Cystic fibrosis MedDRA version: 20.0 Level: LLT Classification code 10028141 Term: Mucoviscidosis System Organ Class: 100000004850
Conditions
Sponsors
Ruprecht-Karls-University Heidelberg, Medical Faculty
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age = 18 years (1st cohort). If justified by interim analysis, 18 > age = 12 years (2nd cohort), 2. Informed consent, 3. Sufficient fluency in German language, 4. Confirmed diagnosis of cystic fibrosis, 6. FEV1 = 50 % pred. at screening, 7.LCl 2.5 = 7.05 at screening, 8. Ability to performed study specific procedures, 9. Oxyhaemoglobin saturation of = 90% on room air at screening, 10. No recent changes in the medication for cystic fibrosis lung disease, 11. Adequate bone marrow function, 12. Adequate liver function, 13. Adequate blood clotting, 14. Negative serology for HIV, HBV, and HCV, negative Interferon-gamma release assay, 15. Negative pregnancy test in women of childbearing potential, 16. Use of adequate contraception in sexually active female subjects. Are the trial subjects under 18? yes Number of subjects for this age range: 27 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 25 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Expected non-compliance, 2. Known allergy to anakinra or any ingredient of the pharmaceutical formulation of Kineret®, 3. Planned (during the treatment) or recently completed immunization with attenuated (live) vaccine(s), 4. Renal failure, 5. History of tuberculosis or repeated detection of non-tuberculous mycobacteria from airway samples in the last 12 months, 6. History of detection of Burkholderia cenocepacia species in the last 12 months, 7. Colonization with multi-resistant Staphylococcus aureus (MRSA) and/or 4-multi-resistant gram negative (MRGN) Pseudomonas aeruginosa, 8. Acute bronchopulmonary exacerbation, 9. Signs of other active infection within 14 days prior to the screening, 10. Immunosuppressive treatment due to organ transplantation, rheumatic or autoimmune diseases as well as treatment with Anakinra in the last 3 months before Day 1 of Period 1 11. Recent Participation in another interventional trial, 12. Current oral corticosteroid use, 13. Current oxygen supplementation, 14. Current treatment with etanercept, 15. Medical history of lung transplantation, 16. Pregnancy or nursing, 17. Known hypersensitivity to hypertonic saline (used for induction of sputum).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • Assessment of absolute pre-post change of the lung clearance index (LCI).;Secondary Objective: • Evaluation of the safety and tolerability of the 28-days-treatment with anakinra. • Investigation of the effects of anakinra on lung function. • Evaluation of the impact of anakinra on the quality-of-life (QoL). ;Primary end point(s): • Absolute pre-post change of the lung clearance index (LCI).;Timepoint(s) of evaluation of this end point: At all trial visits, i.e. at 6 timepoints: Screening visit, directly prior and directly after administration of IMP and Placebo, End of trial visit. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Evaluation of the safety and tolerability of the 28-days-treatment with anakinra by means of: o Physical examination o (Serious) adverse events o Laboratory safety parameters • Investigation of the effects of anakinra on lung function by means of absolute change in percentage points predicted forced expiratory volume in 1 second (FEV1 and FEV1 % pred). • Evaluation of the impact of anakinra on the quality-of-life (QoL) in the considered population by means of the Cystic Fibrosis Questionnaire – Revised (CFQ-R, German version). • Investigation of the effects of anakinra on lung function by means of absolute change in forced expiratory flow75 in liters/second and percent predicted (FEF75 and FEF75 % pred) and forced vital capacity in liter and percent predicted (FVC and FVC % pred). Substudies: • Assessment of the influence of anakinra on lung structure and perfusion determined by chest MRI. • Assessment of the influence of anakinra on airway inflammation by means of the following parameters: o Absolute and differential cell count in sputum samples, o Inflammatory markers in sputum samples. • Assessment of the influence of anakinra on the bronchial infection status by means of sputum microbiology.;Timepoint(s) of evaluation of this end point: At all trial visits, i.e. at 6 timepoints: Screening visit, directly prior and directly after administration of IMP and Placebo, End of trial visit. | — |
Countries
Germany
Contacts
Public ContactDivision of Pediatric Pulmonology
University Hospital Heidelberg, Department of Pediatrics III
Outcome results
None listed