Prospective validation and clinical evaluation of a new posaconazole dosing regimen for children and adolescents with cystic fibrosis and Aspergillus infection.

Phase 1

• Conditions: Cystic Fibrosis and Aspergillus infection.; MedDRA version: 20.0Level: LLTClassification code 10003488Term: AspergillosisSystem Organ Class: 100000004862; Therapeutic area: Diseases [C] - Bacterial Infections and Mycoses [C01]

• Registration Number: EUCTR2019-004511-31-IT
• Lead Sponsor: IRCCS, OSPEDALE PEDIATRICO BAMBINO GESÙ DI ROMA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-10-07
• Last Update Posted: 2 March 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 135

Inclusion Criteria

Inclusion criteria for the screening phase:
1. Diagnosed with cystic fibrosis (genetic diagnosis and clinical phenotype of lung disease)
2. Age = 8 yrs and < 18 yrs
3. Able to produce a sputum sample (spontaneous or induced sputum)
4. Informed Consent given
Inclusion criteria for the intervention phase:
1. Diagnosed with cystic fibrosis (genetic diagnosis and/or abnormal sweat test and clinical phenotype of lung disease)
2. Age = 8 yrs and < 18 yrs
3. Presence of Aspergillus infection as defined for this study
4. Clinically stable condition without a significant change in lung function (FEV1 +/- 10%) or significant worsening of respiratory symptoms over the previous month
5. Able to perform lung function test (FEV1)
6. Able to produce a sputum sample (spontaneous or induced sputum)
7. Informed Consent given
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 for the trial:
1. Age < 8 yrs or = 18 yrs
2. Unable to perform lung function test (FEV1)
3. Unable to produce a sputum sample (spontaneous or induced sputum)
4. Clinically unstable condition with significant change in lung function or significant worsening of respiratory symptoms
5. Unable to tolerate oral medication
6. Known hypersensitivity to the drug/excipients
7. On active transplant list or transplant recipient
8. Azole resistant Aspergillus sp. cultured
9. Patients receiving terfenadine, ergot alkaloids, astemizole, cisapride, pimozide, halofantrine, quinidine, or HMG-CoA reductase inhibitors metabolised through CYP3A4
10. Patients receiving omalizumab
11. Received systemic mould-active antifungals in the last month
12. Shortened or elongated QT interval
13. Cardiac failure
14. ALT = 200 U/L
15. AST = 225 U/L
16. Alkaline phosphatase = 460 U/L
17. Bilirubin = 50 umol/L
18. Pregnancy
19. Informed Consent not given

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified