Chronic Granulomatous DiseasePhase 2iPODNCT00799071
Pharmacokinetics of Posaconazole in Children With Chronic Granulomatous Disease (CGD)
A Phase 2 study testing posaconazole (PSZ) in people with Chronic Granulomatous Disease, run by Radboud University Medical Center at 2 sites in Netherlands.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 16 years
- Conditions the study is for
- Chronic Granulomatous Disease
- What is being tested?
- posaconazole (PSZ)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesNetherlands
- Study dates
- Feb 2009to Aug 2010
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
Netherlands2 sites
AMC
Amsterdam
Radboud University Medical Centre Nijmegen
Nijmegen, Gelderland
About this study
Official title: 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.
Read the 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.
- Conditions
- Chronic Granulomatous Disease
- Being tested
- posaconazole (PSZ)
- Keywords
- prophylaxis, CGD, pharmacokinetics
- Medical subject headings
- Granulomatous Disease, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
posaconazole (PSZ)Drug
Also called Noxafil
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.
| Study group | Role | What they receive |
|---|---|---|
| posaconazole | Experimental | posaconazole as antifungal prophylaxis Receives: Drug: posaconazole (PSZ) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Posaconazole trough levels
Measured: Day 10; 20; 30
Secondary outcomes (1)
adverse events monitoring
Measured: entire study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Nov 2008 (estimated)
- Study startFeb 2009
- Primary completionJul 2010
- Study completionAug 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Chronic Granulomatous DiseaseStudies of the same condition, open and under way.
- More trials run by Radboud University Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Radboud University Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- David M Burger, PharmD PhD, Principal investigator, Radboud University Medical Centre Nijmegen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00799071
- Sponsor's protocol ID
- UMCN-AKF 08.01
- Acronym
- iPOD
- Status last verified
- Nov 2020
- First submitted
- 26 Nov 2008
- Last updated
- 30 Nov 2020
- Results posted
- No
- Data monitoring committee
- No
Publications
Welzen ME, Bruggemann RJ, Van Den Berg JM, Voogt HW, Gilissen JH, Pajkrt D, Klein N, Burger DM, Warris A. A twice daily posaconazole dosing algorithm for children with chronic granulomatous disease. Pediatr Infect Dis J. 2011 Sep;30(9):794-7. doi: 10.1097/INF.0b013e3182195808.
PubMed 21772229
Study at a glance
From the registry record, last updated by the sponsor on 30 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Investigation of POsaconazole Prophylaxis in Children With Chronic Granulomatous Disease (CGD): Pharmacokinetics and Tolerability (iPOD)
- ClinicalTrials.gov ID
- NCT00799071
- Acronym / sponsor ID
- iPOD / UMCN-AKF 08.01
- Conditions
- Chronic Granulomatous Disease
- Keywords
- prophylaxis, CGD, pharmacokinetics
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Radboud University Medical CenterUniversity, hospital or other
- Study start
- Feb 2009
- Primary completion
- Jul 2010 (actual)
- Study completion
- Aug 2010 (actual)
- First posted
- 27 Nov 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| posaconazole | Experimental | Drug: posaconazole (PSZ)posaconazole as antifungal prophylaxis |
Who may qualify
- Ages
- 2 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| posaconazole (PSZ)Also: NoxafilIntake 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. | Drug | posaconazole |
Primary outcome
Posaconazole trough levels
Time frame: Day 10; 20; 30
1 secondary outcome
adverse events monitoring
Time frame: entire study
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Nov 2020.
- Results publication: PubMed 21772229Welzen ME, Bruggemann RJ, Van Den Berg JM, Voogt HW, Gilissen JH, Pajkrt D, Klein N, Burger DM, Warris A. A twice daily posaconazole dosing algorithm for children with chronic granulomatous disease. Pediatr Infect Dis J. 2011 Sep;30(9):794-7. doi: 10.1097/INF.0b013e3182195808.
- Pediatr Infect Dis J. 2011 Sep;30(9):794-7. A twice daily posaconazole dosing algorithm for children with chronic granulomatous disease.Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David M Burger, PharmD PhD principal investigator
Radboud University Medical Centre Nijmegen
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing posaconazole (PSZ) in people with Chronic Granulomatous Disease, run by Radboud University Medical Center at 2 sites in Netherlands.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Posaconazole trough levels (time frame: Day 10; 20; 30). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00799071, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Nov 2020.