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

Phase 3

Completed

• Conditions: Congenital Bleeding Disorder; Haemophilia A
• Interventions: Drug: turoctocog alfa

• Registration Number: NCT01138501
• Lead Sponsor: Novo Nordisk A/S

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2010-05-28
• Study Start: 2010-06
• Study First Submitted QC: 2010-06-04
• Study First Posted: 2010-06-07
• Primary Completion: 2011-11
• Study Completion: 2011-11
• Results First Submitted: 2013-11-14
• Results First Submitted QC: 2014-05-12
• Results First Posted: 2014-06-11
• Status Verified: 2017-02
• Last Update Submitted: 2017-02-09
• Last Update Posted: 2017-03-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 65

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

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
rFVIII	turoctocog alfa	-

Primary Outcome Measures

Name	Time	Method
The Incidence Rate of FVIII Inhibitors (Greater Than or Equal to 0.6 Bethesda Units (BU))	The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject.	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.

Secondary Outcome Measures

Name	Time	Method
Frequency of Adverse Events (AEs)	The adverse events were collected throughout the trial, corresponding to an average of 138 days per subject	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.

Trial Locations

Locations (1)

Novo Nordisk Investigational Site; 🇹🇷 Izmit, Turkey