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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
ClinicalTrials.gov Identifier:
NCT00713648
First received: July 7, 2008
Last updated: September 22, 2011
Last verified: September 2011
History of Changes
  Purpose

The trial is conducted in Asia, Europe and North America. The aim of this trial is to evaluate Recombinant FXIII treatment in patients with FXIII Inherited Deficiency. It is expected that Recombinant FXIII can be used as prevention of bleeding episodes.


Condition Intervention Phase
Congenital FXIII Deficiency
 Drug: recombinant factor XIII
 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:

U.S. FDA Resources

Further study details as provided by Novo Nordisk:

Primary Outcome Measures:
  • Rate (number per subject year) of bleeding episodes requiring treatment with a FXIII containing product during the rFXIII treatment period [ Time Frame: after one year ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Percentage of subjects having a normal clot solubility one hour after rFXIII administration and 28 days after rFXIII administration [ Time Frame: after one year ] [ Designated as safety issue: No ]
  • Level of FXIII activity one hour after rFXIII administration and 28 days after rFXIII administration [ Time Frame: after one year ] [ Designated as safety issue: No ]
  • Number of subjects with rFXIII antibody development [ Time Frame: after one year ] [ Designated as safety issue: Yes ]

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: A Drug: recombinant factor XIII
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:

  • Congenital FXIII A-Subunit deficiency
  • If on regular replacement therapy, then at least one treatment-requiring bleeding episode followed by at least 6 months' treatment, or a documented family history of Congenital FXIII A-Subunit deficiency
  • If on on-demand treatment, then at least two treatment-requiring bleeding episodes within last 12 months

Exclusion Criteria:

  • Known neutralizing antibodies (inhibitors) towards FXIII
  • Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
  • Documented history of 2 or more treatment-requiring bleeding episodes per year during previous regular replacement therapy with FXIII containing blood products
  • Known or suspected allergy to trial product(s) or related products
  • Planned major surgery during the trial period
  • Renal insufficiency defined as current dialysis therapy
  • Any history of confirmed venous or arterial thrombo-embolic events
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00713648

  Show 20 Study Locations
Sponsors and Collaborators
Novo Nordisk
Investigators
Study Director: Gita Ohlsson, MSc, PhD Novo Nordisk
  More Information

Additional Information:
Clinical Trials at Novo Nordisk  This link exits the ClinicalTrials.gov site

No publications provided by Novo Nordisk

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Novo Nordisk
ClinicalTrials.gov Identifier: NCT00713648     History of Changes
Other Study ID Numbers: F13CD-1725, 2006-003148-51
Study First Received: July 7, 2008
Last Updated: September 22, 2011
Health Authority: United States: Institutional Review Board
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, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on May 19, 2013