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

Congenital Bleeding Disorder Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00713648
• Other study ID #: F13CD-1725
• Secondary ID: 2006-003148-51
• Status: Completed
• Phase: Phase 3
• First received: July 7, 2008
• Last updated: November 25, 2015
• Start date: August 2008
• Est. completion date: April 2010

Study information

• Verified date: November 2015
• Source: Novo Nordisk A/S
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationAustria: Agency for Health and Food SafetyFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)Germany: Paul-Ehrlich-InstitutItaly: The Italian Medicines AgencySpain: Spanish Agency of MedicinesSwitzerland: Federal Office of Public HealthUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyIsrael: Israeli Health Ministry Pharmaceutical AdministrationCanada: Health CanadaFinland: Finnish Medicines Agency
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 41
• Est. completion date: April 2010
• Est. primary completion date: April 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years and older

Eligibility

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Prevention

Related Conditions & MeSH terms

• Blood Coagulation Disorders
• Congenital Bleeding Disorder
• Congenital FXIII Deficiency
• Factor XIII Deficiency
• Hemostatic Disorders

Intervention

Drug:
• catridecacog
35 IU/kg body weight, i.v. administration, once every 4 weeks

Locations

Country	Name	City	State
United States	Novo Nordisk Clinical Trial Call Center	Atlanta	Georgia
United States	Novo Nordisk Clinical Trial Call Center	Boston	Massachusetts
United States	Novo Nordisk Clinical Trial Call Center	Columbus	Ohio
United States	Novo Nordisk Clinical Trial Call Center	Minneapolis	Minnesota
United States	Novo Nordisk Clinical Trial Call Center	Oklahoma City	Oklahoma
United States	Novo Nordisk Clinical Trial Call Center	Orange	California
United States	Novo Nordisk Clinical Trial Call Center	Phoenix	Arizona
United States	Novo Nordisk Clinical Trial Call Center	Seattle	Washington
United States	Novo Nordisk Clinical Trial Call Center	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Countries where clinical trial is conducted

United States,  Austria,  Canada,  Finland,  France,  Germany,  Israel,  Italy,  Spain,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Rate (Number Per Subject Year) of Bleeding Episodes Requiring Treatment With a FXIII Containing Product During the Treatment Period	It represents the incidence of bleeding episodes requiring treatment with a FXIII-containing product.	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).	No
Secondary	Percentage of Subjects Having a Normal Clot Solubility One Hour After rFXIII Administration and 28 Days After rFXIII Administration for All Dosing Visits	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).	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).	No
Secondary	Level of FXIII Activity One Hour After rFXIII Administration and 28 Days After rFXIII Administration for All Dosing Visits	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).	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).	No
Secondary	Number of Subjects With rFXIII Antibody Development	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)	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).	Yes

External Links

•   Clinical Trials at Novo Nordisk