NCT01230021|CompletedPhase 3Results

Safety of a Single Intravenous Dose of Recombinant Factor XIII in Children With Congenital FXIII A-subunit Deficiency

mentor™4

A Phase 3b Trial Investigating the Pharmacokinetics and Safety Profile of a Single Intravenous Dose of rFXIII in Paediatric (1 to Less Than 6 Years Old) Subjects With Congenital FXIII A-subunit Deficiency

Novo Nordisk A/S ClinicalTrials.gov

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3 other identifiers

F13CD-3760U1111-1116-25332009-016869-28

Study Type

interventional

Target

Locations

3 countries

Sites

Timeline

RegisteredOct 2010

StartedNov 2010

CompletionJan 2012

Brief Summary

This trial is conducted in Europe and United States of America (USA). The aim of this clinical trial is to investigate the pharmacokinetics (at which rate the substance is distributed and eliminated from the body) and the safety profile of catridecacog (recombinant factor XIII (rFXIII)) in children with congenital FXIII A-subunit deficiency. Young children (1 to less than 6 years old) with congenital FXIII deficiency are evaluated.

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 Nov 2010

Shorter than P25 for phase_3

Geographic Reach

3 countries

10 active sites

Status

completed

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

Trial Relationships

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Study Timeline

Key milestones and dates

1.2 years study duration

First Submitted

Initial submission to the registry

October 27, 2010

Completed

1 day until next milestone

First Posted

Study publicly available on registry

October 28, 2010

Completed

4 days until next milestone

Study Start

First participant enrolled

November 1, 2010

Completed

1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2012

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2012

Completed

2.4 years until next milestone

Results Posted

Study results publicly available

June 12, 2014

Completed

Last Updated

February 24, 2017

Status Verified

January 1, 2017

Enrollment Period

1.2 years

First QC Date

October 27, 2010

Results QC Date

January 22, 2014

Last Update Submit

January 10, 2017

Conditions

Congenital Bleeding Disorder Congenital FXIII Deficiency

Outcome Measures

Primary Outcomes (1)

Area Under the Concentration vs. Time Curve (AUC)
A measure of the exposure. Blood samples for the PK assessment were drawn pre-dose and up to 30 days after dosing. The PK of FXIII in children was assessed after a single i.v. dose of rFXIII 35 IU/kg.
At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing

Secondary Outcomes (16)

Area Under the Concentration vs. Time Curve (AUC0-∞)
From day 0 to day 30
Maximum Plasma Concentration (Cmax) for FXIII
At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing
Terminal Half-life (t½)
From day 0 to day 30
Mean Residence Time (MRT)
From day 0 to day 30
Total Plasma Clearance (CL)
From day 0 to day 30
+11 more secondary outcomes

Study Arms (1)

recombinant factor XIII

EXPERIMENTAL

Drug: catridecacog

Interventions

catridecacogDRUG

Intravenous injection of a single dose of recombinant factor XIII, 35 IU/kg bodyweight

recombinant factor XIII

Eligibility Criteria

Age1 Year - 6 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

Signed Informed Consent by subject's parents or subject's legally acceptable representative before any trial related activities. Trial related activities are any procedures that would not have been performed during the normal management of the subject
Age 1 to less than 6 years old at the time of enrolment
Congenital FXIII subunit-A deficiency previously documented by genotyping or evaluated by genotyping through blood sampling at screening visit
Body weight at least 10 kg

You may not qualify if:

Known antibodies to FXIII
Hereditary or acquired coagulation disorder other than FXIII A-subunit congenital deficiency
Platelet count (thrombocytes) of less than 50 × 10\^9/L (at screening visit)
Previous history of autoimmune disorder involving autoantibodies e.g., systemic lupus erythematosus
Previous history of arterial or venous thromboembolic events e.g., cerebrovascular accident or deep vein thrombosis
Known or suspected allergy to trial product or related products
Any surgical procedure in the 30 days prior to enrolment and any planned surgery during the trial period
Any disease or condition which, judged by the Investigator, could imply a potential hazard to the subject or interfere with the trial participation or trial outcome including renal and/or liver dysfunction

Contact the study team to confirm eligibility.