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

Phase 3

Completed

• Conditions: Congenital Bleeding Disorder; Congenital FXIII Deficiency
• Interventions: Drug: catridecacog

• Registration Number: NCT00713648
• Lead Sponsor: Novo Nordisk A/S

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2008-07-07
• Study First Submitted QC: 2008-07-10
• Study First Posted: 2008-07-11
• Study Start: 2008-08
• Primary Completion: 2010-04
• Study Completion: 2010-04
• Disposition First Submitted: 2010-11-04
• Disposition First Submitted QC: 2011-01-07
• Disposition First Posted: 2011-01-20
• Results First Submitted: 2014-01-22
• Results First Submitted QC: 2014-06-11
• Results First Posted: 2014-07-14
• Status Verified: 2017-01
• Last Update Submitted: 2017-01-10
• Last Update Posted: 2017-02-24

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 41

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)

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
rFXIII	catridecacog	-

Primary Outcome Measures

Name	Time	Method
Rate (Number Per Subject Year) of Bleeding Episodes Requiring Treatment With a FXIII Containing Product During the Treatment Period	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

Name	Time	Method
Percentage of Subjects Having a Normal Clot Solubility One Hour After rFXIII Administration and 28 Days After rFXIII Administration for All Dosing Visits	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).
Level of FXIII Activity One Hour After rFXIII Administration and 28 Days After rFXIII Administration for All Dosing Visits	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).
Number of Subjects With rFXIII Antibody Development	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)

Trial Locations

Locations (1)

Novo Nordisk Investigational Site; 🇬🇧 Newcastle upon Tyne, United Kingdom