Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency: An Extension to Trial F13CD-3760/Mentor™4 (mentor™5)
This study is ongoing, but not recruiting participants.
Sponsor:
Novo Nordisk
Information provided by (Responsible Party):
Novo Nordisk
ClinicalTrials.gov Identifier:
NCT01253811
First received: December 1, 2010
Last updated: April 18, 2013
Last verified: April 2013
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Purpose
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 (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
| Condition | Intervention | Phase |
|---|---|---|
|
Congenital Bleeding Disorder Congenital FXIII Deficiency |
Drug: recombinant factor XIII |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| 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 |
Resource links provided by NLM:
MedlinePlus related topics:
Bleeding Disorders
Drug Information available for:
Factor XIII
U.S. FDA Resources
Further study details as provided by Novo Nordisk:
Primary Outcome Measures:
- Number of treatment emergent (serious and non-serious) adverse events [ Time Frame: every 4th week, from 0 to week 56 ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Frequency of development of anti-rFXIII antibodies, including inhibitors [ Time Frame: every 4th week, from week 0 to week 56 ] [ Designated as safety issue: No ]
- Clinical laboratory assessments: Biochemistry, haematology [ Time Frame: every 6th month, from 0 to week 56 ] [ Designated as safety issue: No ]
- Physical examinations [ Time Frame: every 4th week, from 0 to week 52 ] [ Designated as safety issue: No ]
- Vital signs (Blood Pressure) [ Time Frame: every 4th week, from week 0 to week 56 ] [ Designated as safety issue: No ]
- Vital signs (Pulse) [ Time Frame: every 4th week, from week 0 to week 56 ] [ Designated as safety issue: No ]
- Rate (number per subject year) of all bleeding episodes requiring treatment with a FXIII containing product other than recombinant factor XIII [ Time Frame: weeks 0-52 ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 6 |
| Study Start Date: | January 2011 |
| Estimated Study Completion Date: | June 2014 |
| Estimated Primary Completion Date: | June 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: recombinant factor XIII |
Drug: recombinant factor XIII
Intravenous injection of a single dose of recombinant factor XIII, 35 IU/kg body weight every 4th week
|
Eligibility| Ages Eligible for Study: | 1 Year to 6 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01253811
Locations
| United States, Minnesota | |
| Novo Nordisk Clinical Trial Call Center | |
| Minneapolis, Minnesota, United States, 55404 | |
| United States, Ohio | |
| Novo Nordisk Clinical Trial Call Center | |
| Columbus, Ohio, United States, 43205 | |
| Israel | |
| Petach Tikva, Israel, 49100 | |
| United Kingdom | |
| Manchester, United Kingdom, M13 9WL | |
Sponsors and Collaborators
Novo Nordisk
Investigators
| Study Director: | Gita Ohlsson | Novo Nordisk |
More Information
Additional Information:
No publications provided
| Responsible Party: | Novo Nordisk |
| ClinicalTrials.gov Identifier: | NCT01253811 History of Changes |
| Other Study ID Numbers: | F13CD-3835, U1111-1117-1063, 2010-020192-23 |
| Study First Received: | December 1, 2010 |
| Last Updated: | April 18, 2013 |
| Health Authority: | United States: Food and Drug Administration United Kingdom: Medicines and Healthcare Regulatory Authority (MHRA) Israel: Ministry of Health |
Additional relevant MeSH terms:
|
Blood Coagulation Disorders Hemostatic Disorders Hemorrhagic Disorders Hemorrhage |
Hematologic Diseases Vascular Diseases Cardiovascular Diseases Pathologic Processes |
ClinicalTrials.gov processed this record on May 23, 2013