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Trial record 6 of 10 for:
"Factor XIII deficiency"
Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725 (mentor™2)
This study has been completed.
Sponsor:
Novo Nordisk A/S
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT00978380
First received: September 15, 2009
Last updated: February 28, 2017
Last verified: February 2017
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Purpose
This trial is conducted in Asia, Europe and North America. The aim of the trial is to investigate the safety of monthly replacement therapy of recombinant factor XIII in patients with congenital FXIII deficiency. The trial continues until the product is commercially available, but an interim assessment will take place when all subjects have completed 52 weeks in the trial.
| Condition | Intervention | Phase |
|---|---|---|
| Congenital Bleeding Disorder Congenital FXIII Deficiency | Drug: catridecacog | Phase 3 |
| Study Type: | Interventional |
| Study Design: | Intervention Model: Single Group Assignment Masking: No masking Primary Purpose: Prevention |
| Official Title: | A Multi-Centre, Open-Label, Single-Arm, and Multiple Dosing Trial on Safety of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Subjects With Congenital Factor XIII Deficiency |
Resource links provided by NLM:
Genetics Home Reference related topics:
factor XIII deficiency
Drug Information available for:
Blood-coagulation factor XIII
U.S. FDA Resources
Further study details as provided by Novo Nordisk A/S:
Primary Outcome Measures:
- Adverse Events (AEs)(Serious and Non-serious) [ Time Frame: All AEs were collected and reported from screening (week 0) for a minimum period of 52 weeks or until the end of trial visit. ]An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Trial AEs (serious) included any event such as death, life-threatening experience, in-subject hospitalisation, significant disability/ congential anomaly experienced from the trial product.
Secondary Outcome Measures:
- Antibody and Inhibitor Development [ Time Frame: From week 0 to week 52 ]All subjects who received rFXIII were monitored for anti-rFXIII antibodies and inhibitor development. Samples passed through 2 tiers of ELISA testing: an initial screen with a specific cut-off point (including ~5% false positives) and a second confirmatory assay for samples yielding a result above the screening cut-off point. If samples were confirmed as antibody positive in the confirmation assay, an inhibitor assay was also carried out to detect functional inhibitors. Percentage of subjects with antibody and inhibitor development were reported.
| Enrollment: | 63 |
| Actual Study Start Date: | September 21, 2009 |
| Study Completion Date: | October 20, 2015 |
| Primary Completion Date: | October 20, 2015 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: A |
Drug: catridecacog
Monthly administration of recombinant factor XIII as preventative treatment of bleeding episodes. Dose: 35 IU/kg body weight intravenous (into the vein)
Other Name: recombinant factor XIII
|
Eligibility| Ages Eligible for Study: | 6 Years and older (Child, Adult, Senior) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- For subjects who participated in F13CD-1725:
- Previous participation (means up to and inclusive Visit 16, (End of Trial)) in F13CD-1725
- For all other subjects:
- Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit or documented results from previously performed genotyping)
- Body weight at least 20 kg
Exclusion Criteria:
- Known neutralizing antibodies (inhibitors) towards FXIII
- Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
- Platelet count (thrombocytes) of less than 50 × 109/L. For subjects who participated in F13CD-1725 platelet count from visit 15 in F13CD-1725 must be used for evaluation.
- Females of childbearing potential who are pregnant, breastfeeding or are not using adequate contraceptive methods
Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00978380
Show 34 Study Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00978380
Show 34 Study Locations
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
| Study Director: | Global Clinical Registry (GCR, 1452) | Novo Nordisk A/S |
More Information
Additional Information:
Publications:
| Responsible Party: | Novo Nordisk A/S |
| ClinicalTrials.gov Identifier: | NCT00978380 History of Changes |
| Other Study ID Numbers: |
F13CD-3720 2008-007883-41 ( EudraCT Number ) U1111-1111-9289 ( Other Identifier: WHO ) JapicCTI-121958 ( Registry Identifier: JAPIC ) |
| Study First Received: | September 15, 2009 |
| Results First Received: | October 19, 2016 |
| Last Updated: | February 28, 2017 |
Additional relevant MeSH terms:
|
Blood Coagulation Disorders Hemostatic Disorders Factor XIII Deficiency Hematologic Diseases Vascular Diseases |
Cardiovascular Diseases Hemorrhagic Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Genetic Diseases, Inborn |
ClinicalTrials.gov processed this record on June 26, 2017