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

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ClinicalTrials.gov Identifier: NCT00799071
Recruitment Status : Completed
First Posted : November 27, 2008
Last Update Posted : November 30, 2020
Sponsor:
Information provided by (Responsible Party):
Radboud University Medical Center

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Chronic Granulomatous Disease Drug: posaconazole (PSZ) Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: Investigation of POsaconazole Prophylaxis in Children With Chronic Granulomatous Disease (CGD): Pharmacokinetics and Tolerability (iPOD)
Study Start Date : February 2009
Actual Primary Completion Date : July 2010
Actual Study Completion Date : August 2010


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 Name: Noxafil




Primary Outcome Measures :
  1. Posaconazole trough levels [ Time Frame: Day 10; 20; 30 ]

Secondary Outcome Measures :
  1. adverse events monitoring [ Time Frame: entire study ]


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Ages Eligible for Study:   2 Years to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00799071


Locations
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Netherlands
Radboud University Medical Centre Nijmegen
Nijmegen, Gelderland, Netherlands
AMC
Amsterdam, Netherlands
Sponsors and Collaborators
Radboud University Medical Center
Investigators
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Principal Investigator: David M Burger, PharmD PhD Radboud University Medical Centre Nijmegen
Additional Information:
Publications of Results:
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Responsible Party: Radboud University Medical Center
ClinicalTrials.gov Identifier: NCT00799071    
Other Study ID Numbers: UMCN-AKF 08.01
First Posted: November 27, 2008    Key Record Dates
Last Update Posted: November 30, 2020
Last Verified: November 2020
Keywords provided by Radboud University Medical Center:
prophylaxis
CGD
pharmacokinetics
Additional relevant MeSH terms:
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Granuloma
Granulomatous Disease, Chronic
Lymphoproliferative Disorders
Lymphatic Diseases
Pathologic Processes
Phagocyte Bactericidal Dysfunction
Leukocyte Disorders
Hematologic Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes
Immune System Diseases
Posaconazole
Antifungal Agents
Anti-Infective Agents
Trypanocidal Agents
Antiprotozoal Agents
Antiparasitic Agents
14-alpha Demethylase Inhibitors
Cytochrome P-450 Enzyme Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Steroid Synthesis Inhibitors
Hormone Antagonists
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs