NCT00713648|CompletedPhase 3Results

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

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

Novo Nordisk A/S ClinicalTrials.gov

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2 other identifiers

F13CD-17252006-003148-51

Study Type

interventional

Target

Locations

11 countries

Sites

Timeline

RegisteredJul 2008

StartedAug 2008

CompletionApr 2010

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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at below P25 for phase_3

Timeline

Completed

Started Aug 2008

Geographic Reach

11 countries

35 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

1.7 years study duration

First Submitted

Initial submission to the registry

July 7, 2008

Completed

4 days until next milestone

First Posted

Study publicly available on registry

July 11, 2008

Completed

21 days until next milestone

Study Start

First participant enrolled

August 1, 2008

Completed

1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2010

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2010

Completed

4.3 years until next milestone

Results Posted

Study results publicly available

July 14, 2014

Completed

Last Updated

February 24, 2017

Status Verified

January 1, 2017

Enrollment Period

1.7 years

First QC Date

July 7, 2008

Results QC Date

January 22, 2014

Last Update Submit

January 10, 2017

Conditions

Congenital Bleeding Disorder Congenital FXIII Deficiency

Outcome Measures

Primary Outcomes (1)

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).

Secondary Outcomes (3)

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).
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).
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).

Study Arms (1)

rFXIII

EXPERIMENTAL

Drug: catridecacog

Interventions

catridecacogDRUG

35 IU/kg body weight, i.v. administration, once every 4 weeks

rFXIII

Eligibility Criteria

Age6 Years+

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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)

You may not qualify if:

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

Contact the study team to confirm eligibility.