Trial record 8 of 8 for:    "Factor XIII deficiency"

Evaluation of Recombinant Factor XIII for Prevention of Bleeding in Patients With FXIII Inherited Deficiency

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT00713648
First received: July 7, 2008
Last updated: June 11, 2014
Last verified: June 2014
  Purpose

The trial is conducted in Europe, North America and Asia. The aim of this trial is to evaluate catridecacog (recombinant factor XIII (rFXIII)) treatment in patients with inherited FXIII deficiency. It is expected that recombinant FXIII can be used for the prevention of bleeding episodes.


Condition Intervention Phase
Congenital Bleeding Disorder
Congenital FXIII Deficiency
Drug: catridecacog
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Prevention
Official Title: A Multi-Centre, Open-Label, Single-Arm and Multiple Dosing Trial on Efficacy and Safety of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Subjects With Congenital Factor XIII Deficiency

Resource links provided by NLM:


Further study details as provided by Novo Nordisk A/S:

Primary Outcome Measures:
  • Rate (Number Per Subject Year) of Bleeding Episodes Requiring Treatment With a FXIII Containing Product During the Treatment Period [ Time Frame: For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16). ] [ Designated as safety issue: No ]
    It represents the incidence of bleeding episodes requiring treatment with a FXIII-containing product.


Secondary Outcome Measures:
  • Percentage of Subjects Having a Normal Clot Solubility One Hour After rFXIII Administration and 28 Days After rFXIII Administration for All Dosing Visits [ Time Frame: For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16). ] [ Designated as safety issue: No ]
    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).

  • Level of FXIII Activity One Hour After rFXIII Administration and 28 Days After rFXIII Administration for All Dosing Visits [ Time Frame: For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16). ] [ Designated as safety issue: No ]
    Subjects entered a 52-week treatment period of monthly (28±2 days) doses of 35 IU/kg rFXIII. Blood samples for analysis of FXIII activity were drawn at each visit; at dosing visits blood was drawn 1 hour after administration and before administration(corresponding to 28 days after the previous dose). All Dosing Visits are visits where a dose is given (i.e. Visit 2-15 except Visit 3).

  • Number of Subjects With rFXIII Antibody Development [ Time Frame: For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16). ] [ Designated as safety issue: Yes ]
    Subjects receiving rFXIII were monitored for the development of binding antibodies. Blood sampling was done before administration of trial product at all visits (Visits 1-16 and unscheduled visit)


Enrollment: 41
Study Start Date: August 2008
Study Completion Date: April 2010
Primary Completion Date: April 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: rFXIII Drug: catridecacog
35 IU/kg body weight, i.v. administration, once every 4 weeks

  Eligibility

Ages Eligible for Study:   6 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit)
  • Treatment with regular FXIII replacement therapy initiated at least 6 months prior to screening and one of the following : a documented history of at least one 1 treatment-requiring bleeding episode prior to initiation of regular replacement therapy or a documented family history of FXIII congenital deficiency (only for subjects on regular replacement therapy prior to screening)
  • Documented history of at least two 2 bleeding episodes requiring treatment with FXIII containing blood products within the last 12 months prior to screening (only for subjects receiving on-demand treatment prior to screening)

Exclusion Criteria:

  • Known neutralizing antibodies (inhibitors) towards FXIII
  • Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
  • Documented history of at least 2 treatment-requiring bleeding episodes per year during previous regular replacement therapy with FXIII containing blood products (fresh frozen plasma (FFP), plasma-derived FXIII (pd FXIII) and cryoprecipitate)
  • Known or suspected allergy to trial product(s) or related products
  • Planned major surgery during the trial period. Catheter, ports and dental extractions do not count as surgeries and will not exclude the subject
  • Renal insufficiency defined as current dialysis therapy
  • Any history of confirmed venous or arterial thrombo-embolic events
  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00713648

  Show 20 Study Locations
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
  More Information

Additional Information:
No publications provided by Novo Nordisk A/S

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT00713648     History of Changes
Other Study ID Numbers: F13CD-1725, 2006-003148-51
Study First Received: July 7, 2008
Results First Received: January 22, 2014
Last Updated: June 11, 2014
Health Authority: United States: Food and Drug Administration
Austria: Agency for Health and Food Safety
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Paul-Ehrlich-Institut
Italy: The Italian Medicines Agency
Spain: Spanish Agency of Medicines
Switzerland: Federal Office of Public Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Israel: Israeli Health Ministry Pharmaceutical Administration
Canada: Health Canada
Finland: Finnish Medicines Agency

Additional relevant MeSH terms:
Factor XIII Deficiency
Blood Coagulation Disorders
Hemorrhage
Hemostatic Disorders
Blood Coagulation Disorders, Inherited
Cardiovascular Diseases
Coagulation Protein Disorders
Genetic Diseases, Inborn
Hematologic Diseases
Hemorrhagic Disorders
Pathologic Processes
Vascular Diseases

ClinicalTrials.gov processed this record on October 23, 2014