E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Candida and Aspergillus infections |
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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] - 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 |
The main objectives of this study are to evaluate the safety, tolerability, and efficacy of caspofungin therapy, administered as 50 mg/m2 IV once daily (maximum 70 mg/day) following a loading dose of 70 mg/m2 (maximum 70 mg/day) on Day 1, in pediatric patients (3 months through 17 years of age) with invasive aspergillosis (IA) who are refractory to or intolerant of standard therapy or those with invasive or esophageal Candida infections. The specific objectives are: Primary: (1) To report the proportion of pediatric patients treated with caspofungin with one or more drug-related clinical or laboratory adverse experience(s). |
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E.2.2 | Secondary objectives of the trial |
1) To report the proportion of patients who discontinue due to a drug-related adverse experience or who have a serious drug-related adverse experience. (2) To report the proportion of children with a favorable efficacy response to caspofungin therapy in each of the different infection types. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
The patient is at least 3 months of age and younger than 18 years of age at time of study drug administration.
Clinical evidence of infection must be present at some time within 48 hours prior to enrollment.
Inclusion Criteria for Patients With Aspergillus Infections: 1) Patients must meet definitions of proven or probable invasive aspergillosis (as specified in criteria developed by the European Organization for Research and Treatment of Cancer/Invasive Fungal Infections Co- Operative Group (EORTC/IFICG) and the National Institute of Allergy and Infectious Diseases - Mycoses Study Group (NIAID-MSG)
Inclusion Criteria for Patients With Esophageal Candida Infections, Patients with a diagnosis of esophageal candidiasis based on symptoms, endoscopy and microbiology or histopathology for whom IV therapy is appropriate.
Inclusion Criteria for Patients With Invasive Candida Infections, Patient must have at least 1 positive culture of a Candida species from blood or other normally sterile body site, which has been obtained within 4 days of study entry.
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E.4 | Principal exclusion criteria |
Patient is <3 months or ≥18 years of age (at time of the first dose of study drug administration).
Patient has any of the following abnormal laboratory values: a) International normalization ratio (INR) >1.6 or, if patient is receiving anticoagulants, INR >4.0. b) Bilirubin >3 times the upper limit of normal for age. c) AST (SGOT) or ALT (SGPT) >5 times the upper limit of normal for age. d) Platelet count <5000/μL.
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E.5 End points |
E.5.1 | Primary end point(s) |
To report the proportion of pediatric patients treated with caspofungin with one or more drug-related clinical or laboratory adverse experience(s). |
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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 report the proportion of patients who discontinue study therapy due to a drug-related adverse experience or who have a serious drug-related adverse experience. (2) To report the proportion of patients with a favorable efficacy response to caspofungin therapy in each of the different infection types. |
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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 | 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 | Yes |
E.6.13.1 | Other scope of the trial description |
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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 |
Germany |
Israel |
Italy |
Taiwan |
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 | 1 |
E.8.9.2 | In all countries concerned by the trial days | 6 |