Clinical trial NCT01716234: A Study of the Safety, Tolerance, and Pharmacokinetics of Oral Posaconazole in Immunocompromised Children (P03579)
Official title: Phase 1B Study of the Safety, Tolerance, and Pharmacokinetics of Oral Posaconazole in Immunocompromised Children With Neutropenia (P03579)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Fungal Infections
Lead sponsor: Merck Sharp & Dohme LLC
Sponsor class: INDUSTRY
First posted: 2012-10-29
Enrollment: 142
Sex: ALL
Minimum age (years): 3
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Average Concentration of Posaconazole (Cavg) on Day 1 (Single Dose)
timeFrame: 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)
measure: Average Concentration of Posaconazole (Cavg) on Day 7 (Steady State)
timeFrame: 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
Secondary outcomes:
measure: Number of Participants With an Adverse Event
timeFrame: Up to Day 58
measure: Number of Participants With an Adverse Event Leading to Study Drug Discontinuation
timeFrame: Up to Day 28
Other outcomes:
Locations:
Study officials:
affiliation: Merck Sharp & Dohme LLC
name: Medical Director
role: STUDY_DIRECTOR
References:
citation: Arrieta AC, Sung L, Bradley JS, Zwaan CM, Gates D, Waskin H, Carmelitano P, Groll AH, Lehrnbecher T, Mangin E, Joshi A, Kartsonis NA, Walsh TJ, Paschke A. A non-randomized trial to assess the safety, tolerability, and pharmacokinetics of posaconazole oral suspension in immunocompromised children with neutropenia. PLoS One. 2019 Mar 26;14(3):e0212837. doi: 10.1371/journal.pone.0212837. eCollection 2019.