E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10018937 |
E.1.2 | Term | Haemophilia A |
E.1.2 | System Organ Class | 100000004850 |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the safety of turoctocog alfa for prevention (only applicable for subjects in the preventive regimen) and treatment of bleeds. |
|
E.2.2 | Secondary objectives of the trial |
• To assess the efficacy of turoctocog alfa for prevention (only applicable for subjects in the preventive regimen) and treatment of bleeds.
• To assess Patient Reported Outcomes over time (only for applicable subjects)
|
|
E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Surgery Sub-Trial: Part of the main protocol for NN7008-3568, version 5, 27 July 2009
Trial phase 3b
Surgery Sub-Trial title:
Efficacy and Safety of N8 in Prevention and Treatment of Bleeding during Surgical Procedures in Subjects with Haemophilia A
Primary Objective for the Surgery Sub-Trial:
• To evaluate the efficacy of turoctocog alfa in surgery.
Secondary Objective for the Surgery Sub-Trial:
• To evaluate the haemostatic response to turoctocog alfa in the post-surgery period.
• To evaluate the safety of turoctocog alfa in surgery.
On-Demand Sub-Trial: Efficacy and safety of turoctocog alfa during 6 months On-Demand treatment in Subjects with Haemophilia A
On-Demand Sub-Trial primary and secondary objectives are the same as E.2.1 and E.2.2
PK Sub-Trial: Part of the main protocol for NN7008-3568, version 5, 27 July 2009
Trial phase 3b, Amendment 17
PK Sub-Trial Title: Single dose pharmacokinetics in patients with BMI ≥ 30 kg/m^2
Primary Objective: To investigate single dose pharmacokinetics in patients with BMI ≥ 30 kg/m^2 |
|
E.3 | Principal inclusion criteria |
1. Informed consent obtained before any trial-related activities. (Trial-related activities are any procedure that would not have been performed during normal management of the subject.)
2. Completion of the Pivotal trial (NN7008-3543), Paediatric trial (NN7008-3545), Japanese trial (NN7008-3600), PK trial (NN7008-3893), PK trial (NN7008-4015) or On-demand Sub-Trial in the Extension trial (NN7008-3568).
Inclusion Criteria for new subjects starting in the 6 months On-Demand Sub-Trial
• Male subjects with the diagnosis of severe (FVIII≤1%) haemophilia A from age 18 to 70 years.
• Documented history of at least 150 exposure days to any other FVIII products (prevention or treatment of bleeds)
• No detectable inhibitors to FVIII (≥0.6 BU/mL) (as assessed by a Central Laboratory at the time of screening)
|
|
E.4 | Principal exclusion criteria |
1. Previous participation in the current trial (defined as withdrawal) or withdrawn subjects from NN7008-3522, NN7008-3543, NN7008-3545, NN7008-3600, NN7008-3893 or NN7008-4015 after administration of trial product, unless the previous trial protocol declares that the subject is allowed to be transferred to NN7008-3568 trial.
Exclusion Criteria for new subjects starting in the 6 months On-Demand Sub-Trial
• Any history of FVIII inhibitors
• Known or suspected allergy to trial product (turoctocog alfa) or related products
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
Frequency of development of FVIII inhibitors (≥0.6 BU/mL) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
• Annualised bleeding rate reported during the prevention period (only applicable for subjects in the preventive regimen)
• Haemostatic response to turoctocog alfa (none, moderate, good or excellent) in treatment of bleeds
• The number of infusions of turoctocog alfa required per bleeding episode
• Time to control of bleeding after the first dose of turoctocog alfa used for treatment of bleeds
• Assessment of the actual consumption of turoctocog alfa (IU/kg BW/bleeding episode)
• Assessment of the actual consumption of turoctocog alfa for prevention (only applicable for subjects in the preventive regimen)
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 12 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Brazil |
Croatia |
European Union |
Israel |
Japan |
Macedonia, the former Yugoslav Republic of |
Malaysia |
Russian Federation |
Serbia |
Switzerland |
Taiwan |
Turkey |
United States |
|
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 7 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 7 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |