Safety of a Single Intravenous Dose of Recombinant Factor XIII in Children With Congenital FXIII A-subunit Deficiency

Novo Nordisk A/S6 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Congenital Bleeding Disorder Congenital FXIII Deficiency

Interventions

catridecacogDRUG

Intravenous injection of a single dose of recombinant factor XIII, 35 IU/kg bodyweight

Eligibility

Sex: ALLMin age: 1 YearMax age: 6 Years

Medical Language ↔ Plain English

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

Locations (10)

Novo Nordisk Investigational Site

Boston, Massachusetts, United States

Novo Nordisk Investigational Site

Detroit, Michigan, United States

Novo Nordisk Investigational Site

Minneapolis, Minnesota, United States

Outcomes

Primary Outcomes

Area Under the Concentration vs. Time Curve (AUC)

A measure of the exposure. Blood samples for the PK assessment were drawn pre-dose and up to 30 days after dosing. The PK of FXIII in children was assessed after a single i.v. dose of rFXIII 35 IU/kg.

Time frame: At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing

Secondary Outcomes

Area Under the Concentration vs. Time Curve (AUC0-∞)

A measure of exposure.

Time frame: From day 0 to day 30

Maximum Plasma Concentration (Cmax) for FXIII

Maximum plasma concentration of the drug reached.

Time frame: At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing

Terminal Half-life (t½)

Time point when half of the maximum plasma concentration is reached.

Time frame: From day 0 to day 30

Mean Residence Time (MRT)

The mean residence time (MRT) of a drug in the body and related functions are derived for drugs which are intravenously administered.

Time frame: From day 0 to day 30

Total Plasma Clearance (CL)

The total plasma clearance is a measure of the elimination of a drug from the body. Drugs are excreted primarily by the kidneys into the urine. Clearance is calculated as 'CL=Dose / AUC0-30 days').

Time frame: From day 0 to day 30

Volume of Distribution at Steady State (Vss)

Volume of distribution at steady state (Vss) is the theoretical volume that the total amount of administered drug would have to occupy (if it were uniformly distributed), to provide the same concentration as it is in blood plasma at steady state. Steady state is achieved when all variables are constant in spite of ongoing processes.

Data from ClinicalTrials.gov

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