E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Subjects with hemophilia A or B with inhibitors |
|
E.1.1.1 | Medical condition in easily understood language |
Subjects with hemophilia A or B with inhibitors |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10053751 |
E.1.2 | Term | Hemophilia A with anti factor VIII |
E.1.2 | System Organ Class | 100000004850 |
|
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10053752 |
E.1.2 | Term | Hemophilia B with anti factor IX |
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 |
Part A:
- To identify the recommended dose by conducting a risk-benefit assessment of four different dose levels of BAY 86-6150 based on safety and dose response assessments in acutely bleeding subjects with hemophilia A or B with inhibitors.
Part B:
- To further investigate the safety and efficacy of the recommended dose of BAY 86-6150 in acutely bleeding subjects with hemophilia A or B with inhibitors. |
|
E.2.2 | Secondary objectives of the trial |
- To assess the potential immunogenicity of BAY 86-6150 in subjects with hemophilia A or B with inhibitors.
- To evaluate the comparative PK/PD (pharmacokinetics/pharmacodynamics) parameters to one fixed dose of eptacog alfa (activated) in a subset of the above cohorts at the same four dose levels of BAY 86-6150. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Male subjects
- 12 to 62 years-of-age
- History of moderate or severe congenital hemophilia A or B with inhibitors to FVIII or FIX
- 4 or more bleeding episodes in the last 6 months before enrollment. |
|
E.4 | Principal exclusion criteria |
- Clinically relevant coagulation disorder other than congenital hemophilia A or B with inhibitors
- History of coronary and/or peripheral atherosclerotic disease
- Disseminated intravascular coagulopathy, or stage 2 hypertension
- Angina pectoris
- Myocardial infarction
- Transient ischemic attack
- Stroke
- Congestive heart failure
- Thromboembolic event |
|
E.5 End points |
E.5.1 | Primary end point(s) |
1. Successful treatments of bleeding episodes. (A bleed was defined as successfully treated, if no administration of rescue medication was required.)
2. Proportion of successful treatments of bleeding episodes on subject level. (Proportion of successful treatments of bleeding
episodes was calculated as number of bleeding episodes treated successfully - without rescue medication - divided by the total number of bleeding episodes on a dose level.) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
1. 10 hours after each bleed
2. 10 hours after each bleed |
|
E.5.2 | Secondary end point(s) |
1. Time to stop the bleed.
2. Number of injections needed to stop the bleeding
episode.
3. Effectiveness of treatment as rated by the subject's assessment (very effective, effective, partially effective, not effective).
4. Participant's reported outcome as assessed by Euro QoL (EQ-5D).
5. Participant's reported outcome as assessed by Brief Pain Inventory.
6. Participant's reported outcome as assessed by Work Productivity and Activity Impairment Questionaire. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
1., 2. & 3.: 10 hours after each bleed
4. & 6.: 14 days after last exposure to BAY86-6150
5.: 7 days after last exposure to BAY86-6150 |
|
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 | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | Yes |
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) | Yes |
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 | Yes |
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 | Yes |
E.8.1.7.1 | Other trial design description |
Part A & B: non-controlled design / PK/PD: randomized, controlled, crossover design |
|
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.2.4 | Number of treatment arms in the trial | 9 |
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 | 25 |
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 |
European Union |
Argentina |
Australia |
Brazil |
Bulgaria |
Chile |
China |
Colombia |
Denmark |
France |
Germany |
Hungary |
India |
Israel |
Italy |
Japan |
Korea, Republic of |
Mexico |
Netherlands |
New Zealand |
Poland |
Romania |
Singapore |
South Africa |
Sweden |
Taiwan |
Turkey |
Ukraine |
United Kingdom |
United States |
|
E.8.7 | Trial has a data monitoring committee | Yes |
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 | 2 |
E.8.9.1 | In the Member State concerned months | 5 |
E.8.9.1 | In the Member State concerned days | 0 |
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 | 11 |
E.8.9.2 | In all countries concerned by the trial days | 0 |