Cystic fibrosis MedDRA version: 20.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Ferinject
Product Name: Ferinject
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Ferric carboxymaltose
CAS
Sponsors
University of Oxford / Clinical Trials and Research Governance
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Age =18 years - Established diagnosis of cystic fibrosis - Iron deficiency (transferrin saturation =16 % or ferritin =65 years) no F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: - Urgent (300 g/l or transferrin saturation >45% - Pregnancy or breast feeding - Previous transplantation - Judged by a designated member of the trial team to be unlikely to comply with safety aspects of the trial protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the early safety profile of IV iron in adults with CF by comparing the incidence of new infective events during the 4 weeks before and after IV iron; Secondary Objective: 1) To compare the incidence of new infective events during the 12 weeks before and after IV iron. 2) To assess the impact of IV iron on airway microbiology, including incidence and severity of exacerbations. 3) To assess the effect of IV iron on systemic iron and inflammatory status. 4) To assess the impact of IV iron on standard indices of CF disease severity. 5) To assess the impact of IV iron on health-related quality of life. 6) To assess the acceptability of the intervention and trial procedures. 7) To explore the feasibility of measuring exercise capacity and lung blood pressure in adults with cystic fibrosis. 8) To assess the effect of IV iron on sputum and systemic iron and inflammatory status during infective exacerbations of CF. ; Primary end point(s): The incidence of new infective events in the four weeks before intravenous iron, compared with the four weeks after intravenous iron, where a new infective event is defined as any of: 1) New microbiological isolate on routine sputum culture (organism not cultured in 12 months prior to study); 2) Clinical infection requiring IV antibiotics (as judged by the clinical team based on routine clinical data); 3) Admission to hospital for infection-related reason (as judged by clinical team based on routine clinical data); 4) Significant deterioration in lung function (>10% fall in FEV1), not otherwise explained (as judged by clinical team, based on routine clinical data). ;Timepoint(s) of evaluation of this end point: One month after administration of IMP. | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Weeks 0, 4, 8 and 16 (the intervention is given at week 4); Secondary end point(s): Incidence of new infective events during the 12-week period before intravenous iron, compared with the 12-week period after intravenous iron. Change in number of IV and oral antibiotic days. Sputum iron levels, sputum culture and cell counts, respiratory microbiota analysis. Change in blood and sputum markers of iron and inflammation. Change in arterial oxygen saturation, body mass index and lung function (forced expiratory volume in 1 second, forced vital capacity). Change in CFQ-R and SF-36 questionnaire scores. Change in exercise capacity (shuttle walk test) and echocardiographic assessment of systolic pulmonary artery pressure. Percentage of eligible patients entering and completing the study. | — |
Countries
United Kingdom
Contacts
Public ContactCTRG
University of Oxford / Clinical Trials and Research Governance
Outcome results
None listed