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Completed

NCT Number: NCT00883090

A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency

Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding. This study will evaluate the safety and recommended (best) amount or level of Factor XIII in a patient's blood. Factor XIII Concentrate (Human) is given to people whose blood is lacking Factor XIII. Factor XIII Concentrate (Human) works by assisting your blood in the usual clotting process, thereby preventing bleeding.

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Key information

Conditions

Factor XIII Deficiency Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Study Site, Santa Cruz de Tenerife, Spain

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Written informed consent/assent for study participation obtained before undergoing any study-specific procedures
  • Documented congenital FXIII deficiency that requires prophylactic treatment with a FXIII containing product.
  • Males and females of any age with congenital FXIII deficiency.
  • Received full hepatitis B vaccination and/or is hepatitis B surface antibody positive

Exclusion criteria

  • Diagnosis of acquired FXIII deficiency
  • Administration of a FXIII-containing product, including blood transfusions or other blood products within 4 weeks prior to the planned Day 0
  • Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
  • Known or suspected to have antibodies towards FXIII
  • Use of any other investigational medicinal product within 4 weeks prior to the Baseline Visit (Day 0)
  • Positive result at screening for human immunodeficiency virus (HIV)
  • Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) concentration >2.5 times the upper limit of normal
  • Fibrinogen < lower limit of normal
  • Active bleeding
  • Pregnant or breast-feeding
  • Intention to become pregnant during the course of the study
  • Female subjects of childbearing potential not using, or not willing to use, a medically reliable method of contraception for the entire duration of the study
  • Surgical procedure anticipated during the study period
  • Suspected inability (e.g., language problems) or unwillingness to comply with study procedures or history of noncompliance

Treatment and study plan

FXIII Concentrate (Human)

Biological

Subjects will receive approximately 40 U/kg of FXIII every 28 days for 3 doses administered as a bolus intravenous (IV) injection at approximately 250 U/minute.

Other names: Fibrogammin®-P

Primary outcomes

  1. Peak FXIII Concentration at Steady State

    Time frame: 12 weeks

  2. Trough FXIII Concentration at Steady State

    Time frame: 12 weeks

  3. Time to Peak Concentration

    Time frame: 12 weeks

  4. Incremental Recovery

    Time frame: 12 weeks

    Incremental recovery (U/mL/U/kg) is defined as the maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of FXIII (U/kg) administered.

  5. Terminal Half-life

    Time frame: 12 weeks

  6. Area Under the Curve at Steady State

    Time frame: 12 weeks

  7. Clearance

    Time frame: 12 weeks

  8. Volume of Distribution at Steady State

    Time frame: 12 weeks

  9. Mean Residence Time

    Time frame: 12 weeks

Secondary outcomes

  1. Adverse Events

    Time frame: 16 weeks

    Number of participants with an adverse event

  2. Laboratory Safety Parameters

    Time frame: 16 weeks

    Number of participants with clinically significant laboratory safety parameter values. The laboratory safety parameters measured included serum chemistries, hematology and urinalysis.

  3. Vital Signs

    Time frame: 16 weeks

    Number of participants with clinically significant vital signs. The vital signs measured included blood pressure, pulse rate and temperature. Clinically significant changes in vital signs were to be reported as adverse events.

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

A 12 Week, Multicenter, Pharmacokinetic and Safety Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

Important dates

Study start
2009
Primary completion
2010
Study completion
2010
First posted
Apr 17, 2009
Registry last updated
Jan 16, 2012

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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