Pharmacokinetics of Posaconazole in Children With Chronic Granulomatous Disease (CGD) (iPOD)

November 26, 2020 updated by: Radboud University Medical Center

Investigation of POsaconazole Prophylaxis in Children With Chronic Granulomatous Disease (CGD): Pharmacokinetics and Tolerability (iPOD)

The purpose of this study is to find a dose for a twice daily regimen for posaconazole (PSZ) as prophylactic treatment in children with CGD, based on the PSZ trough level.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

At this moment itraconazole is the drug of first choice in the prophylaxis of fungal infections in children with CGD. Breakthrough fungal infections while on itra-conazole prophylaxis are described in literature indicating the need for a drug with a broader antifungal spectrum. PSZ might provide in this need. PSZ may also have a clinical safety and tolerability advantage over other antifungal agents. Because PSZ is metabolized through phase II glucuronidation it is less common to be subject to drug interactions. PSZ is known to be a CYP3A4 inhibitor, but does not inhibit other CYP enzymes, therefore it may exhibit fewer drug interactions as compared with other azole antifungal agents.

Treatment of children is still off-label use. No data have been published to date on the exposure of PSZ in children under the age of 8 or in children with CGD. There is an urgent need to study the use of PSZ in these young children. Furthermore, the current regimen for antifungal prophylaxis requires a three times daily administration of PSZ. For this specific purpose less complex dosing schedules are warranted thus defining the need to examine a twice daily schedule.

As the tolerability and pharmacokinetics are unknown in patients under the age of 8 years and only limited data are available for age groups 8 to 16 years, we propose a feasibility study of a twice daily regimen of PSZ prophylaxis in CGD patients. With this information available we can suggest a dosage for future prophylaxis in this patient group.

Study Type

Interventional

Enrollment (Actual)

12

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Netherlands
      • Amsterdam, Netherlands
        • AMC
    • Gelderland
      • Nijmegen, Gelderland, Netherlands
        • Radboud University Medical Centre Nijmegen

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 16 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Patient has CGD, rendering him/her at risk for invasive fungal infections hence requiring antifungal prophylaxis.
  • Patient is at least 2 years of age and younger than 17 years of age on the day of the first dosing.
  • Parents or legal representative, and children where appropriate, willing and able to give informed consent.

Exclusion Criteria:

  • Patient is suspected of an invasive fungal infection.
  • Therapy with any medicinal product for which an effect on PSZ is expected. If patient is undergoing therapy with any medicinal product which may be effected by PSZ, the patient is included on condition that the investigator judges that the effects are not clinically relevant. This should be clearly recorded.
  • Documented history of sensitivity/idiosyncrasy to PSZ.
  • Results of serum biochemistry and hematology testing are not higher than 3x the upper limit of normal. If the results exceed these limits, the patient is included on condition that the investigator judges that the deviations are not clinically relevant. This should be clearly recorded.
  • Relevant history or current condition that might interfere with drug absorption, distribution, metabolism or excretion.
  • Relevant history or presence of cardiovascular disorder or renal and hepatic disorder.
  • History of or current abuse of drugs, alcohol or recreational substances.
  • Participation in a trial with an investigational drug within 60 days prior to the first dose.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: posaconazole
posaconazole as antifungal prophylaxis
Drug: posaconazole (PSZ)
Intake of PSZ oral suspension 40mg/ml twice daily with food. Starting dose is based on bodyweight. The dosage will be adjusted if the exposure is not adequate based on PSZ trough level on Day 10 and 20.
Other Names:
  • Noxafil

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Posaconazole trough levels
Time Frame: Day 10; 20; 30
Day 10; 20; 30

Secondary Outcome Measures

Outcome Measure
Time Frame
adverse events monitoring
Time Frame: entire study
entire study

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Radboud University Medical Center

Investigators

  • Principal Investigator: David M Burger, PharmD PhD, Radboud University Medical Centre Nijmegen

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2009

Primary Completion (Actual)

July 1, 2010

Study Completion (Actual)

August 1, 2010

Study Registration Dates

First Submitted

November 26, 2008

First Submitted That Met QC Criteria

November 26, 2008

First Posted (Estimate)

November 27, 2008

Study Record Updates

Last Update Posted (Actual)

November 30, 2020

Last Update Submitted That Met QC Criteria

November 26, 2020

Last Verified

November 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • UMCN-AKF 08.01

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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