Clinical trial NCT01253811: Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency
Official title: A Multi-Centre, Multinational, Open-Label, Single-Arm and Multiple Dosing Trial on Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency. Safety Extension Trial to F13CD-3760
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Congenital Bleeding Disorder
Congenital FXIII Deficiency
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2010-12-03
Enrollment: 6
Sex: ALL
Minimum age (years): 1
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This trial will be conducted in Asia, Europe and the United States of America (USA).

The aim of this clinical trial is to investigate long-term safety of rFXIII when administered for prevention of bleeding episodes in children aged between 1 and 6 years with congenital FXIII A-subunit deficiency. This trial is an extension to trial F13CD-3760 (mentor™4, NCT01230021). If applicable the trial will be extended up to maximum 3 years dependent on when recombinant factor XIII will be commercially available in subject's respective country for use in children of 1-6 years of age.


Eligibility criteria: Inclusion Criteria:

* Completed participation in trial F13CD-3760 (NCT01230021)

Exclusion Criteria:

* Known or suspected hypersensitivity to trial product or related products
* Known history of development of inhibitors against FXIII (factor XIII)
* Hereditary or acquired coagulation disorder other than FXIII congenital deficiency
* Platelet count (thrombocytes) less than 50X10e9 / L
* 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
* Any disease or condition which, judged by the trial physician, could imply a potential hazard to the subject, interfere with the trial participation or trial outcome including renal and/or liver dysfunction

Primary outcomes:
measure: Number of Treatment Emergent (Serious and Non-serious) Adverse Events
timeFrame: Week 0 to end of trial visit (week 173) for a minimum period of 52 weeks.
Secondary outcomes:
measure: Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors.
timeFrame: Week 0 to end of trial visit (week 173).
measure: Clinical Laboratory Assessments: Biochemistry: Creatinine
timeFrame: Every 6th month, from week 24 to end of trial visit (week 173).
measure: Clinical Laboratory Assessments: Biochemistry: Urea
timeFrame: Every 6th month, week 24 to end of trial visit (week 173).
measure: Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)
timeFrame: Every 6th month, from week 24 to end of trial visit (week 173).
measure: Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)
timeFrame: Every 6th month, from week 24 to end of trial visit (week 173).
measure: Clinical Laboratory Assessments: Haematology: Haemoglobin
timeFrame: Every 6th month, from week 0 to end of trial visit (week 173).
measure: Clinical Laboratory Assessments: Haematology: Leucocytes
timeFrame: Every 6th month, from week 0 to end of trial visit (week 173).
measure: Clinical Laboratory Assessments: Haematology: Thrombocytes
timeFrame: Every 6th month, from week 0 to end of trial visit (week 173).
measure: Clinical Laboratory Assessments: Haematology: Erythrocytes
timeFrame: Every 6th month, from week 0 to end of trial visit (week 173).
measure: Clinical Laboratory Assessments: Haematology: Haematocrit
timeFrame: Every 6th month, from week 0 to end of trial visit (week 173).
measure: Physical Examinations
timeFrame: Week 0 to end of trial visit (week 173).
measure: Vital Signs: Systolic BP (Blood Pressure)
timeFrame: Week 0 to end of trial visit (week 173).
measure: Vital Signs: Diastolic BP (Blood Pressure)
timeFrame: Week 0 to end of trial visit (week 173).
measure: Vital Signs: Pulse
timeFrame: Week 0 to end of trial visit (week 173).
measure: Rate (Number Per Subject Year) of All Bleeding Episodes Requiring Treatment With a FXIII Containing Product Other Than Recombinant Factor XIII.
timeFrame: Weeks 0 to end of trial visit (week 173).
Other outcomes:
Locations:
city: Boston
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: Massachusetts
city: Columbus
country: United States
facility: Novo Nordisk Clinical Trial Call Center
state: Ohio
city: Petach Tikva
country: Israel
city: Leicester
country: United Kingdom
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References:
citation: Kerlin BA, Inbal A, Will A, Williams M, Garly ML, Jacobsen L, Kearney SL. Recombinant factor XIII prophylaxis is safe and effective in young children with congenital factor XIII-A deficiency: international phase 3b trial results. J Thromb Haemost. 2017 Aug;15(8):1601-1606. doi: 10.1111/jth.13748. Epub 2017 Jul 10.