E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
The objective of this prospective stratified cluster randomized cross-over trial is to determine whether pharmacologic optimization of voriconazole by means of therapeutic drug monitoring (TDM) results in improved outcome in patient with haematological malignancies (efficacy and safety) and is more cost-effective compared to the current standard of care. |
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MedDRA Classification |
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 |
The objective of this study proposal is to determine whether pharmacologic optimization of voriconazole by means of therapeutic drug monitoring (TDM) results in improved patient outcomes (efficacy and safety). |
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E.2.2 | Secondary objectives of the trial |
The objective of this study proposal is to determine whether pharmacologic optimization of voriconazole by means of therapeutic drug monitoring (TDM) is more cost-effective compared to the current standard of care.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1) are at least 18 years of age 2) have received chemotherapy for haematological malignancies or have received a hematopoietic stem cell transplant 3) proven, probable or possible invasive fungal disease according to the EORTC/MSG criteria 4) treatment with voriconazole
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E.4 | Principal exclusion criteria |
1) allergic to voriconazole or its excipients 2) age below 18 years
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is a reduction in risk of failure on day 28 of treatment with voriconazole. This failure is based on the categorized global response
1) Complete Response: resolution of all clinical signs and symptoms and more than 90 percent of the IA related lesions that were visible on radiographic studies at baseline; 2) Partial Response: Clinical improvement and greater than 50 percent improvement in radiological findings present at baseline; 3) Stable: No change from baseline, or an improvement of less than 50 percent in radiological findings; 4) Failure: Worsening disease.
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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 | 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 | No |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
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 | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | Yes |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
patients are not randomised but centres are randomised |
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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
| Yes |
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 | 13 |
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 | 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
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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 | 3 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |