E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
treatment and prophylaxis of invasive fungal infections; oropharyngeal candidiasis (OPC) and refractory OPC |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10050346 |
E.1.2 | Term | Oropharyngeal candidiasis |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | HLGT |
E.1.2 | Classification code | 10017528 |
E.1.2 | Term | Fungal infectious disorders |
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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 |
The primary objective of this study is to evaluate the pharmacokinetics (PK) of posaconazole (POS) administered orally at three dosage levels to immunocompromised children aged 3 months to <18 years with neutropenia or expected neutropenia |
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E.2.2 | Secondary objectives of the trial |
The secondary objective is to evaluate the safety and tolerability of POS administered orally at three dosage levels to immunocompromised children with neutropenia or expected neutropenia aged 3 months to <18 years and to compare the exposures to POS in pediatric subjects to those from an adult population with similar underlying conditions. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
. Children of either sex and of any race, 3 months to <18 years of age. 2. Subjects must have documented or anticipated neutropenia (ANC <500/mm3 [0.5 x 109/L]) expected to last for at least 7 days and only in the following clinical situations: Acute leukemia (including new and relapse), Myelodysplasia, Severe aplastic anemia, Autologous HSCT recipients, High risk neuroblastoma, Advanced stage non-Hodgkins lymphoma |
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E.4 | Principal exclusion criteria |
1. Subjects with proven IFI, as defined by the MSG/EORTC criteria prior to study entry. 2. Subjects with Grade 3 or Grade 4 nausea and/or vomiting at Screening. 3. Subjects receiving weekly vincristine. 4. Subjects receiving systemic antifungal other than POS (oral, non-absorbable antifungals are permitted). 5. Subjects who have received POS within the past 10 days prior to Screening. 6. Subjects receiving or planning to receive prohibited drugs during POS administration (please refer to Table 3). 7. Subjects whose laboratory tests are outside normal limits, as follows: a. AST or ALT >5 times the upper limit of normal (ULN) b. Serum total bilirubin >2.5 x ULN c. Calculated creatinine clearance <30 mL/min. Creatinine clearance will be calculated using the following equation: Creatinine clearance = k*height (cm)/serum creatinine (mg/dL) Where k = 0.45 for a full term baby less than 1 year old; 0.55 for children up to 12 years old; 0.55 for females between the ages of 13 and 21 years; 0.7 for males between the ages of 13 and 21 years. 8. Subjects with QTc prolongation: a. Symptomatic QTc prolongation >450 msec (males) or >470 msec (females) b. Any QTc prolongation of >500 msec 9. Subjects who are unable to receive study drug enterally. 10. Female subjects who are pregnant, intend to become pregnant during the course of the study, or are breast-feeding. 11. Subjects with a history of anaphylaxis attributed to the azole class of antifungal agents. 12. Subjects with any clinically significant condition or situation, other than the condition being studied that, in the opinion of the investigator, would interfere with the study evaluations or optimal participation in the study, including receiving less than 7 days of POS. 13. Subjects who have already participated in this study or are participating in any Phase 1 clinical study or any study for a medication that has not yet received regulatory approval. 14. Subjects who are part of the study staff personnel or family members of the study staff personnel |
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E.5 End points |
E.5.1 | Primary end point(s) |
pharmacokinetics, safety and tollerance |
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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 | Yes |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
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) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
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E.7.2 | Therapeutic exploratory (Phase II) | No |
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 | Yes |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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 | Yes |
E.8.1.7.1 | Other trial design description |
Age Group 1: 2 ≤7 , Age Group2: 7 ≤18; Age Group 3: 3 mesi≤2 anni |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 4 |
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 | Information not present in EudraCT |
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 |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |