E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10060612 |
E.1.2 | Term | Hemophilia A |
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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 |
Recovery Study Part The primary objective of this clinical research part is to compare the recovery of FACTANE 200 IU/ml with FACTANE 100 IU/ml, when given as a 4 ml/minute intravenous bolus injection. Safety Study Part The primary objective of this clinical research part is to assess the safety of FACTANE 200 IU/ml for at least 9 injections or 3 months whichever is sooner during standard-of-care treatment (on-demand, prophylaxis).
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E.2.2 | Secondary objectives of the trial |
Recovery Study Part The secondary objective of this clinical research part is to compare the local and immediate safety of FACTANE 200 IU/ml with FACTANE 100 IU/ml, when given as a 4 ml/minute intravenous bolus injection.
Safety Study Part The secondary objective of this clinical research part is to evaluate the recovery of FACTANE 200 IU/ml after the last injection (re-test recovery). |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Severe haemophilia A patients (basal levels FVIII:C <1 IU/dl). 2. Male patient older than 18 years. 3. Patient usually treated with FACTANE 100 IU/ml. 4. Patient anticipated to be treated with 9 injections of FACTANE 200 IU/ml within the 3 months following the recovery study.
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E.4 | Principal exclusion criteria |
1. Present or past factor VIII inhibitor titre (defined as ≥0.6 Bethesda Units) (last test ≤ 3 months). 2. Known intolerance to any of the ingredients of FACTANE 3. Participation in another clinical study involving an investigational drug within the past 30 days or on going participation in another clinical study. 4. Any other condition that the Investigator believes could interfere with the study results or would not be in the best interest of the patient.
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E.5 End points |
E.5.1 | Primary end point(s) |
• Recovery Study Part Recovery at T15 min after the end of injection of the IMPs in 12 patients is the primary endpoint. Recovery studies will be based on results using both chromogenic and one-stage clotting methods.
• Safety Study Part The safety assessment will be based on the exposure at least 9 consecutive injections by patient of FACTANE 200 IU/ml in 12 patients. Overall tolerability by patient will be assessed by the investigator at the end of study visit using the same 4-point verbal scale. Tolerability will be based on adverse drug reactions reported by the patient. Each adverse event will be analysed with regards to causality, seriousness and expectedness. Immunogenicity will be based on - inhibitor screening at the last injection. A FVIII inhibitor titre ≥0.6 Bethesda Unit at the central laboratory will be considered as positive result. - control recovery after the last injection. A recovery ratio (control/initial) more than 0.66 will be considered as a stable recovery.
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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 | No |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | Yes |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Yes |
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
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 9 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
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
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | |
E.8.9.1 | In the Member State concerned months | 12 |
E.8.9.1 | In the Member State concerned days | |