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Completed

NCT Number: NCT01230021

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

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.

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Key information

Conditions

Congenital Bleeding Disorder Congenital FXIII Deficiency

Age range

1 year–6 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Novo Nordisk Investigational Site, Petah Tikva, Israel

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Who can participate

Healthy volunteers accepted: No

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

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

Treatment and study plan

catridecacog

Drug

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

Primary outcomes

  1. Area Under the Concentration vs. Time Curve (AUC)

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

    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.

Secondary outcomes

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

    Time frame: From day 0 to day 30

    A measure of exposure.

  2. Maximum Plasma Concentration (Cmax) for FXIII

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

    Maximum plasma concentration of the drug reached.

  3. Terminal Half-life (t½)

    Time frame: From day 0 to day 30

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

  4. Mean Residence Time (MRT)

    Time frame: From day 0 to day 30

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

  5. Total Plasma Clearance (CL)

    Time frame: From day 0 to day 30

    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').

  6. Volume of Distribution at Steady State (Vss)

    Time frame: At steady state

    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.

  7. Percentage of Subjects With One or More Adverse Events (AEs) Recorded

    Time frame: From day 0 to day 30

  8. Percentage of Subjects With One or More Serious Adverse Events (SAEs)

    Time frame: From day 0 to day 30

  9. Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors (Neutralising Antibodies Against Factor XIII)

    Time frame: At screening and day 30

  10. Coagulation Related Parameters - Fibrinogen

    Time frame: Day 0 and at day 30

  11. Coagulation Related Parameters - Activated Partial Thromboplastin Time (aPTT, Seconds)

    Time frame: Day 0 and day 30

  12. Coagulation Related Parameters - Prothrombin Time (PT) (Seconds)

    Time frame: Day 0 and day 30

  13. Clot Solubility Test (Evaluated as Normal/Abnormal)

    Time frame: Day 0 and day 30

    Blood samples for clot solubility drawn at each visit (1 hour before and after dose administration). A clot solubility assay was used to screen for FXIII deficiency. The assay is based on the ability of urea to dissolve fibrin clots that have not undergone FXIII-induced stabilization. Normal blood clots generally remain stable for 24 hours or more, while clots in which fibrin molecules have not been cross-linked are soluble within minutes. The outcome of the test is normal (FXIII present; a clot is observed in the test tube) or abnormal (FXIII absent or very low level; no clot in test tube).

  14. Vital Signs - Pulse

    Time frame: Day 0 and day 30

  15. Vital Signs - Blood Pressure (Systolic and Diastolic)

    Time frame: Day 0 and day 30

  16. Physical Examination (Evaluated as Normal/Abnormal)

    Time frame: From day 0 to day 30

Sponsors and collaborators

Lead sponsor

Novo Nordisk A/S

Industry

Registry information

Official study 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

Acronym: mentor™4

Important dates

Study start
2010
Primary completion
2012
Study completion
2012
First posted
Oct 28, 2010
Registry last updated
Feb 24, 2017

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

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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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