E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
invasive aspergillosis,
invasive mucormycosis |
|
E.1.1.1 | Medical condition in easily understood language |
invasive fungal infection |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Bacterial Infections and Mycoses [C01] |
MedDRA Classification |
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 safety and tolerability of isavuconazonium sulfate in pediatric subjects.
To assess the efficacy of isavuconazonium sulfate for the treatment of invasive aspergillosis (IA) or
invasive mucormycosis (IM) in pediatric subjects.
To evaluate the pharmacokinetics of isavuconazole by monitoring the plasma concentrations
in pediatric subjects during treatment with isavuconazonium sulfate. |
|
E.2.2 | Secondary objectives of the trial |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Institutional Review Board (IRB)-approved written informed consent.
Male or female subject 1 year to < 18 years of age diagnosed with IA or IM.
Subject has sufficient venous access to permit intravenous administration of study drug
or the ability to swallow oral capsules.
A female subject is eligible to participate if she is not pregnant, follows contraceptive guidance, agrees not to breastfeed and does not donate ova.
A male subject with female partner(s) of childbearing potential must agree to use contraception and to not donate sperm. |
|
E.4 | Principal exclusion criteria |
Subject has familial short QT syndrome, is receiving medications that are known to
shorten the QT interval, or has a clinically significant abnormal ECG.
Subject has evidence of hepatic dysfunction, known cirrhosis or chronic hepatic failure.
Subject has used strong cytochrome P450 (CYP3A4) inhibitors or inducers.
Subject has chronic aspergillosis, aspergilloma or allergic bronchopulmonary
aspergillosis.
Subject is unlikely to survive 30 days in the investigator’s opinion. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
All-cause mortality through day 42.
Nature, frequency, and severity of treatment-emergent Adverse Events (TEAEs).
Laboratory Assessments.
Vital Sign measurements.
Routine 12-lead ECGs.
Physical Examination.
Plasma concentration of isavuconazole (Ctrough).
|
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
All-cause mortality: through day 42
TEAEs: throughout the entire study period
Laboratory Assessments: day 7, 28, 56 and 84 or EOT
Vital Sign measurements: day 1, 2, 3, 7, 14, 21, 28, 35, 42, 49, 56, 63, 70, 77 and 84 or EOT.
Routine 12-lead ECGs: day 1, 7, 14, 28, 56 and 84 or EOT.
Physical Examination: day 84 or EOT.
Ctrough: day 7, 14, 21, 42 and 84 or EOT.
|
|
E.5.2 | Secondary end point(s) |
All-cause mortality through day 84 and end of treatment (EOT)
Overall, clinical, radiological and mycological response |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
All-cause mortality: day 84 and EOT
Overall, clinical, radiological and mycological Response: through day 42, day 84 and at EOT. |
|
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 | 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 | 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) | 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 | 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 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
|
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.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |