Safety and Efficacy of Turoctocog Alfa During Long-Term Treatment of Severe and Moderately Severe Haemophilia A

Completed

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

• Registration Number: NCT02035384
• Lead Sponsor: Novo Nordisk A/S

Brief Summary

This study is conducted in Europe, and North and South America. The aim of this study is to provide additional documentation of the immunogenicity, and obtain additional clinical data, of turoctocog alfa in the setting of normal clinical practise in patients previously treated with a factor VIII agent (FVIII).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2014-01-10
• Study First Submitted QC: 2014-01-10
• Study First Posted: 2014-01-14
• Study Start: 2014-06-05
• Primary Completion: 2020-01-15
• Study Completion: 2020-01-15
• Status Verified: 2020-04
• Last Update Submitted: 2020-04-24
• Last Update Posted: 2020-04-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 69

Inclusion Criteria

• Informed consent obtained before any study-related activities. Study-related activities are any procedure related to recording of data according to the protocol
• Previously FVIII treated (150 exposure days at the time of first dosing with turoctocog alfa) male patients with the diagnosis of severe and moderately severe haemophilia A (FVIII below or equal to 2%)
• The decision to initiate treatment with commercially available turoctocog alfa has been made by the patient/parent and the patient's treating physician before and independently from the decision to include the patient in this study
• A negative FVIII inhibitor test obtained not more than four weeks prior to first dosing with turoctocog alfa

Exclusion Criteria

• Contraindications for use according to the approved product information text (US Package insert (PI), European Summary of Product Characteristics (SmPC), or corresponding local prescribing information)
• Treatment with any investigational drug within 30 days prior to enrolment into the study
• Previous participation in any clinical trial with turoctocog alfa
• Treatment with other FVIII products after initiation of treatment with turoctocog alfa

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
All patients	turoctocog alfa	-

Primary Outcome Measures

Name	Time	Method
Incidence rate of FVIII inhibitors (at least 0.6 Bethesda Units (BU) for central laboratory analyses, or above the specific local laboratory reference range) represented as the percentage of patients developing inhibitors	Within approximately 7 years

Secondary Outcome Measures

Name	Time	Method
Haemostatic effect of turoctocog alfa in the treatment of bleeds as assessed by the patient or the physician according to a predefined four point scale: Excellent, Good, Moderate, or None	Within approximately 7 years
Number of adverse reactions reported	During approximately 7 years
Number of serious adverse reactions reported	During approximately 7 years
Haemostatic effect of turoctocog alfa during surgical procedures as assessed by an evaluation according to a predefined four point scale: Excellent, Good, Moderate, or None	Within approximately 7 years
Annualised bleeding rate for patients using turoctocog alfa for preventive treatment	Within approximately 7 years
Annualised bleeding rate for patients using turoctocog alfa for on-demand treatment	Within approximately 7 years

Trial Locations

Locations (1)

Novo Nordisk Investigational Site; 🇨🇭 Zürich, Switzerland