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 |
Bleeding disorder, inherited deficiency in clotting factor VIII |
|
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 | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10018938 |
E.1.2 | Term | Haemophilia A (Factor VIII) |
E.1.2 | System Organ Class | 100000004850 |
|
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the clinical efficacy of turoctocog alfa in treatment of bleeding episodes in Chinese
patients with severe haemophilia A (FVIII≤1%) |
|
E.2.2 | Secondary objectives of the trial |
- To assess the safety of turoctocog alfa in terms of immunogenicity
- To evaluate the clinical efficacy of turoctocog alfa during prophylaxis treatment
- To evaluate the consumption of turoctocog alfa during prophylaxis treatment and treatment of bleeding episodes |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Male patients
- Age from 0 years
- With severe congenital haemophilia A (FVIII≤1%)
- History of exposure days (ED) to any FVIII products fulfilling the criteria of previously treated
patients:
* Patients of 12 years or above: 100 exposures days (ED) or more
*Patients below 12 years: 50 exposure days (ED) or more |
|
E.4 | Principal exclusion criteria |
- Inhibitors to factor VIII (≥0.6 BU) at screening as assessed by central laboratory
- Known history of FVIII inhibitors |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Haemostatic effect of turoctocog alfa when used for treatment of bleeds, assessed on a four-point
scale for haemostatic response (excellent, good, moderate and none) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
During the main phase (6 months duration per patient) |
|
E.5.2 | Secondary end point(s) |
1. Haemostatic effect of turoctocog alfa when used for treatment of bleeds, assessed on a fourpoint scale for haemostatic response (excellent, good, moderate and none)
2. Incidence rate of inhibitory antibodies against FVIII (≥0.6 Bethesda unit (BU))
3. Number of bleeds (total bleeds assessed as annual bleeding rate) per patient
4. Consumption of turoctocog alfa
5. Consumption of turoctocog alfa for bleeding treatment (average dose to treat a bleed, number of injections and IU/kg per bleed)
6. Consumption of turoctocog alfa during prophylaxis treatment (average prophylaxis dose and IU/kg per month and per year) per patient
7. Total consumption of turoctocog alfa (IU/kg per month and per year) per patient
8. Frequency of adverse events (AEs) and serious adverse events (SAEs) reported |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. During the trial period of 24 months
2. During both the main phase of 6 months and during the trial period of 24 months
3. During both the main phase of 6 months and during the trial period of 24 months
4. During both the main phase of 6 months and during the trial period of 24 months
5. During both the main phase of 6 months and during the trial period of 24 months
6. During both the main phase of 6 months and during the trial period of 24 months
7. During both the main phase of 6 months and during the trial period of 24 months
8. During both the main phase of 6 months and during the trial period of 24 months |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | Yes |
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 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
|
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.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |