E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
HAEMOPHILIA A |
Hemofilia A |
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E.1.1.1 | Medical condition in easily understood language |
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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 | 10018938 |
E.1.2 | Term | Haemophilia A (Factor VIII) |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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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 efficacy of FACTANE 200 IU/ml administered by continuous infusion for the perioperative and postoperative prophylaxis of bleeding during major surgical procedures in severe haemophilia A patients. |
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E.2.2 | Secondary objectives of the trial |
To evaluate the safety of FACTANE 200 IU/ml administered by continuous infusion. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Patient who has provided written signed and dated informed consent prior to any study procedures
− Previously-treated severe haemophilia A patients (basal level FVIII:C<1 IU/dl)
− Male patient older than 18 years
− Patient scheduled for an elective major surgery |
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E.4 | Principal exclusion criteria |
− Present or past factor VIII inhibitor titre (defined as ≥0.6 Bethesda Units)
− Platelets <100 x 109/l
− Medication with interferon / ribavirin or protease inhibitors
− AST and ALT > 5 times the upper limit of normal range
− Current participation or in the last 3 months in another clinical study
− Problematic venous access
− Known hypersensitivity to any of the ingredients of FACTANE 200 IU/ml
− CD4 lymphocytes < 400/mm3
− Any other condition that in the Investigator's opinion may 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) |
Clinical efficacy
Haemostasis rating based on the following scale:
“excellent”, “good”, “moderate”, “none”.
− excellent: similar haemostasis to that of a non bleeding disorder patient during the same surgery
− good: slight oozing at surgical incision
− moderate: mild, but controlled bleeding
− none: severe, uncontrolled bleeding
'Responders' are patients who obtain a score of “excellent” or “good” on these 3 assessments. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
The haemostasis rating will be performed by the surgeon on D0 (day of the surgery) and on day of drain tube removal (or on D2 if no drain) and by the Investigator on D7. |
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E.5.2 | Secondary end point(s) |
− Daily clearance (CL) during the continuous infusion.
− Blood loss and transfusion requirements.
− IMP consumption: evolution of the infusion rate (IU/kg/h), total dose per surgery (IU/kg) and number of exposure days.
− Safety: overall safety including adverse events, immunogenicity and local tolerability judged by the Investigator on Dn +30 (±3) according to the following scale: "excellent", "good", "moderate", "none". |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
For daily clearance, Blood loss and transfusion requirements, IMP consumption from D0 (day of the surgery) to Dn (end of the continuous infusion).
For overall safety: during all the study |
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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 | 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 | 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 | No |
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 | 0 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 8 |
E.8.9.2 | In all countries concerned by the trial days | 0 |