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Trial record 17 of 460 for:    Inherited Bleeding Disorder

Evaluation of Recombinant Factor XIII for Prevention of Bleeding in Patients With FXIII Inherited Deficiency

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ClinicalTrials.gov Identifier: NCT00713648
Recruitment Status : Completed
First Posted : July 11, 2008
Results First Posted : July 14, 2014
Last Update Posted : February 24, 2017
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Prevention
Conditions Congenital Bleeding Disorder
Congenital FXIII Deficiency
Intervention Drug: catridecacog
Enrollment 41
Recruitment Details Of a total of 29 initiated trial sites, 23 sites enrolled and dosed at least one patient. The country distribution was (number of actively recruiting sites per country in parenthesis): Austria (1), Canada (1), Finland (1), France (1), Germany (3), Israel (2), Italy (1), Spain (1), Switzerland (1), UK (3) and United States of America (8)
Pre-assignment Details After screening, eligible subjects entered a 4-week run-in period followed by a 52-week recombinant factor XIII (rFXIII) treatment period. Subjects who before entering the trial were receiving regular replacement therapy with a FXIII-containing product were to receive their last standard replacement dose just before the screening visit.
Arm/Group Title rFXIII
Hide Arm/Group Description Subjects received 35 IU/kg rFXIII every 4th week (28±2 days) during a treatment period of 52 weeks. In case of acute bleeding episodes, any additional treatment as per investigator judgment was to be according to local standard practice. Additional doses of rFXIII could therefore not be used to treat such breakthrough bleedings
Period Title: Overall Study
Started 41
Completed 33
Not Completed 8
Reason Not Completed
Adverse Event             1
Unclassified             2
According to Protocol             2
Development of antibodies             3
Arm/Group Title rFXIII
Hide Arm/Group Description Subjects received 35 IU/kg rFXIII every 4th week (28±2 days) during a treatment period of 52 weeks. In case of acute bleeding episodes, any additional treatment as per investigator judgment was to be according to local standard practice. Additional doses of rFXIII could therefore not be used to treat such breakthrough bleedings
Overall Number of Baseline Participants 41
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 41 participants
26.4  (15.9)
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 41 participants
Female
18
  43.9%
Male
23
  56.1%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 41 participants
Black or African American 2
White 28
Asian 5
Other 5
Unknown 1
1.Primary Outcome
Title Rate (Number Per Subject Year) of Bleeding Episodes Requiring Treatment With a FXIII Containing Product During the Treatment Period
Hide Description It represents the incidence of bleeding episodes requiring treatment with a FXIII-containing product.
Time Frame For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set - consisting of a total of 41 subjects who received at least one dose of trial product.
Arm/Group Title rFXIII
Hide Arm/Group Description:
Subjects received 35 IU/kg rFXIII every 4th week (28±2 days) during a treatment period of 52 weeks. In case of acute bleeding episodes, any additional treatment as per investigator judgment was to be according to local standard practice. Additional doses of rFXIII could therefore not be used to treat such breakthrough bleedings
Overall Number of Participants Analyzed 41
Mean (Full Range)
Unit of Measure: bleeding episodes per subject per year
0.138
(0 to 2)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection rFXIII
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value [Not Specified]
Comments [Not Specified]
Method Poisson model (log-link)
Comments The estimated rate was adjusted for age and overdispersion which was estimated by Pearson’s chi-square statistic divided by its degrees of freedom.
Method of Estimation Estimation Parameter Mean (Lambda)
Estimated Value 0.048
Confidence Interval 95%
0.0094 to 0.2501
Estimation Comments Mean (Lambda) refer to the estimate of the annualised bleeding rate
2.Secondary Outcome
Title Percentage of Subjects Having a Normal Clot Solubility One Hour After rFXIII Administration and 28 Days After rFXIII Administration for All Dosing Visits
Hide Description Blood samples for clot solubility drawn at each visit (1 hour before and after dose administration). A clot solubility assay was used to screen for FXIII deficiency. The assay is based on the ability of urea to dissolve fibrin clots that have not undergone FXIII-induced stabilization. Normal blood clots generally remain stable for 24 hours or more, while clots in which fibrin molecules have not been cross-linked are soluble within minutes. The outcome of the test is normal (FXIII present; a clot is observed in the test tube) or abnormal (FXIII absent or very low level; no clot in test tube).
Time Frame For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set - consisting of a total of 41 subjects who received at least one dose of trial product. For All Dosing Visits, the participants analyzed (N) are the 'All Visit Subjects Count' i.e. the sum of subject counts across all dosing visits combined.
Arm/Group Title rFXIII
Hide Arm/Group Description:
Subjects received 35 IU/kg rFXIII every 4th week (28±2 days) during a treatment period of 52 weeks. In case of acute bleeding episodes, any additional treatment as per investigator judgment was to be according to local standard practice. Additional doses of rFXIII could therefore not be used to treat such breakthrough bleedings
Overall Number of Participants Analyzed 41
Measure Type: Number
Unit of Measure: percentage (%) of subjects
After 1 hour post-dosing (N=444) 98.2
After 28 days of treatment (N=419) 91.3
3.Secondary Outcome
Title Level of FXIII Activity One Hour After rFXIII Administration and 28 Days After rFXIII Administration for All Dosing Visits
Hide Description Subjects entered a 52-week treatment period of monthly (28±2 days) doses of 35 IU/kg rFXIII. Blood samples for analysis of FXIII activity were drawn at each visit; at dosing visits blood was drawn 1 hour after administration and before administration(corresponding to 28 days after the previous dose). All Dosing Visits are visits where a dose is given (i.e. Visit 2-15 except Visit 3).
Time Frame For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set - consisting of a total of 41 subjects who received at least one dose of trial product. For All Dosing Visits, the participants analyzed (N) are the 'All Visit Subjects Count' i.e. the sum of subject counts across all dosing visits combined.
Arm/Group Title rFXIII
Hide Arm/Group Description:
Subjects received 35 IU/kg rFXIII every 4th week (28±2 days) during a treatment period of 52 weeks. In case of acute bleeding episodes, any additional treatment as per investigator judgment was to be according to local standard practice. Additional doses of rFXIII could therefore not be used to treat such breakthrough bleedings
Overall Number of Participants Analyzed 41
Mean (Standard Deviation)
Unit of Measure: U/kg
After 1 hour post-dosing (N=453) 0.782  (0.342)
After 28 days of treatment (N=459) 0.189  (0.102)
4.Secondary Outcome
Title Number of Subjects With rFXIII Antibody Development
Hide Description Subjects receiving rFXIII were monitored for the development of binding antibodies. Blood sampling was done before administration of trial product at all visits (Visits 1-16 and unscheduled visit)
Time Frame For a period of 322 days (approximately one year) comprised of a screening visit (Visit 1), treatment period (Visits 2-15), unscheduled visit and end-of-trial visit (Visit 16).
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set - Subjects who received at least one dose of trial product.
Arm/Group Title rFXIII
Hide Arm/Group Description:
Subjects received 35 IU/kg rFXIII every 4th week (28±2 days) during a treatment period of 52 weeks. In case of acute bleeding episodes, any additional treatment as per investigator judgment was to be according to local standard practice. Additional doses of rFXIII could therefore not be used to treat such breakthrough bleedings
Overall Number of Participants Analyzed 41
Measure Type: Number
Unit of Measure: participants
4
Time Frame Adverse events (AEs) were collected and reported during the entire study period i.e. approximately one year (322 days)
Adverse Event Reporting Description All AEs observed by the investigator or reported spontaneously by the subjects, were recorded by the investigator at each contact with the site (visit or telephone, excluding safety visits, where the subject was not seeing the Investigator or his staff (e.g.visits to the laboratory))
 
Arm/Group Title rFXIII
Hide Arm/Group Description Subjects received 35 IU/kg rFXIII every 4th week (28±2 days) during a treatment period of 52 weeks. In case of acute bleeding episodes, any additional treatment as per investigator judgment was to be according to local standard practice. Additional doses of rFXIII could therefore not be used to treat such breakthrough bleedings
All-Cause Mortality
rFXIII
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
rFXIII
Affected / at Risk (%) # Events
Total   6/41 (14.63%)    
Gastrointestinal disorders   
Small intestinal obstruction  1  1/41 (2.44%)  1
General disorders   
Non-cardiac chest pain  1  1/41 (2.44%)  1
Infections and infestations   
Diverticulitis  1  1/41 (2.44%)  1
Injury, poisoning and procedural complications   
Road traffic accident  1  1/41 (2.44%)  1
Investigations   
Antibody test positive  1  3/41 (7.32%)  3
Nervous system disorders   
Headache  1  1/41 (2.44%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 13.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
rFXIII
Affected / at Risk (%) # Events
Total   25/41 (60.98%)    
Gastrointestinal disorders   
Diarrhoea  1  3/41 (7.32%)  3
Toothache  1  3/41 (7.32%)  5
Vomiting  1  3/41 (7.32%)  3
General disorders   
Pyrexia  1  7/41 (17.07%)  7
Infections and infestations   
Influenza  1  4/41 (9.76%)  4
Nasopharyngitis  1  8/41 (19.51%)  11
Urinary tract infection  1  3/41 (7.32%)  3
Injury, poisoning and procedural complications   
Excoriation  1  3/41 (7.32%)  3
Incorrect dose administered  1  4/41 (9.76%)  11
Musculoskeletal and connective tissue disorders   
Arthralgia  1  5/41 (12.20%)  5
Pain In Extremity  1  4/41 (9.76%)  4
Nervous system disorders   
Headache  1  11/41 (26.83%)  20
Respiratory, thoracic and mediastinal disorders   
Cough  1  3/41 (7.32%)  4
Nasal congestion  1  5/41 (12.20%)  6
Oropharyngeal pain  1  4/41 (9.76%)  6
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 13.0
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Novo Nordisk (NN) reserves the right not to release data until after specified milestones, e.g., finalisation of clinical trial report. This includes the right not to release interim results of clinical trials, as release of such information can invalidate the trial results. At end of trial, one or more manuscripts will be prepared collaboratively between Investigator(s) and NN. NN reserves the right to postpone publication and/or communication for up to 60 days to protect intellectual property.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Public Access to Clinical Trials
Organization: Novo Nordisk A/S
EMail: clinicaltrials@novonordisk.com
Layout table for additonal information
Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT00713648     History of Changes
Other Study ID Numbers: F13CD-1725
2006-003148-51 ( EudraCT Number )
First Submitted: July 7, 2008
First Posted: July 11, 2008
Results First Submitted: January 22, 2014
Results First Posted: July 14, 2014
Last Update Posted: February 24, 2017