Trial Outcomes & Findings for Safety and Efficacy of Turoctocog Alfa in Haemophilia A Subjects (NCT NCT00840086)
NCT ID: NCT00840086
Last Updated: 2017-03-17
Results Overview
The incidence rate of FVIII inhibitors was calculated by including all patients with inhibitors in the nominator and including all patients with a minimum 50 exposure plus any patients with less than 50 exposures but with inhibitors in denominator.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
150 participants
Primary outcome timeframe
The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.
Results posted on
2017-03-17
Participant Flow
The patients were recruited at 48 sites in 15 countries: Republic of Serbia (5), Turkey (5), Germany (4), Japan (8), the United Kingdom (3) and the United States of America (10). Two sites each in Brazil, Italy, Spain and Croatia. One site each in Switzerland, Taiwan, Israel, Malaysia and Russian Federation.
Participant milestones
| Measure |
All Subjects Treated With Turoctocog Alfa
All subjects participating in the study (Part A + Part B + Part C).
|
|---|---|
|
Overall Study
STARTED
|
150
|
|
Overall Study
COMPLETED
|
146
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
| Measure |
All Subjects Treated With Turoctocog Alfa
All subjects participating in the study (Part A + Part B + Part C).
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Protocol Violation
|
2
|
|
Overall Study
Lost to Follow-up
|
1
|
Baseline Characteristics
Safety and Efficacy of Turoctocog Alfa in Haemophilia A Subjects
Baseline characteristics by cohort
| Measure |
All Subjects Treated With Turoctocog Alfa
n=150 Participants
All subjects participating in the study (Part A + Part B + Part C).
|
|---|---|
|
Age, Continuous
|
28 years
STANDARD_DEVIATION 11.79 • n=93 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
150 Participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Hispanic Or Latino
|
25 participants
n=93 Participants
|
|
Race/Ethnicity, Customized
Not Hispanic Or Latino
|
125 participants
n=93 Participants
|
|
Region of Enrollment
Brazil
|
16 participants
n=93 Participants
|
|
Region of Enrollment
Croatia
|
11 participants
n=93 Participants
|
|
Region of Enrollment
Germany
|
10 participants
n=93 Participants
|
|
Region of Enrollment
Israel
|
12 participants
n=93 Participants
|
|
Region of Enrollment
Italy
|
7 participants
n=93 Participants
|
|
Region of Enrollment
Japan
|
9 participants
n=93 Participants
|
|
Region of Enrollment
Malaysia
|
5 participants
n=93 Participants
|
|
Region of Enrollment
Russia
|
5 participants
n=93 Participants
|
|
Region of Enrollment
Serbia
|
19 participants
n=93 Participants
|
|
Region of Enrollment
Spain
|
4 participants
n=93 Participants
|
|
Region of Enrollment
Switzerland
|
5 participants
n=93 Participants
|
|
Region of Enrollment
Taiwan
|
4 participants
n=93 Participants
|
|
Region of Enrollment
Turkey
|
11 participants
n=93 Participants
|
|
Region of Enrollment
United Kingdom
|
3 participants
n=93 Participants
|
|
Region of Enrollment
United States
|
29 participants
n=93 Participants
|
PRIMARY outcome
Timeframe: The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.Population: The safety analysis set includes all 150 subjects who received at least one dose of the investigational product. The analysis of the primary endpoint included all subjects with at least 50 exposure days and/or with inhibitors. A total of 148 subjects had 50 exposure days (EDs).
The incidence rate of FVIII inhibitors was calculated by including all patients with inhibitors in the nominator and including all patients with a minimum 50 exposure plus any patients with less than 50 exposures but with inhibitors in denominator.
Outcome measures
| Measure |
All Subjects Treated With Turoctocog Alfa
n=148 Participants
All subjects participating in the study (Part A + Part B + Part C).
|
Part C
Surgery sub-trial: This part of the trial included any of the patients from Part A and Part B that during the course of the trial needed to undergo a major or minor surgical procedure requiring at least 7 days of daily FVIII treatment, including the day of surgery. Patients received a preoperative loading dose of turoctocog alfa immediately prior to the surgical procedure. On the day of surgery and until Day 7 (included) turoctocog alfa was dose adjusted aiming for a trough level above 0.50 IU/mL. Day 8 to last day of the surgical recovery period (if relevant) turoctocog alfa was dosed according to local guidelines.
|
All Subjects Treated With Turoctocog Alfa
All subjects participating in the study (Part A + Part B + Part C).
|
|---|---|---|---|
|
The Incidence Rate of FVIII Inhibitors (Greater Than or Equal to 0.6 Bethesda Units (BU))
|
0 N with Inhibitors / N with ≥50 EDs
|
—
|
—
|
SECONDARY outcome
Timeframe: The adverse events were collected throughout the trial, corresponding to an average of 188 days per subjectPopulation: Safety analysis set includes all subjects who received at least one dose of the investigational product.
Adverse event was defined as events occurring after administration of trial product. Severe AEs: considerable interference with subject's daily activities, unacceptable. Moderate AEs: Marked symptoms, moderate interference with the patient's daily activities. Mild AEs: No or transient symptoms, no interference with the patient's daily activities. Serious AEs: AE that at any dose results in any of the following: death, a life-threatening experience, in-subject hospitalization/prolongation of existing hospitalization, persistent/significant disability/incapacity/congenital anomaly/birth defect.
Outcome measures
| Measure |
All Subjects Treated With Turoctocog Alfa
n=150 Participants
All subjects participating in the study (Part A + Part B + Part C).
|
Part C
n=9 Participants
Surgery sub-trial: This part of the trial included any of the patients from Part A and Part B that during the course of the trial needed to undergo a major or minor surgical procedure requiring at least 7 days of daily FVIII treatment, including the day of surgery. Patients received a preoperative loading dose of turoctocog alfa immediately prior to the surgical procedure. On the day of surgery and until Day 7 (included) turoctocog alfa was dose adjusted aiming for a trough level above 0.50 IU/mL. Day 8 to last day of the surgical recovery period (if relevant) turoctocog alfa was dosed according to local guidelines.
|
All Subjects Treated With Turoctocog Alfa
n=150 Participants
All subjects participating in the study (Part A + Part B + Part C).
|
|---|---|---|---|
|
Frequency of Adverse Events (AEs)
All AEs
|
222 events
|
3 events
|
225 events
|
|
Frequency of Adverse Events (AEs)
Severe AEs
|
8 events
|
0 events
|
8 events
|
|
Frequency of Adverse Events (AEs)
Moderate AEs
|
51 events
|
1 events
|
52 events
|
|
Frequency of Adverse Events (AEs)
Mild AEs
|
163 events
|
2 events
|
165 events
|
|
Frequency of Adverse Events (AEs)
Serious AEs
|
9 events
|
0 events
|
9 events
|
|
Frequency of Adverse Events (AEs)
Probably or Possibly Related
|
17 events
|
0 events
|
17 events
|
Adverse Events
All Subjects Treated With Turoctocog Alfa
Serious events: 7 serious events
Other events: 36 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
All Subjects Treated With Turoctocog Alfa
n=150 participants at risk
All subjects participating in the study (Part A + Part B + Part C).
|
|---|---|
|
Cardiac disorders
Sinus tachycardia
|
0.67%
1/150 • Number of events 1 • The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.
Safety analysis set includes all subjects who received at least one dose of the investigational product.
|
|
Gastrointestinal disorders
Melaena
|
1.3%
2/150 • Number of events 2 • The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.
Safety analysis set includes all subjects who received at least one dose of the investigational product.
|
|
Gastrointestinal disorders
Upper gastrointestinal haemorrhage
|
0.67%
1/150 • Number of events 1 • The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.
Safety analysis set includes all subjects who received at least one dose of the investigational product.
|
|
Injury, poisoning and procedural complications
Fall
|
0.67%
1/150 • Number of events 1 • The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.
Safety analysis set includes all subjects who received at least one dose of the investigational product.
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
0.67%
1/150 • Number of events 1 • The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.
Safety analysis set includes all subjects who received at least one dose of the investigational product.
|
|
Investigations
Hepatic enzyme increased
|
0.67%
1/150 • Number of events 1 • The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.
Safety analysis set includes all subjects who received at least one dose of the investigational product.
|
|
Psychiatric disorders
Insomnia
|
0.67%
1/150 • Number of events 1 • The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.
Safety analysis set includes all subjects who received at least one dose of the investigational product.
|
|
Vascular disorders
Hypertension
|
0.67%
1/150 • Number of events 1 • The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.
Safety analysis set includes all subjects who received at least one dose of the investigational product.
|
Other adverse events
| Measure |
All Subjects Treated With Turoctocog Alfa
n=150 participants at risk
All subjects participating in the study (Part A + Part B + Part C).
|
|---|---|
|
Infections and infestations
Nasopharyngitis
|
8.0%
12/150 • Number of events 13 • The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.
Safety analysis set includes all subjects who received at least one dose of the investigational product.
|
|
Injury, poisoning and procedural complications
Incorrect dose administered
|
10.0%
15/150 • Number of events 19 • The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.
Safety analysis set includes all subjects who received at least one dose of the investigational product.
|
|
Nervous system disorders
Headache
|
9.3%
14/150 • Number of events 18 • The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.
Safety analysis set includes all subjects who received at least one dose of the investigational product.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The information obtained during the conduct of this trial is considered confidential and can be used by Novo Nordisk for regulatory purposes and for the general development of the trial product. The information obtained during this trial may be made available to other physicians who are conducting other clinical trials with the trial product, if deemed necessary by Novo Nordisk.
- Publication restrictions are in place
Restriction type: OTHER