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

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ClinicalTrials.gov Identifier: NCT00713648
Recruitment Status : Completed
First Posted : July 11, 2008
Results First Posted : July 14, 2014
Last Update Posted : February 24, 2017
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Study Description
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Brief Summary:
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 or disease Intervention/treatment Phase
Congenital Bleeding Disorder Congenital FXIII Deficiency Drug: catridecacog Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 41 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : August 2008
Actual Primary Completion Date : April 2010
Actual Study Completion Date : April 2010

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Bleeding

Arms and Interventions
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Arm Intervention/treatment
Experimental: rFXIII Drug: catridecacog
35 IU/kg body weight, i.v. administration, once every 4 weeks


Outcome Measures
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Primary Outcome Measures :
  1. 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). ]
    It represents the incidence of bleeding episodes requiring treatment with a FXIII-containing product.


Secondary Outcome Measures :
  1. 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). ]
    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).

  2. 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). ]
    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).

  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). ]
    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)


Eligibility Criteria
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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
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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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00713648


Locations
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United States, Arizona
Novo Nordisk Investigational Site
Phoenix, Arizona, United States, 85016-7710
United States, California
Novo Nordisk Investigational Site
Orange, California, United States, 92868
United States, Florida
Novo Nordisk Investigational Site
Tampa, Florida, United States, 33607
United States, Georgia
Novo Nordisk Investigational Site
Atlanta, Georgia, United States, 30322
United States, Massachusetts
Novo Nordisk Investigational Site
Boston, Massachusetts, United States, 02115
United States, Michigan
Novo Nordisk Investigational Site
Detroit, Michigan, United States, 48201
Novo Nordisk Investigational Site
East Lansing, Michigan, United States, 48823
United States, Minnesota
Novo Nordisk Investigational Site
Minneapolis, Minnesota, United States, 55404
United States, Ohio
Novo Nordisk Investigational Site
Columbus, Ohio, United States, 43205
United States, Oklahoma
Novo Nordisk Investigational Site
Oklahoma City, Oklahoma, United States, 73104
United States, Washington
Novo Nordisk Investigational Site
Seattle, Washington, United States, 98104
Austria
Novo Nordisk Investigational Site
Graz, Austria, 8036
Novo Nordisk Investigational Site
Klagenfurt, Austria, A-9020
Novo Nordisk Investigational Site
Wien, Austria, 1090
Canada, Ontario
Novo Nordisk Investigational Site
Toronto, Ontario, Canada, M5G 1X8
Finland
Novo Nordisk Investigational Site
Helsinki, Finland, 00290
France
Novo Nordisk Investigational Site
Le Kremlin Bicetre, France, 94270
Novo Nordisk Investigational Site
Marseille, France, 13385
Novo Nordisk Investigational Site
Montpellier, France, 34295
Germany
Novo Nordisk Investigational Site
Bonn, Germany, 53127
Novo Nordisk Investigational Site
Braunschweig, Germany, 38118
Novo Nordisk Investigational Site
Duisburg, Germany, 47051
Israel
Novo Nordisk Investigational Site
Petach Tikva, Israel, 49100
Novo Nordisk Investigational Site
Tel-Hashomer, Israel, 52621
Italy
Novo Nordisk Investigational Site
Vicenza, Italy, 36100
Spain
Novo Nordisk Investigational Site
Barcelona, Spain, 08035
Novo Nordisk Investigational Site
Sevilla, Spain, 41013
Switzerland
Novo Nordisk Investigational Site
Zürich, Switzerland, 8091
United Kingdom
Novo Nordisk Investigational Site
Aberdeen, United Kingdom, AB25 2ZN
Novo Nordisk Investigational Site
Birmingham, United Kingdom, B4 6NH
Novo Nordisk Investigational Site
Bradford, United Kingdom, BD9 6RJ
Novo Nordisk Investigational Site
Bristol, United Kingdom, BS2 8ED
Novo Nordisk Investigational Site
Liverpool, United Kingdom, L12 2AP
Novo Nordisk Investigational Site
London, United Kingdom, WC1N 3JH
Novo Nordisk Investigational Site
Newcastle upon Tyne, United Kingdom, NE1 4LP
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
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Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
More Information
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Additional Information:

Publications of Results:

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Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT00713648    
Other Study ID Numbers: F13CD-1725
2006-003148-51 ( EudraCT Number )
First Posted: July 11, 2008    Key Record Dates
Results First Posted: July 14, 2014
Last Update Posted: February 24, 2017
Last Verified: January 2017
Additional relevant MeSH terms:
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Hemostatic Disorders
Blood Coagulation Disorders
Hemorrhage
Pathologic Processes
 Hematologic Diseases
Vascular Diseases
Cardiovascular Diseases
Hemorrhagic Disorders