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

Congenital Bleeding Disorder Clinical Trial

— guardian™ 3  

Official title:

A Multi-Centre, Open-Label, Non-Controlled Trial on Safety and Efficacy of N8 in Previously Treated Paediatric Patients With Haemophilia A

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01138501
• Other study ID #: NN7008-3545
• Secondary ID: U1111-1113-71822
• Status: Completed
• Phase: Phase 3
• First received: May 28, 2010
• Last updated: April 8, 2015
• Start date: June 2010
• Est. completion date: November 2011

Study information

• Verified date: April 2015
• Source: Novo Nordisk A/S
• Contact: n/a
• Is FDA regulated: No
• Health authority: Brazil: National Health Surveillance AgencyItaly: Ministry of HealthLithuania: Lithuanian Bioethics CommitteeMacedonia, The Former Yugoslav Republic of: Ministry of Health of Republic of MacedoniaMalaysia: Ministry of HealthPoland: Ministry of HealthRussia: Federal Service for Control of Health Care and Social DevelopmentSerbia: Agency for Drugs and Medicinal DevicesTaiwan: Department of HealthTurkey: Ministry of HealthUnited States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

This trial is conducted in Asia, Europe, and North and South America. The aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated paediatric subjects with haemophilia A.

Other known NCT identifiers

• NCT01250028

Recruitment information / eligibility

• Status: Completed
• Enrollment: 65
• Est. completion date: November 2011
• Est. primary completion date: November 2011
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A to 12 Years

Eligibility

Inclusion Criteria:

• Male patients with severe (baseline FVIII less than or equal to 1%) haemophilia A

• Age below 12 years and weight at least 11 kg

Exclusion Criteria:

• Surgery planned to occur during trial participation (exceptions are port placement, dental extractions, and minor, uncomplicated emergent procedures)

• Congenital or acquired coagulation disorders other than haemophilia A

• Any history of FVIII inhibitors (greater than or equal to 0.6 BU/mL)

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Blood Coagulation Disorders
• Congenital Bleeding Disorder
• Haemophilia A
• Hemophilia A
• Hemostatic Disorders

Intervention

Drug:
• turoctocog alfa
Subjects will be treated 3 times per week or every second day with intravenous injections of turoctocog alfa. The dose range for preventive treatment is 25-60 IU/kg body weight depending on treatment regimen.
• turoctocog alfa
Subjects will undergo half-life evaluation of their current factor VIII product and pharmacokinetic session with turoctocog alfa before entering preventive treatment (subjects with available evaluation of terminal half-life within the last year are to be excluded from receiving factor VIII). Intravenous injections. The dose range for preventive treatment is 25-60 IU/kg body weight depending on treatment regimen.

Locations

Country	Name	City	State
United States	Novo Nordisk Clinical Trial Call Center	Boston	Massachusetts
United States	Novo Nordisk Clinical Trial Call Center	Brooklyn	New York
United States	Novo Nordisk Clinical Trial Call Center	Detroit	Michigan
United States	Novo Nordisk Clinical Trial Call Center	East Lansing	Michigan
United States	Novo Nordisk Clinical Trial Call Center	Houston	Texas
United States	Novo Nordisk Clinical Trial Call Center	Iowa City	Iowa
United States	Novo Nordisk Clinical Trial Call Center	Phoenix	Arizona
United States	Novo Nordisk Clinical Trial Call Center	Tampa	Florida
United States	Novo Nordisk Clinical Trial Call Center	Torrance	California
United States	Novo Nordisk Clinical Trial Call Center	Valhalla	New York

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Countries where clinical trial is conducted

United States,  Brazil,  Italy,  Lithuania,  Macedonia, The Former Yugoslav Republic of,  Malaysia,  Poland,  Russian Federation,  Serbia,  Taiwan,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The Incidence Rate of FVIII Inhibitors (Greater Than or Equal to 0.6 Bethesda Units (BU))	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.	The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject.	No
Secondary	Frequency of Adverse Events (AEs)	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.	The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject	No

External Links

•   Clinical Trials at Novo Nordisk