6 years old) with congenital FXIII deficiency are evaluated.


Eligibility criteria: Inclusion Criteria:

* Signed Informed Consent by subject's parents or subject's legally acceptable representative before any trial related activities. Trial related activities are any procedures that would not have been performed during the normal management of the subject
* Age 1 to less than 6 years old at the time of enrolment
* Congenital FXIII subunit-A deficiency previously documented by genotyping or evaluated by genotyping through blood sampling at screening visit
* Body weight at least 10 kg

Exclusion Criteria:

* Known antibodies to FXIII
* Hereditary or acquired coagulation disorder other than FXIII A-subunit congenital deficiency
* Platelet count (thrombocytes) of less than 50 × 10\^9/L (at screening visit)
* 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
* Known or suspected allergy to trial product or related products
* Any surgical procedure in the 30 days prior to enrolment and any planned surgery during the trial period
* Any disease or condition which, judged by the Investigator, could imply a potential hazard to the subject or interfere with the trial participation or trial outcome including renal and/or liver dysfunction

Primary outcomes:
measure: Area Under the Concentration vs. Time Curve (AUC)
timeFrame: At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing
Secondary outcomes:
measure: Area Under the Concentration vs. Time Curve (AUC0-∞)
timeFrame: From day 0 to day 30
measure: Maximum Plasma Concentration (Cmax) for FXIII
timeFrame: At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing
measure: Terminal Half-life (t½)
timeFrame: From day 0 to day 30
measure: Mean Residence Time (MRT)
timeFrame: From day 0 to day 30
measure: Total Plasma Clearance (CL)
timeFrame: From day 0 to day 30
measure: Volume of Distribution at Steady State (Vss)
timeFrame: At steady state
measure: Percentage of Subjects With One or More Adverse Events (AEs) Recorded
timeFrame: From day 0 to day 30
measure: Percentage of Subjects With One or More Serious Adverse Events (SAEs)
timeFrame: From day 0 to day 30
measure: Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors (Neutralising Antibodies Against Factor XIII)
timeFrame: At screening and day 30
measure: Coagulation Related Parameters - Fibrinogen
timeFrame: Day 0 and at day 30
measure: Coagulation Related Parameters - Activated Partial Thromboplastin Time (aPTT, Seconds)
timeFrame: Day 0 and day 30
measure: Coagulation Related Parameters - Prothrombin Time (PT) (Seconds)
timeFrame: Day 0 and day 30
measure: Clot Solubility Test (Evaluated as Normal/Abnormal)
timeFrame: Day 0 and day 30
measure: Vital Signs - Pulse
timeFrame: Day 0 and day 30
measure: Vital Signs - Blood Pressure (Systolic and Diastolic)
timeFrame: Day 0 and day 30
measure: Physical Examination (Evaluated as Normal/Abnormal)
timeFrame: From day 0 to day 30
Other outcomes:
Locations:
city: Boston
country: United States
facility: Novo Nordisk Investigational Site
state: Massachusetts
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: Petach Tikva
country: Israel
facility: Novo Nordisk Investigational Site
city: Birmingham
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: Leicester
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
city: Reading
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: Williams M, Will A, Stenmo C, Rosholm A, Tehranchi R. Pharmacokinetics of recombinant factor XIII in young children with congenital FXIII deficiency and comparison with older patients. Haemophilia. 2014 Jan;20(1):99-105. doi: 10.1111/hae.12224. Epub 2013 Jul 9.
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.