E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Empirical therapy for fungal infections |
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E.1.1.1 | Medical condition in easily understood language |
Treatment of fungal infections |
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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 | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate and compare Day 1 C24 hr caspofungin plasma concentrations obtained from neonates and infants <3 months of age administered caspofungin at a 25-mg/m2 intravenous (IV) dose to historical data from adult patients administered caspofungin at a 50-mg daily IV dose. |
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E.2.2 | Secondary objectives of the trial |
1) To evaluate and compare Day 1 C1 hr caspofungin plasma concentrations obtained from neonates and infants <3 months of age administered caspofungin at a 25-mg/m2 IV dose to historical data from adult patients administered caspofungin at a 50-mg daily IV dose; 2) To evaluate and compare Day 4 C1 hr and C24 hr caspofungin plasma concentrations obtained from neonates and infants <3 months of age administered caspofungin at a 25-mg/m2 daily IV dose to historical data from adult patients administered a 50-mg daily IV dose; 3) To evaluate the safety and tolerability of caspofungin administered at 25 mg/m2/day in neonates and infants <3 months of age. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Patient is <3 months (90 days) of age on day of informed consent.
• Patient has a documented (culture-confirmed) or suspected invasive Candida infection, as defined below.
• Patient must be receiving an intravenous amphotericin B formulation (amphotericin B deoxycholate or lipid preparation of amphotericin B) for the treatment of a highly-suspected or documented invasive Candida infection at the time of study entry and will remain on an intravenous amphotericin B formulation for the duration of caspofungin study therapy. |
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E.4 | Principal exclusion criteria |
• Patient is ≥3 months (≥90 days) of age at the time of informed consent.
• Patient has exclusionary laboratory values as listed below: Hematocrit <35% for patients with a body weight of <1500 grams, AST (SGOT) >2 times the upper limit of normal, for age, ALT (SGPT) >2 times the upper limit of normal, for age.
• Patient is hemodynamically unstable, exhibits hemodynamic compromise, or is not expected to survive at least 5 days.
• Patient has a diagnosis of acute hepatitis or cirrhosis due to any cause.
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E.5 End points |
E.5.1 | Primary end point(s) |
To evaluate and compare Day 1 C(24-hr) caspofungin plasma concentrations obtained from neonates and infants <3 months of age administered caspofungin at a 25-mg/m2 intravenous (IV) dose |
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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) |
To evaluate and compare Day 1 C(1-hr) caspofungin plasma concentrations obtained from neonates and infants <3 months of age administered caspofungin at a 25-mg/m2 IV dose |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | 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 | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
Colombia |
India |
Mexico |
Panama |
United States |
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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.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 3 |