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A Study of the Safety, Tolerance, and Pharmacokinetics of Oral Posaconazole in Immunocompromised Children (P03579)

Registration Number
NCT01716234
Lead Sponsor
Merck Sharp & Dohme LLC
Brief Summary

The purpose of this dose-escalation study is to evaluate the pharmacokinetics, safety, and tolerability of oral posaconazole in immunocompromised children with neutropenia or expected neutropenia.

Detailed Description

Not available

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
142
Inclusion Criteria
  • Documented or anticipated neutropenia expected to last at least 7 days and only in the following clinical situations: acute leukemia; myelodysplasia; severe aplastic anemia; autologous hematopoietic stem cell transplantation (HSCT) recipients; high risk neuroblastoma; advanced stage non-Hodgkin's lymphoma; recipients of allogeneic HSCT during the pre-engraftment (neutropenia) period
  • Participants of child-bearing potential must use a medically accepted method of

contraception throughout the study and for at least 30 days after stopping study medication, unless they are surgically or medically sterile or agree to remain abstinent.

Exclusion Criteria
  • Proven invasive fungal infection (IFI) before study entry
  • Severe nausea and/or vomiting at screening
  • Received posaconazole within 10 days before screening
  • Unable to receive study drug by mouth or via an intestinal (enteral) tube
  • Females who are pregnant, intend to become pregnant during the study, or are breastfeeding
  • History of anaphylaxis attributed to the azole class of antifungal agents

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
POS 18 BID 2 to <7 YearsPosaconazole 18 mg/kg/day BIDParticipants aged 2 to \<7 years received posaconazole oral suspension 18 mg/kg/day divided into 2 doses (BID) until recovery from neutropenia or up to 28 days.
POS 12 TID 3 months to <2 YearsPosaconazole 12 mg/kg/day TIDParticipants aged 3 months to \<2 years received posaconazole oral suspension 12 mg/kg/day divided into 3 doses (TID) until recovery from neutropenia or up to 28 days.
POS 18 BID 7 to <18 YearsPosaconazole 18 mg/kg/day BIDParticipants aged 7 to \<18 years received posaconazole oral suspension 18 mg/kg/day divided into 2 doses (BID) until recovery from neutropenia or up to 28 days.
POS 18 TID 7 to <18 YearsPosaconazole 18 mg/kg/day TIDParticipants aged 7 to \<18 years received posaconazole oral suspension 18 mg/kg/day divided into 3 doses (TID) until recovery from neutropenia or up to 28 days.
POS 12 BID 2 to <7 YearsPosaconazole 12 mg/kg/day BIDParticipants aged 2 to \<7 years received posaconazole oral suspension 12 mg/kg/day divided into 2 doses (BID) until recovery from neutropenia or up to 28 days.
POS 12 BID 7 to <18 YearsPosaconazole 12 mg/kg/day BIDParticipants aged 7 to \<18 years received posaconazole oral suspension 12 mg/kg/day divided into 2 doses (BID) until recovery from neutropenia or up to 28 days.
POS 18 TID 2 to <7 YearsPosaconazole 18 mg/kg/day TIDParticipants aged 2 to \<7 years received posaconazole oral suspension 18 mg/kg/day divided into 3 doses (TID) until recovery from neutropenia or up to 28 days.
Primary Outcome Measures
NameTimeMethod
Average Concentration of Posaconazole (Cavg) on Day 1 (Single Dose)Up to 12 hours after the first dose (BID dose groups) or up to 8 hours after the first dose (TID dose (TID dose groups)

Blood samples for determination of plasma posaconazole concentration were collected predose and approximately 3, 5, 8, and 12 hours after the first dose on Day 1. The 12-hour sample was not obtained for the TID dose groups. Day 1 pharmacokinetic samples were not collected for participants 3 months to \<2 years of age weighing \<6.5 kg.

Average Concentration of Posaconazole (Cavg) on Day 7 (Steady State)Up to 12 hours after the first dose on Day 7 (BID dose groups) or up to 8 hours after the first dose on Day 7 (TID dose

Blood samples for determination of plasma posaconazole concentration were collected predose and approximately 3, 5, 8, and 12 hours after the first dose on Day 7 (steady state). The 12-hour sample was not obtained for the TID dose groups. The target Cavg range was 500 to \<2500 ng/mL.

Secondary Outcome Measures
NameTimeMethod
Number of Participants With an Adverse EventUp to Day 58

An adverse event is any untoward medical occurrence in a participant administered a pharmaceutical product, biologic (at any dose), or medical device, which does not necessarily have a causal relationship with the treatment. Adverse events may include the onset of new illness and the exacerbation of preexisting conditions.

Number of Participants With an Adverse Event Leading to Study Drug DiscontinuationUp to Day 28

An adverse event is any untoward medical occurrence in a participant administered a pharmaceutical product, biologic (at any dose), or medical device, which does not necessarily have a causal relationship with the treatment. Adverse events may include the onset of new illness and the exacerbation of preexisting conditions.

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