Children with chronic granulomatous disease (CGD). MedDRA version: 9.1 Level: LLT Classification code 10008906 Term: Chronic granulomatous disease
Conditions
Interventions
Trade Name: Noxafil 40 mg/ml oral solution
Pharmaceutical Form: Oral suspension
Sponsors
Radboud University Nijmegen Medical Centre
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients with CGD, rendering them at risk for invasive fungal infections, hence requiring antifungal prophylaxis. 2. Patient is at least 2 years of age and younger than 17 years of age on the day of the first dosing. 3. Parents or legal representative, and children where appropriate, willing and able to give informed consent. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Patients suspected of an invasive fungal infection. 2. Therapy with any medicinal product for which an effect on PSZ is expected (see Clinical Trial Protocol, appendix B, Table 1). 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 (see Clinical Trial Protocol, appendix B, Table 2). This should be clearly recorded. 3. Documented history of sensitivity/idiosyncrasy to PSZ. 4. Results of serum biochemistry and hematology testing are not higher than 3x the upper limit of normal (see Clinical Trial Protocl, appendix A). If the results exceed these limits, the subject is included on condition that the investigator judges that the deviations are not clinically relevant. This should be clearly recorded. 5. Relevant history or current condition that might interfere with drug absorption, distribution, metabolism or excretion. 6. Relevant history or presence of cardiovascular disorder or renal and hepatic disorder. 7. History of or current abuse of drugs, alcohol or recreational substances. 8. Participation in a trial with an investigational drug within 60 days prior to the first dose.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Dose finding for a twice daily regimen for posaconazole (PSZ) as prophylactic treatment in children with CGD, based on the PSZ trough level.;Secondary Objective: To determine tolerability of PSZ as prophylactic treatment in children with CGD.;Primary end point(s): Individual trough PSZ plasma concentrations, on basis of which the PSZ dosage for individual patients will be adjusted. Based on the results a dosage for future prophylaxis with PSZ in children with CGD will be defined. | — |
Countries
Netherlands, United Kingdom
Outcome results
None listed