Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency

NCT01253811 | Completed Phase 3 Results

• 3 other identifiers: F13CD-3835U1111-1117-10632010-020192-23
• Study Type: interventional
• Target: 6
• Locations: 3 countries
• Sites: 4

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.

Conditions

Congenital Bleeding Disorder; Congenital FXIII Deficiency

Outcome Measures

Primary Outcomes (1)

• Number of Treatment Emergent (Serious and Non-serious) Adverse Events

  An adverse event was described as any untoward medical occurrence in a subject or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with this treatment. Treatment emergent adverse events (serious and non-serious), defined as adverse events occurring from first trial product administration to the end of the subject's participation in the trial.

  Week 0 to end of trial visit (week 173) for a minimum period of 52 weeks.

Secondary Outcomes (15)

• Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors.

  Week 0 to end of trial visit (week 173).

• Clinical Laboratory Assessments: Biochemistry: Creatinine

  Every 6th month, from week 24 to end of trial visit (week 173).

• Clinical Laboratory Assessments: Biochemistry: Urea

  Every 6th month, week 24 to end of trial visit (week 173).

• Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)

  Every 6th month, from week 24 to end of trial visit (week 173).

• Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)

  Every 6th month, from week 24 to end of trial visit (week 173).

Study Arms (1)

rFXIII 35 IU/kg

EXPERIMENTAL

Drug: catridecacog

Interventions

catridecacog DRUG

Intravenous injection of a single dose of recombinant factor XIII, 35 IU/kg body weight every 4th week

Also known as: recombinant factor XIII

rFXIII 35 IU/kg

Eligibility Criteria

• Age: 1 Year - 6 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Completed participation in trial F13CD-3760 (NCT01230021)

You may not qualify if:

• 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

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (4)

Novo Nordisk Clinical Trial Call Center

Boston, Massachusetts, 02115, United States

Location

Novo Nordisk Clinical Trial Call Center

Columbus, Ohio, 43205, United States

Location

Unknown Facility

Petah Tikva, 49100, Israel

Location

Unknown Facility

Leicester, LE1 5WW, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 28581691 DERIVED

Related Links

• Clinical Trials at Novo Nordisk

MeSH Terms

Interventions

recombinant factor XIII-A2

Limitations and Caveats

Limitations of the trial included the small number of subjects analysed and the sensitivity of the Berichrom® FXIII activity assay. However, congenital FXIII deficiency is a rare disease and there were no bleeds requiring haemostatic treatment.

Results Point of Contact

• Title: Public Access to Clinical Trials
• Organization: Novo Nordisk A/S

clinicaltrials@novonordisk.com

Study Officials

• Global Clinical Registry (GCR, 1452)

  Novo Nordisk A/S

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 1, 2010
• First Posted: December 3, 2010
• Study Start: January 1, 2011
• Primary Completion: March 1, 2015
• Study Completion: March 1, 2015
• Last Updated: June 24, 2016
• Results First Posted: June 24, 2016

Record last verified: 2016-05

Locations

US (2); IL (1); GB (1)