Trial Outcomes & Findings for Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A (NCT NCT01138501)

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

65 participants

Primary outcome timeframe

The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject.

Results posted on

2017-03-17

Participant Flow

Of a total of 39 initiated trial sites, 26 sites enrolled and dosed at least one patient. The country distribution was as follows: Brazil (3), Italy (1), Lithuania (1), Macedonia (1), Malaysia (1), Poland (2), Russia (2), Serbia (1), Taiwan (1), Turkey (3) and the United States of America (10).

Participant milestones

Participant milestones
Measure	All Subjects Treated With Turoctocog Alfa The patients received bleeding preventive treatment with a single dose of turoctocog alfa of 25-50 IU/kg every second day or 25-60 IU/kg three times weekly. Turoctocog alfa was administered as a slow bolus i.v. injection (approximately 1-2 mL/min). Pharmacokinetic assessments were performed in at least 13 patients from each age cohort. Each patient participating in the pharmacokinetic assessments received one dose of previous factor VIII (FVIII) and one dose of turoctocog alfa.
Overall Study STARTED	65
Overall Study Exposed	63
Overall Study COMPLETED	60
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Reasons for withdrawal
Measure	All Subjects Treated With Turoctocog Alfa The patients received bleeding preventive treatment with a single dose of turoctocog alfa of 25-50 IU/kg every second day or 25-60 IU/kg three times weekly. Turoctocog alfa was administered as a slow bolus i.v. injection (approximately 1-2 mL/min). Pharmacokinetic assessments were performed in at least 13 patients from each age cohort. Each patient participating in the pharmacokinetic assessments received one dose of previous factor VIII (FVIII) and one dose of turoctocog alfa.
Overall Study Protocol Violation	2
Overall Study Withdrawal by Subject	3

Baseline Characteristics

Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Subjects Treated With Turoctocog Alfa n=63 Participants The patients received bleeding preventive treatment with a single dose of turoctocog alfa of 25-50 IU/kg every second day or 25-60 IU/kg three times weekly. Turoctocog alfa was administered as a slow bolus i.v. injection (approximately 1-2 mL/min). Pharmacokinetic assessments were performed in at least 13 patients from each age cohort. Each patient participating in the pharmacokinetic assessments received one dose of previous factor VIII (FVIII) and one dose of turoctocog alfa.
Age, Continuous	6.08 years STANDARD_DEVIATION 2.91 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	63 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	16 participants n=5 Participants
Race/Ethnicity, Customized Not Hispanic or Latino	47 participants n=5 Participants
Region of Enrollment Brazil	9 participants n=5 Participants
Region of Enrollment Serbia	5 participants n=5 Participants
Region of Enrollment Italy	2 participants n=5 Participants
Region of Enrollment Lithuania	4 participants n=5 Participants
Region of Enrollment Macedonia, The Former Yugoslav Republic of	5 participants n=5 Participants
Region of Enrollment Malaysia	5 participants n=5 Participants
Region of Enrollment Poland	5 participants n=5 Participants
Region of Enrollment Russia	8 participants n=5 Participants
Region of Enrollment Taiwan	1 participants n=5 Participants
Region of Enrollment Turkey	7 participants n=5 Participants
Region of Enrollment United States	12 participants n=5 Participants

PRIMARY outcome

Timeframe: The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject.

Population: The safety analysis set includes all 63 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 59 subjects had 50 exposure days (EDs).

Outcome measures

Outcome measures
Measure	All Subjects Treated With Turoctocog Alfa n=59 Participants The patients received bleeding preventive treatment with a single dose of turoctocog alfa of 25-50 IU/kg every second day or 25-60 IU/kg three times weekly. Turoctocog alfa was administered as a slow bolus i.v. injection (approximately 1-2 mL/min). Pharmacokinetic assessments were performed in at least 13 patients from each age cohort. Each patient participating in the pharmacokinetic assessments received one dose of previous factor VIII (FVIII) and one dose of turoctocog alfa.
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 138 days per subject

Population: 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 AE: AE that at any dose results in any of the following: death, a life-threatening experience, in-subject hospitalization/prolongation of existing hospitalisation, persistent/significant disability/incapacity/congenital anomaly/birth defect.

Outcome measures

Outcome measures
Measure	All Subjects Treated With Turoctocog Alfa n=63 Participants The patients received bleeding preventive treatment with a single dose of turoctocog alfa of 25-50 IU/kg every second day or 25-60 IU/kg three times weekly. Turoctocog alfa was administered as a slow bolus i.v. injection (approximately 1-2 mL/min). Pharmacokinetic assessments were performed in at least 13 patients from each age cohort. Each patient participating in the pharmacokinetic assessments received one dose of previous factor VIII (FVIII) and one dose of turoctocog alfa.
Frequency of Adverse Events (AEs) All AEs	86 events
Frequency of Adverse Events (AEs) Severe AEs	0 events
Frequency of Adverse Events (AEs) Moderate AEs	11 events
Frequency of Adverse Events (AEs) Mild AEs	74 events
Frequency of Adverse Events (AEs) Serious AEs	3 events
Frequency of Adverse Events (AEs) Probably or possibly related	2 events

Adverse Events

All Subjects Treated With Turoctocog Alfa

Serious events: 3 serious events

Other events: 14 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	All Subjects Treated With Turoctocog Alfa n=63 participants at risk The patients received bleeding preventive treatment with a single dose of turoctocog alfa of 25-50 IU/kg every second day or 25-60 IU/kg three times weekly. Turoctocog alfa was administered as a slow bolus i.v. injection (approximately 1-2 mL/min). Pharmacokinetic assessments were performed in at least 13 patients from each age cohort. Each patient participating in the pharmacokinetic assessments received one dose of previous factor VIII (FVIII) and one dose of turoctocog alfa.
Infections and infestations Device related infection	1.6% 1/63 • Number of events 1 • The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject. Safety analysis set includes all subjects who received at least one dose of the investigational product.
Infections and infestations Gastroenteritis viral	1.6% 1/63 • Number of events 1 • The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject. Safety analysis set includes all subjects who received at least one dose of the investigational product.
Injury, poisoning and procedural complications Soft tissue injury	1.6% 1/63 • Number of events 1 • The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject. Safety analysis set includes all subjects who received at least one dose of the investigational product.

Other adverse events

Other adverse events
Measure	All Subjects Treated With Turoctocog Alfa n=63 participants at risk The patients received bleeding preventive treatment with a single dose of turoctocog alfa of 25-50 IU/kg every second day or 25-60 IU/kg three times weekly. Turoctocog alfa was administered as a slow bolus i.v. injection (approximately 1-2 mL/min). Pharmacokinetic assessments were performed in at least 13 patients from each age cohort. Each patient participating in the pharmacokinetic assessments received one dose of previous factor VIII (FVIII) and one dose of turoctocog alfa.
Gastrointestinal disorders Vomiting	6.3% 4/63 • Number of events 4 • The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject. Safety analysis set includes all subjects who received at least one dose of the investigational product.
Infections and infestations Nasopharyngitis	7.9% 5/63 • Number of events 6 • The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject. Safety analysis set includes all subjects who received at least one dose of the investigational product.
Infections and infestations Upper respiratory tract infection	7.9% 5/63 • Number of events 5 • The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject. Safety analysis set includes all subjects who received at least one dose of the investigational product.
Injury, poisoning and procedural complications Contusion	6.3% 4/63 • Number of events 4 • The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject. Safety analysis set includes all subjects who received at least one dose of the investigational product.
Nervous system disorders Headache	6.3% 4/63 • Number of events 5 • The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject. Safety analysis set includes all subjects who received at least one dose of the investigational product.

Additional Information

Public Access to Clinical Trials

Novo Nordisk A/S

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Novo Nordisk maintains the right to be informed of any Investigator plans for publication and to review any scientific paper, presentation, communication or other information concerning the investigation described in this protocol.
Publication restrictions are in place

Restriction type: OTHER