E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Invasive fungal disease caused by Aspergillus species in patients with renal impairment as well as disease caused by rare moulds, yeasts or dimorphic fungi. |
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E.1.1.1 | Medical condition in easily understood language |
Infection caused by fungus |
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E.1.1.2 | Therapeutic area | Diseases [C] - Bacterial Infections and Mycoses [C01] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 15.0 |
E.1.2 | Level | HLT |
E.1.2 | Classification code | 10003486 |
E.1.2 | Term | Aspergillus infections |
E.1.2 | System Organ Class | 10021881 - Infections and infestations |
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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 |
To describe the safety and efficacy of isavuconazole in the treatment of invasive aspergillosis in patients with renal impairment or in patients with invasive fungal disease caused by rare moulds, yeasts or dimorphic fungi. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives are to determine clinical and mycological response rate by pathogen.
To evaluate the survival status at Day 42, 84, 120 and 180 |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Pharmacokinetics:
To characterize the PK of study drug and metabolites if warranted in the PK subpopulations .
Pharmacogenomics:
Based upon metabolic profiling, PK/PD patterns or safety findings genotype analysis may be conducted.
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E.3 | Principal inclusion criteria |
1. Patients with proven/probable invasive fungal disease caused by Aspergillus who also have renal impairment (including dialysis)
2. Patients with proven/probable invasive fungal disease caused by rare moulds, yeasts and dimorphic fungi, whether really impaired or not (including dialysis)
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E.4 | Principal exclusion criteria |
1. A known condition of the patient that may jeopardize adherence to the protocol requirements
2. Patients who are unlikely to survive 30 days
3. Patients with a body weight < 40 kg
4. Women who are pregnant or breastfeeding
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E.5 End points |
E.5.1 | Primary end point(s) |
Overall outcome of treatment evaluated by Data Review Committee (DRC).
Based on the assessments of clinical, mycological and radiological response
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
•Overall outcome of treatment evaluated by Investigator
Based on the assessments of clinical, mycological and radiological response
•Overall outcome at end of treatment and Day 84 assessed by DRC and Investigator by sub-group
Based on the assessments of clinical, mycological and radiological response
•Clinical response assessed by DRC and Investigator by sub-group
•Mycological response by DRC and Investigator by sub-group
•Survival rate |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
•Overall outcome of treatment evaluated by Investigator [ Time Frame: Day 42 ]
•Overall outcome at end of treatment and Day 84 assessed by DRC and Investigator [ Time Frame: Day 84 and end of treatment (up to Day 180) ]
•Clinical response assessed by DRC and Investigator [ Time Frame: Day 42, Day 84, end of treatment (up to Day 180) and 4 weeks after last study dose ]
•Mycological response by DRC and Investigator [ Time Frame: Day 42, Day 84, end of treatment (up to Day 180) and 4 weeks after last study dose ]
•Survival rate [ Time Frame: Day 42, Day 84, Day 120 and Day 180 ]
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
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 | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | Yes |
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 | 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 | 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
| 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 | 8 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 55 |
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 |
Argentina |
Australia |
Brazil |
Canada |
Chile |
China |
Egypt |
India |
Israel |
Mexico |
Russian Federation |
South Africa |
United States |
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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 | |
E.8.9.1 | In the Member State concerned months | 16 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 52 |