A Study Of The Safety, Tolerability And Effective Of Voriconazole For The Treatment Of Serious Candida Infection And Candida Infection Of The Throat In Pediatric Patients

Study Overview
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Brief Summary
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The purpose of this study is to determine whether voriconazole is safe and effective for the treatment of serious Candida infection and Candida infection of the esophagus in children and adolescents.

Official Title
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A Prospective, Open-label, Non-comparative Study To Assess The Safety, Tolerability And Efficacy Of Voriconazole For The Primary And Salvage Treatment Of Invasive Candidiasis, Candidemia, And Esophageal Candidiasis In Pediatric Subjects

Conditions
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Candidiasis

Intervention / Treatment
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* Drug: voriconazole


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female 2 to <18 years of age; Subjects 2 to <12 years of age will be permitted to enroll in this study only after a Pfizer sponsored pharmacokinetic study confirms the proposed dosage corresponding to this age group is appropriate. Patients with confirmed Candida infection of the blood, body tissues, or the esophagus. Patient's doctor feels voriconazole is an appropriate choice of therapy. Exclusion Criteria: A known allergy to voriconazole or to azole to antifungal drugs. Females who are pregnant, lactating (breast feeding) or planning a pregnancy during the course of the study, or who are of childbearing potential and not using highly effective method of birth control. A patient who is receiving treatment with a drug know to interfere with the heart's electrical system (QTc prolongation). A patient who is receiving treatment with a drug that is not permitted to be used with voriconazole. For primary therapy: a patient who has received more than 48 hours of antifungal therapy for the current episode of Candida infection. A patient with significant underlying liver disease at the time of enrollment in the study. A patient with significant renal disease (CrCl < 50 ml/min) at the time of enrollment in the study. A patient with a high likelihood of death within 72 hours of study enrollment due to factors unrelated to Candida infection.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Active voriconazole<br>All subjects in this study will receive active voriconazole in an open-label fashion; there is no comparator in this study. | Drug: voriconazole<br>* Subjects 12 - <18 yrs (excluding subjects 12 - 14 yrs weighing <50kg): For IC, the loading dose is 6 mg/kg IV q12h on Day 1, followed by 4 mg/kg IV q12h thereafter. Oral therapy, if given, will be administered at a dose of 200 mg q12h. For EC, no loading dose is required, and subjects will be initiated on 3 mg/kg IV q12h. Oral therapy, if given, will be administered at a dose of 200 mg q12h. Subjects 2 - <12 yrs, and subjects 12 - 14 weighing <50kg: For IC, the loading dose is 9 mg/kg IV q12h on Day 1, followed by 8 mg/kg IV q12h thereafter. For EC, no loading dose is required, and subjects will be initiated on 4 mg/kg IV q12. Oral therapy, if given, will be administered at a dose of 9 mg/kg q12h (maximum initial dose of 300 mg). Subjects will be treated for a minimum of 14 days for invasive candidiasis/candidemia or a minimum of 7 days for esophageal candidiasis. Subjects will be treated for up to a maximum of 42 days.<br>* Other names: Vfend;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants With Adverse Events - Overall Summary | Percentage of participants with adverse events (AEs), serious adverse events (SAEs), severe AEs, who discontinued due to AEs, or who had dose redued or temporarily discontinued due to AEs. | Baseline up to 1 month follow-up | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants With a Global Response of Success at End of Treatment (EOT) | Global response was determined programmatically based on investigator assessment of clinical and microbiological response. Global response of success was defined as clinical cure or improvement AND microbiological eradication or presumed eradication. Exact 95 percent (%) confidence interval for binomial proportions using Clopper-Pearson method. | EOT (from 7 to 42 days of treatment) | 
| All-Cause Mortality - Number of Participant Deaths |  | Day 28 and 1 Month Follow-up | 
| Time to Death |  | Baseline up to 1 month follow-up | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Invasive Candidiasis Candidemia Esophageal Candidiasis