Phase 1B Study of the Safety, Tolerance, and Pharmacokinetics of Oral Posaconazole in Immunocompromised Children With Neutropenia

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 108

Inclusion Criteria

1. Children of either sex and of any race, 3 months to <18 years of age.

2. Subjects must have documented or anticipated neutropenia (ANC less than or equal to 500/mm3 [0.5 x 10^9/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-Hodgkin’s lymphoma. Recipients of allogeneic HSCT during the pre-engraftment period (neutropenia period).

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 who have received POS within the past 10 days prior to Screening

4. Subjects receiving prohibited drugs (please refer to Table 3)

5. Subjects whose laboratory tests are outside normal limits as follows:
a. AST ot ALT>5 times th eupper limit of normal (ULN)
b. Serum total bilirubin >2.5 x ULN
c. Calculated creatinine clearance <30mL/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

6. Subjects with QTc prolongation:
a. Symptomatic QTc prolongation >450 msec (males) or >470 msec (females)
b. Any QTc prolongation of >500 msec

7. Subjects who are unable to receive drug internally

8. Female subjects who are pregnant, intend to become pregnant during the course of the study, or are breast-feeding

9. Subjects with a history of anaphylaxis attributed to the azole class of antifungal agents.

10. 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.

11. 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.

12. Subjects who are part of the study staff personnel or family members of the study staff personnel.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: 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.;Secondary Objective: 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.;Primary end point(s): Pharmacokinetics, safety and tolerance.

Secondary Outcome Measures

Name	Time	Method

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