Safety and Efficacy of turoctocog alfa pegol (N8-GP) in Previously Untreated Patients with Haemophilia A
- Conditions
- Haemophilia AMedDRA version: 20.0Level: LLTClassification code 10018938Term: Haemophilia A (Factor VIII)System Organ Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2013-004025-88-AT
- Lead Sponsor
- ovo Nordisk A/S
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 125
- Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial
- Male, age < 6 years at the time of signing informed consent
- Diagnosis of severe haemophilia A (factor VIII activity level < 1%) based on medical records or central laboratory results
- No prior use of purified clotting factor products (5 previous exposure to blood components is acceptable)
Are the trial subjects under 18? yes
Number of subjects for this age range: 125
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- Any history of FVIII inhibitor (defined by medical records)
- Known or suspected hypersensitivity to trial product or related products
- Previous participation in this trial. Participation is defined as administration of trial product
- Receipt of any investigational medicinal product within 30 days before screening
- Congenital or acquired coagulation disorder other than haemophilia A. Any chronic disorder or severe disease which, in the opinion of the Investigator, might jeopardise patient’s safety or compliance with the protocol.
- Patient’s parent(s)/legally acceptable representative(s) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Secondary Objective: - To evaluate safety other than immunogenicity of N8-GP (turoctocog alfa pegol) in PUPs with severe haemophilia A<br>- To evaluate efficacy of N8-GP (turoctocog alfa pegol) in PUPs with severe haemophilia A<br> o in long-term prophylaxis treatment (bleeding preventive effect)<br> o in the treatment of bleeding episodes;Main Objective: To evaluate immunogenicity of N8-GP (turoctocog alfa pegol) in previously untreated patients (PUPs) with severe haemophilia A;Primary end point(s): Incidence of FVIII inhibitors;Timepoint(s) of evaluation of this end point: The primary endpoint will be evaluated when the first 50 PUPs have<br>reached at least 50 exposure days and at end of trial. End of trial will be<br>up to 4 years after the first patient has reached 100 exposure days
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - Frequency of adverse events including serious adverse events and<br>medical events of special interest.<br>- Incidence of confirmed high titre inhibitors (defined as inhibitor titre ><br>5BU).<br>- Number of breakthrough bleeding episodes during prophylaxis with<br>N8-GP (annualised bleeding rate).<br>- Haemostatic effect of N8-GP in treatment of bleeding episodes,<br>assessed by a predefined 4-point haemostatic response scale<br>excellent, good, moderate and none). ;Timepoint(s) of evaluation of this end point: The secondary endpoints will be evaluated when the first 50 PUPs have<br>reached at least 50 exposure days and at end of trial. End of trial will be<br>up to 4 years after the first patient has reached 100 exposure days.